공시 • Feb 28
Avidity Biosciences, Inc.(NasdaqGM:RNAM) dropped from NASDAQ Biotechnology Index Avidity Biosciences, Inc. has been removed from NASDAQ Biotechnology Index . 공시 • Feb 20
Avidity Biosciences, Inc. Announces Final Results from the Completed Phase 1/2 Marine Trial of Delpacibart Etedesiran (Del-Desiran) for Treatment of Myotonic Dystrophy Type 1 Avidity Biosciences, Inc. announced that the final results from the completed Phase 1/2 MARINA® trial of delpacibart etedesiran (del-desiran) in people living with myotonic dystrophy type 1 (DM1) will be published in the February 19 issue of The New England Journal of Medicine (NEJM). The manuscript is titled ‘An Antibody Oligonucleotide Conjugate for Myotonic Dystrophy Type 1.’DM1 is an underrecognized, progressive and often fatal neuromuscular disease with no disease modifying therapies. Del-desiran is an investigational treatment designed to address the underlying genetic root cause of DM1 by reducing total levels of the toxic, DMPK (myotonic dystrophy protein kinase) mRNA. The accumulation of these toxic mRNA sequester key RNA-regulatory proteins that subsequently lead to missplicing of several downstream genes, resulting in the diverse clinical manifestations of disease. The Phase 1/2 MARINA trial was a randomized, double-blind, placebo-controlled study designed to evaluate the safety and tolerability of single and multiple ascending doses of del-desiran administered intravenously in adults with DM1 for six months. Data were assessed from 38 participants who were randomized 3:1 to receive one dose of 1 mg/kg del-desiran, three doses of either 2 mg/kg del-desiran or 4 mg/kg del-desiran (reflected as siRNA dose), or placebo. The primary endpoint of the study was to evaluate the safety and tolerability of del-desiran. Exploratory endpoints were to evaluate the clinical activity of del-desiran across multiple efficacy measures. Results from the Phase 1/2 MARINA study of del-desiran published in NEJM demonstrated: Effective delivery of del-desiran (siRNA) to muscle, with a mean ~ 40% reduction in DMPK mRNA across all treated participants. Splicing improvements in a key set of muscle-specific genes following treatment with del-desiran 2 mg/kg and 4 mg/kg. Improvements in exploratory functional measures including: Hand function/myotonia (video hand opening time, or vHOT); Muscle strength (Quantitative Muscle Testing, or QMT total score); Mobility (10-Meter Walk/Run Test, or 10mWRT, and Timed Up and Go test, or TUG); DM1-Activ, a patient-reported outcome that measures activities of daily living (e.g., taking a shower, visiting family or friends, and walking up stairs). Acceptable safety and tolerability with most treatment emergent adverse events (TEAEs) mild or moderate in participants with DM1 and did not result in discontinuation. Two severe, serious AEs occurred in two participants in the 2 mg/kg and 4 mg/kg dose cohorts, with one participant discontinuing the study in the 4 mg/kg cohort. One of these SAEs was deemed drug-related. Del-desiran (4 mg/kg) is currently being assessed in the global Phase 3 HARBOR™ study in people living with DM1 who are age 16 and older and in the ongoing HARBOR open-label extension (HARBOR-OLE™) trial with all the participants who completed the Phase 1/2 MARINA trial. The global Phase 3 HARBOR study completed enrollment in July 2025 and a 54-week topline data readout is expected in the second half of 2026. 공시 • Nov 19
Avidity Biosciences, Inc. Announces U.S. Managed Access Program for Investigational Therapy Del-Zota in DMD44 Avidity Biosciences, Inc. announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the United States. Under an FDA-authorized treatment protocol, Avidity will provide del-zota to eligible boys and men with DMD44 through participating healthcare providers. Enrollment is anticipated to begin by year end, and participants in EXPLORE44-OLE will have the option to transition to the MAP as they complete 2 years of treatment. Avidity aligned with FDA on a path forward for a BLA submission for del-zota following an October 2025 pre-BLA meeting, with the submission planned for 2026 for accelerated approval. Participants will transition to commercial drug supply upon future potential FDA approval and product availability. One participant discontinued from EXPLORE44-Ole following an event of hypersensitivity. Del-zota has received Rare Pediatric Disease, Orphan Drug, Fast Track and Breakthrough Therapy designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). 공시 • Oct 27
Novartis AG (SWX:NOVN) entered into an agreement to acquire Avidity Biosciences, Inc. (NasdaqGM:RNA) from RTW Biotech Opportunities Ltd (LSE:RTW) and others for $10.6 billion. Novartis AG (SWX:NOVN) entered into an agreement to acquire Avidity Biosciences, Inc. (NasdaqGM:RNA) from RTW Biotech Opportunities Ltd (LSE:RTW) and others for $10.6 billion on October 26, 2025. A cash consideration valued at $72 per share will be paid by Novartis AG of Consideration totals $12 billion in cash. The acquisition will follow the separation of Avidity’s early-stage precision cardiology programs. The proposed acquisition aligns with the long-term neuroscience strategy of Novartis, expanding the company’s pipeline with potential near-term launches in genetically defined diseases with high unmet need. Prior to the closing of the merger, Avidity will transfer to SpinCo, a wholly owned subsidiary of Avidity, the early-stage precision cardiology programs and collaborations of Avidity.
The transfer includes certain Avidity assets whose transfer will trigger a right of first negotiation with an existing collaboration partner of Avidity. As part of the agreement, Avidity will separate its early-stage precision cardiology programs into a new company (“SpinCo”) prior to closing.
Under the terms of the transactions, which have been unanimously approved by the Boards of Directors of both companies, Novartis, through a merger with a newly formed indirect wholly owned subsidiary, will acquire all outstanding shares of Avidity. The acquisition by Novartis of Avidity is subject to the completion of a spin-off or a sale of SpinCo until closing, Novartis and Avidity will continue to operate as separate and independent companies and other customary closing conditions, including the receipt of regulatory approvals and the approval of Avidity stockholders. The companies expect the merger to close in the first half of 2026.
Goldman Sachs & Co. LLC and Barclays Capital Inc. are serving as financial advisors to Avidity, and Kirkland & Ellis LLP as its legal advisor. 공시 • Oct 14
Avidity Biosciences, Inc. Announces Positive Pre-BLA Meeting with U.S. FDA for del-zota in DMD44 with a Submission Planned for First Quarter of 2026 Avidity Biosciences, Inc. announced that the Company completed a positive pre-BLA meeting with the U.S. Food and Drug Administration (FDA) regarding its upcoming BLA submission of delpacibart zotadirsen (del-zota). Del-zota, which received Breakthrough Therapy designation, is an investigational drug being evaluated as a potential treatment for people living with Duchenne muscular dystrophy who have gene mutations amenable to exon 44 skipping (DMD44).
The timing for the BLA submission has been updated to First Quarter 2026 from previous guidance of year end 2025 to ensure the FDA receives additional data to support the chemistry, manufacturing, and controls (CMC) package at time of submission. Avidity remains highly confident in the potential of del-zota and looks forward to filing a BLA for del-zota in First Quarter 2026, which will be the Company's first of three planned BLA submissions over a 12-month period. Additionally, Avidity continues to prepare a confirmatory study to support full global approval of del-zota. 공시 • Sep 12
Avidity Biosciences, Inc. has completed a Follow-on Equity Offering in the amount of $600 million. Avidity Biosciences, Inc. has completed a Follow-on Equity Offering in the amount of $600 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 15,000,000
Price\Range: $40
Discount Per Security: $2.3 공시 • Aug 07
Novartis Reportedly Weighs Deal for Biotech Avidity to Boost Drug Pipeline Novartis AG (SWX:NOVN) has made a takeover approach for rare disease-focused biotech Avidity Biosciences, Inc. (NasdaqGM:RNA), as Big Pharma continues to hunt for deals to offset looming patent cliffs, according to people familiar with the matter. Novartis has been evaluating a bid for Avidity, which had a market value of about $4.7 billion on August 6, 2025, and expressed interest in an acquisition in recent weeks, in a move aimed at boosting the drugmaker’s pipeline of medicines targeting rare genetic disorders, the people said. San Diego-based Avidity has three medicines in clinical trials, which could be approved as early as 2027, that treat different forms of muscular dystrophy — a potentially fatal muscle-wasting disease. Avidity is working with advisers to assess options, the people added. There were no guarantees that a deal would be secured, however, as discussions were at an early stage, the people said. They cautioned that another suitor could emerge or talks could collapse. Novartis and Avidity declined to comment. Shares in Avidity traded up 23% after the Financial Times reported Novartis’s interest. Its stock is up 50% so far this year. 공시 • Jul 23
Avidity Biosciences, Inc. Receives FDA Breakthrough Therapy Designation for Delpacibart Zotadirsen (Del-zota) for the Treatment of DMD in People with Mutations Amenable to Exon 44 Skipping Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart zotadirsen (del-zota) for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44). Del-zota is currently being assessed in the Phase 2 EXPLORE44 Open-Label Extension (EXPLORE44-OLE™?) trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. In the completed Phase 1/2 EXPLORE44®? trial for people living with D MD44, del-zota demonstrated statistically significant increases in exon skipping, a substantial increase in dystrophin production, a significant and sustained reduction in creatine kinase levels to near normal and consistent favorable safety and tolerability. Avidity plans to present topline and functional data from the ongoing, fully enrolled Phase 2 EXPLORE44-OLE trial in the fourth quarter of 2025. Avidity's commercial preparations for a potential U.S. launch of del-zota in DMD44 following FDA approval are underway. In addition to receiving Breakthrough Therapy designation, del-zota has previously been granted Orphan designation by the FDA and the European Medicines Agency (EMA) and Rare Pediatric Disease and Fast Track designation by the FDA for the treatment of DMD44. The EXPLORE44™? trial was a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial that enrolled 26 participants with Duchenne muscular dystroph mutations amenable to exon44 skipping (DMD44). Enrollment has been completed in the EXPLORE44-Ole study, with 23 participants who were previously enrolled in the Phase 1/2 EXPLore44®? trial and 16 participants who directly enrolled in the EXPLORE44®? study. Del-zota has received Rare Pediatric Disease, Orphan Drug, Fast Track and Breakthrough Therapy designation by the U.S. Food & Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EM). Avidity's plans for potentially three potential launches and the timing thereof; Avidity's plans to present topline & functional data from the ongoing EXPLORE44-O LE™? study and the timing thereof; the characterization of data associated with del-zota and the impact of such data on the advancement of del-zota; Avidity's plans for DMD candidates beyond del-zota for DMD44; the design, goals and status of the EXPLORE44- OLE study; Avidity's plans and expectations to advance its clinical programs, and the timing thereof; and Avidity's platform, planned operations and programs. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: preliminary results of a clinical trial are not necessarily indicative of final results; further analysis of existing clinical data and analysis of new data may lead to conclusions different from those established as of the data cutoff dates in the clinical trial of del-zota, and such data may not meet Avidity's or regulators' expectations; unexpected adverse side effects to, or inadequate efficacy of, del-zota that may delay or limit its development, regulatory approval and/or commercialization; later developments with the FDA and other global regulators that that may be developed for DMD44. 공시 • Apr 30
Avidity Biosciences, Inc., Annual General Meeting, Jun 10, 2025 Avidity Biosciences, Inc., Annual General Meeting, Jun 10, 2025. 공시 • Apr 09
Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran (del-desiran) for Treatment of Myotonic Dystrophy Type 1 Avidity Biosciences, Inc. announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation (ODD) to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1), an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA). Avidity has aligned with global regulators on the registrational path for del-desiran for the treatment of DM1, which informed the design of the ongoing Phase 3 HARBOR™ study. Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan. Japan's MHLW grants Orphan Drug designation to drugs in development for the treatment of diseases that affect fewer than 50,000 patients in Japan and for which there is a high unmet medical need. An investigational therapy is eligible to qualify for Orphan Drug designation if there is no approved alternative treatment option or if there is high efficacy or safety expected compared to existing treatment options. Orphan Drug designation provides certain benefits, including prioritized consultation regarding clinical development, reduced consultation fees, tax incentives, priority review of applications, reduced application fees, and extended registration validity period. 공시 • Mar 31
Avidity Biosciences, Inc. Completes Enrollment in Biomarker Cohort in Phase 1/2 Fortuda™? Trial for Delpacibart Braxlosiran (Del-Brax) in People Living with Facioscapulohumeral Muscular Dystrophy Avidity Biosciences, Inc. announced the completion of enrollment in the biomarker cohort in the Phase 1/2 FORTITUDE clinical trial of delpacibart braxlosiran (del-brax) in people living with facioscapulohumeral muscular dystrophy (FSHD). A total of 51 participants were enrolled in the FORTITUDE biomarker cohort. Avidity is on track to deliver multiple updates from the del-brax program in second quarter including: Regulatory alignment on a potential accelerated approval path in the U.S. for the ongoing FORTITUDE biomarker cohort; Regulatory alignment on the design of the global Phase 3 trial as well as initiation of the trial; and Topline data from the dose escalation cohorts in the FORTITUDE trial. The FORTITUDE trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate single and multiple doses of delpacibart braXlosiran or del-brax in 90 participants with facioscapulohUMeral muscular dystrophy (FSHD). Avidity has completed enrollment in the dose escalation cohorts and identified 2 mg/kg every six weeks of del-brax as the dose for future clinical trials. This trial will continue to evaluate the safety, tolerability, PK, PD, and efficacy of del-brax in participants who enrolled in the randomized, placebo-controlled, Phase 1/2 FORTITuDE clinical trial. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted Orphan designation for del-brax and the FDA has granted del-brax Fast Track designation. FSHD is an autosomal dominant disease caused by the aberrant expression of the DUX4 (double homeobox 4) gene in the skeletal muscle, which activates genes that are toxic to muscle cells and leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function.letal muscle weakness results in physical limitations throughout the whole body, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions and serious speech impediments. Such forward-looking statements include, but are not limited to, statements regarding: Avidity's plans to present topline data from the dose escalation cohorts of the FORTITUDE study and the timing thereof; Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the data and results produced in the FOR TITUDE trial and FORTITUDE-OLE as of the most recent respective cutoff updates may not be indicative of final results, may not support BLA submissions or accelerated approvals, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; even if approved, Avidity may not be able to execute a successful product launch for del-brax; unexpected adverse side effects to, or inadequate efficacy of, or inadequate efficacy of, and other regulatory update. 공시 • Mar 18
Avidity Biosciences, Inc. Announces Positive Topline Del-Zota Data Demonstrating Consistent, Statistically Significant Improvements in Dystrophin, Exon Skipping and Creatine Kinase in People Living with Duchenne Muscular Dystrophy Avidity Biosciences, Inc. announced positive del-zota topline data from the Phase 1/2 EXPLORE44®? trial in people living with Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44) demonstrating consistent, statistically significant improvements in dystrophin, exon skipping and creatine kinase as well as favorable safety and tolerability across the dose cohorts. The data will be highlighted in an oral and poster presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, being held March 16-19, 2025, in Dallas, Texas. Del-zota has been granted Orphan designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has also granted del-zota Rare Pediatric Disease and Fast Track designations. Based on interactions with FDA on accelerated approval, believe dystrophin data from EXPLORE44 combined with the safety data from fully enrolled EXPLORE44-OLE trial will support planned BLA submission at the end of this year. The data presented at MDA highlight the consistent data across all parameters in both the 5 mg/kg and 10 mg/kg cohorts of del-zota, including: Targeted delivery of PMOs resulting in tissue concentrations of approximately 200nM in skeletal muscle; Following alignment with FDA on the accelerated approval path late last year, including dose selection, Avidity's commercial preparations for a potential U.S. launch of del-zota in DMD44 are well underway. The EXPLORE44®? Phase 1/2 Trial was a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial that enrolled 26 participants with Duchenne muscular dy Strophy mutations amenable to exon44 skipping (DMD44). Enrollment has been completed in the EXPLORE44-Ole study, with 23 participants who were previously enrolled in the Phase 1/2 EXplORE44®? trial and 16 participants who directly enrolled in the EXPLORE44®? study. Del-zota has received Rare Pediatric Disease, Orphan Drug and Fast Track designations by the U.S. food and Drug Administration (FDA). Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in Avidity's business and beyond its control, including, without limitation: the data and results produced in the EXPLORE44 trial as of the most recent cutoff date may not be indicative of final results, may not support a BLA submission or accelerated approval, may not be satisfactory to the FDA and other regulators, and new analyses of existing data and results may produce different conclusions than established as of the date hereof; the FDA and other regulators may change their positions on the status of del-zota and its clinical development; even if approved, Avidity may not be able to execute any successful product launches; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven; potential potential potential U.S. Food and drug candidates based on its AOC platforms is unproven; potential potential U.S. and the European Medicines Agency. 공시 • Aug 16
Avidity Biosciences, Inc. has completed a Follow-on Equity Offering in the amount of $300.12 million. Avidity Biosciences, Inc. has completed a Follow-on Equity Offering in the amount of $300.12 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 7,320,000
Price\Range: $41
Discount Per Security: $2.46 공시 • Aug 10
Avidity Biosciences, Inc. has filed a Follow-on Equity Offering in the amount of $400 million. Avidity Biosciences, Inc. has filed a Follow-on Equity Offering in the amount of $400 million.
Security Name: Common Stock
Security Type: Common Stock
Transaction Features: At the Market Offering 공시 • Aug 09
Avidity Biosciences Announces Positive AOC 1044 Data Demonstrated Significant Increase of 25% in Dystrophin Production and Reduction of Creatine Kinase Levels to Near Normal in People Living with Duchenne Muscular Dystrophin Amenable to Exon 44 Skipping in the Phase 1/2 EXPLORE44 Trial Avidity Biosciences, Inc. announced positive AOC 1044 5 mg/kg data demonstrated unsurpassed delivery of phosphorodiamidate morpholino oligomers (PMO) concentrations to skeletal muscle, statistically significant increase of 25% of normal in dystrophin production and statistically significant increase of 37% in exon 44 skipping in people living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the Phase 1/2 EXPLORE44 clinical trial. Del-zota has been granted Orphan designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA has also granted del-zota Rare Pediatric and Fast Track designations. In the Phase 1/2 EXplORE44 study, del-zota demonstrated: Unsurpassed delivery of PMO of 200 nM in skeletal muscle; Statistically significant 37% increase in exon 44 skipping and up to 66% exon 44 skipping; Statistically significant increase of 25% of regular dystrophin production and restored total dystrophin up to 54% of normal; Reduction in creatine kinase levels to near normal with greater than 80% reduction compared to baseline; Favorable safety and tolerability with most treatment emergent adverse events (AEs) mild or moderate in participants with DMD44. 공시 • Aug 06
Avidity Biosciences, Inc. Announces Executive Changes Avidity Biosciences, Inc. announced appointment of John B. Moriarty, Jr., J.D., as Chief Legal Officer and Corporate Secretary, effective immediately. With almost 30 years of industry experience, Mr. Moriarty brings extensive legal expertise and a proven track record of successfully guiding leading global biotech companies through transformational growth. Mr. Moriarty succeeds John W. Wallen III, Ph.D., J.D., who served as General Counsel since 2019 and has been head of intellectual property since Avidity's inception in 2014. Dr. Wallen will remain with the company through year-end and will transition as a consultant. Mr. Moriarty was most recently the Executive Vice President, Chief Legal Officer and Corporate Secretary at Mirati Therapeutics where he led the global legal team, executed business transactions and enhanced corporate governance, which was acquired by Bristol Myers Squibb. Previously, he was the Chief Legal Officer at Olema Oncology, leading all legal, compliance, and governance functions during its evolution from a private startup to a public development-stage company. Prior to that, he was the Executive Vice President and General Counsel for Portola Pharmaceuticals, Inc., which was acquired by Alexion Pharmaceuticals, Inc. Prior to Portola, Mr. Moriarty was General Counsel of Alexion and was General Counsel and Chief Legal Officer at Elan Corporation plc. Mr. Moriarty earned a B.A. from the University of Virginia and a J.D. from the University of Georgia School of Law cum laude. 공시 • Jun 14
Avidity Biosciences, Inc. has filed a Follow-on Equity Offering in the amount of $300 million. Avidity Biosciences, Inc. has filed a Follow-on Equity Offering in the amount of $300 million.
Security Name: Common Stock
Security Type: Common Stock 공시 • May 16
Avidity Biosciences, Inc. Appoints Simona Skerjanec as Board of Directors Avidity Biosciences, Inc. announced the appointment of Simona Skerjanec, M.Pharm, MBA to its board of directors. Ms. Skerjanec brings nearly three decades of global experience in the pharmaceutical industry with a strong track record in developing and launching therapies, as well as shaping corporate strategy to deliver transformative treatments to people living with serious diseases. Ms. Skerjanec has led multiple research and development efforts that have resulted in regulatory approvals and launches of commercial therapies in therapeutic areas including neurology and cardiology in the U.S. and other countries. Most recently, Ms. Skerjanec was the Senior Vice President and Global Neuroscience Head at Roche in Switzerland and led the business and global strategy for Roche's portfolio of neurological and rare diseases, including Ocrevus® for the treatment of multiple sclerosis and a novel monoclonal antibody for the treatment of Alzheimer's disease. During her nine-year tenure at Roche, she also served as a General Manager of Roche in Portugal. Prior to joining Roche, Simona was Senior Vice President and Cardiovascular franchise head at The Medicines Company where she held various roles of increasing responsibilities in development and commercialization. She also held positions at Eli Lilly, Pfizer and Johnson & Johnson. Reported Earnings • May 10
First quarter 2024 earnings: EPS and revenues exceed analyst expectations First quarter 2024 results: US$0.79 loss per share (further deteriorated from US$0.74 loss in 1Q 2023). Revenue: US$3.54m (up 59% from 1Q 2023). Net loss: US$68.9m (loss widened 31% from 1Q 2023). Revenue exceeded analyst estimates by 38%. Earnings per share (EPS) also surpassed analyst estimates by 8.7%. Revenue is forecast to grow 64% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in the US. Over the last 3 years on average, earnings per share has fallen by 10% per year but the company’s share price has increased by 8% per year, which means it is well ahead of earnings. 공시 • May 09
Avidity Biosciences, Inc. Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1 Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1). Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT) and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity recently reported positive long-term MARINA-OLE™ data demonstrating reversal of disease progression in adults living with DM1 across multiple endpoints including vHOT, muscle strength and DM1-Activ when compared to natural history data. In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1. New Risk • Apr 05
New minor risk - Insider selling There has been significant insider selling in the company's shares over the past 3 months. Total value of shares sold: US$638k This is considered a minor risk. There are several reasons why an insider may be selling, including to cover a tax obligation or pay for some other expense. However, we generally consider it a negative if insiders have been selling, especially if they do so below the current price. It implies that they considered a lower price to be reasonable. This is a weak signal, but if there is a pattern of unexplained selling, it can be a sign the insider believes the company's stock is overpriced. Note: We only include open market transactions and private dispositions of directly owned stock by individuals, not by corporations or trusts. Currently, the following risks have been identified for the company: Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$301m net loss in 3 years). Share price has been volatile over the past 3 months (12% average weekly change). Shareholders have been diluted in the past year (35% increase in shares outstanding). Significant insider selling over the past 3 months (US$638k sold). Recent Insider Transactions • Apr 05
Director recently sold US$542k worth of stock On the 3rd of April, Arthur Levin sold around 20k shares on-market at roughly US$27.11 per share. This transaction amounted to 7.3% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Insiders have been net sellers, collectively disposing of US$638k more than they bought in the last 12 months. 공시 • Mar 20
Avidity Biosciences, Inc. announced that it has received $400.208766 million in funding On March 19, 2024, Avidity Biosciences, Inc. closed the transaction. The transaction included participation from 45 investors. Major Estimate Revision • Mar 06
Consensus revenue estimates increase by 140% The consensus outlook for revenues in fiscal year 2024 has improved. 2024 revenue forecast increased from US$5.50m to US$13.2m. Forecast losses expected to reduce from -US$3.41 to -US$3.13 per share. Biotechs industry in the US expected to see average net income decline 8.7% next year. Consensus price target up from US$37.50 to US$44.50. Share price rose 25% to US$19.14 over the past week. 공시 • Mar 05
Avidity Biosciences Announces Positive Aoc 1001 Long-Term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 Harbor™ Trial Avidity Biosciences, Inc. announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024. Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran. Del-desiran (AOC 1001) is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Recent Insider Transactions Derivative • Mar 04
President notifies of intention to sell stock Sarah Boyce intends to sell 84k shares in the next 90 days after lodging an Intent To Sell Form on the 1st of March. If the sale is conducted around the recent share price of US$18.30, it would amount to US$1.5m. For the year to December 2019, Sarah's total compensation was 11% salary and 89% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2023, Sarah's direct individual holding has increased from 3.35k shares to 44.01k. Company insiders have collectively sold US$39k more than they bought, via options and on-market transactions in the last 12 months. Reported Earnings • Mar 01
Full year 2023 earnings: EPS and revenues miss analyst expectations Full year 2023 results: US$2.91 loss per share. Net loss: US$212.2m (loss widened 22% from FY 2022). Revenue missed analyst estimates by 51%. Earnings per share (EPS) also missed analyst estimates by 5.4%. 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. 공시 • Feb 21
Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, the company's investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) in people living with mutations amenable to exon 44 skipping (DMD44). AOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA. DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts at a very young age. Currently, there are no therapies approved targeting exon 44. In December 2023, Avidity reported positive AOC 1044 data in healthy volunteers from the EXPLORE44 trial. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMO) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production. AOC 1044 delivered unprecedented concentrations of PMO in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024. The FDA defines a "rare pediatric disease" as a serious or life-threatening disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval for a drug or biologic for a rare pediatric disease may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product. 공시 • Dec 13
Avidity Biosciences Reports Positive Data Demonstrating Aoc 1044 Delivers Unprecedented Concentrations of Pmo in Muscle Following A Single Dose in Healthy Volunteers from Phase 1/2 Explore44™ Trial for Duchenne Muscular Dystrophy Avidity Biosciences, Inc. announced positive AOC 1044 data in healthy volunteers from the Phase 1/2 EXPLORE44™ clinical trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44). AOC 1044 delivered unprecedented concentrations of phosphorodiamidate morpholino oligomers (PMO) in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle following a single dose compared to peptide conjugated PMOs in healthy volunteers. AOC 1044 was well tolerated, demonstrated statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 and increased exon skipping in all participants. Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024. AOC 1044 is designed to deliver PMO to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production. AOC 1044 has been granted Orphan Designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and Fast Track Designation by the FDA. AOC 1044 is the first of multiple AOCs the company is developing for DMD. Phase 1/2 EXPLORE44 Healthy Volunteer Data, AOC 1044 delivered unprecedented, dose-dependent increases in PMO concentrations in skeletal muscle following a single dose of 5 mg/kg or 10 mg/kg, providing up to 50-times greater concentrations of PMO in skeletal muscle when compared to a single dose of peptide conjugated PMOs in healthy volunteers. AOC 1044 produced statistically significant exon 44 skipping compared to placebo of up to 1.5% in healthy volunteers after a single dose of 10 mg/kg AOC 1044 at Day 29. AOC 1044 increased exon skipping in all participants. AOC 1044 was well tolerated in healthy volunteers. All treatment-emergent adverse events in participants dosed with AOC 1044 were mild to moderate. There were no symptomatic hemoglobin changes, no hypomagnesemia and no renal events. In addition to AOC 1044, Avidity is also advancing AOC 1001 in the MARINA open-label extension (MARINA-OLE™) study for people living with myotonic dystrophy type 1 (DM1) and AOC 1020 in the Phase 1/2 FORTITUDE™ trial for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The EXPLORE44™ Phase 1/2 Trial of AOC 1044 The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in healthy volunteers and participants with DMD mutations amenable to exon 44 skipping (DMD44). EXPLORE44 will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 administered intravenously. EXPLORE44 is expected to enroll approximately 40 healthy volunteers and 24 participants with DMD44, ages seven to 27 years old. The EXPLORE44 trial will assess exon skipping and dystrophin protein levels in participants with DMD44. Participants with DMD44 will have the option to enroll into an extension study. For more information about the EXPLORE44 trial, visit the EXPLORE44 study website or visit http://www.clinicaltrials.gov and search for NCT05670730. About Duchenne muscular dystrophy (DMD) Duchenne muscular dystrophy (DMD) causes a lack of functional dystrophin that leads to stress and tears of muscle cell membranes, resulting in muscle cell death and the progressive loss of muscle function. The dystrophin protein maintains the integrity of muscle fibers and acts as a shock absorber through its role as the foundation of a group of proteins that connects the inner and outer elements of muscle cells. People living with DMD suffer from progressive muscle weakness that typically starts at a very young age. Over time, people with Duchenne will develop problems walking and breathing, and eventually, the heart and respiratory muscles will stop working. Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy. While there are treatments approved to treat people with DMD, there remains a very high unmet need. DMD is a monogenic, X-linked, recessive disease that primarily affects males, with one in 3,500 to 5,000 boys born worldwide having Duchenne. About AOC 1044 AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production in people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44). DMD is characterized by progressive muscle degeneration and weakness due to alterations of a protein called dystrophin that protects muscle cells from injury during contraction. AOC 1044 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a PMO targeting exon 44. In a preclinical model of DMD, a murine active AOC produced durable exon skipping and functional dystrophin protein in skeletal muscle and heart tissue following a single intravenous dose. AOC 1044 is currently in Phase 1/2 development as part of the EXPLORE44™ trial for the treatment of DMD mutations amenable to exon 44 skipping. Major Estimate Revision • Nov 29
Consensus revenue estimates increase by 104% The consensus outlook for revenues in fiscal year 2023 has improved. 2023 revenue forecast increased from US$9.59m to US$19.6m. Forecast losses expected to reduce from -US$2.89 to -US$2.75 per share. Biotechs industry in the US expected to see average net income growth of 10% next year. Consensus price target of US$37.00 unchanged from last update. Share price rose 26% to US$7.81 over the past week. 공시 • Nov 29
Avidity Biosciences, Inc. announced that it expects to receive $39.999993 million in funding from Bristol-Myers Squibb Company Avidity Biosciences, Inc. announced that it has entered into a securities purchase agreement to issue 5,075,304 common shares at a price of $7.8813 per share for the gross proceeds of $39,999,993 on November 28, 2023. The transaction will include participation from new investor, Bristol-Myers Squibb Company. The shares are subject to lock-up restrictions, which, without prior approval of the company, prohibit company from transferring the shares for a period of 180 days after the effective date. These lockup restrictions are subject to certain exceptions and shall terminate upon the occurrence of certain trigger events, including a change of control of the company. Major Estimate Revision • Nov 15
Consensus revenue estimates increase by 17% The consensus outlook for revenues in fiscal year 2023 has improved. 2023 revenue forecast increased from US$8.22m to US$9.59m. Forecast losses expected to reduce from -US$2.96 to -US$2.89 per share. Biotechs industry in the US expected to see average net income growth of 5.7% next year. Consensus price target of US$40.80 unchanged from last update. Share price rose 6.9% to US$6.23 over the past week. Price Target Changed • Nov 10
Price target increased by 10% to US$40.80 Up from US$37.00, the current price target is an average from 5 analysts. New target price is 645% above last closing price of US$5.48. Stock is down 62% over the past year. The company is forecast to post a net loss per share of US$2.89 next year compared to a net loss per share of US$3.34 last year. New Risk • Oct 30
New minor risk - Share price stability The company's share price has been volatile over the past 3 months. It is more volatile than 75% of American stocks, typically moving 9.3% a week. This is considered a minor risk. Share price volatility indicates the stock is highly sensitive to market conditions or economic conditions rather than being sensitive to its own business performance, which may also be inconsistent. It also increases the risk of potential losses in the short term as the stock tends to have larger drops in price more frequently than other stocks. Currently, the following risks have been identified for the company: Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$283m net loss in 3 years). Share price has been volatile over the past 3 months (9.3% average weekly change). Shareholders have been diluted in the past year (42% increase in shares outstanding). 공시 • Oct 11
Avidity Biosciences, Inc. Presents New Data Regarding Its Clinical Study of AOC 1001 for Adults with Myotonic Dystrophy Type 1 Avidity Biosciences, Inc. presented new data regarding its clinical study of AOC 1001 for adults with myotonic dystrophy type 1 (DM1) at the 28th Annual Congress of the World Muscle Society ("WMS"). New data presented at WMS demonstrate improvement in additional functional measures augmenting previously reported positive data which showed improvements in myotonia, muscle strength and mobility. These new data include: Multiple additional measures of strength: Hand grip. Manual Muscle Testing (MMT) composite score. Both upper and lower Quantitative Muscle Testing total score composites. DM1-Activ, a patient reported outcome (PRO) that measures activities of daily living (e.g., taking a shower, visiting family or friends, and walking up stairs). New data presented at WMS also addressed favorable long-term safety and tolerability of AOC 1001 from the MARINA-OLE™ study, with over 200 infusions totaling 46.2 patient-years of exposure. The most common adverse events (AEs) in the MARINA-OLE study were procedural pain (22%), pain in extremity (such as arm, leg or foot pain/soreness) (16%) and headache (16%). There was one resolved AE of mild increase in liver enzymes. There have been no reported AEs of anemia in the MARINA-OLE study. In the Phase 1/2 MARINA ® clinical program, anemia has been asymptomatic except for one participant who did not require treatment. There have been no discontinuations in the MARINA-OLE study. The Company has completed the dose-escalation of 12 participants from 2 mg/kg to 4 mg/kg of AOC 1001 in the MARINA-OLE study, as permitted by the U.S. Food and Drug Administration ("FDA") in connection with the FDA's easing of the partial clinical hold on AOC 1001 in May 2023. Data from this dose escalation showed no neurological events and no MRI changes following dosing. The Company continues to work with the FDA regarding a resolution to the partial clinical hold. The Company remains on track to share a first look at efficacy data of AOC 1001 from the MARINA-OLE study during the first half of 2024. Price Target Changed • Oct 09
Price target increased by 10% to US$40.80 Up from US$37.00, the current price target is an average from 5 analysts. New target price is 563% above last closing price of US$6.15. Stock is down 62% over the past year. The company is forecast to post a net loss per share of US$2.96 next year compared to a net loss per share of US$3.34 last year. Major Estimate Revision • Aug 15
Consensus revenue estimates increase by 11% The consensus outlook for revenues in fiscal year 2023 has improved. 2023 revenue forecast increased from US$7.50m to US$8.31m. Forecast losses expected to reduce from -US$3.20 to -US$2.93 per share. Biotechs industry in the US expected to see average net income decline 13% next year. Consensus price target broadly unchanged at US$36.71. Share price was steady at US$8.77 over the past week. Reported Earnings • Aug 09
Second quarter 2023 earnings: EPS and revenues exceed analyst expectations Second quarter 2023 results: US$0.66 loss per share. Net loss: US$47.0m (loss widened 2.9% from 2Q 2022). Revenue exceeded analyst estimates by 23%. Earnings per share (EPS) also surpassed analyst estimates by 17%. Revenue is forecast to grow 63% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in the US. 공시 • May 18
Avidity Biosciences, Inc. Provides an Update to the Status of the Partial Clinical Hold Related to Its AOC 1001 Product Candidate On May 17, 2023, Avidity Biosciences, Inc. provided an update to the status of the partial clinical hold related to its AOC 1001 product candidate. The Company announced that the U.S. Food and Drug Administration (the "FDA") has eased the partial clinical hold related to AOC 1001, allowing Avidity to double the number of participants receiving 4 mg/kg of AOC 1001 in the MARINA Open-Label Extension (MARINA-OLE) study by dose escalating approximately 12 participants currently receiving the 2 mg/kg level to the 4 mg/kg level of AOC 1001. All other participants will remain on their current dosage level of either 2 mg/kg or 4 mg/kg of Aoc 1001. This action by the FDA also allows the Company to enroll new participants at the 2 mg/kg dosage level. Data from the MARINA-OLE study will be used to finalize the AOC 1001 pivotal dose and Phase 3 study design for adults with myotonic dystrophy type 1 (DM1). Avidity remains on track to share a first look at data from theMARINA-OLE study at the end of 2023. Major Estimate Revision • May 16
Consensus revenue estimates increase by 11% The consensus outlook for revenues in fiscal year 2023 has improved. 2023 revenue forecast increased from US$6.74m to US$7.50m. Forecast losses expected to reduce from -US$3.45 to -US$3.23 per share. Biotechs industry in the US expected to see average net income decline 85% next year. Consensus price target of US$37.00 unchanged from last update. Share price was steady at US$11.25 over the past week. Reported Earnings • May 10
First quarter 2023 earnings: EPS and revenues exceed analyst expectations First quarter 2023 results: US$0.74 loss per share (further deteriorated from US$0.71 loss in 1Q 2022). Net loss: US$52.4m (loss widened 53% from 1Q 2022). Revenue exceeded analyst estimates by 27%. Earnings per share (EPS) also surpassed analyst estimates by 13%. Revenue is forecast to grow 81% p.a. on average during the next 3 years, compared to a 18% growth forecast for the Biotechs industry in the US. Price Target Changed • Mar 30
Price target decreased by 7.8% to US$40.57 Down from US$44.00, the current price target is an average from 7 analysts. New target price is 141% above last closing price of US$16.80. Stock is down 9.0% over the past year. The company is forecast to post a net loss per share of US$3.45 next year compared to a net loss per share of US$3.34 last year. Reported Earnings • Mar 01
Full year 2022 earnings released: US$3.34 loss per share (vs US$2.85 loss in FY 2021) Full year 2022 results: US$3.34 loss per share (further deteriorated from US$2.85 loss in FY 2021). Net loss: US$174.0m (loss widened 47% from FY 2021). Revenue is forecast to grow 56% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in the US. Recent Insider Transactions Derivative • Feb 20
Chief Scientific Officer exercised options and sold US$224k worth of stock On the 14th of February, Arthur Levin exercised 10k options at a strike price of around US$1.24 and sold these shares for an average price of US$23.66 per share. This trade did not impact their existing holding. Since June 2022, Arthur's direct individual holding has decreased from 276.53k shares to 275.20k. Company insiders have collectively sold US$5.2m more than they bought, via options and on-market transactions in the last 12 months. 공시 • Feb 15
Avidity Biosciences, Inc. Receives FDA Orphan Drug Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious, rare, hereditary muscle-weakening condition marked by life-long, progressive loss of muscle function that causes significant pain, fatigue, and disability. AOC 1020 is being evaluated in the Phase 1/2 FORTITUDE™ clinical trial in adults with FSHD and is the company's second muscle-targeting small interfering RNA (siRNA) AOC in clinical development. Avidity plans to share data from a preliminary assessment of AOC 1020 in approximately half of study participants from the FORTITUDE trial in the first half of 2024. The FDA's Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials. About Facioscapulohumeral Muscular Dystrophy (FSHD): Facioscapulohumeral muscular dystrophy (FSHD) is characterized by progressive and often asymmetric skeletal muscle loss that typically causes weakness initially in muscles in the face, shoulders, arms and trunk and progresses to weakness in muscles in the lower body. FSHD is an autosomal dominant genetic disease. The abnormal expression of DUX4 (double homeobox 4) leads to a series of downstream events that result in skeletal muscle wasting and progressive loss of muscle function, including an inability to lift arms for more than a few seconds, loss of ability to show facial expressions, and serious speech impediments. These symptoms cause many people affected by FSHD to become dependent on the use of a wheelchair for mobility. Currently, there are no approved treatments for people living with FSHD. About AOC 1020: AOC 1020 is designed to treat the underlying cause of FSHD, which is caused by the abnormal expression of a gene called double homeobox 4 or DUX4. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. AOC 1020 aims to reduce the expression of DUX4 mRNA and DUX4 protein in muscles in patients with FSHD. AOC 1020 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DUX4 mRNA. In preclinical studies, a single intravenous dose with the murine version of AOC 1020 prevented development of muscle weakness demonstrated by three functional assays - treadmill running, in vivo force and compound muscle action potential. AOC 1020 is currently in Phase 1/2 development as part of the FORTITUDE™ trial in adults with FSHD. Recent Insider Transactions Derivative • Feb 05
President notifies of intention to sell stock Sarah Boyce intends to sell 50k shares in the next 90 days after lodging an Intent To Sell Form on the 1st of February. If the sale is conducted around the recent share price of US$23.70, it would amount to US$1.2m. For the year to December 2019, Sarah's total compensation was 10% salary and 90% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2022, Sarah's direct individual holding has increased from 2.23k shares to 3.35k. Company insiders have collectively sold US$3.8m more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Jan 19
Chief Scientific Officer exercised options and sold US$208k worth of stock On the 12th of January, Arthur Levin exercised 10k options at a strike price of around US$1.24 and sold these shares for an average price of US$22.00 per share. This trade did not impact their existing holding. Since June 2022, Arthur's direct individual holding has decreased from 276.53k shares to 275.20k. Company insiders have collectively sold US$3.8m more than they bought, via options and on-market transactions in the last 12 months. 공시 • Jan 19
Avidity Biosciences Granted FDA Fast Track Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious, rare, hereditary muscle-weakening condition marked by life-long, progressive loss of muscle function that causes significant pain, fatigue, and disability. AOC 1020 is being studied in the Phase 1/2 FORTITUDE(TM) clinical trial in adults with FSHD and is the company's second muscle-targeting small interfering RNA (siRNA) AOC in clinical development. Avidity plans to share data from a preliminary assessment of AOC 1020 in approximately half of study participants from the FORTITUDE trial in the first half of 2024. Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Currently, there are no FDA-approved treatments for people living with FSHD. Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies (mAbs) with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. AOC 1020 consists of a proprietary mAb that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting double homeobox 4 (DUX4) mRNA. The abnormal expression of DUX4 protein leads to changes in gene expression in muscle cells that are associated with the life-long, progressive loss of muscle function in patients with FSHD. Avidity has three distinct rare disease programs in the clinic. In addition to AOC 1020, the company is also evaluating AOC 1001 in the Phase 1/2 MARINA(TM) and MARINA open-label extension (MARINA-OLE(TM)) clinical trials for the treatment of myotonic dystrophy type 1 (DM1) and AOC 1044 in the Phase 1/2 EXPLORE44(TM) trial for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44). The FORTITUDE(TM) Phase 1/2 Trial of AOC 1020 in Adults with FSHD The FORTITUDE(TM) trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate AOC 1020 in approximately 70 adult participants with FSHD. FORTITUDE will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of AOC 1020 administered intravenously, with the primary objective being thesafety and tolerability of AOC 1020 in FSHD patients. Activity of AOC 1020 will be assessed using key biomarkers, including magnetic resonance imaging (MRI) measures of muscle volume and composition. Though the Phase 1/2 trial is not statistically powered to assess functional benefit, it will explore the clinical activity of AOC 1020 including measures of mobility and muscle strength as well as patient reported outcomes and quality of life measures. Participants will have the option to enroll in an open-label extension study at the end of the treatment period in the FORTITUDE study. Recent Insider Transactions Derivative • Jan 05
Chief Scientific Officer exercised options and sold US$831k worth of stock On the 29th of December, Arthur Levin exercised 40k options at a strike price of around US$1.24 and sold these shares for an average price of US$22.01 per share. This trade did not impact their existing holding. Since June 2022, Arthur's direct individual holding has decreased from 276.53k shares to 275.20k. Company insiders have collectively sold US$3.6m more than they bought, via options and on-market transactions in the last 12 months. Price Target Changed • Dec 15
Price target increased to US$44.00 Up from US$37.86, the current price target is an average from 7 analysts. New target price is 142% above last closing price of US$18.15. Stock is down 19% over the past year. The company is forecast to post a net loss per share of US$3.35 next year compared to a net loss per share of US$2.85 last year. 공시 • Dec 15
Avidity Biosciences, Inc. Announces Positive AOC 1001 Phase 1/2 Marina(Tm) Data Demonstrating First-Ever Successful Targeted Delivery of RNA to Muscle - Revolutionary Advancement for the Field of RNA Therapeutics Avidity Biosciences, Inc. announced positive AOC 1001 data from the preliminary assessment of the Phase 1/2 MARINA(TM) trial demonstrating the first-ever successful targeted delivery of RNA into muscle, a revolutionary advancement for the field of RNA therapeutics. The effective targeted delivery of siRNA into muscle further reinforces the broad and disruptive potential of Avidity's proprietary AOC platform and expands the ability to address targets and diseases previously unreachable with existing RNA therapies. AOC 1001, Avidity's lead clinical program utilizing its AOC platform, is designed to address the root cause of myotonic dystrophy type 1 (DM1), an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Reported Earnings • Nov 11
Third quarter 2022 earnings: EPS and revenues exceed analyst expectations Third quarter 2022 results: US$0.82 loss per share (further deteriorated from US$0.68 loss in 3Q 2021). Net loss: US$43.6m (loss widened 49% from 3Q 2021). Revenue exceeded analyst estimates by 16%. Earnings per share (EPS) also surpassed analyst estimates by 12%. Revenue is forecast to grow 43% p.a. on average during the next 3 years, compared to a 14% growth forecast for the Biotechs industry in the US. 공시 • Oct 15
Avidity Biosciences, Inc. Announces Planned Explore44 Trial of AOC 1044 in Healthy Volunteers and Participants with Duchenne Muscular Dystrophy On October 13, 2022, Avidity Biosciences, Inc. announced its planned EXPLORE44 trial of AOC 1044 in healthy volunteers and participants with Duchenne muscular dystrophy. The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 administered intravenously, with the primary objective being the safety and tolerability of AOC 1044 in adult healthy volunteers and pediatric and adult participants with DMD mutations amenable to exon 44 skipping. The EXPLORE44 trial will assess exon skipping and dystrophin protein levels in participants with DMD44 and will also explore measures of muscle function, patient-reported outcomes and quality of life. DMD44 is ultra rare, constituting approximately 7% of DMD patients, approximately 900 of which are in the United States. Participants with DMD44 will have the option to enroll in an extension study at the end of the treatment period in the EXPLORE44 trial. The overall development program for AOC 1044, which includes the EXPLORE44 trial and the extension study is designed to potentially support accelerated approval in the United States. In the second half of 2023, the Company plans to present results from the healthy volunteer portion of the EXPLORE44 trial. Preclinical studies showed that an antibody oligonucleotide conjugate approach targeting exon 23 in a murine model of DMD demonstrated exon skipping, dystrophin restoration and improved muscle function and serum biomarkers of muscle damage. Additionally, data from a preclinical study of AOC 1044 in patient-derived myotubes showed dose-dependent dystrophin restoration, and a preclinical study in non-human primate (“NHP”) observed dose-dependent exon skipping in skeletal and heart muscle with AOC 1044. In addition to the preclinical studies described above, Avidity completed an IND-enabling good laboratory practice (“GLP”) toxicology study of AOC 1044 in NHP. Results from the IND-enabling study showed that AOC 1044 was generally well tolerated and supported advancement into the clinic, with no dose limiting toxicity observed in NHP at the high dose tested. The study did not observe any treatment-related histopathologic toxicity, platelet or renal toxicity or any changes in safety pharmacology parameters (cardiac, respiratory and neurological). The high dose tested in both NHP and murine models was the maximum feasible dose and was the no-observed adverse effect level (“NOAEL”). Results of a nine-month chronic toxicology study of AOC 1044 in NHPs was consistent with the results of the IND-enabling study. 공시 • Oct 12
Avidity Biosciences, Inc. Announces Phase 1/2 EXPLORE44 Trial of AOC 1044 for Duchenne Muscular Dystrophy Mutations Amenable to Exon 44 Skipping Avidity Biosciences, Inc. announced the Phase 1/2 EXPLORE44 clinical trial of AOC 1044 in healthy volunteers and participants with Duchenne muscular dystrophy (DMD), a rare, genetic condition that is characterized by progressive muscle damage and weakness. AOC 1044 is designed for people living with DMD amenable to exon 44 skipping (DMD44) and is the first of multiple AOCs the company is developing for DMD. Currently, there are no approved therapies to treat the underlying mechanism of disease for people living with DMD mutations amenable to exon 44 skipping. Avidity has advanced three distinct rare disease programs - DM1, FSHD and DMD44 – into clinical development in a 14-month period. DMD is an irreversible, progressive disease caused by a genetic mutation that prevents the body from producing a protein called dystrophin, which is an important protein that protects muscle cells from injury during contraction. The lack of functional dystrophin leads to stress and tears of muscle cell membranes, resulting in muscle cell death and progressive loss of muscle function. Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMO) to skeletal muscle and heart tissue to specifically skip exon 44 of DMD to enable dystrophin production. AOC 1044 is Avidity's first AOC engineered to deliver PMO and the company has two additional programs for DMD, which target exon 45 and exon 51. Avidity has three distinct rare disease programs in the clinic – AOC 1001 for myotonic dystrophy type 1 (DM1) is currently being evaluated in the MARINA™ Phase 1/2 clinical trial and an open label-extension trial called MARINA-OLE; AOC 1020 is advancing into the Phase 1/2 FORTITUDE trial for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 is advancing into the Phase 1/2 EXPLORE44 trial for the treatment of DMD44. The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in healthy volunteers and participants with DMD mutations amenable to exon 44 skipping (DMD44). EXPLORE44 will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 administered intravenously. EXPLORE44 is expected to enroll approximately 40 healthy volunteers and 24 participants with DMD44, ages seven to 27 years old. The EXPLORE44 trial will assess exon skipping and dystrophin protein levels in participants with DMD44. Participants with DMD44 will have the option to enroll into an extension study. 공시 • Sep 30
Avidity Biosciences Announces the Phase 1/2 Fortitude(Tm) Trial of AOC 1020 in Adults with Facioscapulohumeral Muscular Dystrophy Avidity Biosciences, Inc. announced the Phase 1/2 FORTITUDE(TM) clinical trial of AOC 1020 in adults with facioscapulohumeral muscular dystrophy (FSHD). FSHD is a rare, hereditary muscle-weakening condition marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability. AOC 1020 is the second muscle-targeting small interfering RNA (siRNA) AOC from Avidity's pipeline to advance into clinical development. Earlier this week, Avidity announced that the U.S. Food and Drug Administration (FDA) cleared the company's investigational new drug (IND) applications of AOC 1020 for FSHD and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (DMD44). The company has now advanced three programs - DM1, FSHD and DMD44 -- into clinical development in a 14-month period. 공시 • Sep 28
Avidity Biosciences, Inc. Announces FDA Partial Clinical Hold on New Participant Enrollment in Phase 1/2 MARINA™ Trial Avidity Biosciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on new participant enrollment in the Phase 1/2 MARINA™ clinical trial of AOC 1001 in adults with myotonic dystrophy type 1 (DM1). Close to 40 participants are currently enrolled in the MARINA and MARINA open label extension (MARINA-OLE™) trials. All participants, whether they are on AOC 1001 or placebo, may continue in their current dosing cohort although no additional participants may be enrolled until the partial clinical hold is resolved. All participants in MARINA may roll over into the MARINA-OLE where they will receive AOC 1001 as planned. DM1 is an underrecognized, progressive and often fatal neuromuscular disease with no approved treatment options. The partial clinical hold is in response to a serious adverse event reported in a single participant in the 4mg/kg cohort of the MARINA study. Avidity is working closely with the FDA and the trial investigator to assess the cause of this event. The company is taking all necessary steps to resolve the partial clinical hold on new participant enrollment as quickly as possible. Avidity remains on track to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants in the MARINA trial in the fourth quarter of 2022. Avidity received IND clearance from the FDA to proceed with the clinical trial of AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) and AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) with mutations amenable to exon 44 skipping. These programs are now advancing into the clinic. Recent Insider Transactions Derivative • Aug 27
President exercised options and sold US$1.1m worth of stock On the 25th of August, Sarah Boyce exercised 50k options at a strike price of around US$1.24 and sold these shares for an average price of US$22.88 per share. This trade did not impact their existing holding. For the year to December 2019, Sarah's total compensation was 10% salary and 90% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2022, Sarah's direct individual holding has decreased from 2.23k shares to 0. Company insiders have collectively sold US$1.7m more than they bought, via options and on-market transactions in the last 12 months. Recent Insider Transactions Derivative • Aug 19
President notifies of intention to sell stock Sarah Boyce intends to sell 50k shares in the next 90 days after lodging an Intent To Sell Form on the 15th of August. If the sale is conducted around the recent share price of US$21.95, it would amount to US$1.1m. For the year to December 2019, Sarah's total compensation was 10% salary and 90% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2022, Sarah's direct individual holding has decreased from 2.23k shares to 0. Company insiders have collectively sold US$669k more than they bought, via options and on-market transactions in the last 12 months. Major Estimate Revision • Aug 16
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 expected loss increased from -US$3.12 to -US$3.52 per share. Revenue forecast unchanged at US$7.96m. Biotechs industry in the US expected to see average net income decline 77% next year. Consensus price target of US$40.71 unchanged from last update. Share price rose 13% to US$21.89 over the past week. Reported Earnings • Aug 10
Second quarter 2022 earnings: Revenues exceed analysts expectations while EPS lags behind Second quarter 2022 results: US$0.92 loss per share (down from US$0.70 loss in 2Q 2021). Net loss: US$45.7m (loss widened 73% from 2Q 2021). Revenue exceeded analyst estimates by 5.6%. Earnings per share (EPS) missed analyst estimates by 22%. Over the next year, revenue is forecast to grow 3.6%, compared to a 51% growth forecast for the industry in the US. 공시 • Aug 03
Avidity Biosciences, Inc. Enrolls Patients in the Marina(Tm) Open-Label Extension Study Avidity Biosciences, Inc. announced that it has commenced enrolling patients from the Phase 1/2 MARINA(TM) study into a Phase 2 open-label extension study (MARINA-OLE(TM)) of AOC 1001 in adults with myotonic dystrophy type 1 (DM1). All patients enrolled in the randomized, placebo-controlled MARINA(TM) clinical trial with AOC 1001 are eligible to enroll in MARINA-OLE. DM1 is an underrecognized, progressive and often fatal neuromuscular disease. It primarily affects skeletal and cardiac muscle with multiple organ involvement and can be highly variable with respect to severity, presentation, and age of onset. There are currently no disease-modifying treatments for people living with DM1. AOC 1001, Avidity's lead program utilizing its AOC platform, is designed to address the root cause of DM1 by reducing levels of DMPK, the disease-related mRNA. AOC 1001 consists of a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA). The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted Orphan Designation for AOC 1001 and the FDA has granted AOC 1001 Fast Track Designation. About the Phase 2 MARINA-OLE(TM) Study MARINA-OLE(TM) is an open-label, multi-center study designed to evaluate the long-term safety and tolerability of AOC 1001 in DM1 patients who were previously enrolled in the MARINA Phase 1/2 study. This study will continue to evaluate the safety, tolerability, PK, PD, and efficacy of AOC 1001 in patients that enrolled in the randomized, placebo-controlled, Phase 1/2 MARINA clinical study. Patients who enroll in the MARINA-OLE study will receive quarterly doses of AOC 1001 regardless of whether they received active treatment or placebo in the MARINA study. The total duration of active treatment with AOC 1001 in the MARINA-OLE is approximately 24 months. Once patients have completed active treatment, there will be a 9-month safety follow-up period. Avidity may extend active treatment beyond 24 months at a future timepoint. 공시 • Jun 26
Avidity Biosciences, Inc.(NasdaqGM:RNA) dropped from Russell 2000 Growth Index Avidity Biosciences, Inc.(NasdaqGM:RNA) dropped from Russell 2000 Growth Index 공시 • Jun 18
Avidity Biosciences, Inc. Supports World Facioscapulohumeral Muscular Dystrophy Day and Presents Preclinical Data from FSHD Program Avidity Biosciences, Inc. called Antibody Oligonucleotide Conjugates, joins in activities to raise awareness for Facioscapulohumeral muscular dystrophy in support of World FSHD Day and highlights preclinical results supporting AOC 1020 for the treatment of FSHD at the 29th Annual FSHD Society International Research Congress (FSHD IRC) in Orlando, Florida. Currently, there are no approved therapies for the treatment of FSHD. FSHD is a rare, hereditary muscle-weakening condition that affects approximately 16,000-38,000 people in the United States. It is marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability. FSHD is an autosomal dominant disease caused by the abnormal expression of DUX4 (double homeobox 4), a gene involved in embryonic development but not typically expressed in muscles. This abnormal expression of DUX4 leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function with onset often in teenage and early adult years. Every June 20th, people around the world join in activities to raise awareness for FSHD through World FSHD Day and to recognize patients and families around the world who are affected by FSHD. To mark FSHD awareness day, Avidity is participating in the FSHD IRC as well as the FSHD Connect Conference, an educational conference specifically for people living with FSHD and their families. Both conferences are being held in Orlando, Florida and organized by the FSHD Society, the world's largest research-focused patient organization for FSHD. On June 17, 2022, Avidity will share preclinical data from the FSHD program during a presentation at the FSHD IRC. Studies were conducted in mouse models of FSHD that are genetically engineered to express human DUX4 in skeletal muscle. AOC 1020 is comprised of a DUX4-targeting siRNA conjugated to the human anti-transferrin receptor 1 monoclonal antibody. The preclinical studies were done using a monoclonal antibody suitable for mice. The preclinical data provides support for AOC 1020 to enter the clinic for the treatment of FSHD by end of 2022. Key highlights from the oral presentation include: Data from a study conducted to assess pharmacology in the mouse model of FSHD showed robust dose-dependent down regulation of DUX4 genes in skeletal muscle for 8 weeks following a single intravenous (IV) dose of the murine version of AOC 1020. Data from a study designed to assess phenotype development in the FSHD mouse model showed that a single IV treatment with the murine version of AOC 1020 prevented muscle weakness development demonstrated by three functional assays - treadmill running, in vivo force and compound muscle action potential. Avidity is also presenting data from a real-world data analysis of patients with FSHD before and after their diagnosis. The results highlight the increased morbidity and medical and financial burden patients face as they progress through their journey living with FSHD. Major Estimate Revision • May 17
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 revenue forecast fell from US$7.68m to US$7.60m. 2022 losses expected to reduce from -US$3.54 to -US$3.14 per share. Biotechs industry in the US expected to see average net income decline 52% next year. Consensus price target down from US$49.80 to US$45.40. Share price rose 22% to US$14.94 over the past week. Price Target Changed • May 14
Price target decreased to US$45.40 Down from US$49.80, the current price target is an average from 5 analysts. New target price is 251% above last closing price of US$12.95. Stock is down 37% over the past year. The company is forecast to post a net loss per share of US$3.28 next year compared to a net loss per share of US$2.85 last year. Reported Earnings • May 11
First quarter 2022 earnings: EPS exceeds analyst expectations while revenues lag behind First quarter 2022 results: US$0.71 loss per share (down from US$0.64 loss in 1Q 2021). Net loss: US$34.2m (loss widened 44% from 1Q 2021). Revenue missed analyst estimates by 5.5%. Earnings per share (EPS) exceeded analyst estimates by 11%. Over the next year, revenue is expected to shrink by 10% compared to a 31% growth forecast for the industry in the US. 공시 • May 02
Avidity Biosciences, Inc., Annual General Meeting, Jun 15, 2022 Avidity Biosciences, Inc., Annual General Meeting, Jun 15, 2022, at 09:00 Pacific Standard Time. Agenda: To elect two directors to serve as Class II directors for a three-year term expiring at the 2025 Annual Meeting of Stockholders and until their respective successors shall have been duly elected and qualified; to ratify the appointment of BDO USA, LLP as our independent registered public accounting firm for the fiscal year ending December 31, 2022; to consider and vote upon, on an advisory basis, the compensation of our named executive officers as disclosed in this proxy statement pursuant to the compensation disclosure rules of the Securities and Exchange Commission; and to consider other matters of the business. 
