Annonce • Jun 12
Abeona Therapeutics Inc. Announces Resignation of Michael Amoroso as Member of Board of Directors, Nominating and Corporate Governance Committee, Effective June 11, 2026 Abeona Therapeutics Inc. announced that Michael Amoroso resigned as a member of the Board of Directors of Abeona Therapeutics Inc. effective June 11, 2026. In connection with his resignation, Mr. Amoroso also resigned from the Nominating and Corporate Governance Committee of the Board on which he served. Mr. Amoroso's resignation was not the result of any disagreement with the Company on any matter relating to the Company's operations, policies or practices but solely for personal reasons. Recent Insider Transactions • Jun 10
President recently sold US$302k worth of stock On the 9th of June, Vishwas Seshadri sold around 56k shares on-market at roughly US$5.36 per share. This transaction amounted to 3.9% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Vishwas has been a net seller over the last 12 months, reducing personal holdings by US$1.2m. Recent Insider Transactions Derivative • Jun 09
President notifies of intention to sell stock Vishwas Seshadri intends to sell 58k shares in the next 90 days after lodging an Intent To Sell Form on the 8th of June. If the sale is conducted around the recent share price of US$5.25, it would amount to US$305k. For the year to December 2021, Vishwas' total compensation was 17% salary and 83% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since September 2025, Vishwas' direct individual holding has increased from 1.19m shares to 1.43m. Company insiders have collectively sold US$2.4m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Jun 04
Abeona Therapeutics Inc Participates in Pre-Investigational New Drug Meeting with Fda Regarding Abo-701 Abeona Therapeutics Inc. participated in a pre-Investigational New Drug application (Pre-IND) meeting with the U.S. Food and Drug Administration (FDA) regarding ABO-701, a recently licensed radically novel engineered T-cell therapy, targeting Prostate-Specific Membrane Antigen to treat prostate cancer. While official minutes of the meeting have not yet been received, the meeting was constructive and continues to target submission of an IND application for ABO-701 in 2027, consistent with the previously stated timeline. There can be no assurance that the FDA will not require additional studies, data, or information before accepting an IND submission for ABO-701, or that any IND submission, if submitted, will be accepted by the FDA or result in authorization to commence clinical trials. Development plans remain subject to ongoing evaluation and may be revised based on, among other things, feedback received from the FDA, results of preclinical studies, manufacturing considerations, and other factors. Annonce • May 04
Abeona Therapeutics Inc. to Report Q1, 2026 Results on May 13, 2026 Abeona Therapeutics Inc. announced that they will report Q1, 2026 results Pre-Market on May 13, 2026 Annonce • Apr 21
Abeona Therapeutics Inc., Annual General Meeting, Jun 12, 2026 Abeona Therapeutics Inc., Annual General Meeting, Jun 12, 2026. Board Change • Apr 08
Insufficient new directors There is 1 new director who has joined the board in the last 3 years. The company's board is composed of: 1 new director. 7 experienced directors. 4 highly experienced directors. Independent Director Keith Goldan was the last director to join the board, commencing their role in 2026. The company’s insufficient board refreshment is considered a risk according to the Simply Wall St Risk Model. Annonce • Apr 08
Abeona Therapeutics Inc. Appoints Keith A. Goldan as A New Independent Member to Its Board of Directors as Chairman of Audit Committee , Effective as of April 1, 2026 Abeona Therapeutics Inc. announced the appointment of Keith A. Goldan as a new independent member to its Board of Directors, effective as of April 1, 2026. Mr. Goldan will also serve as Chairman of Abeona’s Audit Committee. Mr. Goldan brings more than two decades of financial leadership experience across publicly traded commercial-stage biotechnology and specialty pharmaceutical companies. His appointment to the Board of Directors reflects Abeona’s continued focus on strengthening its leadership team to advance both strategic and financial objectives. Mr. Goldan is an accomplished financial leader, currently serving as Chief Financial Officer of Syndax Pharmaceuticals since 2022 where he has provided leadership through their first two product approvals and commercialization. Prior to his current role, Mr. Goldan served as Chief Financial Officer of Optinose, a publicly traded specialty pharmaceutical company, where he played a key role in building the infrastructure to support the successful US launch of its lead product. Prior to Optinose, he was Chief Financial Officer and Senior Vice President of Fibrocell, a publicly traded cell and gene therapy company.Mr. Goldan also held Chief Financial Officer roles at NuPathe, PuriCore plc and Biosyn, and served in the financial leadership positions at ViroPharma and KPMG. Across these roles, he led finance, accounting, IT, HR and corporate development functions; successfully raised capital through multiple IPOs, capital market transactions and related financing vehicles; and executed several merger and acquisition transactions. Recent Insider Transactions Derivative • Apr 07
President notifies of intention to sell stock Vishwas Seshadri intends to sell 30k shares in the next 90 days after lodging an Intent To Sell Form on the 31st of March. If the sale is conducted around the recent share price of US$4.38, it would amount to US$131k. For the year to December 2021, Vishwas' total compensation was 18% salary and 82% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2025, Vishwas' direct individual holding has increased from 1.30m shares to 1.43m. Company insiders have collectively sold US$2.9m more than they bought, via options and on-market transactions in the last 12 months. Price Target Changed • Mar 18
Price target decreased by 7.1% to US$19.57 Down from US$21.07, the current price target is an average from 7 analysts. New target price is 330% above last closing price of US$4.55. Stock is down 13% over the past year. The company is forecast to post a net loss per share of US$0.69 compared to earnings per share of US$1.34 last year. Major Estimate Revision • Mar 10
Consensus revenue estimates fall by 14% The consensus outlook for revenues in fiscal year 2025 has deteriorated. 2025 revenue forecast decreased from US$6.05m to US$5.22m. EPS estimate fell from US$1.22 to US$1.18 per share. Net income forecast to shrink 98% next year vs 8.8% decline forecast for Biotechs industry in the US. Consensus price target up from US$20.64 to US$21.07. Share price rose 4.0% to US$5.14 over the past week. Annonce • Mar 10
Abeona Therapeutics Inc. Provides Update On Zevaskyn Commercial Launch Abeona Therapeutics Inc. announced an update on the building momentum in the Company’s launch of FDA-approved ZEVASKYN (prademagene zamikeracel), a first-of-its-kind, autologous gene therapy for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Following the optimization of a release assay in 2025, ZEVASKYN commercial launch activities commenced in the fourth quarter, with the first patient treatment completed in December prior to a mandatory scheduled facility shutdown. Since resuming manufacturing in late January 2026, multiple biopsies have been collected, a patient has completed treatment with ZEVASKYN, and there are ongoing manufacturing runs. Additional biopsies are scheduled for the remainder of this quarter. Policies covering ZEVASKYN have been published by all major commercial payers including United Healthcare, Cigna, Aetna, Anthem, and most Blue Cross Blue Shield plans, that account for 80% of lives covered by commercial insurance, signaling broad and early market acceptance. ZEVASKYN has also secured coverage across all Medicaid programs. In addition, the Centers for Medicare and Medicaid Services (CMS) has established a permanent Healthcare Common Procedure Coding System (HCPCS) J-code for ZEVASKYN, J3389 (Topical administration, prademagene zamikeracel, per treatment), effective January 1, 2026. ZEVASKYN is the first and only autologous cell sheet-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin disease caused by a defect in both copies of the COL7A1 gene resulting in the inability to produce functional type VII collagen. Without functional type VII collagen and anchoring fibrils, the skin is fragile and blisters easily, leading to wounds that continually open and close, or fail to heal altogether. Patients often have large open wounds that can lead to serious life-threatening complications. ZEVASKYN incorporates the functional type VII collagen-producing COL7A1 gene into a patient’s own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds. ZEVASKYN has demonstrated clinically meaningful wound healing and pain reduction with a single surgical application. ZEVASKYN (prademagene zamikeracel) is an autologous cell sheet-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Serious allergic reactions to ZEVASKYN can occur. Patients should get medical help right away if they experience symptoms like itching, swelling, hives, difficulty breathing, runny nose, watery eyes, or nausea. In rare cases, a severe reaction called anaphylaxis may happen. There is a potential risk that treatment with ZEVASKYN may contribute to the development of cancer because of how the therapy works. Patients should be monitored for the rest of their lives to check for any signs of cancer. ZEVASKYN is made using human and animal materials. Although these materials are tested before use, the risk of passing on infections cannot be eliminated. The most common side effects are pain from the procedure and itching. This is not a complete list of side effects. Patients should call their care team for medical advice about side effects. Side effects may be reported to Abeona at 1-844-888-2236 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. See full Prescribing Information. Recent Insider Transactions • Feb 03
Independent Director recently sold US$92k worth of stock On the 2nd of February, Leila Alland sold around 18k shares on-market at roughly US$5.09 per share. This transaction amounted to 9.4% of their direct individual holding at the time of the trade. In the last 3 months, there was an even bigger sale from another insider worth US$372k. Insiders have been net sellers, collectively disposing of US$2.8m more than they bought in the last 12 months. Recent Insider Transactions • Jan 22
Independent Director recently sold US$71k worth of stock On the 21st of January, Mark Alvino sold around 14k shares on-market at roughly US$5.23 per share. This transaction amounted to 22% of their direct individual holding at the time of the trade. In the last 3 months, they made an even bigger sale worth US$71k. Insiders have been net sellers, collectively disposing of US$2.0m more than they bought in the last 12 months. Recent Insider Transactions Derivative • Dec 31
President notifies of intention to sell stock Vishwas Seshadri intends to sell 25k shares in the next 90 days after lodging an Intent To Sell Form on the 29th of December. If the sale is conducted around the recent share price of US$5.31, it would amount to US$133k. For the year to December 2021, Vishwas' total compensation was 18% salary and 82% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since March 2025, Vishwas' direct individual holding has decreased from 1.36m shares to 1.19m. Company insiders have collectively sold US$1.9m more than they bought, via options and on-market transactions in the last 12 months. Annonce • Dec 16
Abeona Therapeutics Inc Appoints Mohamad Tabrizi as Senior Vice President and Chief Business Officer Abeona Therapeutics Inc. announced the appointment of Mohamad Tabrizi, M.S., M.B.A., as Senior Vice President, Chief Business Officer (CBO). In this role, Mr. Tabrizi will lead the Company's corporate strategy and business development functions, as well as drive operating efficiency for the Company. Mr. Tabrizi transitions to Abeona from the venture capital sector, where he served as a General Partner and Managing Director at Pandect Bioventures and the Berkeley Catalyst Fund. In these capacities, he led investment activities and portfolio management while also stepping into operational leadership roles for portfolio companies, including serving as Chief Executive Officer of Azkarra Therapeutics and Interim Chief Financial Officer of Circle Pharma. His strategic commercial acumen is anchored by a rigorous scientific foundation, including research fellowships and associate roles at the Howard Hughes Medical Institute and Stanford University Medical School. Previously, Mr. Tabrizi established a significant track record in corporate development and capital markets. While serving as Executive Director of Corporate Business Development at Nektar Therapeutics during 2007 to 2017, he executed 20 transactions, securing collaborations with major pharmaceutical and biotechnology companies such as AstraZeneca, Bristol-Myers Squibb, Pfizer, Merck and Amgen. Earlier in his career, he served as a healthcare investment banker at RBC Capital Markets and Needham & Company, leading over 40 transactions and raising more than $5 billion in public and private financings. Mr. Tabrizi holds an M.B.A. from Cornell University, an M.S. in Biological Sciences from Stanford University, and a B.S. in Genetics from the University of California, Davis. Annonce • Dec 11
Abeona Therapeutics Announces New Qualified Treatment Center for Zevaskyn® in Texas Abeona Therapeutics Inc. has announced the activation of The University of Texas Medical Branch (UTMB) in Galveston, Texas, as a new Qualified Treatment Center (QTC) for administering ZEVASKYN (prademagene zamikeracel). ZEVASKYN is an FDA-approved gene therapy for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB). This expansion aims to enhance patient access to ZEVASKYN across Texas and the Gulf Coast region. The therapy involves gene-modified cellular sheets that incorporate the functional type VII collagen-producing COL7A1 gene into a patient’s skin cells to promote wound healing. Valuation Update With 7 Day Price Move • Dec 11
Investor sentiment improves as stock rises 16% After last week's 16% share price gain to US$5.47, the stock trades at a forward P/E ratio of 25x. Average trailing P/E is 19x in the Biotechs industry in the US. Total returns to shareholders of 84% over the past three years. Annonce • Dec 09
Abeona Therapeutics Inc. Announces First Patient Treatment with ZEVASKYN® Gene Therapy Abeona Therapeutics Inc. announced the first commercial patient treatment with FDA-approved ZEVASKYN (prademagene zamikeracel), a first-of-its-kind, autologous gene therapy for treating wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). ZEVASKYN was administered at Lucile Packard Children’s Hospital Stanford in Palo Alto, CA. Recessive dystrophic epidermolysis bullosa (RDEB), a rare blistering disorder without a cure, is characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in both copies of the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. ZEVASKYN is the first and only autologous cell sheet-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a severe skin disease caused by a defect in both copies of the COL7A1 gene resulting in the inability to produce functional type VII collagen. Without functional type VII collagen and anchoring fibrils, the skin is fragile and blisters easily, leading to wounds that continually open and close, or fail to heal altogether. Patients often have large open wounds that can lead to serious life-threatening complications. ZEVASKYN incorporates the functional type VII collagen-producing COL7A1 gene into a patient’s own skin cells, ex vivo, using a replication-incompetent retroviral vector to produce functional type VII collagen in treated wounds. ZEVASKYN has demonstrated clinically meaningful wound healing and pain reduction with a single surgical application. Valuation Update With 7 Day Price Move • Nov 26
Investor sentiment improves as stock rises 23% After last week's 23% share price gain to US$5.15, the stock trades at a forward P/E ratio of 23x. Average trailing P/E is 19x in the Biotechs industry in the US. Total returns to shareholders of 37% over the past three years. Major Estimate Revision • Nov 19
Consensus revenue estimates fall by 78% The consensus outlook for revenues in fiscal year 2025 has deteriorated. 2025 revenue forecast decreased from US$27.5m to US$6.05m. EPS estimate fell from US$1.28 to US$1.20 per share. Net income forecast to shrink 82% next year vs 8.5% decline forecast for Biotechs industry in the US. Consensus price target broadly unchanged at US$20.21. Share price rose 7.4% to US$4.48 over the past week. Recent Insider Transactions • Nov 16
Independent Director recently sold US$71k worth of stock On the 13th of November, Mark Alvino sold around 15k shares on-market at roughly US$4.74 per share. This transaction amounted to 19% of their direct individual holding at the time of the trade. In the last 3 months, there was an even bigger sale from another insider worth US$104k. Insiders have been net sellers, collectively disposing of US$1.9m more than they bought in the last 12 months. Recent Insider Transactions Derivative • Nov 14
Independent Director notifies of intention to sell stock Mark Alvino intends to sell 15k shares in the next 90 days after lodging an Intent To Sell Form on the 13th of November. If the sale is conducted around the recent share price of US$4.74, it would amount to US$71k. Since March 2025, Mark's direct individual holding has decreased from 100.44k shares to 77.25k. Company insiders have collectively sold US$1.9m more than they bought, via options and on-market transactions in the last 12 months. Valuation Update With 7 Day Price Move • Nov 06
Investor sentiment deteriorates as stock falls 18% After last week's 18% share price decline to US$4.19, the stock trades at a forward P/E ratio of 6x. Average trailing P/E is 17x in the Biotechs industry in the US. Negligible returns to shareholders over past three years. Annonce • Oct 31
Abeona Therapeutics Inc. Announces Permanent J-Code for ZEVASKYN® (prademagene Zamikeracel) Abeona Therapeutics Inc. announced that the Centers for Medicare and Medicaid Services (CMS) has established a permanent Healthcare Common Procedure Coding System (HCPCS) J-code for ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets, the Company's autologous gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). The new J-code for ZEVasKYN, J3389 (Topical administration, prademagene zamikerACel, per treatment) becomes effective on January 1, 2026. J-codes are unique identifiers designed to identify non-orally administered medications in healthcare settings. The most common side effects are pain from the procedure and itching. This is not a complete list of side effects. Patients should call their care team for medical advice about side effects. Side effects may be reported to Abeona at 1-844-888-2236 or FDA at 1-800-FDA-1088 or see full Prescribing Information. Annonce • Oct 21
Abeona Therapeutics Inc. Appoints James A. Gow as Senior Vice President, Head of Clinical Development & Medical Affairs, Effective October 20, 2025 Abeona Therapeutics Inc. announced the appointment of James A. Gow, MD, MBA, MS, MHCM, as the Senior Vice President, Head of Clinical Development & Medical Affairs, effective October 20, 2025. Dr. Gow has over 20 years of industry experience in clinical development and medical affairs and is a recognized expert in gene therapy, especially in ophthalmology. His track record in clinical development includes leading programs from Phase 1 through post-marketing studies, which led to the FDA approvals of Xibrom, Bromday, Prolensa, Bepreve and global regulatory approvals of Xiidra. Prior to Abeona, Dr. Gow has held executive leadership roles at several pharmaceutical and biotechnology companies, including Shire, Takeda, Novartis, Biogen and SparingVision. Dr. Gow earned his MD from the University of Manitoba, an MBA from Cornell University, a Master of Science in Healthcare Policy and Research from Cornell University, and a Master in Health Care Management from Harvard University. Annonce • Oct 13
Abeona Therapeutics®? Announces Abo-503 Gene Therapy for X-Linked Retinoschisis (Xlrs) Selected by Fda for Rare Disease Endpoint Advancement (Rdea) Pilot Program Abeona Therapeutics Inc. announced that its ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected to participate in the U.S. Food and Drug Administration (FDA) Rare Disease Endpoint Advancement (RDEA) Pilot Program. The RDEA program facilitates the development and timely approval of rare disease therapies by supporting the use of novel efficacy endpoints in clinical trials. As part of the RDEA program, Abeona will have opportunities for enhanced communication and collaboration with the FDA, including frequent advice and regular ad-hoc conversations to accelerate the development and validation of product-specific novel efficacy endpoints for Abeona's XLRS program. ABO-503 is composed of a functional human RS1 gene packaged in the novel AIM™? capsid AAV204. Preclinical studies have demonstrated structural and functional improvements following robust RS1 expression throughout the retina, including improved cone photoreceptor density and overall photoreceptor cell survival, restoration of outer retina architecture by eliminating cysts characteristic of XLRS, and improvements in visual function as demonstrated by electroretinogram (ERG). Abeona anticipates completing IND-enabling studies in the second half of 2026. The FDA launched the RDEA Pilot Program to support the development of novel efficacy endpoints for rare disease treatments. Under the pilot program, between 2023 and 2027, the FDA will accept up to one RDEA proposal per quarter with a maximum of three proposals per year. To be considered, sponsors must submit a proposal detailing the data they plan to collect, the novelty of the endpoint, and its potential to establish substantial evidence of effectiveness. Annonce • Oct 08
Abeona Therapeutics Inc. and Children's Hospital Colorado Announce Newest Treatment Center for Zevaskyn Gene Therapy Abeona Therapeutics Inc. and Children's Hospital Colorado announced activation of Children's Colorado as the newest Qualified Treatment Center (QTC) for ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets. This first-of-its-kind therapy is FDA-approved to treat wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Children's Colorado in Aurora, CO, one of the pioneering institutions that provide advanced levels of care for people with epidermolysis Bullosa (EB), has completed QTC start-up activities enabling it to begin patient identification for scheduling of ZEVASKYN treatment. The most common side effects are pain from the procedure and itching. This is not a complete list of side effects. Patients should call their care team for medical advice about side effects. Side effects may be reported to Abeona at 1-844-888-2236 or FDA at 1-800-FDA-1088 or See full Prescribing Information. Recent Insider Transactions • Oct 02
President recently sold US$104k worth of stock On the 29th of September, Vishwas Seshadri sold around 19k shares on-market at roughly US$5.39 per share. This transaction amounted to 1.6% of their direct individual holding at the time of the trade. In the last 3 months, they made an even bigger sale worth US$407k. Vishwas has been a net seller over the last 12 months, reducing personal holdings by US$853k. Recent Insider Transactions Derivative • Sep 30
President notifies of intention to sell stock Vishwas Seshadri intends to sell 44k shares in the next 90 days after lodging an Intent To Sell Form on the 29th of September. If the sale is conducted around the recent share price of US$5.39, it would amount to US$239k. For the year to December 2021, Vishwas' total compensation was 18% salary and 82% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since December 2024, Vishwas' direct individual holding has increased from 903.89k shares to 1.23m. Company insiders have collectively sold US$1.7m more than they bought, via options and on-market transactions in the last 12 months. Valuation Update With 7 Day Price Move • Sep 16
Investor sentiment deteriorates as stock falls 17% After last week's 17% share price decline to US$5.68, the stock trades at a forward P/E ratio of 8x. Average trailing P/E is 16x in the Biotechs industry in the US. Total returns to shareholders of 53% over the past three years. Recent Insider Transactions • Aug 17
Chief Financial Officer recently sold US$110k worth of stock On the 15th of August, Joseph Vazzano sold around 15k shares on-market at roughly US$7.36 per share. This transaction amounted to 3.1% of their direct individual holding at the time of the trade. In the last 3 months, there was an even bigger sale from another insider worth US$407k. Joseph has been a net seller over the last 12 months, reducing personal holdings by US$389k. Annonce • Jul 15
Abeona Therapeutics Inc. Announces Activation of the Newest Qualified Treatment Center for FDA-Appointment of ZEVASKYN Abeona Therapeutics Inc. announced activation of the newest Qualified Treatment Center (QTC) for FDA-approved ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets. This first-of-its-kind therapy, an outcome of a decade of research by Abeona and two decades of research at Stanford Medicine, where the technology originated, will be used to treat wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). Lucile Packard Children's Hospital Stanford has completed QTC start-up activities and is now ready to accept patients for treatment with ZEVASKYN. Abeona is committed to enabling access to ZEVASKYN for eligible patients in the U.S. and has deployed services to provide information and resources to make informed decisions about treatment with ZEVASKY N for RDEB wounds. Abeona's comprehensive patient support program, Abeona Assist™?, offers personalized support, including helping patients understand their insurance benefits and financial assistance options, and providing travel and logistical assistance. Recent Insider Transactions • Jul 11
President recently sold US$407k worth of stock On the 9th of July, Vishwas Seshadri sold around 69k shares on-market at roughly US$5.86 per share. This transaction amounted to 5.3% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. Vishwas has been a net seller over the last 12 months, reducing personal holdings by US$749k. Recent Insider Transactions Derivative • Jul 10
President notifies of intention to sell stock Vishwas Seshadri intends to sell 69k shares in the next 90 days after lodging an Intent To Sell Form on the 9th of July. If the sale is conducted around the recent share price of US$5.86, it would amount to US$405k. For the year to December 2021, Vishwas' total compensation was 18% salary and 82% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since September 2024, Vishwas' direct individual holding has increased from 904.14k shares to 1.30m. Company insiders have collectively sold US$634k more than they bought, via options and on-market transactions in the last 12 months. Annonce • Jun 24
Abeona Therapeutics®? Announces Publication in the Lancet of Phase 3 Viital™? Study Data in Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced that The Lancet has published results from the pivotal Phase 3 VIITAL study (NCT04227106) evaluating the efficacy and safety of ZEVASKYN (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). This article, titled, "Prademagene Zamikeracel for Recessive Dystrophic Epidermolysis Bullosa Wounds (VIITAL): A Two-Centre, Randomised, Open-Label, Intrapatient-Controlled Phase 3 Trial," representing the primary publication of the full VIITAL study data, is now available online and will be published in a future print issue of The Lancet publication said, "People with RDEB endure incredibly fragile skin, leading to severe, painful wounds that can last for years, and lead to systemic complications impacting the length and quality of life. RDEB patients also have high risk of developing squamous cell carcinoma (SCC). There is no cure for RDEB and ZEVASKYN is the only product approved by the U.S. Food and Drug Administration (FDA) to treat RDEB wounds with a single application. These wounds, each at least 20 cm2 and open for a median duration of 5 years (ranging from 6 months to 21 years), were assessed against two co-primary endpoints at six months (week 24), comparing treated to matched untreated (control) wounds. The VIITAL study builds on the clinical experience from the Phase 1/2a study of ZEVASKYN ("NCT01263379), a single center, open label study in 38 chronic wounds across 7 patients. The Phase 1/2a study showed that a single surgical application of ZEVASKYN was associated with long-term improvement at treated sites over a median follow-up of 6.9 years (range 4 to 8 years). Side effects may be reported to Abeona at 1-844-888-2236 or FDA at 1-800-FDA-1088 or. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, ability to commercialize ZEVASKYN, the therapeutic potential of ZEVASKYN, whether the unmet need and market opportunity for ZEVASKYN are consistent with the Company's expectations, continued interest in rare disease portfolio; ability to enroll patients in clinical trials; the outcome of future meetings with and expectations from the FDA or other regulatory agencies, including those relating to preclinical programs; the ability to achieve or obtain necessary regulatory approvals; the impact of any changes in the financial markets; and the impact of any changes in The Lancet's financial markets; and the impact to any changes in the financial markets. Recent Insider Transactions • Jun 08
President recently sold US$343k worth of stock On the 6th of June, Vishwas Seshadri sold around 51k shares on-market at roughly US$6.76 per share. This transaction amounted to 3.7% of their direct individual holding at the time of the trade. This was the largest sale by an insider in the last 3 months. This was Vishwas' only on-market trade for the last 12 months. Breakeven Date Change • Apr 30
Forecast breakeven date moved forward to 2025 The 7 analysts covering Abeona Therapeutics previously expected the company to break even in 2027. New consensus forecast suggests the company will make a profit of US$3.39m in 2025. Earnings growth of 64% is required to achieve expected profit on schedule. Annonce • Apr 30
U.S. Food and Drug Administration Approves Abeona Therapeutics' ZEVASKYN accelerates momentum and expands treatment options for the EB community In a significant advancement for the Recessive Dystrophic Epidermolysis Bullosa (RDEB) community, the U.S. Food and Drug Administration (FDA) has approved a third treatment for this rare condition. The milestone approval of Abeona Therapeutics' ZEVASKYN not only represents a continued commitment to innovation in RDEB care, but reinforces the growing momentum toward improved quality of life and expanded treatment choices for individuals and families affected by RDEB. ZEVASKYN is the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene. With this FDA approval, EBRP gains continued momentum in its mission to discover treatments and a cure for EB. This milestone reinforces the validity of their scalable, sustainable Venture Philanthropy model, and, most importantly, provides treatment options and ignites hope for families around the world affected by EB. EBRP invested in the ground-breaking work that led to ZEVASKYN at Stanford University under their Venture Philanthropy Model. With skin this fragile, those with EB face severe pain, open external and internal wounds, and grueling bandaging processes. For the 500,000 people around the world living with EB, everyday activities like eating, sleeping, walking and playing can become monumental tasks that often require modification. Over the last decade, EBRP has made undeniable progress in its mission to find a cure for this community, including: Raising over $70 million; Funding 160+ EB projects; Contributing to a 25x growth in the EB clinical trial landscape; Directly funding two FDA-approved EB treatments. FDA approval of the first autologous, cells-based gene therapy for R DEB is an exciting proof point of this progress. EBRP will continue to build off this momentum and is dedicated to delivering a cure. ZEVASKYN's approval arrives during an unprecedented wave of awareness for EB, fueled by recent high-impact initiatives led by EBRP and their Co-Founders Eddie and Jill Vedder. The upcoming premiere of "Matter of Time", a compelling documentary capturing the electrifying energy of Eddie Vedder's October 2023 solo concerts in Seattle, is bringing new levels of EB visibility on a global scale. Premiering in June 2025 at the Tribeca Festival, "Matter of Time" weaves together powerful music and the moving stories of individuals impacted by EB - including families, researchers, and thought leaders - and highlights how innovation and determination are driving progress toward a cure. One such researcher chronicled throughout the film is Dr. Jean Tang of Stanford University, who was lead principal investigator on the clinical of this third FDA approved treatment. The documentary is a testament to the power of music, community, and unwavering determination to drive change and inspire hope. Public awareness of EB has never been stronger--and this most recent FDA approval underscores the momentum building around research, advocacy, and support. Annonce • Apr 12
Abeona Therapeutics Inc., Annual General Meeting, May 19, 2025 Abeona Therapeutics Inc., Annual General Meeting, May 19, 2025. Recent Insider Transactions Derivative • Apr 01
President notifies of intention to sell stock Vishwas Seshadri intends to sell 25k shares in the next 90 days after lodging an Intent To Sell Form on the 31st of March. If the sale is conducted around the recent share price of US$4.78, it would amount to US$120k. For the year to December 2021, Vishwas' total compensation was 19% salary and 81% other compensation. This indicates that these sales could comprise a meaningful part of their income for the year. Since June 2024, Vishwas' direct individual holding has increased from 498.94k shares to 1.38m. Company insiders have collectively sold US$121k more than they bought, via options and on-market transactions in the last 12 months. Major Estimate Revision • Mar 27
Consensus revenue estimates decrease by 33%, EPS upgraded The consensus outlook for fiscal year 2025 has been updated. 2025 revenue forecast fell from US$18.3m to US$12.3m. EPS estimate increased from -US$0.752 to -US$0.718 per share. Biotechs industry in the US expected to see average net income decline 11% next year. Consensus price target up from US$15.92 to US$16.75. Share price was steady at US$5.04 over the past week. Breakeven Date Change • Dec 31
Forecast to breakeven in 2027 The 6 analysts covering Abeona Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$20.6m in 2027. Average annual earnings growth of 57% is required to achieve expected profit on schedule. Major Estimate Revision • Nov 21
Consensus EPS estimates fall by 14% The consensus outlook for fiscal year 2024 has been updated. 2024 expected loss increased from -US$1.50 to -US$1.71 per share. Revenue forecast of US$83.3k unchanged since last update. Biotechs industry in the US expected to see average net income decline 13% next year. Consensus price target up from US$15.92 to US$16.75. Share price fell 2.7% to US$5.79 over the past week. Annonce • Nov 13
Abeona Therapeutics Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review Abeona’s resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), its investigational autologous cell-based gene therapy, as a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025. The BLA resubmission is supported by clinical efficacy and safety data after a one-time administration of pz-cel from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379) with up to 8 years of follow-up. If approved, pz-cel would be the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene. The Company’s BLA for pz-cel was previously accepted for Priority Review by the FDA for patients with RDEB. Abeona may be eligible for a Priority Review Voucher (PRV) should pz-cel be approved. About prademagene zamikeracel (pz-cel): Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene therapy, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. As a result of this defect, cells are unable to express functional collagen VII protein which is needed to form anchoring fibrils that bond the epidermis to the dermis. Lack of anchoring fibrils leads to fragile skin that blisters easily and patients suffer from years of painful wounds, itch and increased risk of infection and squamous cell carcinoma. Pz-cel is made from patients' own skin cells that are genetically corrected with a functional COL7A1 gene integrated into the skin cells’ genome to express collagen VII. These gene-corrected cells are expanded to form keratinocyte sheets to cover wound areas in a single surgical application. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. Major Estimate Revision • Aug 19
Consensus revenue estimates decrease by 33%, EPS upgraded The consensus outlook for fiscal year 2024 has been updated. 2024 revenue forecast fell from US$250.0k to US$170.0k. EPS estimate increased from -US$1.92 to -US$1.50 per share. Biotechs industry in the US expected to see average net income decline 14% next year. Consensus price target down from US$16.42 to US$15.92. Share price was steady at US$5.00 over the past week. Annonce • Aug 14
Abeona Therapeutics Inc. Announces Board Appointments Abeona Therapeutics Inc. announced the appointment of Bernhardt G. Zeiher, MD, FCCP, FACP, and Eric Crombez, MD as new independent members to its Board of Directors. Dr. Zeiher brings more than 20 years of drug development experience where, in various roles, he oversaw the approval of 15 new treatments that addressed unmet needs in serious diseases with few to no treatment options. Dr. Crombez currently serves as Chief Medical Officer of Ultragenyx Pharmaceutical Inc. and brings extensive expertise in the development and execution of clinical development programs for rare genetic disorders. Dr. Zeiher spent more than 10 years at Astellas Pharma, holding multiple roles of increasing responsibility in drug development, leading up to his role as CMO, where he led early- and late-stage drug development, medical and regulatory affairs, pharmacovigilance, and quality assurance. Prior to Astellas, Dr. Zeiher held various roles leading drug development at other pharmaceutical companies including Pfizer and Eli Lilly and Company. He also practiced medicine at a tertiary medical center in Indianapolis. Dr. Zeiher currently serves on multiple public company boards, including Entrada Therapeutics and Amylyx Pharmaceuticals Inc. He previously served on the boards of TransCelerate Biopharma, Biotechnology Innovation Organization and Astellas Global Health Foundation. Dr. Zeiher received a B.S. in biology from the University of Toledo and an MD from Case Western Reserve University School of Medicine. He completed his internal medicine residency and chief residency at University Hospitals of Cleveland and then finished his physician training as a Pulmonary and Critical Care Fellow at University of Iowa Hospitals and Clinics. Dr. Crombez joined Ultragenyx following the acquisition of Dimension Therapeutics in November 2017. As Chief Medical Officer of Ultragenyx, Dr. Crombez is responsible for strategic leadership of the clinical development and translational research programs, and oversees global development functions including Clinical Development, Clinical Operations, BioMetrics, Endpoint Development and Strategy, Regulatory Affairs and Drug Safety/Pharmacovigilance. At Dimension Therapeutics, Dr. Crombez served as Chief Medical Officer and led the clinical development efforts for their gene therapy programs. Dr. Crombez is also an appointed industry representative on the FDA Cellular, Tissue and Gene Therapies Advisory Committee. Before joining industry, he was assistant professor, Department of Pediatrics, Division of Medical Genetics at the David Geffen School of Medicine at the University of California, Los Angeles (UCLA). Dr. Crombez is a board-certified clinical geneticist and completed residencies in pediatrics and medical genetics and a fellowship in clinical biochemical genetics at the UCLA School of Medicine. Dr. Crombez obtained his B.S. degree in biology from the University of Michigan, Ann Arbor, and his M.D. degree from Wayne State University School of Medicine, Detroit. Recent Insider Transactions Derivative • Jun 19
Independent Director notifies of intention to sell stock Mark Alvino intends to sell 28k shares in the next 90 days after lodging an Intent To Sell Form on the 17th of June. If the sale is conducted around the recent share price of US$4.57, it would amount to US$126k. Since September 2023, Mark's direct individual holding has decreased from 69.90k shares to 58.69k. Company insiders have collectively bought US$208k more than they sold, via options and on-market transactions, in the last 12 months. Recent Insider Transactions • May 21
Independent Director recently bought US$52k worth of stock On the 16th of May, Leila Alland bought around 11k shares on-market at roughly US$4.71 per share. This transaction amounted to 15% of their direct individual holding at the time of the trade. This was the largest purchase by an insider in the last 3 months. Insiders have collectively bought US$208k more in shares than they have sold in the last 12 months. New Risk • May 17
New major risk - Negative shareholders equity The company has negative equity. Total equity: -US$8.9m 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 (20% average weekly change). Negative equity (-US$8.9m). Shareholders have been substantially diluted in the past year (111% increase in shares outstanding). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$6.7m net loss in 3 years). Revenue is less than US$5m (US$3.5m revenue). New Risk • May 16
New major risk - Shareholder dilution The company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 111% 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 (20% average weekly change). Shareholders have been substantially diluted in the past year (111% increase in shares outstanding). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$21m net loss in 3 years). Revenue is less than US$5m (US$3.5m revenue). Major Estimate Revision • May 16
Consensus EPS estimates upgraded to US$1.36 loss The consensus outlook for fiscal year 2024 has been updated. 2024 losses forecast to reduce from -US$2.14 to -US$1.36 per share. Revenue forecast unchanged from US$666.7k at last update. Biotechs industry in the US expected to see average net income decline 9.5% next year. Consensus price target of US$19.50 unchanged from last update. Share price rose 3.8% to US$4.62 over the past week. Annonce • May 04
Abeona Therapeutics Inc. has completed a Follow-on Equity Offering in the amount of $75.000174 million. Abeona Therapeutics Inc. has completed a Follow-on Equity Offering in the amount of $75.000174 million.
Security Name: Common Stock
Security Type: Common Stock
Securities Offered: 12,285,056
Price\Range: $4.07
Security Name: Pre-Funded Warrants
Security Type: Equity Warrant
Securities Offered: 6,142,656
Price\Range: $4.0699 Price Target Changed • Apr 28
Price target decreased by 7.1% to US$19.50 Down from US$21.00, the current price target is an average from 4 analysts. New target price is 498% above last closing price of US$3.26. Stock is up 6.2% over the past year. The company is forecast to post a net loss per share of US$2.14 next year compared to a net loss per share of US$2.53 last year. Major Estimate Revision • Apr 24
Consensus revenue estimates fall by 10% The consensus outlook for revenues in fiscal year 2024 has deteriorated. 2024 revenue forecast decreased from US$17.9m to US$16.0m. Forecast losses increased from -US$1.92 to -US$2.18 per share. Biotechs industry in the US expected to see average net income decline 9.7% next year. Consensus price target down from US$21.00 to US$20.50. Share price fell 57% to US$3.41 over the past week. New Risk • Apr 23
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$37m 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$37m free cash flow). Shareholders have been substantially diluted in the past year (53% increase in shares outstanding). Minor Risks Share price has been volatile over the past 3 months (13% average weekly change). Revenue is less than US$5m (US$3.5m revenue). Annonce • Apr 23
Abeona Therapeutics Inc. Provides Regulatory Update on Pz-Cel Abeona Therapeutics Inc. announced a regulatory update for prademagene zamikeracel (pz-cel). The U.S. Food and Drug Administration has issued a Complete Response Letter in response to the Company’s Biologics License Application (BLA) for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). The CRL follows the completion of Abeona’s Late Cycle Review Meeting with the FDA in March 2024. At the Late Cycle Review Meeting and in a subsequent information request, the FDA noted that certain additional information needed to satisfy Chemistry Manufacturing and Controls (CMC) requirements must be satisfactorily resolved before the application can be approved. In response, the Company submitted plans to the FDA with the commitment to provide CMC data prior to BLA approval, and full validation reports after approval in mid-2024. In addition, the Company discussed these plans with the FDA in a subsequent informal meeting. In the CRL, the FDA indicated that the proposed timing of the data submission by Abeona would not allow sufficient time for the FDA to complete its review by the May 25, 2024 PDUFA date. The information needed to satisfy the CMC requests in the CRL pertains to validation requirements for certain manufacturing and release testing methods, including some that were captured in the observations during the FDA’s pre-license inspection (PLI). The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The BLA for pz-cel was accepted for filing and granted priority review designation by the FDA in November 2023. The application is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379). Abeona believes that both studies demonstrate that a single application of pz-cel on large and chronic wounds will deliver sustained wound healing and pain reduction. Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder caused by a defect in the COL7A1 gene that results in the inability to produce Type VII collagen. Pz-cel is designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome. Pz-cel is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing and pain reduction in even the toughest-to-treat RDEB wounds after a one-time application procedure. The pivotal Phase 3 VIITAL™ study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of pz-cel in 43 large chronic wound pairs in 11 subjects with RDEB. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. Abeona produces pz-cel for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio. Annonce • Mar 20
Abeona Therapeutics Inc., Annual General Meeting, Apr 24, 2024 Abeona Therapeutics Inc., Annual General Meeting, Apr 24, 2024, at 09:00 Eastern Standard Time. Agenda: To elect Vishwas Seshadri and Leila Alland as Class 2 directors to hold office for a term of three years and until their successors are elected and qualified; to approve an increase in the number of shares reserved for issuance under the Amended and Restated Abeona Therapeutics Inc. 2023 Equity Incentive Plan from 1,700,000 to 3,200,000 shares; to approve, on an advisory basis, the compensation of the Company’s named executive officers; to ratify the appointment of Deloitte & Touche LLP as our independent registered public accounting firm for the fiscal year ending December 31, 2024; and to discuss other maters. Reported Earnings • Mar 19
Full year 2023 earnings: EPS misses analyst expectations Full year 2023 results: US$2.53 loss per share. Net loss: US$54.2m (loss widened 25% from FY 2022). Revenue is forecast to grow 47% p.a. on average during the next 3 years, compared to a 17% growth forecast for the Biotechs industry in the US. New Risk • Feb 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: Minor Risks Share price has been volatile over the past 3 months (11% average weekly change). Shareholders have been diluted in the past year (44% increase in shares outstanding). Revenue is less than US$5m (US$3.6m revenue). Recent Insider Transactions • Feb 08
President recently bought US$88k worth of stock On the 6th of February, Vishwas Seshadri bought around 20k shares on-market at roughly US$4.39 per share. This transaction amounted to 3.9% of their direct individual holding at the time of the trade. This was the largest purchase by an insider in the last 3 months. Vishwas has been a buyer over the last 12 months, purchasing a net total of US$169k worth in shares. Breakeven Date Change • Dec 31
Forecast to breakeven in 2026 The 4 analysts covering Abeona Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$42.3m in 2026. Average annual earnings growth of 55% is required to achieve expected profit on schedule. Annonce • Nov 29
Abeona Therapeutics Inc. Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA) Abeona Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application. The BLA is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and confirmatory evidence from a Phase 1/2a study (NCT01263379). Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction. Data from the VIITAL™ study were presented during the inaugural International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Long-term follow up data up to eight years and quality of life data from the Phase 1/2a study were published in Orphanet Journal of Rare Diseases. Pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder caused by a defect in the COL7A1 gene that results in the inability to produce Type VII collagen. Pz-cel is designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome. Pz-cel is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing and pain reduction in even the toughest-to-treat RDEB wounds after a one-time application procedure. The pivotal Phase 3 VIITAL™ study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of pz-cel in 43 large chronic wound pairs in 11 subjects with RDEB. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. Abeona produces pz-cel for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio. Breakeven Date Change • Nov 17
Forecast to breakeven in 2025 The 3 analysts covering Abeona Therapeutics expect the company to break even for the first time. New consensus forecast suggests the company will make a profit of US$16.1m in 2025. Average annual earnings growth of 69% is required to achieve expected profit on schedule. New Risk • Nov 04
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 9.5% 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 Risk Shareholders have been substantially diluted in the past year (316% increase in shares outstanding). Minor Risks Less than 1 year of cash runway based on current free cash flow (-US$43m). Share price has been volatile over the past 3 months (9.5% average weekly change). Significant insider selling over the past 3 months (US$141k sold). Revenue is less than US$5m (US$3.6m revenue). New Risk • Oct 01
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$141k 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: Major Risk Shareholders have been substantially diluted in the past year (316% increase in shares outstanding). Minor Risks Less than 1 year of cash runway based on current free cash flow (-US$43m). Share price has been volatile over the past 3 months (9.2% average weekly change). Significant insider selling over the past 3 months (US$141k sold). Revenue is less than US$5m (US$3.6m revenue). Annonce • Sep 27
Abeona Therapeutics Submits Biologics License Application to U.S. FDA Seeking Priority Review and Approval of EB-101 for the Treatment of Patients with Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced the Company has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of EB-101, its investigational autologous, engineered cell therapy, as a treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). As part of the submission, Abeona requested a Priority Review, which, if granted, would shorten the FDA’s review period to six months from the filing acceptance of the BLA, instead of 10 months under standard review. The BLA submission for EB-101 followed ongoing discussions with the FDA and is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL study (NCT04227106) and confirmatory evidence from a Phase 1/2a study (NCT01263379). Data from the VIITAL™ study were presented during the inaugural International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Long-term follow up data up to eight years and quality of life data from the Phase 1/2a study were published in Orphanet Journal of Rare Diseases. The FDA’s decision on BLA acceptance is typically made during the 60-day window following submission. If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024, at which time, Abeona believes that it would be eligible to receive a Priority Review Voucher. The voucher, if granted, could be used by the Company to accelerate the review of a future BLA or New Drug Application, or be sold to a third party. EB-101 has been granted Rare Pediatric Disease, Regenerative Medicine Advanced Therapy, Breakthrough Therapy and Orphan Drug designations. Annonce • Sep 13
Abeona Therapeutics Inc. Appoints Madhav Vasanthavada as Chief Commercial Officer Abeona Therapeutics Inc. announced the appointment of Madhav Vasanthavada, Ph.D., M.B.A. to the role of Chief Commercial Officer (CCO) and Head of Business Development (BD), effective immediately. In this capacity, in addition to his current BD responsibilities, Dr. Vasanthavada will oversee all aspects of commercial strategy, planning and operations as Abeona prepares for a potential launch of EB-101, its investigational, genetically engineered autologous cell therapy for recessive dystrophic epidermolysis bullosa (RDEB). Dr. Vasanthavada is a seasoned commercial executive bringing over 20 years of experience with leadership roles in sales, marketing, and market access in the life sciences industry, including launch experience with autologous cell therapies. Over the past year, as Abeonas Head of BD, Dr. Vasanthavadas efforts to assess EB-101s commercial opportunity informed the Companys strategy to prepare for a U.S. commercial launch without depending on a partner. Madhavs appointment as Chief Commercial Officer is timely as Abeona prepares for the transition into a commercial-stage organization with the potential approval and launch of EB-101 in the U.S. next year, said Vish Seshadri, Chief Executive Officer of Abeona. His diverse commercial leadership experience, coupled with his strong track record launching autologous cell therapies with a heavy focus on customer experience, makes Madhav the ideal candidate to lead the staged build-out of highly focused, nimble commercial organization and for maximizing the commercial opportunity for EB-101. Prior to Abeona, Dr. Vasanthavada served in commercial leadership roles at Bristol Myers Squibb (BMS) and Celgene, where he led the marketing team in the Global CAR-T Cell Therapy Franchise to launch two autologous cell therapies, Breyanzi (lisocabtagene maraleucel) and Abecma (idecabtagene vicleucel), in key worldwide markets. Previously, Dr. Vasanthavada served in a variety of U.S. commercial roles at Bayer in marketing, market access and sales, and was ultimately the brand leader for Xofigo (radium Ra 223 dichloride). He began his career as a scientist in Novartis R&D where his work led to multiple patents and publications. Dr. Vasanthavada holds a Ph.D. in Pharmaceutical Sciences from the University of Rhode Island, and an M.B.A. from the Harvard Business School. Annonce • Sep 01
Abeona Therapeutics Announces Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission Abeona Therapeutics Inc. announced the Company's intention to proceed with the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for EB-101, its investigational autologous, engineered cell therapy, for patients with recessive dystrophic epidermolysis bullosa (RDEB) after the recent completion of a pre-BLA meeting with the FDA. At the meeting, Abeona reached alignment with the FDA that the EB-101 clinical efficacy and safety data appear adequate to support a BLA submission. The Agency also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book. The FDA requested that Abeona include within its BLA submission additional background and data supporting the scientific rationale underlying its EB-101 potency and identity assays so that they can be fully evaluated by the Agency post-submission. The Agency also requested that supplemental data pertaining to certain chemistry, manufacturing, and controls and clinical topics be included in the BLA package. Abeona believes that it has the necessary supporting data in-hand to generate these additional reports, including those regarding potency and identity, to address the Agency's requests. EB-101 has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the FDA. Reported Earnings • Aug 08
Second quarter 2023 earnings released: US$0.92 loss per share (vs US$2.08 loss in 2Q 2022) Second quarter 2023 results: US$0.92 loss per share. Net loss: US$16.7m (loss widened 38% from 2Q 2022). Revenue is forecast to grow 62% p.a. on average during the next 3 years, compared to a 15% growth forecast for the Biotechs industry in the US. Annonce • May 12
Abeona Therapeutics Announces Additional Phase 3 Viital Study Results for Eb-101 Presented At the International Societies for Investigative Dermatology 2023 Meeting Abeona Therapeutics Inc. announced an oral presentation of additional data from the Company's pivotal Phase 3 VIITAL™ study evaluating its investigational EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The data presentation occurred earlier on May 11, 2023 during the inaugural International Societies for Investigative Dermatology (ISID) Meeting in Tokyo, Japan. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin. There is currently no approved treatment for RDEB. EB-101 is an autologous, engineered cell therapy currently being developed for the treatment of recessive dystrophic epidersolysis bullosa (R DEB), a rare connective tissue disorder without an approved therapy. The pivotal Phase 3 VIital™ study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB. Treatment with EB-101 involves using gene transfer to deliver the functional COL7A1 gene into a patient's own skin cells (keratinocytes and its proteins) and transplanting those cells back to the patient. EB-101 is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing. EB-101 has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designation by the U.S. FDA. Abeona produces EB-101 for the VIITAL study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio. EB-101 is an investigational product not yet approved by the FDA. Price Target Changed • Nov 16
Price target increased to US$29.00 Up from US$15.67, the current price target is an average from 2 analysts. New target price is 638% above last closing price of US$3.93. Stock is down 82% over the past year. The company is forecast to post a net loss per share of US$5.73 next year compared to a net loss per share of US$21.57 last year. Board Change • Nov 16
High number of new and inexperienced directors There are 7 new directors who have joined the board in the last 3 years. The company's board is composed of: 7 new directors. 5 experienced directors. No highly experienced directors. Independent Director Todd Wider is the most experienced director on the board, commencing their role in 2015. The following issues are considered to be risks according to the Simply Wall St Risk Model: Lack of board continuity. Lack of experienced directors. Major Estimate Revision • Nov 04
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 losses forecast to reduce from -US$5.33 to -US$4.39 per share. Revenue forecast unchanged from US$1.98m at last update. Biotechs industry in the US expected to see average net income decline 73% next year. Consensus price target of US$15.67 unchanged from last update. Share price fell 12% to US$3.87 over the past week. Seeking Alpha • Oct 19
Abeona: Pivotal Data Readout EB-101 Before Year End Could Boost Shareholder Value Summary
Results from the pivotal VITAL study, using EB-101 for the treatment of patients with RDEB are expected to be released before the end of 2022.
Should EB-101 be approved by FDA for RDEB, it would give company a Priority Review Voucher, which could be sold for around $100 million or more to another pharmaceutical company.
ABO-102, which is being developed to treat patients with Sanfilippo syndrome type A has been offloaded to Ultragenyx Pharmaceutical for advancement.
Abeona has $26 million in cash as of June 30, 2022. It believes enough cash to fund operations until Q2 of 2023, but will likely have to raise cash before or after data from this late-stage study.
Abeona Therapeutics (ABEO) is a great speculative biotech play to look into. That's because it is gearing up to report results from its phase 3 pivotal study known as VITAL. This late-stage study is using its cell therapy known as EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). Results from this trial are expected any day now before the end of 2022, therefore, this provides traders/investors with a huge catalyst to look forward to. Abeona wants to focus on this program for its pipeline, therefore, it chose to offload ABO-102 (now UX111) for Sanfilippo syndrome type A ((MPS IIIA)) to Ultragenyx Pharmaceutical (RARE). That is, Ultragenyx will be responsible for taking over this program and moving it forward in the clinic. In return, Abeona will be receiving tiered royalties and commercial milestone payments should this drug ultimately be approved by regulators. With proof of concept established with EB-101 in a prior study, plus year-end 2022 data readout of EB-101 for RDEB, these are the reasons why I believe it is a great speculative biotech play to look into.
EB-101 Could Possibly Provide A Great Long-Term Durable Treatment Option For RDEB Patients
The main program in the pipeline is EB-101, which is being developed for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is a terrible skin condition by which there is lack of collagen protein in the skin. It is a condition by which wounds are large and blisters tend to form. Patients with RDEB suffer greatly because of pain and complications which occur because of it. That's not the only problem to consider though for this patient population. The average life expectancy of someone who has it is 50 years of age. There are not currently any cures available for these patients, but there are treatments that attempt to control symptoms and pain. The main problem is that a mutation in the COL7A1 gene doesn't allow the formation of Type VII collagen, which is necessary for the skin to be healthy. EB-101 is a cell therapy which is being developed to provide functional collagen VII to the patient's own cells. The therapy is autologous, that is, cells are extracted from the patient and then transduced with the correct collagen gene necessary for proper formation. The final outcome is a fully formed sheet which is 5 to 7 cell layers thick. EB-101 is eventually applied to the wound in surgery which helps the patient to restore their functionality of COL7A1 gene encoding provided by the cells. In turn, EB-101 holds the potential to heal and provide pain relief for the patient. Even better, it provides a "one and done" scenario whereby it may be possible to avoid daily wound care. Abeona expects that EB-101 could produce estimated cumulative revenues of $1.5 billion over the products life cycle.
As I highlighted in the beginning Abeona Therapeutics is currently running its phase 3 VITAL study, which is using EB-101 for the treatment of patients with RDEB. It has been reported that the final patient completed their 6-month follow-up visit for this late-stage trial on October 4, 2022. This means within the next 3 to 4 weeks, investors should get an idea on whether or not this cell therapy succeeds in meeting the co-primary endpoints for this study. This means before the end of 2022, there will be a data readout from this VITAL study which could possibly boost shareholder value. Of course, both co-primary endpoints must be met for this to happen. Developing a treatment for large chronic wounds is not easy, as such, Abeona and the FDA came to an agreement on what the co-primary endpoints need to be in order for EB-101 to be approved for RDEB. The two co-primary endpoints necessary for regulatory approval are:
Proportion of chronic wounds with ≥50% healing from baseline at week 24 (Those treated with EB-101 versus those not treated at all with EB-101)
Pain reduction at dressing changes assesses by a mean difference in scores using the Wong-Baker FACES scale at week 24 (Treated EB-101 versus untreated EB-101) Major Estimate Revision • Aug 18
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 revenue forecast increased from US$1.30m to US$2.30m. EPS estimate fell from -US$2.67 to -US$3.32 per share. Biotechs industry in the US expected to see average net income decline 57% next year. Consensus price target down from US$20.00 to US$13.50. Share price fell 6.4% to US$4.36 over the past week. Major Estimate Revision • Jul 07
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 losses forecast to reduce from -US$7.62 to -US$2.67 per share. Revenue forecast unchanged from US$1.30m at last update. Biotechs industry in the US expected to see average net income decline 65% next year. Consensus price target down from US$37.50 to US$20.00. Share price fell 13% to US$4.52 over the past week. Major Estimate Revision • May 20
Consensus revenue estimates increase by 95% The consensus outlook for revenues in 2022 has improved. 2022 revenue forecast increased from US$670.0k to US$1.30m. Forecast losses expected to reduce from -US$0.33 to -US$0.30 per share. Biotechs industry in the US expected to see average net income decline 52% next year. Consensus price target down from US$2.00 to US$1.50. Share price fell 3.6% to US$0.16 over the past week. Price Target Changed • Apr 27
Price target decreased to US$2.00 Down from US$3.00, the current price target is an average from 3 analysts. New target price is 809% above last closing price of US$0.22. Stock is down 87% over the past year. The company is forecast to post a net loss per share of US$0.33 next year compared to a net loss per share of US$0.86 last year. Board Change • Apr 27
High number of new and inexperienced directors There are 7 new directors who have joined the board in the last 3 years. The company's board is composed of: 7 new directors. 5 experienced directors. No highly experienced directors. Independent Director Todd Wider is the most experienced director on the board, commencing their role in 2015. The following issues are considered to be risks according to the Simply Wall St Risk Model: Lack of board continuity. Lack of experienced directors. Major Estimate Revision • Apr 01
Consensus forecasts updated The consensus outlook for 2022 has been updated. 2022 expected loss increased from -US$0.40 to -US$0.44 per share. Revenue forecast of US$1.91m unchanged since last update. Biotechs industry in the US expected to see average net income decline 46% next year. Consensus price target down from US$3.00 to US$2.67. Share price rose 8.8% to US$0.32 over the past week. Price Target Changed • Dec 01
Price target decreased to US$4.50 Down from US$5.00, the current price target is an average from 4 analysts. New target price is 553% above last closing price of US$0.69. Stock is down 55% over the past year. The company is forecast to post a net loss per share of US$0.60 next year compared to a net loss per share of US$0.91 last year. Board Change • Nov 02
High number of new directors There are 6 new directors who have joined the board in the last 3 years. Chairman Michael Amoroso was the last director to join the board, commencing their role in 2021. The company’s lack of board continuity is considered a risk according to the Simply Wall St Risk Model. Price Target Changed • Jul 20
Price target decreased to US$4.67 Down from US$6.00, the current price target is an average from 4 analysts. New target price is 279% above last closing price of US$1.23. Stock is down 61% over the past year.