View ValuationIntellia Therapeutics 将来の成長Future 基準チェック /56Intellia Therapeutics利益と収益がそれぞれ年間65.7%と55.6%増加すると予測されています。EPS は年間 増加すると予想されています。自己資本利益率は 3 年後に13.6% 66.5%なると予測されています。主要情報65.7%収益成長率66.48%EPS成長率Biotechs 収益成長25.2%収益成長率55.6%将来の株主資本利益率13.57%アナリストカバレッジGood最終更新日22 May 2026今後の成長に関する最新情報分析記事 • May 14Earnings Update: Intellia Therapeutics, Inc. (NASDAQ:NTLA) Just Reported Its First-Quarter Results And Analysts Are Updating Their ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its quarterly results, which were...分析記事 • May 11Analysts Have Made A Financial Statement On Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) First-Quarter ReportA week ago, Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) came out with a strong set of first-quarter numbers that could...分析記事 • Mar 02Earnings Release: Here's Why Analysts Cut Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Price Target To US$46.08Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its yearly results, which were ahead...分析記事 • Aug 11Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportThe analysts might have been a bit too bullish on Intellia Therapeutics, Inc. ( NASDAQ:NTLA ), given that the company...分析記事 • Feb 24Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders are probably feeling a little disappointed, since its shares...すべての更新を表示Recent updatesBoard Change • May 20Insufficient new directorsThere is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 6 experienced directors. 5 highly experienced directors. Independent Director Brian Goff was the last director to join the board, commencing their role in 2024. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model.ナラティブの更新 • May 14NTLA: Clinical Hold Resolution Will Reopen ATTR Phase 3 Upside PotentialAnalysts have lifted the fair value estimate for Intellia Therapeutics to $26.70 from $25.29. This reflects a series of higher price targets across the Street as recent FDA decisions on the MAGNITUDE program ease regulatory concerns and support more optimistic assumptions on revenue growth, profitability, and long-term P/E potential.分析記事 • May 14Earnings Update: Intellia Therapeutics, Inc. (NASDAQ:NTLA) Just Reported Its First-Quarter Results And Analysts Are Updating Their ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its quarterly results, which were...ライブニュース • May 13Intellia Therapeutics Achieves Phase 3 CRISPR Milestone and Advances Toward FDA ApprovalIntellia Therapeutics reported positive topline Phase 3 results for lonvoguran ziclumeran (lonvo-z), its one-time in vivo CRISPR therapy for hereditary angioedema, with the trial meeting its primary and all key secondary endpoints and showing an 87% reduction in mean monthly HAE attacks versus placebo. The company has started a rolling Biologics License Application with the FDA for lonvo-z, with plans to complete the filing in the second half of 2026 and targeting a potential U.S. launch in the first half of 2027, subject to regulatory approval. The FDA has lifted clinical holds on Intellia’s Phase 3 MAGNITUDE and MAGNITUDE-2 trials for transthyretin amyloidosis, and ARK Invest has significantly increased its stake in the company, buying about 3.78 million shares worth nearly US$48m across direct holdings and ETFs. Taken together, the Phase 3 success, BLA progress and resumption of key ATTR trials indicate that Intellia’s in vivo CRISPR platform is moving deeper into late-stage and potential commercial territory, which can be a key focus point for investor attention. At the same time, the stock’s recent volatility, ongoing clinical and regulatory work, and the company’s need to fund its pipeline mean investors still have to weigh execution and approval risk carefully, despite growing institutional interest.お知らせ • May 04Intellia Therapeutics, Inc., Annual General Meeting, Jun 09, 2026Intellia Therapeutics, Inc., Annual General Meeting, Jun 09, 2026.お知らせ • Apr 30Intellia Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $180.00001 million.Intellia Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $180.00001 million. Security Name: Common Stock Security Type: Common Stock Securities Offered: 16,744,187 Price\Range: $10.75 Discount Per Security: $0.645お知らせ • Apr 29Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to Fda for Lonvoguran Ziclumeran as A One-Time Treatment for Hereditary AngioedemaIntellia Therapeutics, Inc. announced it has initiated a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of lonvo-z (formerly known as NTLA-2002) for hereditary angioedema (HAE). Designed as a one-time treatment that is administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels. Intellia also separately announced positive topline data from the Phase 3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all key secondary endpoints, demonstrating that a one-time dose of lonvo-z led to freedom from both HAE attacks and the use of ongoing therapy for most patients during the six-month primary observation period. Pursuant to the Regenerative Medicine Advanced Therapy (RMAT) designation that the FDA granted to lonvo-z for the treatment of HAE, a rolling BLA allows Intellia to submit portions of the BLA on an ongoing basis and provides the FDA with an opportunity to expedite its review. In addition to the RMAT program, Intellia participated in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot. This program allows sponsors to discuss their CMC product development strategies and goals with FDA review staff and address their questions. The increased communication is intended to help sponsors complete CMC activities to expedite their drug development programs to support application submission and earlier patient access. Intellia anticipates completing its BLA submission in the second half of 2026. If the filing is accepted by the FDA, the agency is expected to determine if it will grant a priority review and provide a target action date to complete its evaluation. If approved, Intellia plans to launch lonvo-z commercially in the first half of 2027. Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an in vivo CRISPR gene editing candidate that is intended to permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose. Lonvo-z has received five notable regulatory designations: Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. There are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include lifelong therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.お知らせ • Apr 28Intellia Therapeutics Reports Positive Phase 3 Results In Hereditary AngioedemaIntellia Therapeutics, Inc. announced positive topline results from the global Phase 3 HAELO clinical trial of lonvoguran ziclumeran (lonvo-z) in hereditary angioedema (HAE). HAE is a rare genetic condition in which patients experience recurrent and potentially life-threatening swelling (angioedema) attacks in various parts of their body, including the face, upper airway, abdomen and extremities due to an overproduction of bradykinin. Designed as a one-time treatment that is administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels. Intellia separately announced that it has initiated a rolling biologics license application (BLA) submission to the FDA to seek regulatory approval. The company is preparing for a potential U.S. launch of lonvo-z in the first half of 2027. HAELO is a randomized, double-blind, placebo-controlled Phase 3 trial designed to evaluate the efficacy and safety of a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 years and older with Type I or Type II HAE. Key endpoints of the trial focused on the number of HAE attacks experienced by patients, quality of life, safety and tolerability. A total of 80 patients were enrolled, with 52 receiving lonvo-z and 28 receiving placebo. Of the total population, 49% of patients were enrolled in the United States and 71% were on long-term prophylaxis (LTP) therapy at study entry. Patients on LTP were required to discontinue those therapies in the weeks prior to dosing. The trial met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% versus placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm.Seeking Alpha • Apr 28Intellia Therapeutics And Wall Street Continue To Play Their D&D Game - Data And DoubtSummary Dungeons & Dragons (D&D) is an incredibly successful role-playing game. Today I introduce a new kind of D&D game. I call it Data & Doubt. There's a heck of a D&D match going on right now between Intellia Therapeutics (representing Team Data) and Wall Street analysts and investors (Team Doubt). This contest really heated up in the past few days. Team Data came out swinging hard, with great results in NTLA's Phase 3 trial of a drug called Lonvo-z. Team Doubt came back with a vengeance. Instead of rising, as one might have hoped, NTLA stock lost 14% on Friday, 4/24/26, and dropped another 4% on Monday, 4/27/26. I can envision Team Data making a comeback in the months ahead. But the ongoing tenacity of Team Doubt prevents me from raising my currently neutral 'Hold' rating for Intellia stock. Read the full article on Seeking Alphaお知らせ • Apr 28Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $150 million.Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $150 million. Security Name: Common Stock Security Type: Common Stockナラティブの更新 • Apr 26NTLA: Clinical Hold Resolution Will Reopen Key ATTR Phase 3 CatalystNarrative Update The analyst price target for Intellia Therapeutics is now set at $25.29. Analysts point to the FDA lifting clinical holds on the MAGNITUDE studies, the resumption of Phase 3 enrollment for nexiguran ziclumeran, and updated trial protocols as key reasons for their revised views.ナラティブの更新 • Apr 11NTLA: Clinical Hold Resolution Will Restore ATTR Phase 3 Value DriverAnalysts moved Intellia Therapeutics' consolidated price target higher toward the mid to high $20s, citing the FDA's decision to lift clinical holds on the MAGNITUDE programs for nexiguran ziclumeran and noting that the updated safety protocols support the continuation of Phase 3 enrollment. Analyst Commentary Recent Street research highlights a clear shift in sentiment around Intellia Therapeutics following the FDA decision to lift clinical holds on the MAGNITUDE programs and allow Phase 3 enrollment to resume under updated safety protocols.ナラティブの更新 • Mar 27NTLA: Clinical Hold Resolution Will Reopen ATTR Phase 3 Value OpportunityAnalysts lifted their price targets on Intellia Therapeutics toward a range of about $15 to $48 after the FDA removed clinical holds on the MAGNITUDE program, citing improved clarity on safety, updated trial protocols, and a higher assumed probability of success for nexiguran ziclumeran in ATTR indications. Analyst Commentary Research updates following the FDA's decision cluster around how much value investors should assign to nexiguran ziclumeran in transthyretin amyloidosis and how execution risk in Phase 3 might affect Intellia Therapeutics' longer term profile.ナラティブの更新 • Mar 12NTLA: Clinical Hold Resolution Will Restore Confidence In ATTR Phase 3 FranchiseAnalysts have raised their blended price target for Intellia Therapeutics from $23.86 to $25.29, citing the FDA's removal of clinical holds on MAGNITUDE studies and renewed confidence in the nex-z program as key reasons for the change. Analyst Commentary Recent research commentary on Intellia Therapeutics clusters around the FDA decision to lift clinical holds on the MAGNITUDE and MAGNITUDE 2 programs for nexiguran ziclumeran, with most firms updating price targets and ratings in response.お知らせ • Mar 03+ 1 more updateIntellia Therapeutics Presents Longer-Term Clinical Data For Lonvoguran ZiclumeranIntellia Therapeutics, Inc. announced details about its presentation of four posters at the 2026 American Academy of Allergy, Asthma & Immunology Annual Meeting (AAAAI) that took place this past weekend in Philadelphia, Pennsylvania. Poster Presentation Details: Title: Long-Term Durability and Safety of Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) 50 mg in Patients with Hereditary Angioedema Session: Allergic Skin Diseases Poster Number: 061 In this pooled Phase 1/2 analysis (n=32), a one-time 50 mg dose of lonvo-z led to deep, stable and durable reductions in plasma kallikrein across all patients with up to three years of follow-up. Across the 32 patients, the mean monthly attack rate was consistently =0.2, representing a mean reduction of 96% in HAE attacks from baseline through last follow-up. Of the 32 patients, 31 (97%) were both attack-free and LTP-free as of the data cutoff, with the attack-free and LTP-free periods ranging from 2 months to 3 years with follow-up ongoing. Title: Evolving Treatment Goals to Achieve Freedom from Attacks and Long-Term Prophylaxis Following a One-Time Treatment with Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) Session: Allergic Skin Diseases Poster Number: 005 Recent research shows that achieving an attack-free status and simultaneously minimizing treatment burden are primary treatment goals for HAE experts and patients. Of the 28 patients with >6 months of follow-up after receiving a one-time 50 mg treatment of lonvo-z in a pooled Phase 1/2 analysis, 86% were attack-free and LTP-free for >6 months, a timeframe suggested by patients to be clinically meaningful. Title: Quantitative Systems Biology Modeling Estimates Extent of Excessive Kallikrein Generation in Hereditary Angioedema Patients Session: Allergic Skin Diseases Poster Number: 003 This model suggested that HAE with significant C1-esterase inhibitor deficiency generates excess plasma kallikrein compared to healthy individuals, directly correlating with bradykinin increases. In this model, an 85% reduction in prekallikrein was shown to reduce peak free kallikrein and peak bradykin to near normal ranges. This level of reduction in prekallikrein is consistent with what has been observed clinically with a 50 mg dose of lonvo-z. Title: Chronic Medications Pose Challenges for People Living with Hereditary Angioedema Session: Bridging Evidence for Real World Impact Poster Number: 716 Among 100 surveyed U.S. patients with HAE, 89% of whom were on long-term prophylaxis therapies and 11% of whom were on on-demand therapies only, 34% reported having at least one attack per month and only 20% reported being attack free in the prior year. Most respondents indicated that eliminating lifetime chronic medication use and enhancing efficacy are the most important ways to improve their current therapy. Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy that is currently being investigated in HAELO, a Phase 3 clinical trial in HAE, and is designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency, Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission.ナラティブの更新 • Feb 25NTLA: Nex Z Clinical Return Will Ease ATTR Concerns And Support RepricingNarrative Update on Intellia Therapeutics The analyst price target for Intellia Therapeutics has been raised, with recent moves such as Baird lifting its target to $7 and H.C. Wainwright increasing its target by $10, as analysts point to progress on nex-z returning to the clinic and a broader reassessment of U.S. biopharma valuations. Analyst Commentary Recent Street research on Intellia Therapeutics reflects a mix of optimism tied to specific catalysts and caution around broader sector risks and execution.ナラティブの更新 • Feb 09NTLA: Nex Z Return To Clinic Will Offset ATTR Safety OverhangAnalysts have lifted their fair value estimate for Intellia Therapeutics by about $1.58 per share, supported by recent price target increases from firms citing potential value from a nex-z return to clinic, even as others trim or withdraw targets on safety concerns and a higher assumed future P/E of about 45.5x. Analyst Commentary Recent Street research on Intellia Therapeutics reflects a split view, with some analysts focusing on potential value creation from a nex z return to clinic and others centering on safety concerns and a narrower path to upside.新しいナラティブ • Feb 02One Time Genetic Medicines And Late Stage Pipeline Will Redefine Long Term PotentialCatalysts About Intellia Therapeutics Intellia Therapeutics develops CRISPR based genetic medicines aimed at one time treatments for serious diseases. What are the underlying business or industry changes driving this perspective?お知らせ • Jan 27Intellia Therapeutics, Inc. Announces FDA Lift of Clinical Hold on Magnitude-2 Phase 3 Clinical Trial in Attrv-PnIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug application (IND) for the MAGNITUDE-2 Phase 3 clinical trial of nexiguran ziclumeran (nex-z) for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Intellia's engagement with FDA is ongoing regarding the clinical hold on the IND for the MAGNITU DE Phase 3 clinical trial of ne xiguran ziclumer an (nex-z) for Patients with hereditary transthy Retin amyloidosis with Polyneuropathy (ATTR v-PN). Intellia' engagement with FDA is ongoing regarding The clinical hold on the IND for The MAGNITUDE Phase 3clinical trial of nex-z for patients with transthyretin Amyloidosis with cardiomyopathy (ATTR-CM). The clinical holds on the INDs for MAGNITUDE and MAGNITUDE- 2 were imposed by the FDA on October 29, 2025, following the observation of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in MAGNITUDE that met the trial's protocol-defined pausing criteria. The company has aligned with the FDA on certain study modifications and mitigation measures related to MAGNITUDE-1 that include enhanced safety monitoring of liver laboratory tests. The company is engaged with clinical trial investigators, ethics committees, international regulatory authorities and other stakeholders to resume enrollment activities in MAGNITUDE- second as quickly as possible.ex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and is being investigated in MAGNITUDE andMAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTRv-PN, respectively. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction.ex-z has received an Orphan Drug and RMAT Designation from the U.S. Food & Drug Administration (FDA) and an Orphan Drug Designation (ODD) from the European Commission. These risks and uncertainties include, but are not limited to: uncertainties related to: uncertainties related to Intellia's ability to resume the MAGNITUDE and MagNITUDE-2 trials; regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including nex -z, will not be successfully developed and commercialized; risks related to Intellia's capabilities and uncertainties, and other important factors, any of which could cause Intellia's actual results to differ from those contained in the U.S. Food, Drug Administration (FDA).ナラティブの更新 • Jan 24NTLA: Hereditary Angioedema Progress Will Offset ATTR Liver Safety ConcernsAnalysts have trimmed their price targets on Intellia Therapeutics across the board, with several cuts into the single to low double digit US$ range. They are reassessing the risk profile of the ATTR cardiomyopathy program following serious liver toxicity events and related trial pauses and holds.ナラティブの更新 • Jan 10NTLA: Hereditary Angioedema Data Will Drive Future Upside Despite Liver Safety HoldIntellia Therapeutics' updated analyst price target edges slightly lower as analysts factor in recent safety driven downgrades and target cuts, reflected in a modest reduction in fair value to about $22.27 and a slightly lower forward P/E assumption of roughly 42x. Analyst Commentary Recent Street research on Intellia Therapeutics clusters around the safety questions raised in its ATTR cardiomyopathy program and how those issues flow through to valuation, probability of approval assumptions, and perceived execution risk across the broader platform.ナラティブの更新 • Dec 25NTLA: Hereditary Angioedema Franchise Will Drive Future Upside Despite Ongoing Liver Safety OverhangAnalysts have modestly reduced their average price target on Intellia Therapeutics, trimming fair value by low single digits to around $22 per share, as mounting safety concerns and FDA holds around nexiguran ziclumeran narrow the perceived risk or reward profile despite some lingering optimism on the broader pipeline. Analyst Commentary Street research has turned more cautious on Intellia following the liver safety events in the transthyretin-mediated amyloidosis program, with multiple downgrades and target cuts reflecting a reassessment of risk, probability of approval, and the contribution of nexiguran ziclumeran to long term growth.ナラティブの更新 • Dec 10NTLA: Hereditary Angioedema Program Will Drive Future Upside Despite ATTR Safety OverhangAnalysts have modestly reduced their average price target on Intellia Therapeutics, trimming projected upside by a few dollars per share as mounting liver safety concerns around nexiguran ziclumeran, multiple downgrades, and lowered probability of approval assumptions for the ATTR program weigh more heavily against otherwise intact long term gene editing optionality and the opportunity in hereditary angioedema. Analyst Commentary Street research has turned more cautious on Intellia in the near term, with multiple downgrades and target cuts reflecting heightened uncertainty around the ATTR program and the broader platform safety profile, even as some analysts still highlight long term value in gene editing and the hereditary angioedema franchise.ナラティブの更新 • Nov 26NTLA: Positive Phase 3 Data And Upcoming Regulatory Filing Will Drive Future UpsideAnalysts have lowered their average price target on Intellia Therapeutics from approximately $24.85 to $22.43 per share. They cited heightened safety concerns related to recent clinical trial liver toxicity events and uncertainty about the future of key pipeline programs.ナラティブの更新 • Nov 12NTLA: Phase 3 Enrollment Milestones And Data Readouts Will Drive Next UpsideAnalysts have reduced their fair value estimate for Intellia Therapeutics from approximately $32.30 to $24.85. They cite heightened safety concerns in key clinical programs and lower projected growth rates following recent adverse events and subsequent trial pauses.お知らせ • Nov 11Intellia Therapeutics, Inc. Presents Positive Longer-Term Phase 1 Data of Nexiguran Ziclumeran (nex-z) in Patients with Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced positive follow-up data from the ongoing Phase 1 clinical trial of its investigational product Nexiguran ziclumeran (nex-z) in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy. In addition, the company are working diligently to address the ongoing clinical hold the FDA placed on its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials. The Phase 1 clinical trial is an open-label, two-part trial evaluating the safety and efficacy of nex-z in patients with ATTR amyloidosis with cardoomyopathy (ATTR-CM). Data presented were as of an August 23, 2025, data cut-off date. Patients dosed with nex-z continued to show evidence of disease stabilization or improvement at month 24 compared to baseline. Phase 1 Safety: Nexiguran ziclumers was generally well tolerated across all patients in the Phase 1 clinical trial. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. This investigational product is being investigated in the ongoing MAGNITUDE andMAGNITUDE- 2 Phase 3 clinical trials in ATTR-CM and ATTRv-PN, respectively, which are currently on clinical hold by the U.S. Food and Drug Administration (FDA). These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to Intellia its relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to company's product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for company's product candidates, including the ability to address the clinical hold that the United States Food and Drug Administration ("FDA") placed on the investigational new drug ("IND") applications for company's global Phase 3 MAGNITUDE study for ATTR amyloidosis With polyneuropathy ("ATTRv-PN"), and to resume those clinical trials; and its belief that greater TTR reduction may lead to greater clinical benefit. These risks and uncertainties include, but are not Limited to: risks related to: risks related to company's ability to protect and maintaining its intellectual property position; risksrelated to Intellia's relationship with third parties, including the ability of its licensors and licensees); risks related to the ability of the licensors to protect and maintaining their intellectual property position; uncertaintiesrelated to regulatory agencies' evaluation of Regulatory filings and other information related to the company's product candidates, including Nex-z; uncertainties related of the authorization, initiation and conduct the studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including risks related to the company's ability to address the clinical held that the FDA placed on the IND applications for the MAGNITUDE Phase 3 study for ATTR-CM and the MAGNITUDE- second study for ATTRv-PN and to resume those clinical trials.お知らせ • Nov 09Intellia Therapeutics Presents Positive Pooled Phase 1/2 Data of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary AngioedemaIntellia Therapeutics, Inc. presented positive clinical data from a pooled analysis of all patients who received a 50 milligram (mg) dose of lonvo-z in the company's ongoing Phase 1/2 clinical trial in patients with hereditary angioedema (HAE). These results were shared in an oral presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting in Orlando, Florida. Intellia's global Phase 1/2 clinical trial is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of lonvo-zin in adults with HAE Types I or II. Today's presentation was based on a pooled analysis of all 32 patients who have received a one-time 50 mg treatment of lonvo-z via intravenous infusion in the Phase 1/2 trial. Of the 32 patients, 15 had initially received the 50 mg dose at study Day 1 (four in Phase 1 and 11 in Phase 2) and 17 were treated after unblinding of the Phase 2 clinical trial for the primary analysis (11 had originally received a 25 mg dose of lonvo-Z, which was determined to be a suboptimal dose, and six had previously received placebo). The data cut-off for the analysis was August 29, 2025. Deep, stable and durable reductions in plasma kallikrein were observed in all patients, with a mean reduction of 89% at month 24. Among the 32 patients, 31 (97%) were both attack-free and LTP-free as of the data cutoff, with 24 (75%) being attack-free and L TP-free for at least seven months (up to 32 months for patients with the longest follow-up). Of the 11 patients who initially received the 50 mg dose of lonvo, 10 were attack-free and LTP -free (nine for 7-32 months and one for 1:2.お知らせ • Oct 30Intellia, Inc. Provides Clinical Hold on the Investigational New Drug Applications for the Magnituda-2 Phase 3 Clinical Trials for Nexigur ZiclumeranOn October 29, 2025, the United States Food and Drug Administration (the FDA") verbally informed Intellia Therapeutics, Inc. that the FDA has placed a clinical hold on the Investigational New Drug applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for nexiguran ziclumeran (nex-z). FDA indicated that it would provide a formal Clinical Hold Letter within 30 calendar days. The clinical hold follows the previously disclosed report of Grade 4 liver transaminases and increased total b birubin in a patient who was dosed with nex-z in the MAGNITUDE trial. As previously announced on October 27, 2025, the Company had temporarily paused dosing and screening in the MAGNITU DE and MAGNITUDE -2 Phase 3 clinical trials for Nex-z based on the MAGNITUDE test's protocol-defined pausing criteria. The Company intends to work with the FDA to address the clinical hold as expeditiously as possible.ナラティブの更新 • Oct 29Paused Trials Will Drive Rebound After Resolution Of Safety ConcernsIntellia Therapeutics' consensus price target has declined from approximately $34.08 to $32.30 per share. Analysts cite heightened safety concerns surrounding recent liver injury events and paused clinical trials as key drivers behind lower valuation estimates.お知らせ • Oct 27Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (Nex-z)Intellia Therapeutics, Inc. announced that the company has temporarily paused patient dosing and screening for its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials of nex-z for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively. This action follows a report on October 24, 2025 of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial on September 30, 2025, meeting the trial's protocol-defined pausing criteria. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and is currently being investigated in MAGNITUDE and MAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTR-PN, respectively. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Nex-z has received an Orphan Drug and RMAT Designation from the U.S. Food and Drug Administration (FDA) and an Orphan Drug Designation (ODD) from the European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc.お知らせ • Sep 26Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced longer-term follow-up data from the ongoing Phase 1 study of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Results were presented in an oral session on Thursday, September 25 at the 5th International ATTR Amyloidosis Annual Meeting for Patients and Doctors in Baveno, Italy. Continuation of Deep and Durable Serum TTR Reduction: Deep, durable and consistent TTR reductions continue to be observed. Across patients who received a one-time dose of 0.3 mg/kg or higher (n=33), the mean serum TTR reduction at 24 months was 92% (corresponding mean absolute serum TTR level of 17.3 g/mL [Mean 95% CI, 12.5 – 22.2]). Among the 12 patients who had reached 36 months of follow-up, the mean serum TTR reduction was 90% (corresponding mean absolute serum TTR level of 20 g/mL [Mean 95% CI, 11.2 – 28.8]). Evidence of Stability or Improvement on Clinical and Biomarker Measures: Favorable trends indicating stability or improvement were observed in most patients with ATTRv-PN after a single dose of nex-z. Stability or improvement was based on evaluation of multiple clinical and biomarker measures, including Neuropathy Impairment Score (NIS), modified Neuropathy Impairment Score +7 (mNIS+7), modified body mass index (mBMI), Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) questionnaire, neurofilament light chain (NfL), and polyneuropathy disability (PND) score. Among the 18 patients in whom a 24-month mNIS+7 assessment was completed by the data cutoff date (April 11, 2025), 13 (72%) showed improvements of a clinically meaningful threshold of =4 points, including most of the patients in the cohort who had progressed on patisiran. Among all 36 patients enrolled in the Phase 1 trial, mean values of the secondary endpoints mBMI, QoL-DN and NfL all trended toward disease improvement and 89% of patients showed improvement or stability in PND scores through 24 months compared to baseline. Safety Summary: Nex-z has been generally well tolerated as of the data cutoff date across all patients and at all dose levels tested. The most commonly reported treatment-related adverse events were infusion-related reactions, which were mild or moderate and did not result in any discontinuations. As previously reported, three participants had Grade >3 liver enzyme elevations that were not considered serious, were asymptomatic and resolved spontaneously without medical intervention or sequelae. Phase 3 MAGNITUDE-2 Trial Advancing Rapidly: Intellia began dosing patients in the Phase 3 MAGNITUDE-2 trial in April 2025. Patient screening is advancing rapidly, and enrollment completion is expected in the first half of 2026. Intellia anticipates submitting a biologics license application (BLA) for ATTRv-PN by 2028. MAGNITUDE-2 is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of nexiguran ziclumeran (nex-z) in approximately 50 patients with hereditary transthyretin ATTR amyloidosis with polyneuropathy (ATTRv-PN). The primary endpoints of the study are a change in modified neuropathy impairment score and a change in serum TTR levels. Adult patients with ATTRv-PN are randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo.お知らせ • Sep 22Intellia Therapeutics, Inc. to Present Longer-Term Data from the Ongoing Phase 1 Clinical Trial of Nexiguran Ziclumeran (Nex-Z) for the Treatment of Hereditary Transthyretin (Attr) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced that longer-term data from the ongoing Phase 1 trial of investigational nex-z for the treatment ofhereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) will be presented at the 5th International ATTR Amyloidosis Meeting for Patients and Doctors, taking place September 25-26 in Baveno, Italy. This presentation will include ATTRv-PN disease-relevant measures, including up to three years of patient follow-up. Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encourages for the transthyretin(TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of Nexiguran Ziclumeran as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc. Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin ("TTR") protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (AT TRv-PN), which can lead to vein damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. This condition, called wild-type ATTR (ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloid diagnosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.お知らせ • Sep 18Intellia Therapeutics, Inc. Completes Enrollment in the Global Phase 3 HAELO Study of Lonvoguran Ziclumeran (lonvo-z) for Hereditary AngioedemaIntellia Therapeutics, Inc. announced it has completed enrollment in the global Phase 3 HAELO study of lonvoguran ziclumeran (lonvo-z) for the treatment of hereditary angioedema (HAE). Intellia is on track to submit a biologics license application (BLA) in the second half of 2026 to support the company's plans for a U.S. launch in the first half of 2027. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to the conduct of clinical studies and other development requirements for its product candidates, including risks related to the ability to develop and commercialize any one or more of Intellia's product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related To the company's plans for aU.S. launch in the second half of 2027.分析記事 • Sep 18Is Intellia Therapeutics (NASDAQ:NTLA) In A Good Position To Deliver On Growth Plans?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...ナラティブの更新 • Aug 10Robust In Vivo CRISPR Trials Will Accelerate Therapeutic SuccessDespite a notable improvement in both future P/E and net profit margin, Intellia Therapeutics’ consensus analyst price target has been revised downward from $37.11 to $35.23. What's in the News Phase 3 HAELO trial for lonvo-z in hereditary angioedema (HAE) is ongoing, showing strong interim efficacy data; drug has received multiple regulatory designations, including FDA Orphan Drug and RMAT.お知らせ • May 29Intellia Therapeutics, Inc. Provides Business Update Regarding Its Ongoing Phase 3 StudiesIntellia Therapeutics, Inc. provided a business update regarding its ongoing Phase 3 studies. Enrollment in global Phase 3 HAELO study of NTLA-2002 for hereditary angioedema is on track. The company expects to complete enrollment in the third quarter of 2025 and submit a biologics license application ("BLA") in the second half of 2026 to support plans for a potential U.S. commercial launch in 2027. En enrollment in global Phase 3 MAGNITUDE-2 study of nex-z for ATTRv-PN is also progressing well. The MAGNITUDE- 2 study is designed to measure clinical outcomes and evaluate how a single dose of nex-z can lead to reduction in serum TTR. The company expects to complete MAGNITUDE- two hundred patients have been dosed with nex-z in the MAGNITUDE study. To date, reported adverse events have been similar in nature to the reported adverse events in the Phase 1 study of nex-z For ATTR amyloidosis, and have included infusion-related reactions and asymptomatic liver transaminase elevations. There has been a single, recent, asymptomatic patient with grade 4 liver transaminase elevations based on laboratory tests, which appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations. The company continue to monitor these events as the MAGNITUDE trial progresses.お知らせ • May 20Intellia Therapeutics, Inc. Announces Positive Two-Year Follow-Up Data from Ongoing Phase 1 Study of Nexiguran Ziclumeran (Nex-Z) in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy At Peripheral Nerve Society Annual MeetingIntellia Therapeutics, Inc. announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran Ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The pivotal Phase 3 MAGNITUDE-2 clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nexiguran ziclumeran in approximately 50 patients with hereditary transthyretin amyloidosis with polyNEuropathy (ATTRv -PN). The primary endpoints of the study are a change in modified neuropathy impairment score and a change in serum TTR levels. These forward-looking statements include, but are not limited to: risks related to Intellia's beliefs and expectations regarding: the safety, tolerability, efficacy, success and advancement of its clinical programs for Nexiguran ziclumer an or "nex-z" (also known as NTLA-2001) for transthyretin ("ATTR") amyloidosis, including the ability to successfully complete its global Phase 3 MAGNITU DE-2 study for hereditary ATTR amyloidOS with polyneuropathy ("ATTRv-PN") pursuant to its clinical trial applications and investigational new drug submissions; its belief that enrollment continues to progress well in the MAGNITUDE- 2 study; its belief that a single dose of nex-z leads to deep, durable and consistent reductions in serum TTR and that increasingly deep reductions in TTR levels leads to improved outcomes; and its expectation to be able to support the submission of a biologics license application for nex-z for the treatment of ATTRv-PN by 2028. These risks and uncertainties include, but are not limited To: risks related to IntellIA's ability to protect and maintain its intellectual property position; risks related to Intellia' relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including nex -z, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates; and risks related to Intellia its reliance on collaborations, including that its collaboration with Regeneron Pharmaceuticals, Inc. will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia's potential to cause Intellia's potential for the company's potential to be able to support a BLA submission by 2028.分析記事 • May 11Analysts Have Made A Financial Statement On Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) First-Quarter ReportA week ago, Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) came out with a strong set of first-quarter numbers that could...分析記事 • May 10Industry Analysts Just Made An Upgrade To Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Revenue ForecastsCelebrations may be in order for Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders, with the analysts delivering...お知らせ • May 01Intellia Therapeutics, Inc., Annual General Meeting, Jun 11, 2025Intellia Therapeutics, Inc., Annual General Meeting, Jun 11, 2025.お知らせ • Apr 03Intellia Therapeutics Announces First Patient Dosed in the MAGNITUDE-2 Phase 3 Study of Nexiguran Ziclumeran (nex-z), a One-Time Gene Editing-Based Treatment for Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The Phase 3 MAGNITUDE-2 study is informed by Intellia’s Phase 1 data, showing that a single dose of nex-z led to consistently rapid, deep and durable reduction in serum TTR. Intellia expects to present longer-term data from the Phase 1 studies of nex-z for both polyneuropathy and cardiomyopathy later this year. The company plans to submit a biologics licensing application (BLA) for ATTRv-PN by 2028.お知らせ • Mar 26Intellia Therapeutics, Inc. Announces FDA Regenerative Medicine Advanced Therapy Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product's biologics license application (BLA). Exex-z has been granted Regenerative Medicine Advanced Therapy designations by the U.S. FDA for both cardiomyopathy and polyneuropathy.ex-z has also been granted Orphan Drug Designation by the U.S.F and European Commission. Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin ("ATTR) amyloidOS. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. These forward-looking statements include, but are not limited to, express or implied statements regarding: the safety, efficacy, success and advancement of its clinical program for nexiguran zic Lumeran (nex-z), also known as NTLA- 2001) for the treatment of trANsthyretin (AT TR) amyloidosis with Cardiomyopathy (ATTR -CM) pursuant to its clinical trial applications and investigational new drug application, including the potential of NTLA-2001 to be a transformative treatment for patients with ATTR-CM; and the expected timing of regulatory filings, completion of clinical trials, submission of marketing authorizations, and commercialization. These risks and uncertainties include, but are not limited To: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia' relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; and uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including nex-z; risks related to the results of preclinical or clinical studies, including that they may not be positive or predictive of future results; the risk that one or more of Intellia's product candidates, including nex -z, will not be successfully developed and commercialized; and risks related toIntellia's reliance on collaborations, including that its collaboration with Regeneron will not continue or will not be successful.新しいナラティブ • Mar 25Phase III Trials And Gene Editing Will Bring Promising Therapies Strategic discontinuation of less promising programs and capital prioritization are expected to improve net margins and enhance commercial prospects. Seeking Alpha • Mar 02Intellia Trades Lower After Q4 Results As Clinical Progress Fails To ImpressSummary Intellia Therapeutics' stock is downgraded to HOLD due to market skepticism and lack of near-term catalysts despite promising Phase 3 trials for HAE and ATTR. NTLA-2002, Intellia's CRISPR/Cas9 drug for HAE, is the closest to commercialization, expected by 2027, but profitability remains distant. Intellia's financials show a cash runway extended through 2027, but additional funding or partnerships will likely be needed for profitability. Gene therapy sector uncertainty, exemplified by bluebird bio's struggles, underscores the risks and potential rewards for Intellia investors willing to wait. Read the full article on Seeking Alpha分析記事 • Mar 02Earnings Release: Here's Why Analysts Cut Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Price Target To US$46.08Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its yearly results, which were ahead...分析記事 • Feb 15Will Intellia Therapeutics (NASDAQ:NTLA) Spend Its Cash Wisely?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...お知らせ • Feb 14Bronstein, Gewirtz & Grossman, LLC Files Class Action Lawsuit Against Intellia Therapeutics, IncBronstein, Gewirtz & Grossman, LLC notified investors that a class action lawsuit has been filed against Intellia Therapeutics, Inc. and certain of its officers. This lawsuit seeks to recover damages against Defendants for alleged violations of the federal securities laws on behalf of all persons and entities that purchased or otherwise acquired Intellia securities between July 30, 2024 and January 8, 2025, both dates inclusive (the “Class Period”). The Complaint alleges that throughout the Class Period, Defendants made false and/or misleading statements and/or failed to disclose that: (1) defendants created the false impression that they possessed reliable information pertaining to the viability of NTLA-3001’s development and eventual marketing, if approved; (2) Intellia’s optimistic reports of timelines, including dosing and future studies of the drug, fell short of reality; the NTLA program was not viable or sustainable for Intellia because viral-based editing programs remained expensive and inefficient in comparison to then-existing non-viral delivery methods; (3) Intellia was not equipped to timely dose patients with NTLA-3001, maintain the drug’s research and development, or even to maintain its full staff in light of the existing scientific landscape surrounding viral-based editing drugs; and (4) even if NTLA-3001 proved successful, the use of viral-based editing drugs is costly, inefficient, and poor mitigators of adverse effects in patients.お知らせ • Jan 22Intellia Therapeutics, Inc. Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary AngioedemaIntellia Therapeutics, Inc. announced the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based therapy in development as a single-dose treatment for this potentially life-threatening disease. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the Company’s plans for a U.S. launch in 2027. The pivotal Phase 3 HAELO clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. Key endpoints include the number of HAE attacks and the number of patients who achieve attack-free status from week 5 through week 28. Based on Nobel-prize winning CRISPR/Cas9 technology, NTLA-2002 has the potential to become the first one-time treatment for hereditary angioedema (HAE). NTLA-2002 is designed to prevent HAE attacks by inactivating the kallikrein B1(KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission.Seeking Alpha • Jan 13Intellia Therapeutics: Buying The Path To CommercializationSummary Intellia Therapeutics, Inc. is undergoing strategic reorganization, halting NTLA-3001, and reducing workforce by 27% to extend cash runway into 2027, focusing on late-stage programs NTLA-2002 and Nexiguran Ziclumeran. Despite industry headwinds and significant layoffs, I see Intellia's cost-cutting measures as prudent, aiming for commercialization of NTLA-2001 and NTLA-2002 in large, promising markets. The gene therapy sector faces turmoil, with multiple companies making tough decisions; however, Intellia's focus on late-stage programs may position it for future growth and potential M&A interest. I initiated a speculative buy on NTLA under $10 per share, anticipating a potential technical rebound and long-term investment opportunities as the company approaches commercialization. Read the full article on Seeking Alphaお知らせ • Nov 26Intellia Therapeutics, Inc. Announces FDA Regenerative Medicine Advanced Therapy Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo CRISPR-based investigational therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of ATTR amyloidosis. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron. The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA). This RMAT designation is the third special regulatory designation received by Intellia for nex-z. Nex-z was also granted Orphan Drug Designation by the U.S. FDA and European Union Orphan Drug Designation by the European Commission. nexiguran ziclumeran (nex-z, also known as NTLA-2001): Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. Transthyretin (ATTR) Amyloidosis: Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.Seeking Alpha • Nov 19Intellia Therapeutics' Gene Therapies Fall Short Of Breakthroughs (Rating Downgrade)Summary Intellia initiated the Phase 3 HAELO trial for NTLA-2002, targeting lifelong control of hereditary angioedema (HAE) with a single-dose CRISPR-based therapy. Positive Phase 2 NTLA-2002 data showed most patients remained attack-free; however, limitations like small sample size and short follow-up require cautious interpretation. Phase 1 data for nexiguran ziclumeran (nex-z) in ATTR amyloidosis showed disease stabilization, but competitive therapies demonstrate more robust improvements. Intellia has strong financial health, with over $900M in liquidity and a cash runway expected to last until late 2026. Intellia's limited transformational data, high manufacturing costs, and competitive landscape justify downgrading the stock from "Buy" to "Hold." Read the full article on Seeking Alphaお知らせ • Nov 17Intellia Therapeutics, Inc. Announces First Clinical Evidence from Ongoing Phase 1 Study That Nexiguran Ziclumeran (nex-z) May Favorably Impact Disease Progression in Transthyretin (ATTR) AmyloidosisIntellia Therapeutics, Inc. announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. These results from the ongoing Phase 1 study increase the belief in the likelihood of success of active Phase 3 studies based on hypothesis that greater TTR reduction may lead to greater clinical benefit in ATTR amyloidosis. Rapid, Deep and Durable Serum TTR Reduction: Across all patients (n=36), a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction, regardless of baseline levels, through the latest follow-up. Evidence of Disease Modification on Clinical Measures: F favorable trends indicating stability or improvement were observed in patients with ATTRv-PN based on evaluation of multiple clinical measures, including Neuropathy Impairment Score (NIS), modified Neuropathy Impairment Score ("mNIS+7) and modified BMI (mBMI). The clinical measure results are detailed in the table below. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to the relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including ability to initiate or enroll the Phase 3 MAGNITUDE study for ATTRv-PN or "nex-z" (f/k/a NTLA-2001), for transthyretin ("ATTR") amyloidosis.Seeking Alpha • Nov 11Intellia Therapeutics: Q3 Earnings Report Puts Phase II Results In FocusSummary Intellia Therapeutics' NTLA-2002 Phase II results last month were underwhelming, however, management reassured investors on the Q3 earnings call setting the stage for a modest rally. While last month's drop is appropriately discounting NTLA-2002, investors should be encouraged that 3 Phase III trials will be engaged by year-end, representing multiple opportunities for substantial upside. Intellia's financial position is robust with $945 million in cash, enough to fund operations into late 2026. NTLA-2002's Phase III study will enroll 60 patients and is anticipated to reach its primary endpoint by April 2026, potentially offering a functional cure for hereditary angioedema (HAE). HAE represents a huge market opportunity estimated to reach $17.4 billion by 2032. Read the full article on Seeking AlphaSeeking Alpha • Nov 05Data Underwhelms - Intellia's Cash, Data And Competition Are A Challenging StorySummary ATTR has become a more competitive space, with uncertainty ahead on Phase III timelines. Recent data on HAE did not provide a differentiated outcome from more advanced competitors. The competitive landscape across lead indications is evolving rapidly and may create unforeseen challenges to trial completion or commercialization. Funding and regular share offerings are diminishing flexibility and potential return for investors. 2026 cash runway may be insufficient to obtain meaningful Phase III clinical readouts. Read the full article on Seeking Alphaお知らせ • Oct 25Intellia Therapeutics, Inc. Presents Positive Results from the Phase 2 Study of NTLA-2002Intellia Therapeutics, Inc. announced positive Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE), with results continuing to indicate that NTLA-2002 has the potential to eliminate HAE attacks following a one-time infusion. NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for HAE, a rare genetic condition that leads to potentially life-threatening swelling attacks. Results were published online on October 24, 2024 in The New England Journal of Medicine and will be presented on October 26 at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting in Boston, Massachusetts. The Phase 2 study is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of NTLA-2002. A total of 27 participants were enrolled and randomized to receive one of two single doses of NTLA-2002 (25 mg or 50 mg) or placebo via intravenous infusion. The data cut-off date for the analysis was April 4, 2024, when the 25th patient completed the 16-week primary observation period. Single dose of NTLA-2002 led to deep attack rate reductions during the primary observation period. The mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1 – 16, and by 80% and 81% during weeks 5 – 16, respectively. In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months) and no subsequent treatment has been required. In contrast, four of the 10 patients in the 25 mg arm experienced a complete response and zero patients in the placebo arm. Similarly, patients who received the 50 mg dose achieved a greater kallikrein protein reduction, with an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16. At both dose levels, NTLA-2002 was well tolerated. The most frequent adverse events (AEs) were headache, fatigue and nasopharyngitis. There have been no serious AEs and all AEs were either Grade 1 or 2, except for one patient in the placebo arm who experienced a serious AE of Grade 4 edema of the tongue with breathing impairment that was attributed to their underlying HAE. No clinically significant laboratory abnormalities were observed. The safety, tolerability and efficacy data from the Phase 2 study are consistent with the long-term Phase 1 data presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in Valencia, Spain on June 2, 2024. Based on these results, Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, which is actively screening patients.Seeking Alpha • Oct 25Intellia Therapeutics: Lower Stock Price On HAE Data Presents A Buying OpportunitySummary Intellia Therapeutics, Inc. achieved positive phase 1/2 results using NTLA-2002 for HAE patients: 8 out of 11 patients taking higher doses of 50 mg of NTLA-2002 were attack-free during the 16-week primary observation period. Despite the stock price trading lower by 20%, the HAE treatment program continues on using NTLA-2002 in the phase 3 HAELO study. The global hereditary angioedema treatment market size is projected to reach $17.31 billion by the end of 2032. NTLA-2001 is another in vivo CRISPR drug being developed to target patients with transthyretin [ATTR] amyloidosis being explored in the phase 3 MAGNITUDE study; Updated phase 1 data is expected 2nd half of 2024. Read the full article on Seeking Alphaお知らせ • Oct 07Intellia Therapeutics, Inc. Announces Initiation of Haelo Phase 3 Study of Ntla-2002Intellia Therapeutics, Inc. announced the initiation of HAELO, a global, pivotal Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for this potentially life-threatening disease. Patient screening is active following Intellia's successful end-of-Phase 2 meeting and submission of an Investigational New Drug Application amendment to the U.S. Food and Drug Administration (FDA). HAELO is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. The primary endpoint is the change in number of HAE attacks from week 5 through week 28. Intellia is initiating the Phase 3 study based on positive safety and efficacy data from the ongoing Phase 1/2 study (NCT05120830) of NTLA-2002. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Intellia previously announced positive toplines results from the Phase 2 portion of the study. The Company plans to present the detailed results at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.Seeking Alpha • Sep 25Intellia - Exciting Promise Of First In-Vivo Gene Therapy Makes Bull CaseSummary Intellia Therapeutics is pioneering in-vivo CRISPR gene editing, aiming for groundbreaking therapies, with NTLA-2001 showing promising results in ATTR amyloidosis, potentially achieving blockbuster status. Despite a 65% stock drop since 2022, Intellia's advancements in Phase 3 trials and strong partnerships, like with Regeneron, make it a compelling buy opportunity. The company's innovative use of lipid nanoparticles for gene delivery and a robust pipeline position it as a leader in the CRISPR space, targeting large markets. With a cash position of $940m and multiple Phase 3 studies in play, Intellia is poised for substantial valuation growth and a possible Big Pharma acquisition. The major risks to the thesis are a safety setback or a failure to deliver on approval-worthy efficacy endpoints, but this is mitigated somewhat by the success of e.g. Crispr Therapeutics / Vertex very effective sickle cell disease therapy Casgevy. Read the full article on Seeking Alpha分析記事 • Sep 07We're Keeping An Eye On Intellia Therapeutics' (NASDAQ:NTLA) Cash Burn RateThere's no doubt that money can be made by owning shares of unprofitable businesses. For example, although Amazon.com...分析記事 • Aug 11Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportThe analysts might have been a bit too bullish on Intellia Therapeutics, Inc. ( NASDAQ:NTLA ), given that the company...Seeking Alpha • Aug 03Intellia Therapeutics Is Expanding The Scope Of Gene Editing (Technical Analysis)Summary The future is here… The CRISPR-Cas9 gene editing platform lets us reshuffle DNA, analogous to how we edit text. The difference is that CRISPR-Cas9 is a much more powerful tool that we’re slowly learning to use. It’s important that we master it… gene editing is the best treatment for many serious diseases. Intellia Therapeutics is blazing new trails in using CRISPR-Cas9 to treat more conditions. But we can’t compute any sort of valuation for the stock, so it’s only suitable as a sentiment play. Read the full article on Seeking Alphaお知らせ • Jul 30Intellia Therapeutics, Inc. Receives Authorization to Initiate Phase 1/2 Clinical Trial of Ntla-3001 for the Treatment of Alpha-1 Antitrypsin DeficiencyIntellia Therapeutics, Inc. announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom's Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. AATD is a rare, genetic disease that most commonly manifests in lung dysfunction due to insufficient levels of alpha- 1 antitrypsin (AAT) protein. NTLA-3001 is a systemically administered in vivo CRISPR/Cas9-based targeted gene insertion candidate. The study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-3001. Beyond its first application in the United Kingdom, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development strategy for NTLA-3001.お知らせ • Jul 09Blueallele Corporation Files Lawsuit for Patent Infringement Against Intellia Therapeutics, Inc., for the Unauthorized Use of Blueallele’S Patented Bi-Directional Insertion TemplatesBlueAllele Corporation filed a patent infringement lawsuit against Intellia Therapeutics in the United States District Court for the District of Delaware. The complaint alleges infringement of BlueAllele’s bi-directional insertion template patents, which cover the company’s transformational platform used in the development and treatment of genetic diseases. The novel design of BlueAllele’s bi-directional insertion template technology has unlocked the full potential of the cell’s highly active NHEJ pathway. Use of the patented bi-directional insertion template technology can increase the efficacy of gene editing events, improve safety, and allow development of therapeutics for a wide range of diseases. As alleged in the complaint, Intellia has utilized BlueAllele’s patented platform technology to advance internal programs as well as external collaboration programs. BlueAllele has expanded upon its bi-directional insertion template technology with the design of additional platforms for treating specific classes of genetic diseases, including bi-directional templates with RNAi cassettes for autosomal dominant diseases and functions to remove unwanted gene products while simultaneously restoring normal protein function. Further, BlueAllele has developed bi-directional templates with dual terminators designed to address repeat expansion diseases and functions to prevent transcription through the expansion. BlueAllele Corporation is being represented in the lawsuit by Fish & Richardson P.C. and Schwartz Law Firm.お知らせ • Jun 27Intellia Therapeutics, Inc. Announces Chief Financial Officer ChangesIntellia Therapeutics, Inc. announced the appointment of Edward Dulac as Chief Financial Officer (CFO), effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. Mr. Dulac is a highly accomplished biotechnology business leader and joins Intellia with more than 20 years of combined finance, business development and corporate strategy experience. Most recently, Mr. Dulac served as CFO of Fate Therapeutics. Prior to that role, Mr. Dulac spent numerous years at Celgene (now Bristol Myers Squibb), where he held multiple positions including as Vice President, Business Development & Strategy. Prior to Celgene, Mr. Dulac worked as a biopharmaceutical equity research analyst at Barclays Capital and Lehman Brothers and in corporate finance at Pfizer. Mr. Dulac holds a Bachelor of Pharmacy from the University of Pittsburgh and an MBA from Indiana University, Kelley School of Business.お知らせ • Jun 26+ 1 more updateIntellia Therapeutics, Inc. Announces Executive ChangesIntellia Therapeutics, Inc. announced the appointment of Edward Dulac as Executive Vice President and Treasurer, effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. Mr. Dulac is a highly accomplished biotechnology business leader and joins Intellia with more than 20 years of combined finance, business development and corporate strategy experience. Most recently, Mr. Dulac served as CFO of Fate Therapeutics. Prior to that role, Mr. Dulac spent numerous years at Celgene (now Bristol Myers Squibb), where he held multiple positions including as Vice President, Business Development & Strategy. Prior to Celgene, Mr. Dulac worked as a biopharmaceutical equity research analyst at Barclays Capital and Lehman Brothers and in corporate finance at Pfizer. Mr. Dulac holds a Bachelor of Pharmacy from the University of Pittsburgh and an MBA from Indiana University, Kelley School of Business.お知らせ • Jun 18Intellia Therapeutics to Present the First-Ever Clinical Data from Patients Redosed with an Investigational in Vivo CRISPR Gene Editing Therapy At the Peripheral Nerve Society Annual Meeting 2024Intellia Therapeutics, Inc., a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, announced the acceptance of an abstract featuring redosing data from the Phase 1 study of NTLA-2001 has been selected for an oral presentation at the Peripheral Nerve Society Annual Meeting, taking place June 22 – 25 in Montreal, Canada. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis. In the dose-escalation portion of the Phase 1 study, the initial three patients received the lowest dose of 0.1 mg/kg and subsequently received a follow-on dose of 55 mg. These data will be the first-ever clinical data from patients redosed with an in vivo CRISPR-based gene editing candidate and provide insight on the safety and pharmacodynamic effect. While repeat dosing is not planned for the NTLA-2001 program for ATTR amyloidosis, a redosing option could be an important advantage of Intellia’s non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired.お知らせ • Jun 15Intellia Therapeutics, Inc. Names Brian Goff to its Board of DirectorsIntellia Therapeutics, Inc. announced the appointment of Brian Goff to its board of directors. Mr. Goff joins the Intellia board of directors with over three decades of commercialization, operations and sales and marketing experience at leading biopharmaceutical companies. He is a seasoned and accomplished executive global leader focused on rare diseases. Since 2022, he has been the chief executive officer of Agios Pharmaceuticals and a member of its board of directors. He previously served as executive vice president, chief commercial and global operations officer of Alexion Pharmaceuticals until its acquisition by AstraZeneca in 2021. Prior to Alexion, Mr. Goff was chief operating officer and a member of the board of directors of Neurovance until its acquisition by Otuska Pharmaceuticals. Before joining Neurovance, Mr. Goff served as executive vice president and president of the Hematology Division at Baxalta until its acquisition by Shire. He previously served with Baxter Healthcare Corporation as global hemophilia franchise head. Earlier in his career, Mr. Goff held positions of increasing seniority in sales and marketing with Novartis Pharmaceuticals and the pharmaceutical division of Johnson & Johnson. Mr. Goff holds a bachelor’s degree from Skidmore College and a Master of Business Administration from the Wharton School at the University of Pennsylvania.分析記事 • Jun 06Here's Why It's Unlikely That Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) CEO Will See A Pay Rise This YearKey Insights Intellia Therapeutics will host its Annual General Meeting on 12th of June Total pay for CEO John Leonard...Seeking Alpha • Jun 05Intellia Therapeutics Presents Promising In Vivo CRISPR ResultsSummary Intellia Therapeutics has released positive data from its Phase I/II study of NTLA-2002, a CRISPR-based gene editing therapy. The study shows promising results for in vivo gene editing, offering potential treatments for genetic diseases. Robust target engagement and efficacy data pave the way for future Phase II results from Intellia later this year. Intellia has the most advanced in vivo gene editing portfolio, which, while speculative, could represent a huge advance for the field in coming years. Read the full article on Seeking Alphaお知らせ • Jun 05Intellia Therapeutics, Inc. Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002Intellia Therapeutics, Inc. announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. As previously announced, Intellia completed enrollment of the randomized, placebo-controlled Phase 2 study further evaluating the 25 mg and 50 mg doses and plans to report topline results mid-year with detailed results expected to be presented at a medical meeting later in the year. Intellia's ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates including NTLA-2002; the risk that the results of preclinical studies or clinical studies, such as the ongoing Phase 1/2 clinical study of NTLA-2002, will not be predictive of future results in connection with this study or future studies for NTLA-2002 or Intellia's other product candidates. For a discussion of these and other important factors, any of which could cause Intellia's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in Intellia's most recent Phase 1/2 clinical study.分析記事 • May 14Industry Analysts Just Made A Dazzling Upgrade To Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Revenue ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders will have a reason to smile today, with the analysts making...Seeking Alpha • May 13Intellia Therapeutics: 3 Near-Term Pivotal In Vivo Gene TherapiesSummary Intellia's longstanding collaboration with Regeneron is important in its value proposition. With three cutting-edge late-stage programs, NTLA is among the top tier of gene editing companies. Solid financials underpin the Company. Intellia presents special risks. Read the full article on Seeking AlphaReported Earnings • May 10First quarter 2024 earnings released: US$1.13 loss per share (vs US$1.18 loss in 1Q 2023)First quarter 2024 results: US$1.13 loss per share. Revenue: US$28.9m (up 130% from 1Q 2023). Net loss: US$107.4m (loss widened 4.2% from 1Q 2023). Revenue is forecast to grow 60% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in the US.お知らせ • May 03Intellia Therapeutics, Inc. to Report Q1, 2024 Results on May 09, 2024Intellia Therapeutics, Inc. announced that they will report Q1, 2024 results on May 09, 2024お知らせ • May 01Intellia Therapeutics, Inc., Annual General Meeting, Jun 12, 2024Intellia Therapeutics, Inc., Annual General Meeting, Jun 12, 2024, at 09:00 US Eastern Standard Time. Agenda: To consider Election of two class II directors to board of directors, each to serve until the 2027 annual meeting of stockholders and until their successor has been duly elected and qualified; to Ratification of the appointment of Deloitte & Touche LLP as independent registered public accounting firm for the fiscal year ending December 31, 2024; to Approval, on a non-binding advisory basis, of the compensation of named executive officers; to Approval of a second amendment to Second Amended and Restated Certificate of Incorporation, as amended, to limit the liability of certain officers of the Company as permitted by recent amendments to the Delaware General Corporation Law; and to Transaction of any other business properly brought before the Annual Meeting or any adjournment or postponement thereof.お知らせ • Mar 19Intellia Therapeutics, Inc. Announces First Patient Dosed in Phase 3 MAGNITUDE Study of NTLA-2001 as Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced the first patient dosed in the global pivotal, Phase 3 MAGNITUDE trial of NTLA-2001. NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis. The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy. The pivotal Phase 3 MAGNITUDE clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2001 in approximately 765 patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The primary endpoint of the study is a composite endpoint of cardiovascular (CV)-related mortality and CV-related events. Adult patients with hereditary or wild type ATTR-CM will be randomized 2:1 to receive a single 55 mg infusion of NTLA-2001 or placebo.Seeking Alpha • Feb 29Intellia Therapeutics Is On The MoveSummary Today, we circle back on gene editing concern Intellia Therapeutics, Inc., whose stock has moved up by a third over the past month. The company has potential cures for HAE and ATTR-CM within its pipeline, but commercialization is years away. An updated analysis around Intellia Therapeutics follows in the paragraphs below. Read the full article on Seeking Alphaお知らせ • Feb 24Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $375.727167 million.Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $375.727167 million. Security Name: Common Stock Security Type: Common Stock Transaction Features: At the Market Offering分析記事 • Feb 24Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders are probably feeling a little disappointed, since its shares...Reported Earnings • Feb 24Full year 2023 earnings: EPS in line with expectations, revenues disappointFull year 2023 results: US$5.42 loss per share. Revenue: US$36.3m (down 30% from FY 2022). Net loss: US$481.2m (loss widened 1.5% from FY 2022). Revenue missed analyst estimates by 28%. Earnings per share (EPS) were mostly in line with analyst estimates. Revenue is forecast to grow 57% p.a. on average during the next 3 years, compared to a 17% growth forecast for the Biotechs industry in the US.New Risk • Feb 23New major risk - Revenue and earnings growthEarnings are forecast to decline by an average of 0.6% per year for the foreseeable future. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are expected to decline, then in most cases the share price will decline over time as well. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 0.6% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$554m net loss in 3 years). Significant insider selling over the past 3 months (US$1.4m sold).お知らせ • Feb 16Intellia Therapeutics, Inc. to Report Q4, 2023 Results on Feb 22, 2024Intellia Therapeutics, Inc. announced that they will report Q4, 2023 results on Feb 22, 2024Seeking Alpha • Feb 09Intellia's Bet On In-Vivo Breakthroughs: A Perspective On CRISPR's Next FrontierSummary Intellia is adopting a distinct strategy by focusing on in-vivo gene editing, potentially redefining industry standards and expectations. Interim results from Intellia's in-vivo gene-editing program for ATTR amyloidosis show promising safety and efficacy profiles. Intellia is still a cash-burning company, while any commercial therapy is still some time away. Read the full article on Seeking Alpha分析記事 • Feb 07Is Intellia Therapeutics (NASDAQ:NTLA) In A Good Position To Deliver On Growth Plans?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...お知らせ • Feb 01Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of MedicineIntellia Therapeutics, Inc. announced that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks. Additionally, the company remain on track to initiate a global pivotal study for NTLA-2002 in the second half of 2024, subject to regulatory feedback. This marks the second consecutive Intellia in vivo CRISPR- based program to have its initial clinical data published in the New England Journal of medicine, further supporting the immense potential impact its proprietary gene editing platform could have on the future of human health. The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. Intellia's ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that NTLA-2002 will not be successfully developed and commercialized; and the risk that the results of preclinical stu dies or clinical studies, such as the clinical study of NTLA-2002, will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates. For a discussion of these and other risks and uncertainties, and other risks and uncertainties, and other risks and uncertainties.Recent Insider Transactions • Jan 07President recently sold US$566k worth of stockOn the 3rd of January, John Leonard sold around 19k shares on-market at roughly US$29.46 per share. This transaction amounted to 2.1% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. This was John's only on-market trade for the last 12 months.分析記事 • Jan 01Is There An Opportunity With Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) 25% Undervaluation?Key Insights The projected fair value for Intellia Therapeutics is US$40.78 based on 2 Stage Free Cash Flow to Equity...Recent Insider Transactions Derivative • Dec 10Independent Director notifies of intention to sell stockJohn Crowley intends to sell 33k shares in the next 90 days after lodging an Intent To Sell Form on the 6th of December. If the sale is conducted around the recent share price of US$29.86, it would amount to US$979k. Since March 2023, John's direct individual holding has increased from 2.09k shares to 7.29k. Company insiders have collectively sold US$940k more than they bought, via options and on-market transactions in the last 12 months.お知らせ • Dec 06Intellia Therapeutics, Inc. Announces Resignation of John F. Crowley from the Board of Directors of Company and Its CommitteesIntellia Therapeutics, Inc. announced that John F. Crowley notified company of his intent to resign from the Board of Directors of the Company and its committees, effective December 5, 2023. Mr. Crowley currently serves on the Nominating and Governance Committee and on the Compensation and Talent Development Committee of the Board. His resignation is not the result of any disagreement with the Company on any matter relating to the Company’s operations, policies or practices. The Company thanks Mr. Crowley for his long, dedicated service on the Board and wishes him well in his future pursuits.Reported Earnings • Nov 10Third quarter 2023 earnings: EPS exceeds analyst expectationsThird quarter 2023 results: US$1.38 loss per share. Revenue: US$12.0m (down 9.6% from 3Q 2022). Net loss: US$122.2m (loss widened 7.9% from 3Q 2022). Revenue was in line with analyst estimates. Earnings per share (EPS) surpassed analyst estimates by 8.1%. Revenue is forecast to grow 60% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in the US.お知らせ • Nov 03Intellia Therapeutics, Inc. Presents New Interim Data from the Ongoing Phase 1 Study of NTLA-2001 At the 4th International ATTR Amyloidosis MeetingIntellia Therapeutics, Inc. presented additional interim results from its ongoing Phase 1 study of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain. In the newly reported dose-expansion portion, administration of NTLA-2001 at the 55 mg and 80 mg dose led to deep serum TTR reductions consistent with the results previously reported [1] from patients in the dose-escalation portion who received the corresponding weight-based dose, 0.7 mg/kg and 1.0 mg/kg, respectively. Across all patients who received a dose of 0.3 mg/kg or higher (n=62), the median serum TTR reduction was 91% and the median absolute residual serum TTR concentration was 17 µg/mL at day 28. The persistently low levels of TTR concentration are expected to reduce the rate of ongoing amyloid formation and hold the possibility for amyloid clearance to reverse the symptoms of the disease. If clinically validated, the use of absolute residual TTR concentration levels could become a new benchmark for evaluating ATTR amyloidosis. The reduction of serum TTR compared to baseline was sustained through the latest follow-up. With 29 patients now reaching at least 12 months of follow-up, all patients continued to show a long-lasting response with no evidence of loss in activity over time. NTLA-2001 was generally well tolerated across all patients and at all dose levels tested. The most commonly reported adverse events were infusion-related reactions, which occurred in 38% of patients. The majority of adverse events, including infusion-related reactions, were Grade 1 or 2 in severity, transient and resolved spontaneously. Other adverse events that were reported in greater than 10% of patients included headache, diarrhea and back pain, and were all Grade 1 or 2. All patients received a full dose of NTLA-2001 and remain on study. No dose-limiting toxicities were observed. Based on the safety and activity of NTLA-2001, the 55mg dose has now been selected to be evaluated in the upcoming pivotal Phase 3 study. These data support NTLA-2001’s continued development as a potential one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein in people living with ATTR amyloidosis. As previously announced [2], Intellia recently received IND clearance from the FDA to begin a Phase 3 trial of NTLA-2001 for ATTR-CM and expects to initiate the global pivotal trial by the end of this year. Additionally, the Company is actively preparing for a Phase 3 trial for ATTRv-PN.Board Change • Nov 01Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 8 experienced directors. 4 highly experienced directors. Independent Director John Crowley was the last director to join the board, commencing their role in 2020. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.分析記事 • Oct 20Intellia Therapeutics (NASDAQ:NTLA) Is In A Good Position To Deliver On Growth PlansEven when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...お知らせ • Oct 19Intellia Therapeutics Announces FDA Clearance of Investigational New Drug Application to Initiate a Pivotal Phase 3 Trial of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy. The global Phase 3 study of NTLA-2001, an in vivo CRISPR-based gene editing candidate, is expected to initiate by year-end 2023. Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical and clinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The trial is now closed for enrollment.New Risk • Aug 06New major risk - Revenue and earnings growthEarnings have declined by 41% per year over the past 5 years. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are declining over an extended period, then in most cases the share price will decline over time unless the company can turn around its fortunes. A trend of falling earnings can be very difficult to turn around. If the company is well already established it may also be a sign the company has matured and is in decline. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risk Earnings have declined by 41% per year over the past 5 years. Minor Risk Shareholders have been diluted in the past year (16% increase in shares outstanding).Reported Earnings • Aug 03Second quarter 2023 earnings: Revenues exceed analysts expectations while EPS lags behindSecond quarter 2023 results: US$1.40 loss per share (further deteriorated from US$1.33 loss in 2Q 2022). Revenue: US$13.6m (down 3.1% from 2Q 2022). Net loss: US$123.7m (loss widened 23% from 2Q 2022). Revenue exceeded analyst estimates by 20%. Earnings per share (EPS) missed analyst estimates by 7.1%. Revenue is forecast to grow 59% p.a. on average during the next 3 years, compared to a 16% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 36% per year but the company’s share price has increased by 25% per year, which means it is well ahead of earnings.お知らせ • Jul 22Intellia Therapeutics, Inc. Appoints Eliana Clark, Phd, Executive Vice President and Chief Technical OfficerCulture Biosciences welcomed Eliana Clark, PhD, Executive Vice President and Chief Technical Officer at Intellia Therapeutics, Inc., to the company’s Advisory Board. The group of seasoned industry leaders provides Culture’s leadership with insights and recommendations as the company establishes new partnerships and pursues new growth opportunities in biotech and biopharma. Prior to her current role at Intellia, Eliana Clark was the company’s Senior Vice President, Technical Operations and Quality. Before she joined Intellia, Eliana held leading roles in Product and Portfolio Management, Manufacturing, Manufacturing Sciences, Process Development and CMC-Regulatory, at Biogen and Sanofi/Genzyme, respectively. Prior to joining industry, she was a professor at Tufts University in the Chemical and Biological Engineering Department. Eliana held post-doc roles in Biochemical Engineering at the University of Delaware and the Worcester Polytechnic Institute. She has a PhD and a Bachelor of Engineering degree in Chemical Engineering from the Universidad Nacional del Litoral in Santa Fe, Argentina, and has graduated from the Greater Boston Executive Program at MIT Sloan.お知らせ • Jun 12Intellia Therapeutics, Inc. Announces New Positive Clinical Data from Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema (HAE)Intellia Therapeutics, Inc. announced updated interim results from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. NTLA-2002 is an in vivo, systemically administered CRISPR candidate being developed as a single-dose treatment for hereditary angioedema (HAE). The data, with a cut-off date of February 17, 2023, were shared in a late-breaking presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023, being held June 9-11 in Hamburg, Germany, and virtually. In the Phase 1 portion of the study, single doses of 25 mg (n=3), 50 mg (n=4) and 75 mg (n=3) of NTLA-2002 were administered via intravenous infusion, and HAE attacks and plasma kallikrein protein levels were measured for each patient. The first analysis of HAE attack rates occurred at the end of the pre-specified 16-week primary observation period. HAE attacks and plasma kallikrein protein levels will continue to be assessed through the end of the study. Across all patients, a 95% mean reduction in monthly attack rate was observed after a single dose of NTLA-2002 through the latest follow-up. The median duration of follow-up was 9.0 months (range of 5.6 - 14.1 months). At each dose level tested, a robust level of HAE attack rate reduction was achieved. Importantly, the elimination of HAE attacks has been sustained and long lasting. The first three patients dosed in the study with the longest follow-up to date have experienced attack-free durations of approximately one year or longer. Additionally, the reduction in HAE attacks has been persistent in patients with the most severe HAE symptoms. The three patients with the highest historic monthly HAE attack rates at the start of the study (16.8, 14.0 and 4.4 attacks per month, respectively) all became attack free by the end of the 16-week primary observation period and remained free of attacks through the latest follow-up. The longest attack-free duration in this patient group is 11.5 months and ongoing. All nine patients who achieved greater than 60% plasma kallikrein reduction, the target level expected to yield a highly meaningful clinical response, remain completely attack free since the 16-week observation period. There was one patient in the lowest 25 mg dose cohort who did not achieve the targeted 60% minimum kallikrein reduction post-NTLA-2002 administration. Following 12.3 months of being attack free, this patient reported a single, mild HAE attack after experiencing minor hand swelling precipitated by a sports injury. The event did not require any medical intervention or acute therapy. The patient has not experienced any subsequent HAE attacks following this event. Six of the 10 patients were receiving long-term HAE prophylaxis medications prior to the administration of NTLA-2002. Subsequently, they were permitted to withdraw their medication at the investigator’s discretion. All six patients have discontinued their prophylactic therapy and have not experienced any subsequent HAE attacks. As previously reported, administration of NTLA-2002 led to dose-dependent, robust and durable reductions in plasma kallikrein. These deep reductions in plasma kallikrein continue to be sustained through the latest follow-up, which ranged from 24 to 48 weeks across all three dose cohorts. At all three dose levels, NTLA-2002 has been well tolerated, and the majority of adverse events were mild in severity. Consistent with previously reported results, the most frequent adverse events were infusion-related reactions and fatigue, which were mostly Grade 1 and resolved within two days. There have been no dose-limiting toxicities, no serious adverse events and no adverse events of Grade 3 or higher observed to date. No clinically significant laboratory abnormalities were observed in any patient. As previously announced, the Phase 2 portion of this Phase 1/2 clinical trial of NTLA-2002 has begun dosing patients, and Intellia expects to complete enrollment in the second half of this year.業績と収益の成長予測NasdaqGM:NTLA - アナリストの将来予測と過去の財務データ ( )USD Millions日付収益収益フリー・キャッシュフロー営業活動によるキャッシュ平均アナリスト数12/31/2028635-77-9-951412/31/2027257-249-224-2372112/31/202660-414-334-297213/31/202666-395-364-363N/A12/31/202568-413-396-395N/A9/30/202558-446-413-411N/A6/30/202553-480-422-419N/A3/31/202546-526-381-377N/A12/31/202458-519-355-349N/A9/30/202443-522-363-357N/A6/30/202446-509-383-373N/A3/31/202453-486-418-405N/A12/31/202336-481-408-394N/A9/30/202352-462-407-391N/A6/30/202353-453-384-368N/A3/31/202353-430-379-363N/A12/31/202252-474-392-333N/A9/30/202251-442-355-298N/A6/30/202245-400-342-285N/A3/31/202238-369-309-252N/A12/31/202133-268-238-225N/A9/30/202127-229-221-211N/A6/30/202142-185-181-174N/A3/31/202152-149-69-64N/A12/31/202058-134-53-50N/A9/30/202062-120-43-38N/A6/30/202051-116-41-35N/A3/31/202046-109-126-120N/A12/31/201943-100N/A-103N/A9/30/201940-90N/A-83N/A6/30/201937-89N/A-70N/A3/31/201933-86N/A-65N/A12/31/201830-85N/A-61N/A9/30/201829-90N/A-74N/A6/30/201829-83N/A-78N/A3/31/201827-76N/A-72N/A12/31/201726-68N/A-65N/A9/30/201725-54N/A-58N/A6/30/201723-46N/A-49N/A3/31/201721-38N/A34N/A12/31/201616-32N/A36N/A9/30/201613-26N/A45N/A6/30/20169-22N/A49N/A3/31/20167-18N/A-16N/A12/31/20156-12N/A-2N/A9/30/20154-14N/A1N/Aもっと見るアナリストによる今後の成長予測収入対貯蓄率: NTLAは今後 3 年間で収益性が向上すると予測されており、これは 貯蓄率 ( 3.5% ) よりも高い成長率であると考えられます。収益対市場: NTLA今後 3 年間で収益性が向上すると予想されており、これは市場平均を上回る成長と考えられます。高成長収益: NTLA今後 3 年以内に収益を上げることが予想されます。収益対市場: NTLAの収益 ( 55.6% ) US市場 ( 11.7% ) よりも速いペースで成長すると予測されています。高い収益成長: NTLAの収益 ( 55.6% ) 20%よりも速いペースで成長すると予測されています。一株当たり利益成長率予想将来の株主資本利益率将来のROE: NTLAの 自己資本利益率 は、3年後には低くなると予測されています ( 13.6 %)。成長企業の発掘7D1Y7D1Y7D1YPharmaceuticals-biotech 業界の高成長企業。View Past Performance企業分析と財務データの現状データ最終更新日（UTC時間）企業分析2026/05/24 13:12終値2026/05/22 00:00収益2026/03/31年間収益2025/12/31データソース企業分析に使用したデータはS&P Global Market Intelligence LLC のものです。本レポートを作成するための分析モデルでは、以下のデータを使用しています。データは正規化されているため、ソースが利用可能になるまでに時間がかかる場合があります。パッケージデータタイムフレーム米国ソース例会社財務10年損益計算書キャッシュ・フロー計算書貸借対照表SECフォーム10-KSECフォーム10-Qアナリストのコンセンサス予想+プラス3年予想財務アナリストの目標株価アナリストリサーチレポートBlue Matrix市場価格30年株価配当、分割、措置ICEマーケットデータSECフォームS-1所有権10年トップ株主インサイダー取引SECフォーム4SECフォーム13Dマネジメント10年リーダーシップ・チーム取締役会SECフォーム10-KSECフォームDEF 14A主な進展10年会社からのお知らせSECフォーム8-K* 米国証券を対象とした例であり、非米国証券については、同等の規制書式および情報源を使用。特に断りのない限り、すべての財務データは1年ごとの期間に基づいていますが、四半期ごとに更新されます。これは、TTM（Trailing Twelve Month）またはLTM（Last Twelve Month）データとして知られています。詳細はこちら。分析モデルとスノーフレーク本レポートを生成するために使用した分析モデルの詳細は当社のGithubページでご覧いただけます。また、レポートの使用方法に関するガイドやYoutubeのチュートリアルも掲載しています。シンプリー・ウォールストリート分析モデルを設計・構築した世界トップクラスのチームについてご紹介します。業界およびセクターの指標私たちの業界とセクションの指標は、Simply Wall Stによって6時間ごとに計算されます。アナリスト筋Intellia Therapeutics, Inc. 21 これらのアナリストのうち、弊社レポートのインプットとして使用した売上高または利益の予想を提出したのは、 。アナリストの投稿は一日中更新されます。35 アナリスト機関Jack AllenBairdBrian SkorneyBairdWilliam PickeringBernstein32 その他のアナリストを表示
分析記事 • May 14Earnings Update: Intellia Therapeutics, Inc. (NASDAQ:NTLA) Just Reported Its First-Quarter Results And Analysts Are Updating Their ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its quarterly results, which were...
分析記事 • May 11Analysts Have Made A Financial Statement On Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) First-Quarter ReportA week ago, Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) came out with a strong set of first-quarter numbers that could...
分析記事 • Mar 02Earnings Release: Here's Why Analysts Cut Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Price Target To US$46.08Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its yearly results, which were ahead...
分析記事 • Aug 11Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportThe analysts might have been a bit too bullish on Intellia Therapeutics, Inc. ( NASDAQ:NTLA ), given that the company...
分析記事 • Feb 24Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders are probably feeling a little disappointed, since its shares...
Board Change • May 20Insufficient new directorsThere is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 6 experienced directors. 5 highly experienced directors. Independent Director Brian Goff was the last director to join the board, commencing their role in 2024. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model.
ナラティブの更新 • May 14NTLA: Clinical Hold Resolution Will Reopen ATTR Phase 3 Upside PotentialAnalysts have lifted the fair value estimate for Intellia Therapeutics to $26.70 from $25.29. This reflects a series of higher price targets across the Street as recent FDA decisions on the MAGNITUDE program ease regulatory concerns and support more optimistic assumptions on revenue growth, profitability, and long-term P/E potential.
分析記事 • May 14Earnings Update: Intellia Therapeutics, Inc. (NASDAQ:NTLA) Just Reported Its First-Quarter Results And Analysts Are Updating Their ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its quarterly results, which were...
ライブニュース • May 13Intellia Therapeutics Achieves Phase 3 CRISPR Milestone and Advances Toward FDA ApprovalIntellia Therapeutics reported positive topline Phase 3 results for lonvoguran ziclumeran (lonvo-z), its one-time in vivo CRISPR therapy for hereditary angioedema, with the trial meeting its primary and all key secondary endpoints and showing an 87% reduction in mean monthly HAE attacks versus placebo. The company has started a rolling Biologics License Application with the FDA for lonvo-z, with plans to complete the filing in the second half of 2026 and targeting a potential U.S. launch in the first half of 2027, subject to regulatory approval. The FDA has lifted clinical holds on Intellia’s Phase 3 MAGNITUDE and MAGNITUDE-2 trials for transthyretin amyloidosis, and ARK Invest has significantly increased its stake in the company, buying about 3.78 million shares worth nearly US$48m across direct holdings and ETFs. Taken together, the Phase 3 success, BLA progress and resumption of key ATTR trials indicate that Intellia’s in vivo CRISPR platform is moving deeper into late-stage and potential commercial territory, which can be a key focus point for investor attention. At the same time, the stock’s recent volatility, ongoing clinical and regulatory work, and the company’s need to fund its pipeline mean investors still have to weigh execution and approval risk carefully, despite growing institutional interest.
お知らせ • May 04Intellia Therapeutics, Inc., Annual General Meeting, Jun 09, 2026Intellia Therapeutics, Inc., Annual General Meeting, Jun 09, 2026.
お知らせ • Apr 30Intellia Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $180.00001 million.Intellia Therapeutics, Inc. has completed a Follow-on Equity Offering in the amount of $180.00001 million. Security Name: Common Stock Security Type: Common Stock Securities Offered: 16,744,187 Price\Range: $10.75 Discount Per Security: $0.645
お知らせ • Apr 29Intellia Therapeutics Initiates Rolling Submission of Biologics License Application to Fda for Lonvoguran Ziclumeran as A One-Time Treatment for Hereditary AngioedemaIntellia Therapeutics, Inc. announced it has initiated a rolling submission of a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of lonvo-z (formerly known as NTLA-2002) for hereditary angioedema (HAE). Designed as a one-time treatment that is administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels. Intellia also separately announced positive topline data from the Phase 3 HAELO clinical trial of lonvo-z in HAE. The trial met its primary and all key secondary endpoints, demonstrating that a one-time dose of lonvo-z led to freedom from both HAE attacks and the use of ongoing therapy for most patients during the six-month primary observation period. Pursuant to the Regenerative Medicine Advanced Therapy (RMAT) designation that the FDA granted to lonvo-z for the treatment of HAE, a rolling BLA allows Intellia to submit portions of the BLA on an ongoing basis and provides the FDA with an opportunity to expedite its review. In addition to the RMAT program, Intellia participated in the FDA’s Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot. This program allows sponsors to discuss their CMC product development strategies and goals with FDA review staff and address their questions. The increased communication is intended to help sponsors complete CMC activities to expedite their drug development programs to support application submission and earlier patient access. Intellia anticipates completing its BLA submission in the second half of 2026. If the filing is accepted by the FDA, the agency is expected to determine if it will grant a priority review and provide a target action date to complete its evaluation. If approved, Intellia plans to launch lonvo-z commercially in the first half of 2027. Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an in vivo CRISPR gene editing candidate that is intended to permanently lower kallikrein by inactivating the kallikrein B1 (KLKB1) gene with a single dose. Lonvo-z has received five notable regulatory designations: Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. Hereditary angioedema (HAE) is a rare, genetic disease characterized by severe, recurring and unpredictable inflammatory attacks in various organs and tissues of the body, which can be painful, debilitating and life-threatening. It is estimated that one in 50,000 people are affected by HAE. There are preventative and on-demand treatment options to help manage the condition, including long- and short-term prophylaxis used to prevent swelling attacks. Current treatment options often include lifelong therapies, which may require chronic intravenous (IV) or subcutaneous (SC) administration as often as twice per week or daily oral administration to ensure constant pathway suppression for disease control. Despite chronic administration, breakthrough attacks still occur. Kallikrein inhibition is a clinically validated strategy for the preventive treatment of HAE attacks.
お知らせ • Apr 28Intellia Therapeutics Reports Positive Phase 3 Results In Hereditary AngioedemaIntellia Therapeutics, Inc. announced positive topline results from the global Phase 3 HAELO clinical trial of lonvoguran ziclumeran (lonvo-z) in hereditary angioedema (HAE). HAE is a rare genetic condition in which patients experience recurrent and potentially life-threatening swelling (angioedema) attacks in various parts of their body, including the face, upper airway, abdomen and extremities due to an overproduction of bradykinin. Designed as a one-time treatment that is administered in an outpatient setting, lonvo-z is an in vivo CRISPR gene editing candidate that is intended to inactivate the kallikrein B1 (KLKB1) gene to permanently lower kallikrein and bradykinin levels. Intellia separately announced that it has initiated a rolling biologics license application (BLA) submission to the FDA to seek regulatory approval. The company is preparing for a potential U.S. launch of lonvo-z in the first half of 2027. HAELO is a randomized, double-blind, placebo-controlled Phase 3 trial designed to evaluate the efficacy and safety of a one-time 50 milligram dose of lonvo-z in adults and adolescents aged 16 years and older with Type I or Type II HAE. Key endpoints of the trial focused on the number of HAE attacks experienced by patients, quality of life, safety and tolerability. A total of 80 patients were enrolled, with 52 receiving lonvo-z and 28 receiving placebo. Of the total population, 49% of patients were enrolled in the United States and 71% were on long-term prophylaxis (LTP) therapy at study entry. Patients on LTP were required to discontinue those therapies in the weeks prior to dosing. The trial met its primary endpoint. For the six-month efficacy evaluation period (weeks 5 to 28), a one-time infusion of lonvo-z reduced attacks by 87% versus placebo, with a mean monthly attack rate of 0.26 in the lonvo-z arm compared with 2.10 in the placebo arm.
Seeking Alpha • Apr 28Intellia Therapeutics And Wall Street Continue To Play Their D&D Game - Data And DoubtSummary Dungeons & Dragons (D&D) is an incredibly successful role-playing game. Today I introduce a new kind of D&D game. I call it Data & Doubt. There's a heck of a D&D match going on right now between Intellia Therapeutics (representing Team Data) and Wall Street analysts and investors (Team Doubt). This contest really heated up in the past few days. Team Data came out swinging hard, with great results in NTLA's Phase 3 trial of a drug called Lonvo-z. Team Doubt came back with a vengeance. Instead of rising, as one might have hoped, NTLA stock lost 14% on Friday, 4/24/26, and dropped another 4% on Monday, 4/27/26. I can envision Team Data making a comeback in the months ahead. But the ongoing tenacity of Team Doubt prevents me from raising my currently neutral 'Hold' rating for Intellia stock. Read the full article on Seeking Alpha
お知らせ • Apr 28Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $150 million.Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $150 million. Security Name: Common Stock Security Type: Common Stock
ナラティブの更新 • Apr 26NTLA: Clinical Hold Resolution Will Reopen Key ATTR Phase 3 CatalystNarrative Update The analyst price target for Intellia Therapeutics is now set at $25.29. Analysts point to the FDA lifting clinical holds on the MAGNITUDE studies, the resumption of Phase 3 enrollment for nexiguran ziclumeran, and updated trial protocols as key reasons for their revised views.
ナラティブの更新 • Apr 11NTLA: Clinical Hold Resolution Will Restore ATTR Phase 3 Value DriverAnalysts moved Intellia Therapeutics' consolidated price target higher toward the mid to high $20s, citing the FDA's decision to lift clinical holds on the MAGNITUDE programs for nexiguran ziclumeran and noting that the updated safety protocols support the continuation of Phase 3 enrollment. Analyst Commentary Recent Street research highlights a clear shift in sentiment around Intellia Therapeutics following the FDA decision to lift clinical holds on the MAGNITUDE programs and allow Phase 3 enrollment to resume under updated safety protocols.
ナラティブの更新 • Mar 27NTLA: Clinical Hold Resolution Will Reopen ATTR Phase 3 Value OpportunityAnalysts lifted their price targets on Intellia Therapeutics toward a range of about $15 to $48 after the FDA removed clinical holds on the MAGNITUDE program, citing improved clarity on safety, updated trial protocols, and a higher assumed probability of success for nexiguran ziclumeran in ATTR indications. Analyst Commentary Research updates following the FDA's decision cluster around how much value investors should assign to nexiguran ziclumeran in transthyretin amyloidosis and how execution risk in Phase 3 might affect Intellia Therapeutics' longer term profile.
ナラティブの更新 • Mar 12NTLA: Clinical Hold Resolution Will Restore Confidence In ATTR Phase 3 FranchiseAnalysts have raised their blended price target for Intellia Therapeutics from $23.86 to $25.29, citing the FDA's removal of clinical holds on MAGNITUDE studies and renewed confidence in the nex-z program as key reasons for the change. Analyst Commentary Recent research commentary on Intellia Therapeutics clusters around the FDA decision to lift clinical holds on the MAGNITUDE and MAGNITUDE 2 programs for nexiguran ziclumeran, with most firms updating price targets and ratings in response.
お知らせ • Mar 03+ 1 more updateIntellia Therapeutics Presents Longer-Term Clinical Data For Lonvoguran ZiclumeranIntellia Therapeutics, Inc. announced details about its presentation of four posters at the 2026 American Academy of Allergy, Asthma & Immunology Annual Meeting (AAAAI) that took place this past weekend in Philadelphia, Pennsylvania. Poster Presentation Details: Title: Long-Term Durability and Safety of Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) 50 mg in Patients with Hereditary Angioedema Session: Allergic Skin Diseases Poster Number: 061 In this pooled Phase 1/2 analysis (n=32), a one-time 50 mg dose of lonvo-z led to deep, stable and durable reductions in plasma kallikrein across all patients with up to three years of follow-up. Across the 32 patients, the mean monthly attack rate was consistently =0.2, representing a mean reduction of 96% in HAE attacks from baseline through last follow-up. Of the 32 patients, 31 (97%) were both attack-free and LTP-free as of the data cutoff, with the attack-free and LTP-free periods ranging from 2 months to 3 years with follow-up ongoing. Title: Evolving Treatment Goals to Achieve Freedom from Attacks and Long-Term Prophylaxis Following a One-Time Treatment with Lonvoguran Ziclumeran (Lonvo-z; NTLA-2002) Session: Allergic Skin Diseases Poster Number: 005 Recent research shows that achieving an attack-free status and simultaneously minimizing treatment burden are primary treatment goals for HAE experts and patients. Of the 28 patients with >6 months of follow-up after receiving a one-time 50 mg treatment of lonvo-z in a pooled Phase 1/2 analysis, 86% were attack-free and LTP-free for >6 months, a timeframe suggested by patients to be clinically meaningful. Title: Quantitative Systems Biology Modeling Estimates Extent of Excessive Kallikrein Generation in Hereditary Angioedema Patients Session: Allergic Skin Diseases Poster Number: 003 This model suggested that HAE with significant C1-esterase inhibitor deficiency generates excess plasma kallikrein compared to healthy individuals, directly correlating with bradykinin increases. In this model, an 85% reduction in prekallikrein was shown to reduce peak free kallikrein and peak bradykin to near normal ranges. This level of reduction in prekallikrein is consistent with what has been observed clinically with a 50 mg dose of lonvo-z. Title: Chronic Medications Pose Challenges for People Living with Hereditary Angioedema Session: Bridging Evidence for Real World Impact Poster Number: 716 Among 100 surveyed U.S. patients with HAE, 89% of whom were on long-term prophylaxis therapies and 11% of whom were on on-demand therapies only, 34% reported having at least one attack per month and only 20% reported being attack free in the prior year. Most respondents indicated that eliminating lifetime chronic medication use and enhancing efficacy are the most important ways to improve their current therapy. Based on Nobel Prize-winning CRISPR/Cas9 technology, lonvo-z has the potential to become the first one-time treatment for hereditary angioedema (HAE). Lonvo-z is an investigational in vivo CRISPR-based gene editing therapy that is currently being investigated in HAELO, a Phase 3 clinical trial in HAE, and is designed to prevent HAE attacks by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency, Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission.
ナラティブの更新 • Feb 25NTLA: Nex Z Clinical Return Will Ease ATTR Concerns And Support RepricingNarrative Update on Intellia Therapeutics The analyst price target for Intellia Therapeutics has been raised, with recent moves such as Baird lifting its target to $7 and H.C. Wainwright increasing its target by $10, as analysts point to progress on nex-z returning to the clinic and a broader reassessment of U.S. biopharma valuations. Analyst Commentary Recent Street research on Intellia Therapeutics reflects a mix of optimism tied to specific catalysts and caution around broader sector risks and execution.
ナラティブの更新 • Feb 09NTLA: Nex Z Return To Clinic Will Offset ATTR Safety OverhangAnalysts have lifted their fair value estimate for Intellia Therapeutics by about $1.58 per share, supported by recent price target increases from firms citing potential value from a nex-z return to clinic, even as others trim or withdraw targets on safety concerns and a higher assumed future P/E of about 45.5x. Analyst Commentary Recent Street research on Intellia Therapeutics reflects a split view, with some analysts focusing on potential value creation from a nex z return to clinic and others centering on safety concerns and a narrower path to upside.
新しいナラティブ • Feb 02One Time Genetic Medicines And Late Stage Pipeline Will Redefine Long Term PotentialCatalysts About Intellia Therapeutics Intellia Therapeutics develops CRISPR based genetic medicines aimed at one time treatments for serious diseases. What are the underlying business or industry changes driving this perspective?
お知らせ • Jan 27Intellia Therapeutics, Inc. Announces FDA Lift of Clinical Hold on Magnitude-2 Phase 3 Clinical Trial in Attrv-PnIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Investigational New Drug application (IND) for the MAGNITUDE-2 Phase 3 clinical trial of nexiguran ziclumeran (nex-z) for patients with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Intellia's engagement with FDA is ongoing regarding the clinical hold on the IND for the MAGNITU DE Phase 3 clinical trial of ne xiguran ziclumer an (nex-z) for Patients with hereditary transthy Retin amyloidosis with Polyneuropathy (ATTR v-PN). Intellia' engagement with FDA is ongoing regarding The clinical hold on the IND for The MAGNITUDE Phase 3clinical trial of nex-z for patients with transthyretin Amyloidosis with cardiomyopathy (ATTR-CM). The clinical holds on the INDs for MAGNITUDE and MAGNITUDE- 2 were imposed by the FDA on October 29, 2025, following the observation of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in MAGNITUDE that met the trial's protocol-defined pausing criteria. The company has aligned with the FDA on certain study modifications and mitigation measures related to MAGNITUDE-1 that include enhanced safety monitoring of liver laboratory tests. The company is engaged with clinical trial investigators, ethics committees, international regulatory authorities and other stakeholders to resume enrollment activities in MAGNITUDE- second as quickly as possible.ex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and is being investigated in MAGNITUDE andMAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTRv-PN, respectively. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction.ex-z has received an Orphan Drug and RMAT Designation from the U.S. Food & Drug Administration (FDA) and an Orphan Drug Designation (ODD) from the European Commission. These risks and uncertainties include, but are not limited to: uncertainties related to: uncertainties related to Intellia's ability to resume the MAGNITUDE and MagNITUDE-2 trials; regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including nex -z, will not be successfully developed and commercialized; risks related to Intellia's capabilities and uncertainties, and other important factors, any of which could cause Intellia's actual results to differ from those contained in the U.S. Food, Drug Administration (FDA).
ナラティブの更新 • Jan 24NTLA: Hereditary Angioedema Progress Will Offset ATTR Liver Safety ConcernsAnalysts have trimmed their price targets on Intellia Therapeutics across the board, with several cuts into the single to low double digit US$ range. They are reassessing the risk profile of the ATTR cardiomyopathy program following serious liver toxicity events and related trial pauses and holds.
ナラティブの更新 • Jan 10NTLA: Hereditary Angioedema Data Will Drive Future Upside Despite Liver Safety HoldIntellia Therapeutics' updated analyst price target edges slightly lower as analysts factor in recent safety driven downgrades and target cuts, reflected in a modest reduction in fair value to about $22.27 and a slightly lower forward P/E assumption of roughly 42x. Analyst Commentary Recent Street research on Intellia Therapeutics clusters around the safety questions raised in its ATTR cardiomyopathy program and how those issues flow through to valuation, probability of approval assumptions, and perceived execution risk across the broader platform.
ナラティブの更新 • Dec 25NTLA: Hereditary Angioedema Franchise Will Drive Future Upside Despite Ongoing Liver Safety OverhangAnalysts have modestly reduced their average price target on Intellia Therapeutics, trimming fair value by low single digits to around $22 per share, as mounting safety concerns and FDA holds around nexiguran ziclumeran narrow the perceived risk or reward profile despite some lingering optimism on the broader pipeline. Analyst Commentary Street research has turned more cautious on Intellia following the liver safety events in the transthyretin-mediated amyloidosis program, with multiple downgrades and target cuts reflecting a reassessment of risk, probability of approval, and the contribution of nexiguran ziclumeran to long term growth.
ナラティブの更新 • Dec 10NTLA: Hereditary Angioedema Program Will Drive Future Upside Despite ATTR Safety OverhangAnalysts have modestly reduced their average price target on Intellia Therapeutics, trimming projected upside by a few dollars per share as mounting liver safety concerns around nexiguran ziclumeran, multiple downgrades, and lowered probability of approval assumptions for the ATTR program weigh more heavily against otherwise intact long term gene editing optionality and the opportunity in hereditary angioedema. Analyst Commentary Street research has turned more cautious on Intellia in the near term, with multiple downgrades and target cuts reflecting heightened uncertainty around the ATTR program and the broader platform safety profile, even as some analysts still highlight long term value in gene editing and the hereditary angioedema franchise.
ナラティブの更新 • Nov 26NTLA: Positive Phase 3 Data And Upcoming Regulatory Filing Will Drive Future UpsideAnalysts have lowered their average price target on Intellia Therapeutics from approximately $24.85 to $22.43 per share. They cited heightened safety concerns related to recent clinical trial liver toxicity events and uncertainty about the future of key pipeline programs.
ナラティブの更新 • Nov 12NTLA: Phase 3 Enrollment Milestones And Data Readouts Will Drive Next UpsideAnalysts have reduced their fair value estimate for Intellia Therapeutics from approximately $32.30 to $24.85. They cite heightened safety concerns in key clinical programs and lower projected growth rates following recent adverse events and subsequent trial pauses.
お知らせ • Nov 11Intellia Therapeutics, Inc. Presents Positive Longer-Term Phase 1 Data of Nexiguran Ziclumeran (nex-z) in Patients with Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced positive follow-up data from the ongoing Phase 1 clinical trial of its investigational product Nexiguran ziclumeran (nex-z) in patients with transthyretin (ATTR) amyloidosis with cardiomyopathy. In addition, the company are working diligently to address the ongoing clinical hold the FDA placed on its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials. The Phase 1 clinical trial is an open-label, two-part trial evaluating the safety and efficacy of nex-z in patients with ATTR amyloidosis with cardoomyopathy (ATTR-CM). Data presented were as of an August 23, 2025, data cut-off date. Patients dosed with nex-z continued to show evidence of disease stabilization or improvement at month 24 compared to baseline. Phase 1 Safety: Nexiguran ziclumers was generally well tolerated across all patients in the Phase 1 clinical trial. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. This investigational product is being investigated in the ongoing MAGNITUDE andMAGNITUDE- 2 Phase 3 clinical trials in ATTR-CM and ATTRv-PN, respectively, which are currently on clinical hold by the U.S. Food and Drug Administration (FDA). These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to Intellia its relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to company's product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for company's product candidates, including the ability to address the clinical hold that the United States Food and Drug Administration ("FDA") placed on the investigational new drug ("IND") applications for company's global Phase 3 MAGNITUDE study for ATTR amyloidosis With polyneuropathy ("ATTRv-PN"), and to resume those clinical trials; and its belief that greater TTR reduction may lead to greater clinical benefit. These risks and uncertainties include, but are not Limited to: risks related to: risks related to company's ability to protect and maintaining its intellectual property position; risksrelated to Intellia's relationship with third parties, including the ability of its licensors and licensees); risks related to the ability of the licensors to protect and maintaining their intellectual property position; uncertaintiesrelated to regulatory agencies' evaluation of Regulatory filings and other information related to the company's product candidates, including Nex-z; uncertainties related of the authorization, initiation and conduct the studies and other development requirements for its product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including risks related to the company's ability to address the clinical held that the FDA placed on the IND applications for the MAGNITUDE Phase 3 study for ATTR-CM and the MAGNITUDE- second study for ATTRv-PN and to resume those clinical trials.
お知らせ • Nov 09Intellia Therapeutics Presents Positive Pooled Phase 1/2 Data of Lonvoguran Ziclumeran (lonvo-z) in Patients with Hereditary AngioedemaIntellia Therapeutics, Inc. presented positive clinical data from a pooled analysis of all patients who received a 50 milligram (mg) dose of lonvo-z in the company's ongoing Phase 1/2 clinical trial in patients with hereditary angioedema (HAE). These results were shared in an oral presentation at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting in Orlando, Florida. Intellia's global Phase 1/2 clinical trial is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of lonvo-zin in adults with HAE Types I or II. Today's presentation was based on a pooled analysis of all 32 patients who have received a one-time 50 mg treatment of lonvo-z via intravenous infusion in the Phase 1/2 trial. Of the 32 patients, 15 had initially received the 50 mg dose at study Day 1 (four in Phase 1 and 11 in Phase 2) and 17 were treated after unblinding of the Phase 2 clinical trial for the primary analysis (11 had originally received a 25 mg dose of lonvo-Z, which was determined to be a suboptimal dose, and six had previously received placebo). The data cut-off for the analysis was August 29, 2025. Deep, stable and durable reductions in plasma kallikrein were observed in all patients, with a mean reduction of 89% at month 24. Among the 32 patients, 31 (97%) were both attack-free and LTP-free as of the data cutoff, with 24 (75%) being attack-free and L TP-free for at least seven months (up to 32 months for patients with the longest follow-up). Of the 11 patients who initially received the 50 mg dose of lonvo, 10 were attack-free and LTP -free (nine for 7-32 months and one for 1:2.
お知らせ • Oct 30Intellia, Inc. Provides Clinical Hold on the Investigational New Drug Applications for the Magnituda-2 Phase 3 Clinical Trials for Nexigur ZiclumeranOn October 29, 2025, the United States Food and Drug Administration (the FDA") verbally informed Intellia Therapeutics, Inc. that the FDA has placed a clinical hold on the Investigational New Drug applications for the MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials for nexiguran ziclumeran (nex-z). FDA indicated that it would provide a formal Clinical Hold Letter within 30 calendar days. The clinical hold follows the previously disclosed report of Grade 4 liver transaminases and increased total b birubin in a patient who was dosed with nex-z in the MAGNITUDE trial. As previously announced on October 27, 2025, the Company had temporarily paused dosing and screening in the MAGNITU DE and MAGNITUDE -2 Phase 3 clinical trials for Nex-z based on the MAGNITUDE test's protocol-defined pausing criteria. The Company intends to work with the FDA to address the clinical hold as expeditiously as possible.
ナラティブの更新 • Oct 29Paused Trials Will Drive Rebound After Resolution Of Safety ConcernsIntellia Therapeutics' consensus price target has declined from approximately $34.08 to $32.30 per share. Analysts cite heightened safety concerns surrounding recent liver injury events and paused clinical trials as key drivers behind lower valuation estimates.
お知らせ • Oct 27Intellia Therapeutics Provides Update on MAGNITUDE Clinical Trials of Nexiguran Ziclumeran (Nex-z)Intellia Therapeutics, Inc. announced that the company has temporarily paused patient dosing and screening for its MAGNITUDE and MAGNITUDE-2 Phase 3 clinical trials of nex-z for patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN), respectively. This action follows a report on October 24, 2025 of Grade 4 liver transaminases and increased total bilirubin in a patient who was dosed with nex-z in the MAGNITUDE trial on September 30, 2025, meeting the trial's protocol-defined pausing criteria. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein and is currently being investigated in MAGNITUDE and MAGNITUDE-2, Phase 3 clinical trials in ATTR-CM and ATTR-PN, respectively. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Nex-z has received an Orphan Drug and RMAT Designation from the U.S. Food and Drug Administration (FDA) and an Orphan Drug Designation (ODD) from the European Commission. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc.
お知らせ • Sep 26Intellia Therapeutics Announces Positive Longer-Term Phase 1 Data for Nexiguran Ziclumeran (nex-z) in Patients with Hereditary Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced longer-term follow-up data from the ongoing Phase 1 study of investigational nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). Results were presented in an oral session on Thursday, September 25 at the 5th International ATTR Amyloidosis Annual Meeting for Patients and Doctors in Baveno, Italy. Continuation of Deep and Durable Serum TTR Reduction: Deep, durable and consistent TTR reductions continue to be observed. Across patients who received a one-time dose of 0.3 mg/kg or higher (n=33), the mean serum TTR reduction at 24 months was 92% (corresponding mean absolute serum TTR level of 17.3 g/mL [Mean 95% CI, 12.5 – 22.2]). Among the 12 patients who had reached 36 months of follow-up, the mean serum TTR reduction was 90% (corresponding mean absolute serum TTR level of 20 g/mL [Mean 95% CI, 11.2 – 28.8]). Evidence of Stability or Improvement on Clinical and Biomarker Measures: Favorable trends indicating stability or improvement were observed in most patients with ATTRv-PN after a single dose of nex-z. Stability or improvement was based on evaluation of multiple clinical and biomarker measures, including Neuropathy Impairment Score (NIS), modified Neuropathy Impairment Score +7 (mNIS+7), modified body mass index (mBMI), Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) questionnaire, neurofilament light chain (NfL), and polyneuropathy disability (PND) score. Among the 18 patients in whom a 24-month mNIS+7 assessment was completed by the data cutoff date (April 11, 2025), 13 (72%) showed improvements of a clinically meaningful threshold of =4 points, including most of the patients in the cohort who had progressed on patisiran. Among all 36 patients enrolled in the Phase 1 trial, mean values of the secondary endpoints mBMI, QoL-DN and NfL all trended toward disease improvement and 89% of patients showed improvement or stability in PND scores through 24 months compared to baseline. Safety Summary: Nex-z has been generally well tolerated as of the data cutoff date across all patients and at all dose levels tested. The most commonly reported treatment-related adverse events were infusion-related reactions, which were mild or moderate and did not result in any discontinuations. As previously reported, three participants had Grade >3 liver enzyme elevations that were not considered serious, were asymptomatic and resolved spontaneously without medical intervention or sequelae. Phase 3 MAGNITUDE-2 Trial Advancing Rapidly: Intellia began dosing patients in the Phase 3 MAGNITUDE-2 trial in April 2025. Patient screening is advancing rapidly, and enrollment completion is expected in the first half of 2026. Intellia anticipates submitting a biologics license application (BLA) for ATTRv-PN by 2028. MAGNITUDE-2 is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of nexiguran ziclumeran (nex-z) in approximately 50 patients with hereditary transthyretin ATTR amyloidosis with polyneuropathy (ATTRv-PN). The primary endpoints of the study are a change in modified neuropathy impairment score and a change in serum TTR levels. Adult patients with ATTRv-PN are randomized 1:1 to receive a single 55 mg infusion of nex-z or placebo.
お知らせ • Sep 22Intellia Therapeutics, Inc. to Present Longer-Term Data from the Ongoing Phase 1 Clinical Trial of Nexiguran Ziclumeran (Nex-Z) for the Treatment of Hereditary Transthyretin (Attr) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced that longer-term data from the ongoing Phase 1 trial of investigational nex-z for the treatment ofhereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN) will be presented at the 5th International ATTR Amyloidosis Meeting for Patients and Doctors, taking place September 25-26 in Baveno, Italy. This presentation will include ATTRv-PN disease-relevant measures, including up to three years of patient follow-up. Based on Nobel Prize-winning CRISPR/Cas9 gene editing technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encourages for the transthyretin(TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of Nexiguran Ziclumeran as part of a multi-target discovery, development and commercialization collaboration with Regeneron Pharmaceuticals, Inc. Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin ("TTR") protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (AT TRv-PN), which can lead to vein damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. This condition, called wild-type ATTR (ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloid diagnosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.
お知らせ • Sep 18Intellia Therapeutics, Inc. Completes Enrollment in the Global Phase 3 HAELO Study of Lonvoguran Ziclumeran (lonvo-z) for Hereditary AngioedemaIntellia Therapeutics, Inc. announced it has completed enrollment in the global Phase 3 HAELO study of lonvoguran ziclumeran (lonvo-z) for the treatment of hereditary angioedema (HAE). Intellia is on track to submit a biologics license application (BLA) in the second half of 2026 to support the company's plans for a U.S. launch in the first half of 2027. Interim Phase 1/2 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Lonvo-z has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration (FDA), the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation (ODD) by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to the conduct of clinical studies and other development requirements for its product candidates, including risks related to the ability to develop and commercialize any one or more of Intellia's product candidates successfully; risks related to the results of preclinical studies or clinical studies not being predictive of future results in connection with future studies; the risk that clinical study results will not be positive; and risks related To the company's plans for aU.S. launch in the second half of 2027.
分析記事 • Sep 18Is Intellia Therapeutics (NASDAQ:NTLA) In A Good Position To Deliver On Growth Plans?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...
ナラティブの更新 • Aug 10Robust In Vivo CRISPR Trials Will Accelerate Therapeutic SuccessDespite a notable improvement in both future P/E and net profit margin, Intellia Therapeutics’ consensus analyst price target has been revised downward from $37.11 to $35.23. What's in the News Phase 3 HAELO trial for lonvo-z in hereditary angioedema (HAE) is ongoing, showing strong interim efficacy data; drug has received multiple regulatory designations, including FDA Orphan Drug and RMAT.
お知らせ • May 29Intellia Therapeutics, Inc. Provides Business Update Regarding Its Ongoing Phase 3 StudiesIntellia Therapeutics, Inc. provided a business update regarding its ongoing Phase 3 studies. Enrollment in global Phase 3 HAELO study of NTLA-2002 for hereditary angioedema is on track. The company expects to complete enrollment in the third quarter of 2025 and submit a biologics license application ("BLA") in the second half of 2026 to support plans for a potential U.S. commercial launch in 2027. En enrollment in global Phase 3 MAGNITUDE-2 study of nex-z for ATTRv-PN is also progressing well. The MAGNITUDE- 2 study is designed to measure clinical outcomes and evaluate how a single dose of nex-z can lead to reduction in serum TTR. The company expects to complete MAGNITUDE- two hundred patients have been dosed with nex-z in the MAGNITUDE study. To date, reported adverse events have been similar in nature to the reported adverse events in the Phase 1 study of nex-z For ATTR amyloidosis, and have included infusion-related reactions and asymptomatic liver transaminase elevations. There has been a single, recent, asymptomatic patient with grade 4 liver transaminase elevations based on laboratory tests, which appear to be resolving without hospitalization or medical intervention and have fallen to grade 3 ALT and grade 2 AST elevations. The company continue to monitor these events as the MAGNITUDE trial progresses.
お知らせ • May 20Intellia Therapeutics, Inc. Announces Positive Two-Year Follow-Up Data from Ongoing Phase 1 Study of Nexiguran Ziclumeran (Nex-Z) in Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy At Peripheral Nerve Society Annual MeetingIntellia Therapeutics, Inc. announced positive two-year follow-up data from the ongoing Phase 1 trial of investigational nexiguran Ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The pivotal Phase 3 MAGNITUDE-2 clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of nexiguran ziclumeran in approximately 50 patients with hereditary transthyretin amyloidosis with polyNEuropathy (ATTRv -PN). The primary endpoints of the study are a change in modified neuropathy impairment score and a change in serum TTR levels. These forward-looking statements include, but are not limited to: risks related to Intellia's beliefs and expectations regarding: the safety, tolerability, efficacy, success and advancement of its clinical programs for Nexiguran ziclumer an or "nex-z" (also known as NTLA-2001) for transthyretin ("ATTR") amyloidosis, including the ability to successfully complete its global Phase 3 MAGNITU DE-2 study for hereditary ATTR amyloidOS with polyneuropathy ("ATTRv-PN") pursuant to its clinical trial applications and investigational new drug submissions; its belief that enrollment continues to progress well in the MAGNITUDE- 2 study; its belief that a single dose of nex-z leads to deep, durable and consistent reductions in serum TTR and that increasingly deep reductions in TTR levels leads to improved outcomes; and its expectation to be able to support the submission of a biologics license application for nex-z for the treatment of ATTRv-PN by 2028. These risks and uncertainties include, but are not limited To: risks related to IntellIA's ability to protect and maintain its intellectual property position; risks related to Intellia' relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including nex -z, will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates; and risks related to Intellia its reliance on collaborations, including that its collaboration with Regeneron Pharmaceuticals, Inc. will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia's potential to cause Intellia's potential for the company's potential to be able to support a BLA submission by 2028.
分析記事 • May 11Analysts Have Made A Financial Statement On Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) First-Quarter ReportA week ago, Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) came out with a strong set of first-quarter numbers that could...
分析記事 • May 10Industry Analysts Just Made An Upgrade To Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Revenue ForecastsCelebrations may be in order for Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders, with the analysts delivering...
お知らせ • May 01Intellia Therapeutics, Inc., Annual General Meeting, Jun 11, 2025Intellia Therapeutics, Inc., Annual General Meeting, Jun 11, 2025.
お知らせ • Apr 03Intellia Therapeutics Announces First Patient Dosed in the MAGNITUDE-2 Phase 3 Study of Nexiguran Ziclumeran (nex-z), a One-Time Gene Editing-Based Treatment for Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced the first patient has been dosed in MAGNITUDE-2, a global, pivotal Phase 3 trial of nexiguran ziclumeran (nex-z) for the treatment of hereditary ATTR amyloidosis with polyneuropathy (ATTRv-PN). The Phase 3 MAGNITUDE-2 study is informed by Intellia’s Phase 1 data, showing that a single dose of nex-z led to consistently rapid, deep and durable reduction in serum TTR. Intellia expects to present longer-term data from the Phase 1 studies of nex-z for both polyneuropathy and cardiomyopathy later this year. The company plans to submit a biologics licensing application (BLA) for ATTRv-PN by 2028.
お知らせ • Mar 26Intellia Therapeutics, Inc. Announces FDA Regenerative Medicine Advanced Therapy Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy (ATTR-CM). The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product's biologics license application (BLA). Exex-z has been granted Regenerative Medicine Advanced Therapy designations by the U.S. FDA for both cardiomyopathy and polyneuropathy.ex-z has also been granted Orphan Drug Designation by the U.S.F and European Commission. Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin ("ATTR) amyloidOS. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Since its inception, Intellia has focused on leveraging gene editing technology to develop novel, first-in-class medicines that address important unmet medical needs and advance the treatment paradigm for patients. These forward-looking statements include, but are not limited to, express or implied statements regarding: the safety, efficacy, success and advancement of its clinical program for nexiguran zic Lumeran (nex-z), also known as NTLA- 2001) for the treatment of trANsthyretin (AT TR) amyloidosis with Cardiomyopathy (ATTR -CM) pursuant to its clinical trial applications and investigational new drug application, including the potential of NTLA-2001 to be a transformative treatment for patients with ATTR-CM; and the expected timing of regulatory filings, completion of clinical trials, submission of marketing authorizations, and commercialization. These risks and uncertainties include, but are not limited To: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to Intellia' relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; and uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including nex-z; risks related to the results of preclinical or clinical studies, including that they may not be positive or predictive of future results; the risk that one or more of Intellia's product candidates, including nex -z, will not be successfully developed and commercialized; and risks related toIntellia's reliance on collaborations, including that its collaboration with Regeneron will not continue or will not be successful.
新しいナラティブ • Mar 25Phase III Trials And Gene Editing Will Bring Promising Therapies Strategic discontinuation of less promising programs and capital prioritization are expected to improve net margins and enhance commercial prospects.
Seeking Alpha • Mar 02Intellia Trades Lower After Q4 Results As Clinical Progress Fails To ImpressSummary Intellia Therapeutics' stock is downgraded to HOLD due to market skepticism and lack of near-term catalysts despite promising Phase 3 trials for HAE and ATTR. NTLA-2002, Intellia's CRISPR/Cas9 drug for HAE, is the closest to commercialization, expected by 2027, but profitability remains distant. Intellia's financials show a cash runway extended through 2027, but additional funding or partnerships will likely be needed for profitability. Gene therapy sector uncertainty, exemplified by bluebird bio's struggles, underscores the risks and potential rewards for Intellia investors willing to wait. Read the full article on Seeking Alpha
分析記事 • Mar 02Earnings Release: Here's Why Analysts Cut Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Price Target To US$46.08Intellia Therapeutics, Inc. ( NASDAQ:NTLA ) defied analyst predictions to release its yearly results, which were ahead...
分析記事 • Feb 15Will Intellia Therapeutics (NASDAQ:NTLA) Spend Its Cash Wisely?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...
お知らせ • Feb 14Bronstein, Gewirtz & Grossman, LLC Files Class Action Lawsuit Against Intellia Therapeutics, IncBronstein, Gewirtz & Grossman, LLC notified investors that a class action lawsuit has been filed against Intellia Therapeutics, Inc. and certain of its officers. This lawsuit seeks to recover damages against Defendants for alleged violations of the federal securities laws on behalf of all persons and entities that purchased or otherwise acquired Intellia securities between July 30, 2024 and January 8, 2025, both dates inclusive (the “Class Period”). The Complaint alleges that throughout the Class Period, Defendants made false and/or misleading statements and/or failed to disclose that: (1) defendants created the false impression that they possessed reliable information pertaining to the viability of NTLA-3001’s development and eventual marketing, if approved; (2) Intellia’s optimistic reports of timelines, including dosing and future studies of the drug, fell short of reality; the NTLA program was not viable or sustainable for Intellia because viral-based editing programs remained expensive and inefficient in comparison to then-existing non-viral delivery methods; (3) Intellia was not equipped to timely dose patients with NTLA-3001, maintain the drug’s research and development, or even to maintain its full staff in light of the existing scientific landscape surrounding viral-based editing drugs; and (4) even if NTLA-3001 proved successful, the use of viral-based editing drugs is costly, inefficient, and poor mitigators of adverse effects in patients.
お知らせ • Jan 22Intellia Therapeutics, Inc. Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary AngioedemaIntellia Therapeutics, Inc. announced the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based therapy in development as a single-dose treatment for this potentially life-threatening disease. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the Company’s plans for a U.S. launch in 2027. The pivotal Phase 3 HAELO clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. Key endpoints include the number of HAE attacks and the number of patients who achieve attack-free status from week 5 through week 28. Based on Nobel-prize winning CRISPR/Cas9 technology, NTLA-2002 has the potential to become the first one-time treatment for hereditary angioedema (HAE). NTLA-2002 is designed to prevent HAE attacks by inactivating the kallikrein B1(KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission.
Seeking Alpha • Jan 13Intellia Therapeutics: Buying The Path To CommercializationSummary Intellia Therapeutics, Inc. is undergoing strategic reorganization, halting NTLA-3001, and reducing workforce by 27% to extend cash runway into 2027, focusing on late-stage programs NTLA-2002 and Nexiguran Ziclumeran. Despite industry headwinds and significant layoffs, I see Intellia's cost-cutting measures as prudent, aiming for commercialization of NTLA-2001 and NTLA-2002 in large, promising markets. The gene therapy sector faces turmoil, with multiple companies making tough decisions; however, Intellia's focus on late-stage programs may position it for future growth and potential M&A interest. I initiated a speculative buy on NTLA under $10 per share, anticipating a potential technical rebound and long-term investment opportunities as the company approaches commercialization. Read the full article on Seeking Alpha
お知らせ • Nov 26Intellia Therapeutics, Inc. Announces FDA Regenerative Medicine Advanced Therapy Designation Granted to Nexiguran Ziclumeran (nex-z) for the Treatment of Hereditary Transthyretin (ATTR) Amyloidosis with PolyneuropathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of hereditary transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN). Nex-z is an in vivo CRISPR-based investigational therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of ATTR amyloidosis. Development and commercialization of nex-z is led by Intellia as part of a multi-target collaboration with Regeneron. The RMAT designation was established under the 21st Century Cures Act to expedite the development and review of promising therapeutic candidates, including genetic therapies, that are intended to treat, modify, reverse or cure a serious or life-threatening disease. RMAT designation includes benefits, such as early interactions with the FDA, including discussions on surrogate or intermediate endpoints that could potentially support accelerated approval and satisfy post-approval requirements, and potential priority review of a product’s biologics license application (BLA). This RMAT designation is the third special regulatory designation received by Intellia for nex-z. Nex-z was also granted Orphan Drug Designation by the U.S. FDA and European Union Orphan Drug Designation by the European Commission. nexiguran ziclumeran (nex-z, also known as NTLA-2001): Based on Nobel Prize-winning CRISPR/Cas9 technology, nex-z has the potential to become the first one-time treatment for transthyretin (ATTR) amyloidosis. Nex-z is designed to inactivate the TTR gene that encodes for the transthyretin (TTR) protein. Interim Phase 1 clinical data showed the administration of nex-z led to consistent, deep and long-lasting TTR reduction. Intellia leads development and commercialization of nex-z as part of a multi-target discovery, development and commercialization collaboration with Regeneron. Transthyretin (ATTR) Amyloidosis: Transthyretin amyloidosis, or ATTR amyloidosis, is a rare, progressive and fatal disease. Hereditary ATTR (ATTRv) amyloidosis occurs when a person is born with mutations in the TTR gene, which causes the liver to produce structurally abnormal transthyretin (TTR) protein with a propensity to misfold. These damaged proteins build up as amyloid in the body, causing serious complications in multiple tissues, including the heart, nerves and digestive system. ATTRv amyloidosis predominantly manifests as polyneuropathy (ATTRv-PN), which can lead to nerve damage, or cardiomyopathy (ATTRv-CM), which can lead to heart failure. Some individuals without the genetic mutation produce non-mutated, or wild-type TTR proteins that become unstable over time, misfolding and aggregating in disease-causing amyloid deposits. This condition, called wild-type ATTR (ATTRwt) amyloidosis, primarily affects the heart. There are an estimated 50,000 people worldwide living with ATTRv amyloidosis and between 200,000 and 500,000 people with ATTRwt amyloidosis. There is no known cure for ATTR amyloidosis and currently available medications are limited to slowing accumulation of misfolded TTR protein.
Seeking Alpha • Nov 19Intellia Therapeutics' Gene Therapies Fall Short Of Breakthroughs (Rating Downgrade)Summary Intellia initiated the Phase 3 HAELO trial for NTLA-2002, targeting lifelong control of hereditary angioedema (HAE) with a single-dose CRISPR-based therapy. Positive Phase 2 NTLA-2002 data showed most patients remained attack-free; however, limitations like small sample size and short follow-up require cautious interpretation. Phase 1 data for nexiguran ziclumeran (nex-z) in ATTR amyloidosis showed disease stabilization, but competitive therapies demonstrate more robust improvements. Intellia has strong financial health, with over $900M in liquidity and a cash runway expected to last until late 2026. Intellia's limited transformational data, high manufacturing costs, and competitive landscape justify downgrading the stock from "Buy" to "Hold." Read the full article on Seeking Alpha
お知らせ • Nov 17Intellia Therapeutics, Inc. Announces First Clinical Evidence from Ongoing Phase 1 Study That Nexiguran Ziclumeran (nex-z) May Favorably Impact Disease Progression in Transthyretin (ATTR) AmyloidosisIntellia Therapeutics, Inc. announced positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z, also known as NTLA-2001) in patients with transthyretin (ATTR) amyloidosis. These results from the ongoing Phase 1 study increase the belief in the likelihood of success of active Phase 3 studies based on hypothesis that greater TTR reduction may lead to greater clinical benefit in ATTR amyloidosis. Rapid, Deep and Durable Serum TTR Reduction: Across all patients (n=36), a single dose of nex-z led to consistently rapid, deep and sustained serum TTR reduction, regardless of baseline levels, through the latest follow-up. Evidence of Disease Modification on Clinical Measures: F favorable trends indicating stability or improvement were observed in patients with ATTRv-PN based on evaluation of multiple clinical measures, including Neuropathy Impairment Score (NIS), modified Neuropathy Impairment Score ("mNIS+7) and modified BMI (mBMI). The clinical measure results are detailed in the table below. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to valid third party intellectual property; risks related to the relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to regulatory agencies' evaluation of regulatory filings and other information related to product candidates, including nex-z; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for product candidates, including uncertainties related to regulatory approvals to conduct clinical trials, including ability to initiate or enroll the Phase 3 MAGNITUDE study for ATTRv-PN or "nex-z" (f/k/a NTLA-2001), for transthyretin ("ATTR") amyloidosis.
Seeking Alpha • Nov 11Intellia Therapeutics: Q3 Earnings Report Puts Phase II Results In FocusSummary Intellia Therapeutics' NTLA-2002 Phase II results last month were underwhelming, however, management reassured investors on the Q3 earnings call setting the stage for a modest rally. While last month's drop is appropriately discounting NTLA-2002, investors should be encouraged that 3 Phase III trials will be engaged by year-end, representing multiple opportunities for substantial upside. Intellia's financial position is robust with $945 million in cash, enough to fund operations into late 2026. NTLA-2002's Phase III study will enroll 60 patients and is anticipated to reach its primary endpoint by April 2026, potentially offering a functional cure for hereditary angioedema (HAE). HAE represents a huge market opportunity estimated to reach $17.4 billion by 2032. Read the full article on Seeking Alpha
Seeking Alpha • Nov 05Data Underwhelms - Intellia's Cash, Data And Competition Are A Challenging StorySummary ATTR has become a more competitive space, with uncertainty ahead on Phase III timelines. Recent data on HAE did not provide a differentiated outcome from more advanced competitors. The competitive landscape across lead indications is evolving rapidly and may create unforeseen challenges to trial completion or commercialization. Funding and regular share offerings are diminishing flexibility and potential return for investors. 2026 cash runway may be insufficient to obtain meaningful Phase III clinical readouts. Read the full article on Seeking Alpha
お知らせ • Oct 25Intellia Therapeutics, Inc. Presents Positive Results from the Phase 2 Study of NTLA-2002Intellia Therapeutics, Inc. announced positive Phase 2 data from the ongoing Phase 1/2 study of NTLA-2002 in patients with hereditary angioedema (HAE), with results continuing to indicate that NTLA-2002 has the potential to eliminate HAE attacks following a one-time infusion. NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a one-time treatment for HAE, a rare genetic condition that leads to potentially life-threatening swelling attacks. Results were published online on October 24, 2024 in The New England Journal of Medicine and will be presented on October 26 at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Scientific Meeting in Boston, Massachusetts. The Phase 2 study is a randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, pharmacodynamics and pharmacokinetics of NTLA-2002. A total of 27 participants were enrolled and randomized to receive one of two single doses of NTLA-2002 (25 mg or 50 mg) or placebo via intravenous infusion. The data cut-off date for the analysis was April 4, 2024, when the 25th patient completed the 16-week primary observation period. Single dose of NTLA-2002 led to deep attack rate reductions during the primary observation period. The mean monthly attack rates relative to placebo were reduced by 75% and 77% for the 25 mg and 50 mg arms during weeks 1 – 16, and by 80% and 81% during weeks 5 – 16, respectively. In the 50 mg arm, eight of 11 patients experienced a complete response after a single dose of NTLA-2002, with no attacks at all during the 16-week primary observation period; these eight patients continued to be attack-free through the latest follow-up (median of eight months) and no subsequent treatment has been required. In contrast, four of the 10 patients in the 25 mg arm experienced a complete response and zero patients in the placebo arm. Similarly, patients who received the 50 mg dose achieved a greater kallikrein protein reduction, with an 86% mean reduction from baseline compared to 55% in the 25 mg arm at week 16. At both dose levels, NTLA-2002 was well tolerated. The most frequent adverse events (AEs) were headache, fatigue and nasopharyngitis. There have been no serious AEs and all AEs were either Grade 1 or 2, except for one patient in the placebo arm who experienced a serious AE of Grade 4 edema of the tongue with breathing impairment that was attributed to their underlying HAE. No clinically significant laboratory abnormalities were observed. The safety, tolerability and efficacy data from the Phase 2 study are consistent with the long-term Phase 1 data presented at the European Academy of Allergy and Clinical Immunology (EAACI) Congress in Valencia, Spain on June 2, 2024. Based on these results, Intellia selected 50 mg for evaluation in the global, pivotal Phase 3 HAELO study, which is actively screening patients.
Seeking Alpha • Oct 25Intellia Therapeutics: Lower Stock Price On HAE Data Presents A Buying OpportunitySummary Intellia Therapeutics, Inc. achieved positive phase 1/2 results using NTLA-2002 for HAE patients: 8 out of 11 patients taking higher doses of 50 mg of NTLA-2002 were attack-free during the 16-week primary observation period. Despite the stock price trading lower by 20%, the HAE treatment program continues on using NTLA-2002 in the phase 3 HAELO study. The global hereditary angioedema treatment market size is projected to reach $17.31 billion by the end of 2032. NTLA-2001 is another in vivo CRISPR drug being developed to target patients with transthyretin [ATTR] amyloidosis being explored in the phase 3 MAGNITUDE study; Updated phase 1 data is expected 2nd half of 2024. Read the full article on Seeking Alpha
お知らせ • Oct 07Intellia Therapeutics, Inc. Announces Initiation of Haelo Phase 3 Study of Ntla-2002Intellia Therapeutics, Inc. announced the initiation of HAELO, a global, pivotal Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for this potentially life-threatening disease. Patient screening is active following Intellia's successful end-of-Phase 2 meeting and submission of an Investigational New Drug Application amendment to the U.S. Food and Drug Administration (FDA). HAELO is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE. Patients will be randomized 2:1 to receive a single 50 mg infusion of NTLA-2002 or placebo. Patients randomized to the placebo arm will be eligible for optional crossover to NTLA-2002 at week 28. The primary endpoint is the change in number of HAE attacks from week 5 through week 28. Intellia is initiating the Phase 3 study based on positive safety and efficacy data from the ongoing Phase 1/2 study (NCT05120830) of NTLA-2002. Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. Intellia previously announced positive toplines results from the Phase 2 portion of the study. The Company plans to present the detailed results at the 2024 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, taking place October 24 – 28 in Boston, Massachusetts.
Seeking Alpha • Sep 25Intellia - Exciting Promise Of First In-Vivo Gene Therapy Makes Bull CaseSummary Intellia Therapeutics is pioneering in-vivo CRISPR gene editing, aiming for groundbreaking therapies, with NTLA-2001 showing promising results in ATTR amyloidosis, potentially achieving blockbuster status. Despite a 65% stock drop since 2022, Intellia's advancements in Phase 3 trials and strong partnerships, like with Regeneron, make it a compelling buy opportunity. The company's innovative use of lipid nanoparticles for gene delivery and a robust pipeline position it as a leader in the CRISPR space, targeting large markets. With a cash position of $940m and multiple Phase 3 studies in play, Intellia is poised for substantial valuation growth and a possible Big Pharma acquisition. The major risks to the thesis are a safety setback or a failure to deliver on approval-worthy efficacy endpoints, but this is mitigated somewhat by the success of e.g. Crispr Therapeutics / Vertex very effective sickle cell disease therapy Casgevy. Read the full article on Seeking Alpha
分析記事 • Sep 07We're Keeping An Eye On Intellia Therapeutics' (NASDAQ:NTLA) Cash Burn RateThere's no doubt that money can be made by owning shares of unprofitable businesses. For example, although Amazon.com...
分析記事 • Aug 11Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportThe analysts might have been a bit too bullish on Intellia Therapeutics, Inc. ( NASDAQ:NTLA ), given that the company...
Seeking Alpha • Aug 03Intellia Therapeutics Is Expanding The Scope Of Gene Editing (Technical Analysis)Summary The future is here… The CRISPR-Cas9 gene editing platform lets us reshuffle DNA, analogous to how we edit text. The difference is that CRISPR-Cas9 is a much more powerful tool that we’re slowly learning to use. It’s important that we master it… gene editing is the best treatment for many serious diseases. Intellia Therapeutics is blazing new trails in using CRISPR-Cas9 to treat more conditions. But we can’t compute any sort of valuation for the stock, so it’s only suitable as a sentiment play. Read the full article on Seeking Alpha
お知らせ • Jul 30Intellia Therapeutics, Inc. Receives Authorization to Initiate Phase 1/2 Clinical Trial of Ntla-3001 for the Treatment of Alpha-1 Antitrypsin DeficiencyIntellia Therapeutics, Inc. announced the authorization of its Clinical Trial Application (CTA) by the United Kingdom's Medicine and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1/2 study evaluating NTLA-3001 for the treatment of alpha-1 antitrypsin deficiency (AATD)-associated lung disease. AATD is a rare, genetic disease that most commonly manifests in lung dysfunction due to insufficient levels of alpha- 1 antitrypsin (AAT) protein. NTLA-3001 is a systemically administered in vivo CRISPR/Cas9-based targeted gene insertion candidate. The study will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of NTLA-3001. Beyond its first application in the United Kingdom, Intellia is submitting additional regulatory applications in other countries as part of its ongoing, multi-national development strategy for NTLA-3001.
お知らせ • Jul 09Blueallele Corporation Files Lawsuit for Patent Infringement Against Intellia Therapeutics, Inc., for the Unauthorized Use of Blueallele’S Patented Bi-Directional Insertion TemplatesBlueAllele Corporation filed a patent infringement lawsuit against Intellia Therapeutics in the United States District Court for the District of Delaware. The complaint alleges infringement of BlueAllele’s bi-directional insertion template patents, which cover the company’s transformational platform used in the development and treatment of genetic diseases. The novel design of BlueAllele’s bi-directional insertion template technology has unlocked the full potential of the cell’s highly active NHEJ pathway. Use of the patented bi-directional insertion template technology can increase the efficacy of gene editing events, improve safety, and allow development of therapeutics for a wide range of diseases. As alleged in the complaint, Intellia has utilized BlueAllele’s patented platform technology to advance internal programs as well as external collaboration programs. BlueAllele has expanded upon its bi-directional insertion template technology with the design of additional platforms for treating specific classes of genetic diseases, including bi-directional templates with RNAi cassettes for autosomal dominant diseases and functions to remove unwanted gene products while simultaneously restoring normal protein function. Further, BlueAllele has developed bi-directional templates with dual terminators designed to address repeat expansion diseases and functions to prevent transcription through the expansion. BlueAllele Corporation is being represented in the lawsuit by Fish & Richardson P.C. and Schwartz Law Firm.
お知らせ • Jun 27Intellia Therapeutics, Inc. Announces Chief Financial Officer ChangesIntellia Therapeutics, Inc. announced the appointment of Edward Dulac as Chief Financial Officer (CFO), effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. Mr. Dulac is a highly accomplished biotechnology business leader and joins Intellia with more than 20 years of combined finance, business development and corporate strategy experience. Most recently, Mr. Dulac served as CFO of Fate Therapeutics. Prior to that role, Mr. Dulac spent numerous years at Celgene (now Bristol Myers Squibb), where he held multiple positions including as Vice President, Business Development & Strategy. Prior to Celgene, Mr. Dulac worked as a biopharmaceutical equity research analyst at Barclays Capital and Lehman Brothers and in corporate finance at Pfizer. Mr. Dulac holds a Bachelor of Pharmacy from the University of Pittsburgh and an MBA from Indiana University, Kelley School of Business.
お知らせ • Jun 26+ 1 more updateIntellia Therapeutics, Inc. Announces Executive ChangesIntellia Therapeutics, Inc. announced the appointment of Edward Dulac as Executive Vice President and Treasurer, effective July 22, 2024. Mr. Dulac will succeed Glenn Goddard who will step down from his role effective June 30, 2024. Mr. Dulac is a highly accomplished biotechnology business leader and joins Intellia with more than 20 years of combined finance, business development and corporate strategy experience. Most recently, Mr. Dulac served as CFO of Fate Therapeutics. Prior to that role, Mr. Dulac spent numerous years at Celgene (now Bristol Myers Squibb), where he held multiple positions including as Vice President, Business Development & Strategy. Prior to Celgene, Mr. Dulac worked as a biopharmaceutical equity research analyst at Barclays Capital and Lehman Brothers and in corporate finance at Pfizer. Mr. Dulac holds a Bachelor of Pharmacy from the University of Pittsburgh and an MBA from Indiana University, Kelley School of Business.
お知らせ • Jun 18Intellia Therapeutics to Present the First-Ever Clinical Data from Patients Redosed with an Investigational in Vivo CRISPR Gene Editing Therapy At the Peripheral Nerve Society Annual Meeting 2024Intellia Therapeutics, Inc., a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, announced the acceptance of an abstract featuring redosing data from the Phase 1 study of NTLA-2001 has been selected for an oral presentation at the Peripheral Nerve Society Annual Meeting, taking place June 22 – 25 in Montreal, Canada. NTLA-2001 is an investigational in vivo CRISPR-based gene editing therapy designed to be a single-dose treatment for transthyretin (ATTR) amyloidosis. In the dose-escalation portion of the Phase 1 study, the initial three patients received the lowest dose of 0.1 mg/kg and subsequently received a follow-on dose of 55 mg. These data will be the first-ever clinical data from patients redosed with an in vivo CRISPR-based gene editing candidate and provide insight on the safety and pharmacodynamic effect. While repeat dosing is not planned for the NTLA-2001 program for ATTR amyloidosis, a redosing option could be an important advantage of Intellia’s non-viral, lipid nanoparticle (LNP)-based delivery platform for future investigational therapies where a target additive effect is desired.
お知らせ • Jun 15Intellia Therapeutics, Inc. Names Brian Goff to its Board of DirectorsIntellia Therapeutics, Inc. announced the appointment of Brian Goff to its board of directors. Mr. Goff joins the Intellia board of directors with over three decades of commercialization, operations and sales and marketing experience at leading biopharmaceutical companies. He is a seasoned and accomplished executive global leader focused on rare diseases. Since 2022, he has been the chief executive officer of Agios Pharmaceuticals and a member of its board of directors. He previously served as executive vice president, chief commercial and global operations officer of Alexion Pharmaceuticals until its acquisition by AstraZeneca in 2021. Prior to Alexion, Mr. Goff was chief operating officer and a member of the board of directors of Neurovance until its acquisition by Otuska Pharmaceuticals. Before joining Neurovance, Mr. Goff served as executive vice president and president of the Hematology Division at Baxalta until its acquisition by Shire. He previously served with Baxter Healthcare Corporation as global hemophilia franchise head. Earlier in his career, Mr. Goff held positions of increasing seniority in sales and marketing with Novartis Pharmaceuticals and the pharmaceutical division of Johnson & Johnson. Mr. Goff holds a bachelor’s degree from Skidmore College and a Master of Business Administration from the Wharton School at the University of Pennsylvania.
分析記事 • Jun 06Here's Why It's Unlikely That Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) CEO Will See A Pay Rise This YearKey Insights Intellia Therapeutics will host its Annual General Meeting on 12th of June Total pay for CEO John Leonard...
Seeking Alpha • Jun 05Intellia Therapeutics Presents Promising In Vivo CRISPR ResultsSummary Intellia Therapeutics has released positive data from its Phase I/II study of NTLA-2002, a CRISPR-based gene editing therapy. The study shows promising results for in vivo gene editing, offering potential treatments for genetic diseases. Robust target engagement and efficacy data pave the way for future Phase II results from Intellia later this year. Intellia has the most advanced in vivo gene editing portfolio, which, while speculative, could represent a huge advance for the field in coming years. Read the full article on Seeking Alpha
お知らせ • Jun 05Intellia Therapeutics, Inc. Announces Positive Long-Term Data from Ongoing Phase 1 Study of NTLA-2002Intellia Therapeutics, Inc. announced long-term data from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. As previously announced, Intellia completed enrollment of the randomized, placebo-controlled Phase 2 study further evaluating the 25 mg and 50 mg doses and plans to report topline results mid-year with detailed results expected to be presented at a medical meeting later in the year. Intellia's ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's ability to protect and maintain its intellectual property position; risks related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates including NTLA-2002; the risk that the results of preclinical studies or clinical studies, such as the ongoing Phase 1/2 clinical study of NTLA-2002, will not be predictive of future results in connection with this study or future studies for NTLA-2002 or Intellia's other product candidates. For a discussion of these and other important factors, any of which could cause Intellia's actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in Intellia's most recent Phase 1/2 clinical study.
分析記事 • May 14Industry Analysts Just Made A Dazzling Upgrade To Their Intellia Therapeutics, Inc. (NASDAQ:NTLA) Revenue ForecastsIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders will have a reason to smile today, with the analysts making...
Seeking Alpha • May 13Intellia Therapeutics: 3 Near-Term Pivotal In Vivo Gene TherapiesSummary Intellia's longstanding collaboration with Regeneron is important in its value proposition. With three cutting-edge late-stage programs, NTLA is among the top tier of gene editing companies. Solid financials underpin the Company. Intellia presents special risks. Read the full article on Seeking Alpha
Reported Earnings • May 10First quarter 2024 earnings released: US$1.13 loss per share (vs US$1.18 loss in 1Q 2023)First quarter 2024 results: US$1.13 loss per share. Revenue: US$28.9m (up 130% from 1Q 2023). Net loss: US$107.4m (loss widened 4.2% from 1Q 2023). Revenue is forecast to grow 60% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in the US.
お知らせ • May 03Intellia Therapeutics, Inc. to Report Q1, 2024 Results on May 09, 2024Intellia Therapeutics, Inc. announced that they will report Q1, 2024 results on May 09, 2024
お知らせ • May 01Intellia Therapeutics, Inc., Annual General Meeting, Jun 12, 2024Intellia Therapeutics, Inc., Annual General Meeting, Jun 12, 2024, at 09:00 US Eastern Standard Time. Agenda: To consider Election of two class II directors to board of directors, each to serve until the 2027 annual meeting of stockholders and until their successor has been duly elected and qualified; to Ratification of the appointment of Deloitte & Touche LLP as independent registered public accounting firm for the fiscal year ending December 31, 2024; to Approval, on a non-binding advisory basis, of the compensation of named executive officers; to Approval of a second amendment to Second Amended and Restated Certificate of Incorporation, as amended, to limit the liability of certain officers of the Company as permitted by recent amendments to the Delaware General Corporation Law; and to Transaction of any other business properly brought before the Annual Meeting or any adjournment or postponement thereof.
お知らせ • Mar 19Intellia Therapeutics, Inc. Announces First Patient Dosed in Phase 3 MAGNITUDE Study of NTLA-2001 as Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced the first patient dosed in the global pivotal, Phase 3 MAGNITUDE trial of NTLA-2001. NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis. The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy. The pivotal Phase 3 MAGNITUDE clinical trial is a randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2001 in approximately 765 patients with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The primary endpoint of the study is a composite endpoint of cardiovascular (CV)-related mortality and CV-related events. Adult patients with hereditary or wild type ATTR-CM will be randomized 2:1 to receive a single 55 mg infusion of NTLA-2001 or placebo.
Seeking Alpha • Feb 29Intellia Therapeutics Is On The MoveSummary Today, we circle back on gene editing concern Intellia Therapeutics, Inc., whose stock has moved up by a third over the past month. The company has potential cures for HAE and ATTR-CM within its pipeline, but commercialization is years away. An updated analysis around Intellia Therapeutics follows in the paragraphs below. Read the full article on Seeking Alpha
お知らせ • Feb 24Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $375.727167 million.Intellia Therapeutics, Inc. has filed a Follow-on Equity Offering in the amount of $375.727167 million. Security Name: Common Stock Security Type: Common Stock Transaction Features: At the Market Offering
分析記事 • Feb 24Intellia Therapeutics, Inc. (NASDAQ:NTLA) Consensus Forecasts Have Become A Little Darker Since Its Latest ReportIntellia Therapeutics, Inc. ( NASDAQ:NTLA ) shareholders are probably feeling a little disappointed, since its shares...
Reported Earnings • Feb 24Full year 2023 earnings: EPS in line with expectations, revenues disappointFull year 2023 results: US$5.42 loss per share. Revenue: US$36.3m (down 30% from FY 2022). Net loss: US$481.2m (loss widened 1.5% from FY 2022). Revenue missed analyst estimates by 28%. Earnings per share (EPS) were mostly in line with analyst estimates. Revenue is forecast to grow 57% p.a. on average during the next 3 years, compared to a 17% growth forecast for the Biotechs industry in the US.
New Risk • Feb 23New major risk - Revenue and earnings growthEarnings are forecast to decline by an average of 0.6% per year for the foreseeable future. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are expected to decline, then in most cases the share price will decline over time as well. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risk Earnings are forecast to decline by an average of 0.6% per year for the foreseeable future. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$554m net loss in 3 years). Significant insider selling over the past 3 months (US$1.4m sold).
お知らせ • Feb 16Intellia Therapeutics, Inc. to Report Q4, 2023 Results on Feb 22, 2024Intellia Therapeutics, Inc. announced that they will report Q4, 2023 results on Feb 22, 2024
Seeking Alpha • Feb 09Intellia's Bet On In-Vivo Breakthroughs: A Perspective On CRISPR's Next FrontierSummary Intellia is adopting a distinct strategy by focusing on in-vivo gene editing, potentially redefining industry standards and expectations. Interim results from Intellia's in-vivo gene-editing program for ATTR amyloidosis show promising safety and efficacy profiles. Intellia is still a cash-burning company, while any commercial therapy is still some time away. Read the full article on Seeking Alpha
分析記事 • Feb 07Is Intellia Therapeutics (NASDAQ:NTLA) In A Good Position To Deliver On Growth Plans?Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...
お知らせ • Feb 01Intellia Therapeutics Announces Publication of Positive Interim Phase 1 Data for NTLA-2002 in Patients with Hereditary Angioedema in the New England Journal of MedicineIntellia Therapeutics, Inc. announced that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks. Additionally, the company remain on track to initiate a global pivotal study for NTLA-2002 in the second half of 2024, subject to regulatory feedback. This marks the second consecutive Intellia in vivo CRISPR- based program to have its initial clinical data published in the New England Journal of medicine, further supporting the immense potential impact its proprietary gene editing platform could have on the future of human health. The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. Intellia's ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE). Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that NTLA-2002 will not be successfully developed and commercialized; and the risk that the results of preclinical stu dies or clinical studies, such as the clinical study of NTLA-2002, will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates. For a discussion of these and other risks and uncertainties, and other risks and uncertainties, and other risks and uncertainties.
Recent Insider Transactions • Jan 07President recently sold US$566k worth of stockOn the 3rd of January, John Leonard sold around 19k shares on-market at roughly US$29.46 per share. This transaction amounted to 2.1% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. This was John's only on-market trade for the last 12 months.
分析記事 • Jan 01Is There An Opportunity With Intellia Therapeutics, Inc.'s (NASDAQ:NTLA) 25% Undervaluation?Key Insights The projected fair value for Intellia Therapeutics is US$40.78 based on 2 Stage Free Cash Flow to Equity...
Recent Insider Transactions Derivative • Dec 10Independent Director notifies of intention to sell stockJohn Crowley intends to sell 33k shares in the next 90 days after lodging an Intent To Sell Form on the 6th of December. If the sale is conducted around the recent share price of US$29.86, it would amount to US$979k. Since March 2023, John's direct individual holding has increased from 2.09k shares to 7.29k. Company insiders have collectively sold US$940k more than they bought, via options and on-market transactions in the last 12 months.
お知らせ • Dec 06Intellia Therapeutics, Inc. Announces Resignation of John F. Crowley from the Board of Directors of Company and Its CommitteesIntellia Therapeutics, Inc. announced that John F. Crowley notified company of his intent to resign from the Board of Directors of the Company and its committees, effective December 5, 2023. Mr. Crowley currently serves on the Nominating and Governance Committee and on the Compensation and Talent Development Committee of the Board. His resignation is not the result of any disagreement with the Company on any matter relating to the Company’s operations, policies or practices. The Company thanks Mr. Crowley for his long, dedicated service on the Board and wishes him well in his future pursuits.
Reported Earnings • Nov 10Third quarter 2023 earnings: EPS exceeds analyst expectationsThird quarter 2023 results: US$1.38 loss per share. Revenue: US$12.0m (down 9.6% from 3Q 2022). Net loss: US$122.2m (loss widened 7.9% from 3Q 2022). Revenue was in line with analyst estimates. Earnings per share (EPS) surpassed analyst estimates by 8.1%. Revenue is forecast to grow 60% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in the US.
お知らせ • Nov 03Intellia Therapeutics, Inc. Presents New Interim Data from the Ongoing Phase 1 Study of NTLA-2001 At the 4th International ATTR Amyloidosis MeetingIntellia Therapeutics, Inc. presented additional interim results from its ongoing Phase 1 study of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. Results were presented in an oral presentation at the 4th International ATTR Amyloidosis Meeting, held Nov. 2–3 in Madrid, Spain. In the newly reported dose-expansion portion, administration of NTLA-2001 at the 55 mg and 80 mg dose led to deep serum TTR reductions consistent with the results previously reported [1] from patients in the dose-escalation portion who received the corresponding weight-based dose, 0.7 mg/kg and 1.0 mg/kg, respectively. Across all patients who received a dose of 0.3 mg/kg or higher (n=62), the median serum TTR reduction was 91% and the median absolute residual serum TTR concentration was 17 µg/mL at day 28. The persistently low levels of TTR concentration are expected to reduce the rate of ongoing amyloid formation and hold the possibility for amyloid clearance to reverse the symptoms of the disease. If clinically validated, the use of absolute residual TTR concentration levels could become a new benchmark for evaluating ATTR amyloidosis. The reduction of serum TTR compared to baseline was sustained through the latest follow-up. With 29 patients now reaching at least 12 months of follow-up, all patients continued to show a long-lasting response with no evidence of loss in activity over time. NTLA-2001 was generally well tolerated across all patients and at all dose levels tested. The most commonly reported adverse events were infusion-related reactions, which occurred in 38% of patients. The majority of adverse events, including infusion-related reactions, were Grade 1 or 2 in severity, transient and resolved spontaneously. Other adverse events that were reported in greater than 10% of patients included headache, diarrhea and back pain, and were all Grade 1 or 2. All patients received a full dose of NTLA-2001 and remain on study. No dose-limiting toxicities were observed. Based on the safety and activity of NTLA-2001, the 55mg dose has now been selected to be evaluated in the upcoming pivotal Phase 3 study. These data support NTLA-2001’s continued development as a potential one-time treatment to permanently inactivate the TTR gene and reduce the disease-causing protein in people living with ATTR amyloidosis. As previously announced [2], Intellia recently received IND clearance from the FDA to begin a Phase 3 trial of NTLA-2001 for ATTR-CM and expects to initiate the global pivotal trial by the end of this year. Additionally, the Company is actively preparing for a Phase 3 trial for ATTRv-PN.
Board Change • Nov 01Insufficient new directorsNo new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 8 experienced directors. 4 highly experienced directors. Independent Director John Crowley was the last director to join the board, commencing their role in 2020. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment.
分析記事 • Oct 20Intellia Therapeutics (NASDAQ:NTLA) Is In A Good Position To Deliver On Growth PlansEven when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...
お知らせ • Oct 19Intellia Therapeutics Announces FDA Clearance of Investigational New Drug Application to Initiate a Pivotal Phase 3 Trial of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis with CardiomyopathyIntellia Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy. The global Phase 3 study of NTLA-2001, an in vivo CRISPR-based gene editing candidate, is expected to initiate by year-end 2023. Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2001 could potentially be the first single-dose treatment for ATTR amyloidosis. NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or through a vein, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical and clinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic. Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron. The global Phase 1 trial is an open-label, multi-center, two-part study of NTLA-2001 in adults with hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) or transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The trial is now closed for enrollment.
New Risk • Aug 06New major risk - Revenue and earnings growthEarnings have declined by 41% per year over the past 5 years. This is considered a major risk. Ultimately, shareholders want to see a good return on their investment and that generally comes from sharing in the company's profits. If profits are declining over an extended period, then in most cases the share price will decline over time unless the company can turn around its fortunes. A trend of falling earnings can be very difficult to turn around. If the company is well already established it may also be a sign the company has matured and is in decline. In addition, if the company pays dividends it will also likely need to reduce or cut them, striking a dual blow to total shareholder returns. Currently, the following risks have been identified for the company: Major Risk Earnings have declined by 41% per year over the past 5 years. Minor Risk Shareholders have been diluted in the past year (16% increase in shares outstanding).
Reported Earnings • Aug 03Second quarter 2023 earnings: Revenues exceed analysts expectations while EPS lags behindSecond quarter 2023 results: US$1.40 loss per share (further deteriorated from US$1.33 loss in 2Q 2022). Revenue: US$13.6m (down 3.1% from 2Q 2022). Net loss: US$123.7m (loss widened 23% from 2Q 2022). Revenue exceeded analyst estimates by 20%. Earnings per share (EPS) missed analyst estimates by 7.1%. Revenue is forecast to grow 59% p.a. on average during the next 3 years, compared to a 16% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 36% per year but the company’s share price has increased by 25% per year, which means it is well ahead of earnings.
お知らせ • Jul 22Intellia Therapeutics, Inc. Appoints Eliana Clark, Phd, Executive Vice President and Chief Technical OfficerCulture Biosciences welcomed Eliana Clark, PhD, Executive Vice President and Chief Technical Officer at Intellia Therapeutics, Inc., to the company’s Advisory Board. The group of seasoned industry leaders provides Culture’s leadership with insights and recommendations as the company establishes new partnerships and pursues new growth opportunities in biotech and biopharma. Prior to her current role at Intellia, Eliana Clark was the company’s Senior Vice President, Technical Operations and Quality. Before she joined Intellia, Eliana held leading roles in Product and Portfolio Management, Manufacturing, Manufacturing Sciences, Process Development and CMC-Regulatory, at Biogen and Sanofi/Genzyme, respectively. Prior to joining industry, she was a professor at Tufts University in the Chemical and Biological Engineering Department. Eliana held post-doc roles in Biochemical Engineering at the University of Delaware and the Worcester Polytechnic Institute. She has a PhD and a Bachelor of Engineering degree in Chemical Engineering from the Universidad Nacional del Litoral in Santa Fe, Argentina, and has graduated from the Greater Boston Executive Program at MIT Sloan.
お知らせ • Jun 12Intellia Therapeutics, Inc. Announces New Positive Clinical Data from Phase 1 Study of NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema (HAE)Intellia Therapeutics, Inc. announced updated interim results from the Phase 1 portion of the ongoing Phase 1/2 study of NTLA-2002. NTLA-2002 is an in vivo, systemically administered CRISPR candidate being developed as a single-dose treatment for hereditary angioedema (HAE). The data, with a cut-off date of February 17, 2023, were shared in a late-breaking presentation at the European Academy of Allergy and Clinical Immunology (EAACI) Hybrid Congress 2023, being held June 9-11 in Hamburg, Germany, and virtually. In the Phase 1 portion of the study, single doses of 25 mg (n=3), 50 mg (n=4) and 75 mg (n=3) of NTLA-2002 were administered via intravenous infusion, and HAE attacks and plasma kallikrein protein levels were measured for each patient. The first analysis of HAE attack rates occurred at the end of the pre-specified 16-week primary observation period. HAE attacks and plasma kallikrein protein levels will continue to be assessed through the end of the study. Across all patients, a 95% mean reduction in monthly attack rate was observed after a single dose of NTLA-2002 through the latest follow-up. The median duration of follow-up was 9.0 months (range of 5.6 - 14.1 months). At each dose level tested, a robust level of HAE attack rate reduction was achieved. Importantly, the elimination of HAE attacks has been sustained and long lasting. The first three patients dosed in the study with the longest follow-up to date have experienced attack-free durations of approximately one year or longer. Additionally, the reduction in HAE attacks has been persistent in patients with the most severe HAE symptoms. The three patients with the highest historic monthly HAE attack rates at the start of the study (16.8, 14.0 and 4.4 attacks per month, respectively) all became attack free by the end of the 16-week primary observation period and remained free of attacks through the latest follow-up. The longest attack-free duration in this patient group is 11.5 months and ongoing. All nine patients who achieved greater than 60% plasma kallikrein reduction, the target level expected to yield a highly meaningful clinical response, remain completely attack free since the 16-week observation period. There was one patient in the lowest 25 mg dose cohort who did not achieve the targeted 60% minimum kallikrein reduction post-NTLA-2002 administration. Following 12.3 months of being attack free, this patient reported a single, mild HAE attack after experiencing minor hand swelling precipitated by a sports injury. The event did not require any medical intervention or acute therapy. The patient has not experienced any subsequent HAE attacks following this event. Six of the 10 patients were receiving long-term HAE prophylaxis medications prior to the administration of NTLA-2002. Subsequently, they were permitted to withdraw their medication at the investigator’s discretion. All six patients have discontinued their prophylactic therapy and have not experienced any subsequent HAE attacks. As previously reported, administration of NTLA-2002 led to dose-dependent, robust and durable reductions in plasma kallikrein. These deep reductions in plasma kallikrein continue to be sustained through the latest follow-up, which ranged from 24 to 48 weeks across all three dose cohorts. At all three dose levels, NTLA-2002 has been well tolerated, and the majority of adverse events were mild in severity. Consistent with previously reported results, the most frequent adverse events were infusion-related reactions and fatigue, which were mostly Grade 1 and resolved within two days. There have been no dose-limiting toxicities, no serious adverse events and no adverse events of Grade 3 or higher observed to date. No clinically significant laboratory abnormalities were observed in any patient. As previously announced, the Phase 2 portion of this Phase 1/2 clinical trial of NTLA-2002 has begun dosing patients, and Intellia expects to complete enrollment in the second half of this year.
