Live News • Jul 09
Vor Biopharma Accelerates Telitacicept Trials for Autoimmune Diseases with $491.5 Million Reserve Vor Biopharma is shifting its core focus from oncology to late-stage autoimmune disease drug development, centering on telitacicept, a dual BAFF/APRIL inhibitor already approved in multiple indications in China and currently in Phase 3 trials for generalized myasthenia gravis and Sjögren’s disease, supported by a cash reserve of $491.5m.
The company’s pipeline and business priorities are now tied closely to telitacicept’s global clinical progress, with current studies targeting readouts into early 2029 and facing competition and regulatory hurdles in autoimmune indications such as gMG, Sjögren’s disease and IgA nephropathy.
Vor Biopharma’s stock trades at $19.75, with the share price up 69.7% year to date, reflecting a market that is already highly sensitive to updates on telitacicept and capital allocation decisions.
This pivot concentrates Vor Biopharma’s risk and potential reward in a single late-stage asset. As a result, trial outcomes, regulatory feedback and partnering terms are likely to have an outsized effect on shareholder value. Live News • Jul 08
Vor Biopharma Adds Biopharma Veteran Zaccardelli to Board as Phase 3 Milestone Nears Vor Biopharma has appointed David Zaccardelli, Pharm.D., to its Board of Directors, adding a biopharmaceutical leader with more than 20 years of experience in late-stage development, regulatory approval and commercial launches.
The company is advancing telitacicept, a therapy for autoimmune diseases, through global Phase 3 trials and views Zaccardelli’s track record with late-stage programs and commercialization as important support as it approaches potential regulatory and launch milestones.
Vor Biopharma’s stock trades at US$20.10, with a return of 72.7% year to date.
The key read-through is that Vor Biopharma is strengthening its governance and commercial experience ahead of potential telitacicept inflection points, which may help execution but also increases the importance of clinical and regulatory outcomes. Announcement • Jul 08
Vor Biopharma Announces Board Changes Vor Biopharma announced the appointment of David Zaccardelli, Pharm.D., to its Board of Directors. With more than 20 years of biopharmaceutical leadership experience, Dr. Zaccardelli has successfully built and led companies through late-stage development, regulatory approval, and commercial launch. Dr. Zaccardelli served as President, Chief Executive Officer, and on the board of directors of Verona Pharma from February 2020 until its acquisition by Merck for $10 billion in October 2025. During his tenure, Verona advanced from a clinical-stage company to a commercial-stage organization, successfully completing two Phase 3 trials, receiving FDA approval, and launching Ohtuvayre for chronic obstructive pulmonary disease. Under his leadership, Verona raised more than $1 billion to support development and commercialization and became one of the top-performing biotechnology companies on Nasdaq in 2024 based on its five-year increase in market value. Dr. Zaccardelli was recognized as Executive of the Year at the 2023 Scrip Awards. Currently, he serves as Chair of the Board of Directors for SAB BIO and Founder and Managing Member of Bull City Select Investments, LLC. Prior to Verona, Dr. Zaccardelli served as President and Chief Executive Officer of Dova Pharmaceuticals, a rare disease company acquired by Swedish Orphan Biovitrum in 2019 for up to $915 million. He previously served as Acting Chief Executive Officer of Cempra through its merger with Melinta Therapeutics and held several senior leadership roles at United Therapeutics Corporation, including Chief Operating Officer, Chief Manufacturing Officer and Executive Vice President, Pharmaceutical Development and Operations. Earlier in his career, Dr. Zaccardelli founded and led a contract research start-up and held clinical research roles at Burroughs Wellcome, Glaxo Wellcome and Bausch & Lomb Pharmaceutical. He received a Pharm.D. from the University of Michigan and completed a fellowship in clinical research and drug development at the University of North Carolina. In connection with Dr. Zaccardelli’s appointment, Andrew Levin, M.D., Ph.D., an Advisor at RA Capital Management, has stepped down from Vor Bio’s Board of Directors. Announcement • May 16
Vor Biopharma Announces Publication of Interim Analysis of Teligan Phase 3 Trial of Telitacicept in Iga Nephropathy Vor Bio announced that results from the Phase 3 TELIGAN trial evaluating telitacicept in IgA nephropathy in China sponsored by its collaborator, RemeGen Co. Ltd., were published in The New England Journal of Medicine (NEJM). The publication reports results from a prespecified interim analysis of the ongoing Phase 3 TELIGAN trial, a multicenter, randomized, double-blind, placebo-controlled trial conducted at 72 sites in China evaluating telitacicept in adults with biopsy-proven IgA nephropathy and persistent proteinuria despite optimized supportive care. The study met its primary endpoint, demonstrating a statistically significant reduction in proteinuria at week 39. Patients treated with telitacicept achieved a 58.9% reduction from baseline in 24-hour urinary protein-to-creatinine ratio (UPCR), compared with an 8.8% reduction for placebo, representing a 55.0% relative difference between groups. Estimated glomerular filtration rate (eGFR) remained stable with telitacicept, with a mean percentage change from baseline of -1.0% compared with -7.7% for placebo at week 39. A confirmed decline in eGFR of =30% occurred in 6.3% of telitacicept-treated patients compared with 27.0% of placebo-treated patients. At week 39, 61.0% of telitacicept-treated patients achieved a UPCR below 0.8 compared with 19.5% of placebo-treated patients. Reductions in proteinuria were observed consistently across prespecified patient subgroups, including baseline kidney function, proteinuria level, and use of SGLT2 inhibitors. Telitacicept treatment was associated with reductions in circulating CD19+ B cells and serum immunoglobulin levels, including a 60.6% mean reduction in serum IgA levels. Safety findings were generally consistent with previous studies of telitacicept. Most adverse events were mild to moderate in severity. Serious adverse events occurred less frequently with telitacicept than placebo (2.5% vs. 8.2%). The most common adverse events associated with telitacicept included upper respiratory tract infection, injection-site reactions, and reductions in immunoglobulin levels. Telitacicept is a novel recombinant fusion protein designed to treat autoimmune diseases through dual inhibition of BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). Additional regulatory filings in China are underway, including biologics license applications for primary Sjögren’s disease (SjD) and IgA nephropathy (IgAN). Vor Bio is advancing telitacicept in global Phase 3 trials in gMG and SjD to support potential regulatory approvals in the United States, Europe, and Japan. Announcement • Apr 28
Vor Biopharma Inc., Annual General Meeting, Jun 11, 2026 Vor Biopharma Inc., Annual General Meeting, Jun 11, 2026. Location: meetnow.global/mpc7qxv., United States Announcement • Mar 30
Vor Bio Doses First Patient in Global Phase 3 UPSTREAM SjD Registrational Trial of Telitacicept in Primary Sjögren’s Disease Vor Bio announced the dosing of the first patient in UPSTREAM SjD, a global, randomized, double-blind, placebo-controlled Phase 3 trial evaluating telitacicept in adult patients with active primary Sjögren’s disease (SjD), formerly known as Sjögren's syndrome. First and only dual BAFF/APRIL inhibitor in primary Sjögren’s disease, one of the largest autoimmune diseases without an approved therapy. In late 2025, RemeGen presented results from a Phase 3 trial conducted in China with telitacicept in the same indication that demonstrated potential best-in-disease activity, with statistically significant and clinically meaningful improvements in both ESSDAI and ESSPRI, the two key, validated EULAR measurements used to assess patient disease burden. These prior results provide important clinical support for dual BAFF/APRIL inhibition as a therapeutic strategy in this disease. UPSTREAM SjD will evaluate the efficacy and safety of telitacicept administered subcutaneously with a pre-filled syringe compared to placebo in approximately 250 adult patients with active primary SjD. The primary endpoint is the change from baseline in the EULAR Sjögren’s Syndrome Disease Activity Index (ESSDAI) score at Week 48. The trial is expected to enroll patients who meet the 2016 ACR/EULAR classification criteria for primary SjD and have active disease as defined by an ESSDAI score =5. Key secondary endpoints in the UPSTREAM SjD trial will evaluate the effect of telitacicept at Week 48 across systemic disease activity, glandular function, and patient-reported symptoms. Sjögren’s disease is a chronic autoimmune condition in which overactive B cells drive inflammation, damaging moisture-producing glands and, in many cases, other organs throughout the body. Hallmark symptoms include dry eyes and dry mouth, alongside fatigue, pain, and systemic complications affecting the skin, lungs, kidneys, and nervous system. About one-third of patients develop significant extraglandular involvement, and the disease carries an elevated lymphoma risk, often leading to substantial impairment in daily life. One of the most common rheumatic autoimmune diseases, Sjögren’s remains underdiagnosed, with roughly half of cases unrecognized and women comprising the vast majority of patients. Despite its prevalence and burden, no approved systemic disease-modifying therapies exist; current care focuses on symptom management with incomplete relief. Telitacicept is a novel, investigational recombinant fusion protein designed to treat autoimmune diseases by selectively inhibiting BLyS (BAFF) and APRIL - two cytokines essential to B cell and plasma cell survival. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. Telitacicept is approved in China for systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). Additional regulatory filings in China are underway, including biologics license applications for primary Sjögren’s disease (SjD) and IgA nephropathy (IgAN). Vor Bio is advancing global development programs across major autoimmune indications, including a global Phase 3 trial in gMG and SjD to support potential regulatory approvals in the United States, Europe, and Japan. Announcement • Mar 27
Vor Biopharma Inc. announced that it expects to receive $74.999996 million in funding from TCG Crossover Management, LLC Vor Biopharma Inc. announced that it has entered into a Securities Purchase Agreement with entities affiliated with TCGX, TCG Crossover Fund II, L.P. for 2,669,039 shares, TCG Crossover Fund III, L.P. for 2,669,039 shares pursuant to which the Company, in a private placement, agreed to issue and sell to the Investors an aggregate of 5,338,078 common shares at a price per Share of $14.05 for gross proceeds of $74,999,995.9 on March 26, 2026. The Purchase Agreement contains customary representations and warranties of the Company, on the one hand, and the Investors, on the other hand, and customary conditions to closing. The closing of the Private Placement is expected to occur on March 30, 2026, subject to customary closing conditions. Upon the closing of the Private Placement, the Company will have 54,185,582 shares of common stock outstanding. The Shares have not been registered under the Securities Act of 1933, as amended (the “Securities Act”), in reliance on the exemption from registration afforded by Section 4(a)(2) of the Securities Act. Each Investor has represented that it is a “qualified institutional buyer,” under the Securities Act or an institutional “accredited investor” (as defined in Rule 501(a) under the Securities Act), Announcement • Jan 03
Vor Biopharma Inc. Announces Separation of Qing Zuraw as Chief Development Officer, Effective December 31, 2025 On December 31, 2025, Vor Biopharma Inc. (the “Company”) entered into a separation agreement (the “Separation Agreement”) with Dr. Qing Zuraw, the Company’s Chief Development Officer, as a result of Dr. Zuraw’s decision to pursue other opportunities. Pursuant to the Separation Agreement, Dr. Zuraw’s employment with the Company ended on December 31, 2025. The Company anticipates entering into a consulting arrangement with Dr. Zuraw pursuant to which Dr. Zuraw will provide consulting services to assist in the transition during the first quarter of 2026. Board Change • Dec 26
Less than half of directors are independent Following the recent departure of a director, there are only 4 independent directors on the board. The company's board is composed of: 4 independent directors. 5 non-independent directors. Independent Director Daniella Beckman was the last independent director to join the board, commencing their role in 2020. The company's minority of independent directors is a risk according to the Simply Wall St Risk Model. Announcement • Dec 24
Vor Biopharma Inc Announces Board Appointments Vor Biopharma Inc. announced the appointment of Andrew Levin, M.D., Ph.D., Partner at RA Capital Management, and Wouter Joustra, General Partner at Forbion, to its Board of Directors. Andrew and Wouter bring highly complementary experience in life sciences investment, financing strategy, and board-level oversight. Dr. Levin is a Partner on the investment team at RA Capital Management, L.P., where he brings deep expertise in life sciences investing and strategic oversight to support innovative biopharma companies across all stages of development. He holds a B.S. in mechanical engineering from Princeton University, an M.D. from Harvard Medical School, and a Ph.D. in biomedical engineering from Massachusetts Institute of Technology. Dr. Levin fills the seat previously held by Sarah Reed, General Counsel at RA Capital, who resigned from the Board. Mr. Joustra is a General Partner at Forbion, where he is responsible for general fund management, late stage private, cross-over and public investments. Mr. Joustra has served on the boards of several high-growth biotech companies through successful acquisitions and currently serves on the boards of multiple public and private life sciences companies. He holds an M.Sc. in Business Administration and a B.Sc. in International Business and Management from the University of Groningen. Price Target Changed • Dec 23
Price target decreased by 11% to US$43.25 Down from US$48.50, the current price target is an average from 4 analysts. New target price is 220% above last closing price of US$13.53. Stock is down 23% over the past year. The company is forecast to post a net loss per share of US$84.64 next year compared to a net loss per share of US$34.03 last year. Announcement • Dec 15
Vor Biopharma Inc. announced that it expects to receive $149.999906 million in funding from RA Capital Management, L.P., Forbion Capital Partners B.V., Caligan Partners, LP, Logos Capital LLC, Frazier Life Sciences Management, LP Vor Biopharma Inc announced a private placement and entered into purchase agreement with a group of institutional and accredited healthcare specialist investors to issue 13,876,032 shares at a price of $10.81 for aggregate gross proceeds of $149,999,905.92 on December 15, 2025. The transaction is expected to close on December 18, 2025, subject to the satisfaction of customary closing conditions and includes participation from RA Capital Management, Forbion, Frazier Life Sciences, Caligan Partners, Logos Capital, and Venrock Healthcare Capital Partners. In addition, as part of the private placement, Forbion is being granted the right to appoint one director to Vor Bio’s board of directors. The offer and sale of the securities to be sold in the private placement have not been registered under the Securities Act of 1933, as amended, or any state or other applicable jurisdiction’s securities laws, and such securities may not be offered or sold in the United States absent registration or an applicable exemption from the registration requirements of the Securities Act and applicable state or other jurisdictions’ securities laws. Announcement • Nov 09
Vor Biopharma Inc. Achieves Primary Endpoint of Reducing Proteinuria in Stage A of A Phase 3 Clinical Study for Iga Nephropathy in China Vor Biopharma Inc. announced that the primary endpoint was achieved in Stage A of a Phase 3 clinical study in China evaluating telitacicept in adults with IgA nephropathy (IgAN). In addition, statistically significant benefits were achieved across all secondary endpoints in the study, which was conducted by RemeGen Co. Ltd. (HKEX: 9995, SHA: 688331), Vor Bio’s collaborator.
This study is a multicenter, randomized, double-blind, placebo-controlled clinical trial in China that enrolled 318 adult patients with IgAN at high risk of progression who had received stable standard therapy. Patients were randomized (1:1) to telitacicept (240 mg) or placebo, subcutaneously once weekly. Primary Endpoint Achieved: In Stage A of the Phase 3 study, telitacicept achieved the primary endpoint of reducing proteinuria, demonstrating a significant reduction in 24h-UPCRvs. Placebo at 39 weeks compared to placebo (-58.9% vs. -8.8%, p<0.0001), 24h-UPCR is an objective and internally recognized regulatory marker for assessing disease activity in IgAN. Statistically Significant Benefits Across all key Secondary Endpoints: Key secondary endpoints evaluated preservation of kidney function—measured by change in estimated glomerular filtration rate (eGFR), the proportion of patients with a =30% decline in eGFR, and remission rates defined by achievement of UPCR threshold <0.8 g/g. Additional endpoints included resolution of hematuria and changes in pharmacodynamic markers such as B-cell counts and serum immunoglobulins. Treatment with telitacicept achieved statistically significant improvements across all key secondary endpoints at Week 39. Compared with placebo, telitacicept stabilized kidney function (GMR of eGFR relative to baseline, showed stabilization in the telitacicept group (-0.10) in contrast to a decline in the placebo group (-0.77)) and reduced the risk of eGFR decline = 30% (6.3% in the telitacicept group vs. 27.0% in the placebo group). 61% of patients on telitacicept vs.19.5% of patients on placebo achieved 24h-UPCR <0.8 g/g, 42.1% of patients on telitacicept vs. 7.5% of patients on placebo achieved <0.5 g/g, and 24.5% of patients on telitacicept vs. 0.6% of patients on placebo achieved <0.3 g/g, thresholds linked to low risk of disease progression. Announcement • Nov 04
Vor Bio Appoints Jeremy Sokolove as Chief Medical Officer Vor Bio announced the appointment of Jeremy Sokolove, M.D., as Chief Medical Officer. Dr. Sokolove brings more than two decades of clinical and translational experience in rheumatology and autoimmune disease research and development to the companys leadership team. Most recently, Dr. Sokolove served as Chief Medical Officer In-Residence at Roivant Sciences where he led strategic assessment and in-licensing of novel therapeutic opportunities while providing operational leadership, clinical guidance, and translational expertise across Roivants emerging and established portfolio companies. Before that, he was Chief Medical Officer at Odyssey Therapeutics, leading the transition of the company to a clinical-stage biotechnology organization focused on next-generation immunomodulators for autoimmune and inflammatory diseases. Earlier, Dr. Sokolove was Senior Vice President and Head of Clinical Pharmacology and Experimental Medicine at GSK, where he led early and translational development across the specialty pharma portfolio, and previously Head of Immunology Translational Science at AbbVie, directing early-phase clinical programs and building portfolio-wide translational medicine strategy. He began his career on the faculty at Stanford University School of Medicine as a practicing rheumatologist and primary investigator, where his research focused on biomarker discovery and immune-mediated disease mechanisms. Dr. Sokolove has practiced extensively as an internist and rheumatologist, treating patients and leading clinical research programs focused on autoimmune and inflammatory disorders. He has published extensively in the fields of immunology and rheumatology, contributing to the understanding of immune-mediated disease mechanisms. His academic credentials include board certification in internal medicine and rheumatology, and he holds an MD from the BostonUniversitySchoolofMedicine. Announcement • Oct 17
Vor Bio Announces Late-Breaking Oral Presentation of China Phase 3 IgA Nephropathy Clinical Study at American Society of Nephrology's Kidney Week 2025 Vor Bio announced that clinical data from Stage A of a Phase 3 study in China sponsored by Vor Bio's collaborator RemeGen Co., Ltd, which evaluated telitacicept in adults with IgA neuropathy (IgAN), will be presented as a late-breaking oral presentation at American Society of Nephrology's (ASN) Kidney Week 2025 being held Nov. 5-9, 2025, in Houston, Texas. In August, Vor Bio announced topline results from the RemeGen-sponsored study, reporting that telitacicept achieved the primary endpoint of reducing proteinuria demonstrating a 55% reduction in 24-hour urine protein-to-creatinine ratio (UPCR) at 39 weeks compared with placebo (p). Announcement • Oct 16
Vor Biopharma Inc. Announces Publication of China Phase 3 Study of Telitacicept in Systemic Lupus Erythematosus in The New England Journal of Medicine Vor Biopharma Inc. announced that results from a Phase 3 study in China evaluating telitacicept in systemic lupus erythematosus (SLE) sponsored by its collaborator, RemeGen Co. Ltd., were published in The New England Journal of Medicine (NEJM). For the first time, a therapy in a Phase 3 trial is delivering more than double the clinical response seen with the current standard of care in lupus. These data present a compelling case for potentially broadening telitacicept's use as a new standard of care worldwide. Lupus has challenged researchers and clinicians for decades. By targeting both BAFF and APRIL, it was possible to achieve excellent clinical results that suggest the effective restoration of immune balance in at least some of the patients. This dual-target mechanism reduces autoreactive B cells and autoantibody production, key drivers of autoimmune pathology. In a Phase 3 clinical trial in generalized myasthenia gravis in China, telitacicept demonstrated a placebo adjusted 4.83-point improvement in MG-ADL (Myasthenia Gravis Activities of Daily Living scale) at 24 weeks, the primary endpoint of the trial. Telitacicept is approved in China for systemic lupus ery thematosus (SLE), rheumatoid arthritis (RA), and generalized myasthenia gravis (gMG). A global Phase 3 clinical trial in gMG is currently underway across the United States, Europe, South America, and Asia-Pacific to support potential approval in the United States, Europe, and Japan. Buy Or Sell Opportunity • Oct 02
Now 25% undervalued after recent price drop Over the last 90 days, the stock has fallen 4.0% to US$36.68. The fair value is estimated to be US$48.82, however this is not to be taken as a buy recommendation but rather should be used as a guide only. Announcement • Sep 23
Vor Biopharma Appoints Navid Z. Khan as Chief Medical Affairs Officer Vor Biopharma announced the appointment of Navid Z. Khan, Ph.D., as Chief Medical Affairs Officer. Dr. Khan joins Vor Bio with over two decades of experience spanning medical affairs, commercial, and R&D functions. He has overseen more than 40 development programs in neurology, immunology, and infectious diseases, and successfully guided seven U.S. and global product launches in rare neurology and immunology indications. Most recently, Dr. Khan was Head of Neuromuscular Therapeutic Area, Medical Affairs at argenx, where he led integrated medical strategy and execution for four launches of VYVGART® and VYVGART Hytrulo®, including two for generalized myasthenia gravis (gMG). Earlier in his career, Dr. Khan held senior leadership positions at Akouos Inc. and Sarepta Therapeutics, where he played a pivotal role in the launch of three Duchenne muscular dystrophy therapies - EXONDYS 51®, VYONDYS 53®, and AMONDYS 45® - and helped transform Sarepta’s Medical Affairs organization in preparation for a wave of gene therapy programs. He also held roles of increasing responsibility at EMD Millipore over more than a decade, spanning R&D, viral vector and vaccine strategy, and commercial leadership.Dr. Khan earned his Ph.D. in Biomedical Engineering and Biotechnology from the University of Massachusetts, Lowell, and his B.S. in Biochemistry and Molecular Biology from the University of Massachusetts, Amherst. Buy Or Sell Opportunity • Aug 25
Now 22% undervalued Over the last 90 days, the stock has risen 1,087% to US$2.06. The fair value is estimated to be US$2.64, however this is not to be taken as a buy recommendation but rather should be used as a guide only. New Risk • Aug 14
New major risk - Negative shareholders equity The company has negative equity. Total equity: -US$1.5b This is considered a major risk. Being in negative equity means that the company's liabilities exceed its assets, meaning it owes more to creditors than it has in owned assets. While this doesn't mean the company is about to collapse, in the long-term, this is unsustainable. The company may have issues meeting financial obligations, is at risk of becoming insolvent and may have difficulty raising capital, especially more debt, if needed. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (37% average weekly change). Negative equity (-US$1.5b). Shareholders have been substantially diluted in the past year (85% increase in shares outstanding). Revenue is less than US$1m. Minor Risk Currently unprofitable and not forecast to become profitable over next 3 years (US$60m net loss in 3 years). Buy Or Sell Opportunity • Aug 06
Now 21% undervalued Over the last 90 days, the stock has risen 1,206% to US$2.09. The fair value is estimated to be US$2.64, however this is not to be taken as a buy recommendation but rather should be used as a guide only. Announcement • Aug 05
Vor Biopharma Inc. Appoints Dallan Murray as Chief Commercial Officer Vor Biopharma Inc. announced the appointment of Dallan Murray as Chief Commercial Officer, effective immediately. Mr. Murray brings more than 25 years of experience leading commercial strategy, global product launches, and customer-facing organizations in biotechnology and pharmaceutical companies. He joins Vor Bio from Sarepta Therapeutics, where he most recently served as Executive Vice President, Chief Customer Officer. In that role, he led the commercial and medical affairs organizations, along with the international business unit. Under his leadership, Sarepta achieved approximately $1.8 billion in net product revenue in 2024. While at Sarepta he oversaw multiple product launches, including the launch of the first RNA therapy and first gene therapy for duchenne muscular dystrophy. Over the course of his career, Mr. Murray has led or supported more than a dozen product launches, including INCIVEK at Vertex Pharmaceutics Inc., the fastest launch to $1 billion in biotech history at the time, and multiple global rollouts at Gilead Sciences Inc., Biogen Inc., and Johnson & Johnson. At Gilead, he oversaw the launch of VIREAD in hepatitis B and maintained leadership in the HBV market. While at Biogen, he played a key role in the reintroduction and global commercialization of TYSABRI for multiple sclerosis, helping drive adoption across North America and international markets. Mr. Murray holds an MBA from Queen’s University in Ontario, Canada, and a Bachelor of Commerce from the University of Alberta. Announcement • Jul 21
Vor Biopharma Inc. Appoints Alexander (Bo) Cumbo and Michel Detheux to Board of Directors, Effective July 16, 2025 Vor Biopharma Inc. announced the appointment of Alexander (Bo) Cumbo and Michel Detheux, Ph.D., to its Board of Directors, effective July 16, 2025. Alexander (Bo) Cumbo currently serves as President and Chief Executive Officer of Solid Biosciences. Prior to that, he was the founding CEO of AavantiBio, a gene therapy company backed by Bain Capital Life Sciences, Perceptive Advisors, and RA Capital. Bo brings more than 30 years of commercial and executive leadership, including nearly eight years at Sarepta Therapeutics, where he most recently served as Executive Vice President and Chief Commercial Officer. He played a central role in launching multiple rare disease therapies and building global commercial operations. He currently serves on the boards of Climb Bio and Verve Therapeutics and previously served on the boards of RA Pharma and CSM through successful transactions. Mr. Cumbo earned a Bachelor of Science in Laboratory Technology from Auburn University. Michel Detheux, Ph.D., is President and Chief Executive Officer of iTeos Therapeutics, which he co-founded and led from discovery to late-stage clinical development. Under his leadership, iTeos raised over $1 billion in capital, completed a successful IPO, and secured a landmark $2 billion partnership with GSK to co-develop and commercialize the company’s lead anti-TIGIT antibody. Prior to founding iTeos, Dr. Detheux was a Director at Ludwig Cancer Research and held scientific and business development roles at Euroscreen (now Ogeda). He currently serves as Chairman of the Board at Egle Therapeutics. Dr. Detheux holds a Ph.D. in Biochemistry and a degree in Bioengineering from Université Catholique de Louvain. Announcement • Jul 17
Vor Biopharma Inc. Appoints Qing Zuraw as Chief Development Officer Vor Biopharma Inc. announced the appointment of Qing Zuraw, M.D., M.P.H., M.B.A., as Chief Development Officer, effective immediately. Dr. Zuraw joins Vor Bio with over 25 years of experience leading complex global and U.S. clinical development programs across autoimmune, inflammatory, and immunologic diseases. Most recently, she served as Chief Development Officer and Head of Global Clinical Development for Autoimmune Diseases at RemeGen Co. Ltd., where she was one of the key leaders of successful development and execution of clinical trials for telitacicept across four key indications—systemic lupus erythematosus (SLE), Sjögren’s syndrome, myasthenia gravis (MG), and rheumatoid arthritis (RA)—culminating in regulatory approvals in China for the treatment of SLE, generalized MG and RA.At RemeGen, Dr. Zuraw built and led a cross-functional global team that managed all aspects of telitacicept development, including clinical trial design, regulatory strategy, site engagement, and execution. She played a central role in regulatory interactions with the U.S. Food & Drug Administration, European Medicines Agency, and China’s Center for Drug Evaluation, achieving Fast Track, Breakthrough Therapy, and Orphan Drug designations for telitacicept across multiple indications. Dr. Zuraw has also previously held senior leadership roles at Janssen Research & Development, Teva Pharmaceutical Industries Ltd., Akebia Therapeutics Inc., Biogen Inc., and Covance Inc., where she led global clinical development programs across rheumatology, nephrology, respiratory, and immunology. She played a key role in the U.S. FDA approval of Guselkumab for psoriatic arthritis and contributed to multiple NDA and BLA submissions across therapeutic areas. Throughout her career, she has built and led high-performing teams to execute complex trials from early development through post-marketing. Recent Insider Transactions Derivative • Jul 01
Strategic Advisor notifies of intention to sell stock Robert Ang intends to sell 41k shares in the next 90 days after lodging an Intent To Sell Form on the 30th of June. If the sale is conducted around the recent share price of US$1.73, it would amount to US$70k. Since September 2024, Robert's direct individual holding has increased from 580.88k shares to 622.96k. There have been no trades via on-market transactions or options from company insiders in the last 12 months. Announcement • May 08
Vor Biopharma Inc. to Report Q1, 2025 Results on May 14, 2025 Vor Biopharma Inc. announced that they will report Q1, 2025 results on May 14, 2025 Announcement • Apr 26
Vor Biopharma Receives A Notice from the Nasdaq Stock Market On April 22, 2025, Vor Biopharma Inc. (the “Company”) received a notice from The Nasdaq Stock Market (“Nasdaq”) that the Company is not in compliance with Nasdaq’s Listing Rule 5450(a)(1), as the minimum bid price of the Company’s common stock has been below $1.00 per share for 30 consecutive business days. The notification of noncompliance has no immediate effect on the listing or trading of the Company’s common stock on The Nasdaq Global Select Market. The Company has 180 calendar days, or until October 20, 2025, to regain compliance with the minimum bid price requirement. To regain compliance, the minimum bid price of the Company’s common stock must meet or exceed $1.00 per share for a minimum of ten consecutive business days during this 180-calendar day grace period. In the event the Company does not regain compliance with the minimum bid price requirement by October 20, 2025, the Company may be eligible for an additional 180-calendar day compliance period if it elects to transfer to The Nasdaq Capital Market to take advantage of the additional compliance period offered on that market. To qualify, the Company would be required to meet the continued listing requirement for market value of publicly held shares and all other initial listing standards for The Nasdaq Capital Market, with the exception of the bid price requirement, and would need to provide written notice of its intention to cure the bid price deficiency during the second compliance period by effecting a reverse stock split if necessary. In the event the Company fails to regain compliance or transfer to The Nasdaq Capital Market before the compliance period expires, the Company will receive a written notification from Nasdaq that its common stock is subject to delisting. If the Company were to receive such a notification, the Company could appeal Nasdaq’s determination to delist its common stock, but there can be no assurance Nasdaq would grant the Company’s request for continued listing. The Company intends to actively monitor the bid price of its common stock and will consider available options to regain compliance with the listing requirements. There can be no assurance that the Company will be able to regain compliance with Nasdaq’s Listing Rule 5450(a)(1) or will otherwise be in compliance with other Nasdaq listing criteria. Announcement • Apr 04
Vor Biopharma Inc. Announces Resignation of Eyal C. Attar as Chief Medical Officer, Effective April 18, 2025 On March 31, 2025, Eyal C. Attar, M.D., notified Vor Biopharma Inc. (the “Company”) of his decision to resign as Chief Medical Officer of the Company, effective as of April 18, 2025, in order to pursue a new opportunity. Announcement • Apr 01
Vor Biopharma Inc., Annual General Meeting, May 22, 2025 Vor Biopharma Inc., Annual General Meeting, May 22, 2025. New Risk • Mar 28
New minor risk - Market cap size The company's market capitalization is less than US$100m. Market cap: US$95.6m This is considered a minor risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$100m free cash flow). Shareholders have been substantially diluted in the past year (83% increase in shares outstanding). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$131m net loss in 3 years). Share price has been volatile over the past 3 months (16% average weekly change). Market cap is less than US$100m (US$95.6m market cap). New Risk • Mar 21
New major risk - Financial position The company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$100m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$100m free cash flow). Share price has been highly volatile over the past 3 months (22% average weekly change). Shareholders have been substantially diluted in the past year (83% increase in shares outstanding). Revenue is less than US$1m. Minor Risk Currently unprofitable and not forecast to become profitable over next 3 years (US$172m net loss in 3 years). Announcement • Feb 15
Vor Bio Presents Novel Research Highlighting Opportunities and Challenges Facing Institutions Enrolling Patients in Cell and Gene Therapy Trials Vor Bio presented novel research evaluating the patient experience and barriers to enrollment and participation in cell and gene therapy (CGT) trials. The data, which was presented at the TANDEM Meetings of ASTCT and CIBMTR, demonstrated the need to improve the process and experience of patients considering and enroll in CGT trials. Despite the growing number of CGT trials, their complexity, logistical challenges including treatment duration and caregiver burden, and the significant resources they require represent important barriers that delay enrollment and limit patient access. The results reveal significant opportunities for institutions and trial sponsors to improve the patient experience, streamline the process, and enroll more patients in CGT trials. Clinical trial sponsors can address the complexity of cell and gene therapies and help patients better understand these options by providing more comprehensive educational content beyond the handouts typically shared. Additionally, as hospitals are often limited in how many CGT trials they can offer, adding a specialized CGT research team could potentially expand their capacity to offer more trials and treat more patients. This research will inform Vor Bio's communications approach for future clinical trials and how the Company can help support patients and partner institutions through the enrollment process. Vor Bio plans to continue this research to uncover additional learnings that may lead to further improvements in the patient experience and CGT clinical trial enrollment. The study coauthors include staff at NMDP (National Marrow Donor Program), MDS Foundation, Memorial Sloan Kettering Cancer Center, University Hospitals Cleveland Medical Center, Miami Cancer Institute, and Stanford Medicine. The words "aim," "anticipate," "can," "continue," "continue," "could," "design," " enable," "intend," "on-track," "ongoing," "plan," "pot potential," "target," update," "will," " would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. New Risk • Jan 10
New major risk - Shareholder dilution The company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 84% This is considered a major risk. Shareholder dilution occurs when there is an increase in the number of shares on issue that is not proportionally distributed between all shareholders. Often due to the company raising equity capital or some options being converted into stock. All else being equal, if there are more shares outstanding then each existing share will be entitled to a lower proportion of the company's total earnings, thus reducing earnings per share (EPS). While dilution might not always result in lower EPS (like if the company is using the capital to fund an EPS accretive acquisition) in a lot cases it does, along with lower dividends per share and less voting power at shareholder meetings. Currently, the following risks have been identified for the company: Major Risks Share price has been highly volatile over the past 3 months (22% average weekly change). Shareholders have been substantially diluted in the past year (84% increase in shares outstanding). Revenue is less than US$1m. Minor Risk Currently unprofitable and not forecast to become profitable over next 3 years (US$115m net loss in 3 years). Announcement • Jan 09
Vor Bio Appoints Erez Kalir to Its Board of Directors Vor Bio announced the appointment of Mr. Erez Kalir to its Board of Directors. The appointment represents a new seat on Vor Bio’s Board of Directors, which has been created in connection with the recent private investment in public equity financing (PIPE) which was led by Reid Hoffman. Mr. Kalir will serve as the director designee of Reprogrammed Interchange, LLC, Mr. Hoffman’s investment fund. Mr. Kalir brings a wealth of experience as an investor, entrepreneur, and thought leader with a deep focus on life sciences and technology. Currently the Managing Member of Martial Eagle Fund and a Venture Partner at FJ Labs—renowned for its leadership in early-stage venture investments—Mr. Kalir is widely recognized for his strategic acumen and ability to drive growth in innovative industries. In addition to his investment leadership, Mr. Kalir authors the influential Biotech Frontiers newsletter for Porter & Co., offering incisive analysis on cutting-edge biotech companies. His interdisciplinary expertise in biology, finance, and law uniquely positions him to contribute to Vor Bio’s mission of advancing breakthrough innovations in cancer and beyond. Prior to his current roles, Mr. Kalir worked with legendary investor Julian H. Robertson at Tiger Management, advising on investments in biotech and health care. He co-founded and led Sabretooth Capital, a hedge fund managing assets for prominent institutions and families, and further honed his expertise at Eton Park and McKinsey & Co. Mr. Kalir is a Rhodes Scholar and holds an MSc in cell biology from Magdalen College, Oxford, a JD from Yale Law School, and an A.B. with highest honors from Stanford University. Announcement • Dec 31
Vor Biopharma Inc. announced that it has received $55.55 million in funding from RA Capital Management, L.P. On December 30, 2024. Vor Biopharma Inc. closed the transaction. The PIPE was led by new investor, Reid Hoffman, and included participation from existing investor and Vor Bio's largest stockholder, RA Capital Management. In addition, as part of the PIPE, each of Mr. Hoffman, or his duly appointed nominee, and RA Capital Management are being granted one board seat and one board observer seat. Price Target Changed • Dec 29
Price target decreased by 11% to US$11.79 Down from US$13.21, the current price target is an average from 7 analysts. New target price is 828% above last closing price of US$1.27. Stock is down 44% over the past year. The company is forecast to post a net loss per share of US$1.44 next year compared to a net loss per share of US$1.75 last year. Announcement • Dec 28
Vor Biopharma Inc. announced that it expects to receive $55.55 million in funding Vor Biopharma Inc. entered into a Securities Purchase Agreement to issue 55,871,260 shares at a price of $0.99425 per share for gross proceeds of $55.55 million and accompanying warrants to purchase up to 69,839,075 shares of Common Stock on December 26, 2024. The closing of the Private Placement is expected to occur on December 30, 2024. Each Common Stock Warrant has an exercise price of $0.838 per Warrant Share. The Common Stock Warrants will be exercisable immediately and will expire seven years from the date of issuance. Announcement • Dec 10
Vor Bio Provides Clinical Update Further Validating Approach of Using Shielded Transplants to Deliver Targeted Therapies; Receives Supportive Feedback from Fda Regarding Registrational Trial Design Vor Bio announced updated clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg. The data, which was presented in a poster at the American Society of Hematology Annual Meeting on Sunday, December 8th, demonstrated durable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of improved relapse free survival compared to published high-risk AML comparators. The data released included 25 patients treated with trem-cel of which 15 had received Mylotarg (six at the 2 mg/m2 dose) as of the data cut-off date of November 1, 2024. The data demonstrated: Preliminary evidence of improved relapse-free survival (median RFS not reached with median follow-up duration of 7.4 months) compared to published groups of AML patients at high risk of relapse post hematopoietic stem cell transplant. Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2. Broadened therapeutic index for Mylotarg when administered after trem-cel. Reliable engraftment, with 100% of patients achieving primary neutrophil engraftment? (median 9.5 days), robust platelet recovery (median 16 days)?, and full myeloid donor chimerism ?at Day 28. Trem-cel continues to be manufactured with high CD33 editing efficiency (median 90%, range 71-94%)?. Company received supportive feedback from the FDA in a Type C meeting, The Company had the opportunity to interact with the FDA regarding data from the trem-cel + Mylotarg study alongside a proposed registrational clinical trial synopsis. The FDA agreed that trem-cel engrafts neutrophils and platelets and has a similar safety profile to unedited CD34+ grafts. In addition, there was agreement with the trem-cel + Mylotarg registrational clinical trial design with respect to study population, control arm, primary endpoint, stratification factors, and statistical design. The Company agreed to provide further updates to the FDA alongside submission of the full clinical trial protocol. Price Target Changed • Nov 10
Price target decreased by 9.5% to US$11.94 Down from US$13.19, the current price target is an average from 8 analysts. New target price is 1,146% above last closing price of US$0.96. Stock is down 46% over the past year. The company is forecast to post a net loss per share of US$1.62 next year compared to a net loss per share of US$1.75 last year. Announcement • Sep 30
Vor Biopharma Inc. Appoints Han Choi as Chief Financial Officer Vor Biopharma Inc. announced the appointment of Han Choi, M.D., LL.M., as its new chief financial officer, effective immediately. Dr. Choi will join the Vor Bio leadership team and will report directly to the company’s President and Chief Executive Officer, Dr. Robert Ang. Dr. Choi brings to Vor Bio over twenty-five years of experience in public and private investment management, business development, and corporate strategy within the pharmaceutical and biotechnology industry. His deep expertise in structuring corporate partnerships and investments, managing complex transactions, and capital markets strategy will play a critical role as Vor Bio advances its clinical pipeline and continues to expand its leadership in the field of cell and genome engineering. Prior to joining Vor Bio, Dr. Choi was a Principal at Oracle Investment Management Inc. For over two decades at Oracle, he was responsible for sourcing, negotiating, and managing investments in the pharmaceutical and biotechnology sectors, and provided strategic advice to portfolio companies on clinical, corporate development, and capital market strategies. Prior to joining Oracle, Dr. Choi held positions of increasing responsibility in licensing and business development at Pharmacia Corporation and Bristol-Myers Squibb Company. He received his M.D. from the Mount Sinai School of Medicine and holds law degrees from Oxford University and Harvard Law School. Dr. Choi is a licensed physician in New York State and a member of the New York State Bar. New Risk • Sep 10
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 11% 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: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$105m free cash flow). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$113m net loss in 3 years). Share price has been volatile over the past 3 months (11% average weekly change). Market cap is less than US$100m (US$74.6m market cap). Announcement • Sep 06
Vor Bio Announces New Clinical Data from Its Ongoing Phase 1/2 VBP101 Study of Patients with Relapsed/Refractory AML Receiving trem-cel Followed by Mylotarg™ Vor Bio announced new clinical data from its ongoing Phase 1/2 VBP101 study of patients with relapsed/refractory AML receiving trem-cel followed by Mylotarg™. The data demonstrated reliable engraftment, shielding from Mylotarg on-target toxicity, a broadened Mylotarg therapeutic window, and early evidence of patient benefit. The data released included 18 patients treated with trem-cel of which ten had received Mylotarg as of the data cut-off date of July 19, 2024. The data demonstrated: Reliable engraftment, with 100% of patients achieving primary neutrophil engraftment? (median 9 days) and robust platelet recovery (median 16.5 days)?. High CD33 editing efficiency (median 89%, range 71-94%)? and full myeloid chimerism ?at Day 28; Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2; Broadened therapeutic index for Mylotarg with drug exposure represented by AUC which is related to efficacy, consistent with labeled Mylotarg doses, and with maximal concentrations, measured by Cmax and related to veno-occlusive disease, well below known toxic range; Early evidence suggesting patient benefit as measured by relapse-free survival when compared to published high-risk AML comparators. Vor Bio plans to approach the U.S. Food & Drug Administration to discuss a pivotal trial design for trem-cel + Mylotarg by around year end. Continued progress with VCAR33ALLO: VCAR33ALLO represents another potentially significant synergistic treatment option after trem-cel; The VBP301 study continues enrolling patients with initial focus on relapsed/refractory AML post-transplant; Vor Bio is encouraged by in vivo CAR-T expansion data from three patients treated to date, all at the lowest dose of 1 x 106 CAR+ cells/kg. Vor Bio announced a new preclinical asset, VADC45, which has a number of potential opportunities in oncology, gene therapy, and autoimmune disorders - VADC45 is an ADC that targets the CD45 protein. CD45 is a well-validated target for a wide variety of blood cancers with clinical proof of concept. The linker-payload used in VADC45 is also clinically validated; VADC45 has the potential to treat a number of diseases, including treatment of hematologic malignancies, as a targeted conditioning agent for gene therapies such as for sickle cell disease, holistic immune reset for autoimmune disorders, and for Vor Bio's approach of combining this asset with epitope modification of CD45 to shield healthy stem cells; and Vor Bio already has robust preclinical data for VADC45 and is progressing IND-enabling studies to enable future Phase 1 studies. Announcement • Sep 01
Vor Biopharma Receives Notice from Nasdaq Due to Non-Compliance with the Minimum Bid Price Requirement On August 29, 2024, Vor Biopharma Inc. (the ‘Company’) received a notice from The Nasdaq Stock Market (‘Nasdaq’) that the Company is not in compliance with Nasdaq’s Listing Rule 5450(a)(1), as the minimum bid price of the Company’s common stock has been below $1.00 per share for 30 consecutive business days. The notification of noncompliance has no immediate effect on the listing or trading of the Company’s common stock on The Nasdaq Global Select Market. The Company has 180 calendar days, or until February 25, 2025, to regain compliance with the minimum bid price requirement. To regain compliance, the minimum bid price of the Company’s common stock must meet or exceed $1.00 per share for a minimum of ten consecutive business days during this 180-calendar day grace period. In the event the Company does not regain compliance with the minimum bid price requirement by February 25, 2025, the Company may be eligible for an additional 180-calendar day compliance period if it elects to transfer to The Nasdaq Capital Market to take advantage of the additional compliance period offered on that market. To qualify, the Company would be required to meet the continued listing requirement for market value of publicly held shares and all other initial listing standards for The Nasdaq Capital Market, with the exception of the bid price requirement, and would need to provide written notice of its intention to cure the bid price deficiency during the second compliance period by effecting a reverse stock split if necessary. In the event the Company fails to regain compliance or transfer to The Nasdaq Capital Market before the compliance period expires, the Company will receive a written notification from Nasdaq that its common stock is subject to delisting. If the Company were to receive such a notification, the Company could appeal Nasdaq’s determination to delist its common stock, but there can be no assurance Nasdaq would grant the Company’s request for continued listing. The Company intends to actively monitor the bid price of its common stock and will consider available options to regain compliance with the listing requirements. There can be no assurance that the Company will be able to regain compliance with Nasdaq’s Listing Rule 5450(a)(1) or will otherwise be in compliance with other Nasdaq listing criteria. New Risk • May 30
New minor risk - Market cap size The company's market capitalization is less than US$100m. Market cap: US$96.2m This is considered a minor risk. Companies with a small market capitalization are most likely businesses that have not yet released a product to market or are simply a very small company without a wide reach. Either way, risk is elevated with these companies because there is a chance the product may not come to fruition or the company's addressable market or demand may not be as large as expected. In addition, if the company's size is the main factor, it is less likely to have many investors and analysts following it and scrutinizing its performance and outlook. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$108m free cash flow). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$119m net loss in 3 years). Market cap is less than US$100m (US$96.2m market cap). New Risk • May 10
New minor risk - Financial position The company has less than a year of cash runway based on its current free cash flow. Free cash flow: -US$108m This is considered a minor risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risk Revenue is less than US$1m. Minor Risks Less than 1 year of cash runway based on current free cash flow (-US$108m). Currently unprofitable and not forecast to become profitable over next 3 years (US$116m net loss in 3 years). Announcement • May 03
Vor Bio Appoints Fouad Namouni to its Board of Directors Vor Bio announced the appointment of Fouad Namouni, M.D., to its Board of Directors. Dr. Namouni currently serves as President of Research & Development at Blueprint Medicines, bringing a wealth of industry experience and expertise to Vor Bio's Board. In his role at Blueprint Medicines, Dr. Namouni has demonstrated exceptional leadership in building the company's fully integrated business and advancing a broad pipeline of innovative medicines to address significant medical needs in oncology/hematology and allergy/inflammation. With over two decades of experience in clinical development, regulatory affairs, and medical affairs within the biotechnology and pharmaceutical industries, Dr. Namouni's insights and strategic guidance will be invaluable to Vor Bio as it continues to advance its novel approach to curing acute myeloid leukemia. Dr. Namouni has over 20 years of oncology and cancer immunotherapy drug development expertise, as well as clinical experience as a pediatric oncologist. Prior to his current role as President of Research & Development, at Blueprint Medicines, Dr. Namouni held multiple senior leadership positions at Bristol-Myers Squibb where he played a pivotal role in the development and commercialization of oncology therapies including Opdivo® and Yervoy®. Throughout his career, Dr. Namouni has been dedicated to driving scientific innovation and improving patient outcomes. Dr. Namouni holds an M.D. from the University of Annaba Medical School in Algeria, and a Pediatrics degree from Université Rene Descartes in Paris, France. In addition, he received a Pediatric Oncology and Hematology degree and an M.S. in clinical and experimental pharmacology from Université Paris-Sud in France. Announcement • Apr 10
Vor Biopharma Inc., Annual General Meeting, May 23, 2024 Vor Biopharma Inc., Annual General Meeting, May 23, 2024, at 12:00 US Eastern Standard Time. Agenda: To elect each of the two Class III director nominees; to approve the amendment and restatement of 2021 Equity Incentive Plan to, among other things, increase the number of shares that will automatically be added to the share reserve thereunder on January 1 of each calendar year from 4% to 5% of the total number of shares of Common Stock outstanding on December 31 of the preceding calendar year through and including calendar year 2034; to ratify the selection by the audit committee of the Board of Directors of Ernst & Young LLP as independent registered public accounting firm for the fiscal year ending December 31, 2024; and to conduct any other business properly brought before the meeting or any adjournment or postponement thereof. New Risk • Mar 22
New major risk - Financial position The company has less than a year of cash runway based on its current free cash flow trend. Free cash flow: -US$101m This is considered a major risk. With less than a year's worth of cash, the company will need to raise capital or take on debt unless its cash flows improve. This would dilute existing shareholders or increase balance sheet risk. Currently, the following risks have been identified for the company: Major Risks Less than 1 year of cash runway based on free cash flow trend (-US$101m free cash flow). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$115m net loss in 3 years). Share price has been volatile over the past 3 months (15% average weekly change). Shareholders have been diluted in the past year (3.8% increase in shares outstanding). Board Change • Feb 01
Insufficient new directors No new directors have joined the board in the last 3 years. The company's board is composed of: No new directors. 12 experienced directors. No highly experienced directors. Scientific & Clinical Advisor Soheil Meshinchi was the last director to join the board, commencing their role in 2021. The following issues are considered to be risks according to the Simply Wall St Risk Model: Insufficient board refreshment. Announcement • Jan 18
Vor Bio Doses First AML Patient with VCAR33 (ALLO) Vor Bio announced it has dosed the first patient in VBP301, its Phase 1/2, multicenter, open-label, first-in-human study of VCAR33ALLO. The Company has extended its cash runway into the second half of 2025. The first patient with relapsed/refractory acute myeloid leukemia (AML) has been dosed with VCAR33ALLO in the VBP301 clinical trial, a significant milestone demonstrating that VCAR33ALLO can be successfully manufactured in Vor Bio’s in-house manufacturing facility. VCAR33ALLO is manufactured from lymphocytes collected from the patient’s original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient’s engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient’s own lymphocytes. While this first patient had relapsed after a standard-of-care transplant, patients who have relapsed after a trem-cel transplant are also eligible to enroll in the VBP301 protocol and to receive VCAR33ALLO. The ability to treat relapsed trem-cel transplant patients with VCAR33ALLO may provide valuable early insights into the potential of the Company’s trem-cel + VCAR33 Treatment System, which pairs VCAR33 after trem-cel to reduce the risk of relapse or treat evidence of relapse. Initial data from VBP301 is expected in the second half of 2024. The Company has further extended its cash runway into the second half of 2025 through an internal review and prioritization process and will continue to invest in its platform and clinical programs while strategically prioritizing late-stage programs and managing employee growth to preserve cash. Announcement • Nov 11
Vor Bio Presents Positive Data Update on Trem-cel at HSCT² Conference Vor Bio presented updated clinical data from patients treated in VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (VOR33) in patients with acute myeloid leukemia (AML). Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida will present these data on November 10, in an oral presentation at the ASTCT/EBMT 6thInternational Conference on Relapse After Transplant and Cellular Therapy (HSCT²) in Los Angeles, California. Primary neutrophil engraftment occurred in all seven patients treated to date with trem-cel with a median time to engraftment of 10 days, further providing evidence that CD33 is biologically dispensable. Additionally, platelet recovery occurred at a median of 15.5 days, excluding one patient with previously documented anti-platelet antibodies (immune thrombocytopenia). All three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity (myelosuppression) typically seen with Mylotarg treatment. The hematological protection exhibited provides support that dose escalation of Mylotarg is warranted and highlights the potential to dose CD33-targeted CAR-T therapy without expected hematologic toxicity. Mylotarg first-dose pharmacokinetics for the three patients treated showed that the 0.5 mg/m2 dose was within the exposure range measured for the therapeutic dose of Mylotarg in relapsed/refractory AML patients, potentially due to the decreased CD33 antigen sink in trem-cel patients. In all three patients, the percentage of CD33-negative donor cells increased following Mylotarg administration, suggesting that Mylotarg treatment at the first cohort level of 0.5 mg/m2 was pharmacologically active and enriched for CD33-edited donor cells. Next steps in the VBP101 study include dose escalation of Mylotarg to 1.0 mg/m2 and providing treatment opportunities for trem-cel patients who become measurable residual disease positive or relapse, such as induction-course Mylotarg and VCAR33ALLO. As previously announced, the Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) released data in an ASH abstract from its Phase 1/2 study (NCT03971799)1 of CD33CART (also known as VCAR33AUTO), which uses the same CAR-T construct as the Company’s VCAR33ALLO. The data show that 2 of 5 (40%) evaluable patients treated at the highest dose level achieved complete remission with a manageable safety profile (four out of 19 evaluable patients had cytokine release syndrome = Grade 3). This data further validates Vor Bio’s approach of using transplant donor cells as CAR-T starting material that are healthy, stem-like, and exactly matched to the patient’s immune system. AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting 20,000 newly diagnosed patients each year in the United States. Approximately half of patients with AML who receive a hematopoietic cell transplant (HCT) suffer a relapse of their leukemia, with two-year survival rates of less than 20%, and relapse rates are higher for patients with certain adverse risk features. The fragility of engrafted hematopoietic stem cells prevents treatment following transplant, giving the cancer a chance to return. VBP101 is a Phase 1/2a, multicenter, open-label, first-in-human study of trem-cel (VOR33) in participants with AML who are undergoing human leukocyte antigen (HLA)-matched allogeneic HCT. Trem-cel is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product, lacking the CD33 protein. It is being investigated for participants with CD33+ AML at high risk for relapse after HCT to allow post-HCT targeting of residual CD33+ acute AML cells using Mylotarg (gemtuzumab ozogamicin) without toxicity to engrafted cells. Participants undergo a myeloablative HCT with matched related or unrelated donor CD34-selected HSPCs engineered to remove CD33 expression (trem-cel drug product). Mylotarg is given after engraftment for up to four cycles. The primary endpoint is the incidence of successful engraftment, defined as the first day of 3 consecutive days of absolute neutrophil count (ANC) =500 cells/mm2 by day 28. Part 1 of this study is evaluating the safety of escalating Mylotarg dose levels to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose. Part 2 will expand the number of participants to evaluate the Mylotarg recommended Phase 2 dose. Tremtelectogene empogeditemcel (trem-cel), formerly VOR33, is a genome-edited hematopoietic stem and progenitor allogeneic donor product candidate where CD33 has been deleted using genome engineering. Transplant with trem-cel is designed to replace standard of care transplants for patients suffering from AML and potentially other blood cancers. Trem-cel has the potential to enable powerful targeted therapies in the post-transplant setting including CD33-targeted CAR-T cells. Announcement • Nov 03
Vor Bio Announces Multiple Clinical and Preclinical Presentations at ASH 2023 Highlight Vor Bio's Novel eHSC and CAR-T Platform VV Bio announced that preclinical and clinical data supporting the Company's novel platform and approach for treating acute myeloid leukemia (AML), will be presented at the 65th American Society of Hematology (ASH) Annual Meeting & Exposition, being held from December 9-12, 2023, in San Diego, CA. An abstract providing a clinical update from the VBP101 clinical trial (NCT048499910), a Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (VOR33) in patients with AML, was accepted by ASH for oral presentation. This data supports robust neutrophil engraftment of trem-cel and provides evidence of hematologic protection from Mylotarg(TM), a CD33-targeted antibody drug conjugate. An updated data release from VBP101 is expected by the Relapse After Transplant and Cellular Therapy (HSCT(2)) conference taking place November 10-11, 2023. CD33CART Study Clinical Data Update The Pediatric Transplantation and Cellular Therapy Consortium (PTCTC) released data providing a clinical update on a Phase 1/2 study of CD33CART (NCT03971799)(1), an autologous CAR-T therapy targeting CD33 (also referred to as VCAR33(AUTO) which uses the same CAR-T construct as VCAR33(A) (LLO). Nineteen pediatric and young adult patients with relapsed/refractory AML with a median age of 16 years were infused in the Phase 1 portion of the study. Sarah K. Tasian, MD, is a co-investigator on the PTCTC-supported clinical trial and led the preclinical testing and translation of CD33CART with Terry Fry, MD, at the University of Colorado. Lastly, a trial-in-progress poster will be presented on the Company's VBP301 clinical trial, a Phase 1/2 multicenter, open- label, first-in-human trial of VCAR33(ALLO) in patients with relapsed or refractory AML after allogeneic stem cell transplantation. Price Target Changed • Aug 13
Price target decreased by 7.2% to US$13.75 Down from US$14.81, the current price target is an average from 8 analysts. New target price is 368% above last closing price of US$2.94. Stock is down 46% over the past year. The company is forecast to post a net loss per share of US$1.82 next year compared to a net loss per share of US$2.33 last year. New Risk • Jun 29
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 10% 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: Major Risks Earnings are forecast to decline by an average of 3.9% per year for the foreseeable future. Shareholders have been substantially diluted in the past year (78% increase in shares outstanding). Revenue is less than US$1m. Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$155m net loss in 3 years). Share price has been volatile over the past 3 months (10% average weekly change). Announcement • Jun 10
Vor Biopharma Inc. Presents Updated Clinical Data from Patients Treated in VBP101 Vor Biopharma Inc. announced VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (formerly VOR33) in patients with acute myeloid leukemia (AML). These data were presented by Guenther Koehne, MD, PhD, Deputy Director and Chief of Blood & Marrow Transplant and Hematologic Oncology at Miami Cancer Institute of Baptist Health South Florida in a poster presentation at the European Hematology Association (EHA) 2023 Congress in Frankfurt, Germany. As previously reported, neutrophil and platelet cell counts were maintained in patient 1 who received multiple Mylotarg doses at 0.5 mg/m2, suggesting protection from Mylotarg-induced hematotoxicity. The Company also announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for VCAR33ALLO, a T-cell therapy derived from allogeneic healthy donors using a chimeric antigen receptor (CAR) specifically binding to CD33. Trem-cel is planned to be studied in the VBP301 clinical trial, which will focus on patients who have relapsed following allogeneic stem cell transplant where T cells harvested from the original donor are used as starting material for the drug product. VBP101 is a Phase 1/2a, multicenter, open- label, first-in-human trial of trem-cel (VOR33) in participants with AML who are undergoing human leukocyte antigen (HLA)-matched allogeneic HCT. Trem-cel is an allogeneic CRISPR/Cas9 genome-edited hematopoietic stem and progenitor cell (HSPC) therapy product, lacking the CD33 protein. Price Target Changed • Mar 24
Price target decreased by 19% to US$14.82 Down from US$18.25, the current price target is an average from 8 analysts. New target price is 152% above last closing price of US$5.88. Stock is down 13% over the past year. Announcement • Feb 18
Vor Biopharma Inc. Presents Clinical Data from VBP101 Vor Biopharma Inc. presented clinical data from VBP101, its Phase 1/2a multicenter, open-label, first-in-human study of trem-cel (previously VOR33) in patients with acute myeloid leukemia (AML). In the first patient, trem-cel maintained hematopoesis through three cycles of Mylotarg (gemtuzumab ozogamicin), which was well-tolerated at the initial dose level of 0.5 mg/m2. A second patient has successfully received a trem-cel transplant and engrafted normally. These data were presented by Miguel-Angel Perales, MD, Chief, Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center in a late-breaking poster at the 2023 Tandem Meetings (Transplantation & Cellular Therapy Meetings of ASTCT™ and CIBMTR®) in Orlando, FL. Trem-cel Safety & Durability: Patient 1 maintained neutrophil and platelet counts approximately five months (147 days) after transplantation with trem-cel. Due to detectable measurable residual disease (MRD), Patient 1 was moved to other therapies following administration of the third dose of Mylotarg, subsequently relapsed, and remains on study for long-term follow-up. Similar to Patient 1, Patient 2 successfully received a trem-cel transplant and showed robust cell recovery with neutrophil engraftment occurring at Day 11 and platelet recovery on Day 17. Trem-cel was well tolerated in both patients, with no related and no unexpected adverse events (AEs) reported. No Hematological Toxicity Observed Through Repeated Doses of Mylotarg: In Patient 1, neutrophil and platelet cell counts were maintained following three sequential Mylotarg doses at 0.5 mg/m2. This suggests potential protection from Mylotarg-related hematotoxicity. The only AE observed possibly related to Mylotarg through dose 3 was low grade nausea and vomiting, a known side-effect of Mylotarg. Mylotarg first-dose pharmacokinetics revealed 0.5 mg/m2 achieved Cmax and AUC parameters equivalent to 1-2 and 4-5 mg/m2 accordingly, potentially due to the decreased CD33 antigen sink. Evidence of Mylotarg Causing CD33-negative Donor Cell Enrichment: In Patient 1, CD33-negative donor hematopoiesis was enriched across hematopoietic cell types following Mylotarg administration. In addition, the CD33 deletion was observed in donor cells of myeloid and lymphoid origin which were both enriched following Mylotarg, suggesting that CD33 is expressed in early hematopoietic cells and that Mylotarg treatment enriches for edited donor cells. Interest in enrollment in VBP101 continues to be strong with a high level of investigator enthusiasm at all nine study sites. The company is moving forward with dose escalation of Mylotarg per the 3+3 dose escalation schema in the protocol. The Company is also on-track to submit an IND in the first half of 2023 for VCAR33ALLO, a CAR-T therapy using allogeneic healthy donor-derived cells, which it intends use in combination with trem-cel as a Treatment System. Announcement • Dec 09
Vor Biopharma Inc. announced that it expects to receive $50 million in funding from RA Capital Management, L.P. Vor Biopharma Inc. entered into a securities purchase agreement with RA Capital Healthcare Fund, L.P., a fund managed by RA Capital Management, L.P. for a private placement of 11,627,907 common shares at a price of $4.30 per share for gross proceeds of approximately $50 million on December 7, 2022. The transaction is expected to close on or about December 9, 2022, subject to customary closing conditions. In the event the registration statement has not become effective by December 31, 2022, the company has agreed to issue to the investor warrants to purchase an aggregate of 116,279 shares of with an exercise price of $4.30 per share. The term of the warrants, if issued, will be one year. Announcement • Dec 08
Vor Bio Announces First AML Patient Successfully Transplanted with VOR Bio's Investigational Trem-Cel (Vor33) and Tolerated Mylotarg(Tm) Vor Bio announced initial clinical data from VBP101, its Phase 1/2a multicenter, open-label, first- in-human study of tremtelectogene empogeditemcel or "trem-cel" (formerly VOR33) in patients with acute myeloid leukemia (AML). The data observed from the first treated patient support the potential of a trem-cel transplant to be successfully manufactured, to engraft normally, and to maintain blood counts following treatment with the CD33-targeted therapy Mylotarg. The clinical trial continues to enroll patients and additional data are expected in 2023. Price Target Changed • Nov 16
Price target decreased to US$18.13 Down from US$21.13, the current price target is an average from 8 analysts. New target price is 293% above last closing price of US$4.61. Stock is down 73% over the past year. The company is forecast to post a net loss per share of US$2.48 next year compared to a net loss per share of US$2.10 last year. Announcement • Oct 13
Vor Bio Appoints Eyal C. Attar, M.D. as Chief Medical Officer Vor Bio announced that Eyal C. Attar, M.D., has joined Vor Bio as Chief Medical Officer (CMO). Dr. Attar brings more than 20 years of demonstrated clinical experience to the position, with previous roles as a clinician, bench researcher and clinical developer of treatments for hematologic malignancies. Dr. Attar most recently served as senior vice president and CMO for Aprea Therapeutics. In this role, he led development of Aprea’s lead programs, including overseeing clinical development, pharmacovigilance and regulatory filings. Dr. Attar worked closely on APR-246 (eprenetapopt), a cancer therapeutic pursuing indications including myelodysplastic syndromes (MDS), AML, and various solid tumors. Before Aprea, he held multiple positions in clinical development at Agios Pharmaceuticals, including Senior Medical Director and IDH Hematology Medical Lead, where he worked on various solid tumor and hematologic malignancy programs and contributed to the successful filing of multiple INDs. Dr. Attar has played a leadership role in all aspects of clinical development from Pre-IND through to marketing authorization for several products to treat patients with newly diagnosed and relapsed/refractory AML. Dr. Attar was previously an attending hematologist/oncologist at Massachusetts General Hospital focusing on leukemia, MDS, and other hematologic malignancies affecting the bone marrow and completed fellowship training at Dana-Farber Cancer Center and residency at Brigham and Women’s Hospital. Dr. Attar received his medical degree from the University of North Carolina School of Medicine. Price Target Changed • Aug 13
Price target decreased to US$21.88 Down from US$24.25, the current price target is an average from 6 analysts. New target price is 305% above last closing price of US$5.40. Stock is down 62% over the past year. The company is forecast to post a net loss per share of US$2.34 next year compared to a net loss per share of US$2.10 last year. Price Target Changed • Jul 26
Price target decreased to US$22.25 Down from US$24.25, the current price target is an average from 6 analysts. New target price is 356% above last closing price of US$4.88. Stock is down 62% over the past year. The company is forecast to post a net loss per share of US$2.67 next year compared to a net loss per share of US$2.10 last year. Announcement • Jun 11
Vor Bio Successfully Demonstrates Multiplex Editing of Hematopoietic Stem Cells for Next-generation AML Treatment Presented at EHA Vor Bio announced successful first of its kind dual editing of CD33 and CLL-1 in human hematopoietic stem cells (HSCs) demonstrating continued progress on its novel approach for the treatment of acute myeloid leukemia (AML). The data is being presented at the European Hematology Association Congress in Vienna, Austria.The pre-clinical data demonstrates that multiplex deletion by CRISPR/Cas9 of CD33 and CLL-1 from human CD34+ hematopoietic stem and progenitor cells (HSPCs) maintained cell function and persisted long-term post engraftment in vivo, with a high-level of editing, no counterselection, and minimum translocation risk when compared to unedited control cells. In addition, genetically modifying HSPCs to remove select cell surface targets does not impair their function and these dual engineered cells showed significant protection from targeted immunotherapy in vitro.AML is the most common type of acute leukemia in adults and is characterized by excessive proliferation of immature myeloid progenitor cells and their failure to properly differentiate into mature blood cells. Healthy donor HSC transplantation is the standard of care and currently around 40% of patients with AML who receive HSC transplantation suffer a relapse of their cancer, with two-year survival rates of less than 20%, highlighting the need for new therapeutic approaches for these patients. Price Target Changed • May 15
Price target decreased to US$24.25 Down from US$28.50, the current price target is an average from 6 analysts. New target price is 475% above last closing price of US$4.22. Stock is down 78% over the past year. The company is forecast to post a net loss per share of US$2.51 next year compared to a net loss per share of US$2.10 last year.