Announcement • 6h
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 €261k worth of stock On the 9th of June, Vishwas Seshadri sold around 56k shares on-market at roughly €4.64 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 €1.0m. Announcement • 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. Announcement • 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 Announcement • Apr 21
Abeona Therapeutics Inc., Annual General Meeting, Jun 12, 2026 Abeona Therapeutics Inc., Annual General Meeting, Jun 12, 2026. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • 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. Announcement • Apr 12
Abeona Therapeutics Inc., Annual General Meeting, May 19, 2025 Abeona Therapeutics Inc., Annual General Meeting, May 19, 2025. Announcement • 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. Announcement • 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. New Risk • Aug 13
New major risk - Revenue size The company makes less than US$1m in revenue. This is considered a major risk. Companies with a small amount of revenue 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 Shareholders have been substantially diluted in the past year (75% increase in shares outstanding). Revenue is less than US$1m. Minor Risks Share price has been volatile over the past 3 months (8.0% average weekly change). Significant insider selling over the past 3 months (€217k sold). New Risk • Jun 21
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: €217k 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 Risks Share price has been highly volatile over the past 3 months (18% average weekly change). Negative equity (-US$8.9m). Shareholders have been substantially diluted in the past year (118% increase in shares outstanding). Minor Risks Currently unprofitable and not forecast to become profitable over next 3 years (US$12m net loss in 3 years). Significant insider selling over the past 3 months (€217k sold). Revenue is less than US$5m (US$3.5m revenue). 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). Announcement • 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 New Risk • Apr 24
New minor risk - Market cap size The company's market capitalization is less than US$100m. Market cap: €87.1m (US$93.3m) 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$37m free cash flow). Share price has been highly volatile over the past 3 months (20% average weekly change). Shareholders have been substantially diluted in the past year (53% increase in shares outstanding). Minor Risks Revenue is less than US$5m (US$3.5m revenue). Market cap is less than US$100m (€87.1m market cap, or US$93.3m). Announcement • 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. 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). Share price has been highly volatile over the past 3 months (14% average weekly change). Shareholders have been substantially diluted in the past year (53% increase in shares outstanding). Minor Risk Revenue is less than US$5m (US$3.5m revenue). Announcement • 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. New Risk • Mar 19
New major risk - Shareholder dilution The company's shareholders have been substantially diluted in the past year. Increase in shares outstanding: 59% 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 (14% average weekly change). Shareholders have been substantially diluted in the past year (59% increase in shares outstanding). Minor Risk Revenue is less than US$5m (US$3.5m revenue). Reported Earnings • Mar 19
Full year 2023 earnings released: US$2.53 loss per share (vs US$5.53 loss in FY 2022) 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 18% growth forecast for the Biotechs industry in Germany. Recent Insider Transactions • Feb 08
President recently bought €82k worth of stock On the 6th of February, Vishwas Seshadri bought around 20k shares on-market at roughly €4.08 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 €159k worth in shares. New Risk • Feb 02
New major risk - Share price stability The company's share price has been highly volatile over the past 3 months. It is more volatile than 90% of German stocks, typically moving 10% a week. This is considered a major risk. Share price volatility 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. It may also indicate 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. Currently, the following risks have been identified for the company: Major Risk Share price has been highly volatile over the past 3 months (10% average weekly change). Minor Risks Shareholders have been diluted in the past year (44% increase in shares outstanding). Revenue is less than US$5m (US$3.6m revenue). 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. Announcement • 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 • 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: €134k 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 Risks Share price has been highly volatile over the past 3 months (11% average weekly change). 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). Significant insider selling over the past 3 months (€134k sold). Revenue is less than US$5m (US$3.6m revenue). Announcement • 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. Announcement • 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. Announcement • 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 09
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 14% growth forecast for the Biotechs industry in Germany. Announcement • 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. 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. Announcement • Nov 05
Abeona Therapeutics Inc. announced that it expects to receive $35.000004 million in funding from Adage Capital Management, L.P., Armistice Capital LLC, EcoR1 Capital, LLC, Nantahala Capital Management, LLC, Deerfield Management Company, L.P. Series C and other investors Abeona Therapeutics Inc. announced that it has entered into a security purchase agreement for the issuance of 7,065,946 common shares at a price of $4.60 per share for gross proceeds of $32,503,351.6, pre-funded warrants exercisable for 543,933 shares of common stock at a price of $4.59 for gross proceeds of $2,496,652.47 and accompanying warrants to purchase 7,609,879 shares of its common stock, for aggregate gross proceeds of $35,000,000 on November 3, 2022. The transaction will include participation from new and existing investors including Adage Capital Management, L.P., Armistice Capital LLC, EcoR1 Capital, LLC, Nantahala Capital Management, LLC, Deerfield Management Company, L.P. Series C as well as other specialist biotech investors. Each accompanying warrant will represent the right to purchase one share of the company common stock at an exercise price of $4.75 per share of common stock. The pre-funded warrants and the accompanying warrants will be exercisable immediately and will expire five years from the date of issuance. The private placement is expected to close on November 7, 2022, subject to the satisfaction of customary closing conditions. All the securities issued under the purchase agreement were offered in reliance on an exemption from registration provided by Section 4(a)(2) of the Securities Act and Rule 506 of Regulation D promulgated thereunder. Announcement • Nov 04
Abeona Therapeutics Announces Positive Topline Results with Both Co-Primary Endpoints Met in Pivotal Phase 3 VIITAL(TM) Study of EB-101 Abeona Therapeutics Inc. announced positive topline data from its pivotal Phase 3 VIITAL study assessing the safety and efficacy of EB-101 for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). The VIITAL study met its two co-primary efficacy endpoints demonstrating statistically significant, clinically meaningful improvements in wound healing and pain reduction in large chronic RDEB wounds. All Evaluated Endpoints Successfully Achieved The pivotal Phase 3 VIITAL study evaluated the efficacy, safety and tolerability of EB-101 in 43 large chronic wound pairs in 11 subjects with RDEB. The large chronic wounds randomized and treated in VIITAL measured greater than 20 cm(2) of surface area and had remained open for a minimum of six months and a maximum of 21 years (mean 6.2 years). The co- primary endpoints of the study were: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing randomized treated with matched untreated (control) wound sites at the six-month timepoint, as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between randomized treated and matched untreated (control) wounds at the six-month timepoint. The study allowed for wounds not included in the randomized primary efficacy analysis to receive EB-101 treatment (n=14 non-randomized wounds). The tables below summarize the topline primary efficacy results: Post-Hoc Analysis of Pain Data In addition to meeting the co-primary pain endpoint, in a post-hoc analysis of the EB-101 treated severe wounds (baseline pain score of 6 or greater), including randomized and non-randomized (n=27), a mean pain reduction from baseline at six months of 5.70 was observed, as compared to a mean pain reduction of 3.51 for all treated randomized and non-randomized wounds for which pain was evaluated (n=53). Safety Results EB-101 was shown to be well-tolerated with no serious treatment-related adverse events observed, consistent with past clinical experience. There were no deaths or instances of positive replication-competent retrovirus (RCR) results, and no systemic immunologic responses were reported during the study, as well as no squamous cell carcinoma (SCC) at treatment sites after application of EB-101. Two subjects reported at least one serious adverse event (SAE) unrelated to EB-101. Four subjects reported related treatment emergent adverse events (TEAEs), including procedural pain, muscle spasms and pruritis. Infections unrelated to EB-101 were observed in eight patients. Abeona anticipates submitting results from this study, including further details with additional exploratory endpoints and the Week 12 results, for presentation at future medical meetings and for publication in a peer-reviewed journal. The Week 12 results are similar to Week 24 results, achieving statistical significance for pain reduction and wound healing at all levels. Jean Tang, M.D., Ph.D., Professor of Dermatology, Stanford University School of Medicine and Principal Investigator of the EB-101 pivotal Phase 3 VIITAL study said, "Large chronic RDEB wounds are the toughest to treat and often associated with intense chronic pain that significantly impacts the quality of life of RDEB patients, necessitating frequent use of opioids. In the Phase 3 VIITAL study, EB-101 has been shown to both heal such large chronic wounds and significantly reduce pain. And continue to see durable clinical benefit of EB-101 with up to eight years of follow-up in Phase 1/2a study. Next Steps Based on the positive topline results, Abeona intends to submit a Biologics License Application (BLA) for EB-101 to the U.S. Food and Drug Administration (FDA) in the second quarter of 2023. EB-101 has been granted Orphan Drug and Rare Pediatric Disease (RPD) designations by the FDA. Among the benefits of Orphan Drug designation are seven years of market exclusivity following FDA approval, potentially preventing FDA approval of another product deemed to be the same as the approved product for the same indication, waiver of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received. A sponsor who receives an approval for a BLA with RPD designation may qualify for a Priority Review Voucher (PRV), subject to final determination by the FDA. The PRV can be used to receive an expedited review process of a subsequent marketing application for a different product or sold to another company. Announcement • Oct 20
Abeona Therapeutics Inc. Announces Database Lock for Pivotal Phase 3 VIITAL Study of EB-101 in Patients with Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced the database lock on October 18, 2022 for the pivotal Phase 3 VIITAL study of its investigational autologous, engineered cell therapy, EB-101, in patients with recessive dystrophic epidermolysis bullosa (RDEB). With the database now locked, the Company remains on track to report topline results in the next two to three weeks, consistent with guidance provided in early-October. Abeona expects the VIITAL study, if positive, to serve as a basis for seeking approval by the U.S. Food and Drug Administration (FDA) of EB-101 for the treatment of patients with RDEB. About Recessive Dystrophic Epidermolysis Bullosa Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder 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 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. About EB-101 EB-101 is an autologous, engineered cell therapy currently being investigated in Abeona's pivotal Phase 3 VIITAL study for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder without an approved therapy. The EB-101 VIITAL study is a randomized clinical trial with target enrollment of at least 10 to 15 RDEB patients with approximately 36 large, chronic wound sites treated in total. Treatment with EB-101 involves using gene transfer to deliver the COL7A1 gene into a patient's own skin cells (keratinocytes and its progenitors) 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. The U.S. FDA has granted Rare Pediatric Disease Designation for EB-101. 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. Announcement • Oct 06
Abeona Therapeutics Inc. Announces Completion of Patient Follow-up in Pivotal Phase 3 VIITAL™ Study of EB-101 in Patients with Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced that the last patient has completed their 6-month follow-up visit in Abeona’s pivotal Phase 3 VIITAL™ study of its investigational autologous, engineered cell therapy, EB-101, in patients with recessive dystrophic epidermolysis bullosa. Abeona has been verifying and preparing the data in real time to ensure that an efficient database lock can be completed within two to three weeks from the last patient’s final visit. Abeona expects the VIITAL™ study, if positive, to serve as the basis for seeking approval by the U.S. Food and Drug Administration (FDA) of EB-101 for the treatment of patients with RDEB. Announcement • Jul 21
Abeona Therapeutics Regains Compliance with Nasdaq Listing Requirements Abeona Therapeutics Inc. announced that it received notice from The Nasdaq Stock Market LLC (Nasdaq) on July 19, 2022 informing Abeona that it has regained compliance with the minimum bid price requirement under Nasdaq Listing Rule 5550(a)(2) (the “Rule”) for continued listing on The Nasdaq Capital Market. In order to regain compliance with the Rule, the Company’s common stock was required to maintain a minimum closing bid price of $1.00 or more for at least 10 consecutive trading days. That requirement was met on July 18, 2022. Announcement • Jul 12
Abeona Therapeutics Inc., Annual General Meeting, Aug 17, 2022 Abeona Therapeutics Inc., Annual General Meeting, Aug 17, 2022, at 09:00 US Eastern Standard Time. Agenda: To consider and elect Mark J. Alvino, Faith L. Charles and Donald A. Wuchterl as Class 3 directors to hold office for a term of three years and until their successors are elected and qualified; to consider and approve an amendment to the Abeona Therapeutics Inc. 2015 Equity Incentive Plan to increase the number of shares of our Common Stock authorized for issuance thereunder from 720,000 to 1,440,000 shares; to consider and approve, on an advisory basis, the compensation of the Company's named executive officers; to consider an advisory vote on the frequency of future advisory votes on the compensation of the Company's named executive officers; and to consider the ratify the appointment of Whitley Penn LLP as independent registered public accounting firm for the fiscal year ending December 31, 2022. Announcement • May 20
Nasdaq Grants Second 180-Day Period to Abeona Therapeutics to Regain Compliance with Minimum Bid Price Rule Abeona Therapeutics Inc. announced that the Company has been granted an additional 180-day period from Nasdaq’s Listing Qualification Department, through November 14, 2022, to regain compliance with the $1.00 minimum bid price requirement for continued listing on The Nasdaq Capital Market. The Company's common stock continues to trade on the Nasdaq Capital Market under the symbol "ABEO." If at any time until November 14, 2022, the closing bid price of the Company’s common stock is at or above $1.00 per share for a minimum of 10 consecutive trading days, Nasdaq will provide the Company with written confirmation of compliance. If compliance cannot be demonstrated during the additional 180-day grace period, Nasdaq will provide written notification that the common stock will be subject to delisting. At such time, the Company may appeal the determination to a Nasdaq Hearings Panel. The Company's common stock would remain listed pending the completion of the appeal process. 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. Announcement • Apr 01
Abeona Therapeutics Inc. Reports Goodwill Impairment Charge for the Fourth Quarter Ended December 31, 2021 Abeona Therapeutics Inc. reported Goodwill impairment charge for the fourth quarter ended December 31, 2021. For the quarter, the company reported Goodwill impairment charge of $32,466,000. 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. Announcement • Jul 24
Abeona Therapeutics Inc. Activates Second Clinical Trial Site in EB-101 Pivotal Phase 3 Viital Study for Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced the activation of a second clinical trial site in its pivotal Phase 3 VIITAL study of its investigational EB-101 treatment for recessive dystrophic epidermolysis bullosa (RDEB) at UMass Memorial Medical Center in Worcester, MA. The EB-101 pivotal VIITAL™ study is currently ongoing at Stanford University Medical Center in Palo Alto, CA. Target enrollment is 10 to 15 RDEB patients with approximately 35 large, chronic wound sites to be treated in total. Treatment with EB-101 uses gene transfer to deliver a functional COL7A1 gene into a patient’s own skin cells (keratinocytes and its progenitors) and transplanting those cells back to the patient. EB-101 is believed to facilitate wound healing by supplementing Type VII collagen expression in RDEB patients who lack a fully functional COL7A1 gene. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6), as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). Recessive dystrophic epidermolysis bullosa (RDEB) is a rare connective tissue disorder 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 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. Breakeven Date Change • May 19
Forecast to breakeven in 2024 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$35.4m in 2024. Average annual earnings growth of 68% is required to achieve expected profit on schedule. Recent Insider Transactions • Feb 20
Independent Director recently sold €258k worth of stock On the 17th of February, Paul Mann sold around 133k shares on-market at roughly €1.94 per share. This was the largest sale by an insider in the last 3 months. Insiders have been net sellers, collectively disposing of €269k more than they bought in the last 12 months. Announcement • Feb 13
Abeona Therapeutics Inc. Announces New Positive Data from Two Ongoing Phase 1/2 Clinical Trials of its Investigational AAV-Based Gene Therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB Abeona Therapeutics Inc. announced new positive data from two ongoing Phase 1/2 clinical trials of the company’s investigational AAV-based gene therapies ABO-102 and ABO-101 in MPS IIIA and MPS IIIB, respectively. The updated results from the Transpher A study evaluating ABO-102 in Sanfilippo syndrome type A (MPS IIIA) demonstrated that neurocognitive development was preserved within normal range of a non-afflicted child for 2.5 years to 3 years (the latest time point measured) after treatment with ABO-102 (3x1013 vg/kg) in three young patients in the high-dose cohort 3. The three young patients were treated with ABO-102 at ages 27 months, 19 months, and 12 months and are now at ages ranging from 3.5 years to 5+ years, the timepoint at which patients with MPS IIIA have already started to experience neurocognitive decline based on the natural history of disease progression. Dose-dependent and statistically significant reductions in cerebrospinal fluid heparan sulfate, denoting enzyme activity in the central nervous system (CNS), and liver volume were sustained for two years after treatment. ABO-102 has been well-tolerated with long-term safety remaining favorable 24-55 months following treatment. There have been no treatment-related severe adverse events and no clinically significant adverse events reported.Results from the Transpher B study evaluating ABO-101 in Sanfilippo syndrome type B (MPS IIIB) showed that treatment with ABO-101 is associated with a dose-dependent and sustained improvement in central nervous system and systemic biomarkers, indicating the potent biologic effect of ABO-101 in patients with MPS IIIB. ABO-101 has been well-tolerated with no infusion related or early acute reactions and no clinically significant adverse events or laboratory abnormalities. Is New 90 Day High Low • Feb 05
New 90-day high: €2.00 The company is up 133% from its price of €0.86 on 06 November 2020. The German market is up 14% over the last 90 days, indicating the company outperformed over that time. It also outperformed the Biotechs industry, which is up 6.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is €0.89 per share. Announcement • Jan 26
Abeona Therapeutics Inc. Announces Successful Type B Meeting with FDA for Pivotal Phase 3 VIITAL Study of EB-101 in Recessive Dystrophic Epidermolysis Bullosa Abeona Therapeutics Inc. announced that the company held a successful Type B meeting with the U.S. Food and Drug Administration (FDA) to align with the Agency on the company’s proposal regarding co-primary endpoints for the pivotal Phase 3 VIITAL™ study of EB-101 in recessive dystrophic epidermolysis bullosa (RDEB). Following the meeting, Abeona is proceeding with its plan to enroll between 10 to 15 patients with RDEB, comprising approximately 35 large chronic wound sites treated in total. The co-primary endpoints of the study are: 1) the proportion of RDEB wound sites with greater than or equal to 50% healing from baseline, comparing treated with untreated wound sites at Week 24 (Month 6) as determined by direct investigator assessment; and 2) pain reduction associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds at Week 24 (Month 6). As previously announced, data from a Phase 1/2a clinical trial presented at the 2020 Society for Pediatric Dermatology Annual Meeting showed that wound healing of 50% or greater following EB-101 treatment in patients with RDEB was associated with no pain at treated sites at three-, four- and five-years post-treatment, compared with presence of pain in 53% of wound sites at baseline. Is New 90 Day High Low • Jan 13
New 90-day high: €1.55 The company is up 33% from its price of €1.16 on 14 October 2020. The German market is up 8.0% over the last 90 days, indicating the company outperformed over that time. It also outperformed the Biotechs industry, which is up 2.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is €0.93 per share. Is New 90 Day High Low • Dec 28
New 90-day high: €1.47 The company is up 50% from its price of €0.98 on 29 September 2020. The German market is up 7.0% over the last 90 days, indicating the company outperformed over that time. It also outperformed the Biotechs industry, which is down 6.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. Announcement • Nov 14
Abeona Therapeutics Receives A Letter from the Nasdaq Stock Market On November 5, 2020, Abeona Therapeutics Inc. (the “Company”) received a letter from the Nasdaq Stock Market (“Nasdaq”) stating that the previously-disclosed departures of two independent directors from the Company’s Board of Directors (the “Board”) resulted in noncompliance with the independent director and audit committee requirements set forth in Listing Rule 5605 (the “Notice”). More specifically, the Board currently is not comprised of a majority of “independent directors” within the meaning of Listing Rule 5605(a)(2), and the Board’s Audit Committee does not have at least three members, each of whom is independent and meets the criteria for independence set forth in Rule 10A-3(b)(1) of the Securities Exchange Act of 1934, as amended. Currently, the Board has two independent members and two non-independent members, and the Audit Committee consists of two independent members. The Notice states that, consistent with Listing Rules 5605(b)(1)(A) and 5605(c)(4), the Company is entitled to cure the noncompliance, and the cure period is defined as (i) until the earlier of the Company’s next annual stockholders’ meeting or September 27, 2021; or (ii) if the next annual stockholders’ meeting is held on or before March 26, 2021, then the Company must evidence compliance no later than March 26, 2021. Is New 90 Day High Low • Nov 04
New 90-day low: €0.87 The company is down 64% from its price of €2.43 on 05 August 2020. The German market is down 3.0% over the last 90 days, indicating the company underperformed over that time. It also underperformed the Biotechs industry, which is down 17% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. Announcement • Oct 29
Abeona Therapeutics Inc. Announces the Appointment of Michael Amoroso as Chief Operating Officer, with effect from November 01, 2020 Abeona Therapeutics Inc. announced that Michael Amoroso, Senior Vice President and Chief Commercial Officer at Abeona, has been promoted to Chief Operating Officer and will serve as the Company’s principal executive and operating officer, effective November 1, 2020. In this newly created role at Abeona, Mr. Amoroso's responsibilities will broaden to include oversight and leadership of all operations; including but not limited to, research and clinical development, regulatory, medical, commercial, corporate affairs and business development. Mr. Amoroso has served as Senior Vice President and Chief Commercial Officer of Abeona since July 2020. Prior to joining Abeona, Mr. Amoroso held various senior level commercial positions at leading biopharmaceutical companies, including Kite, Eisai Inc., Celgene Corporation (now a subsidiary of Bristol-Myers Squibb Company), and Sanofi. Previously, Mr. Amoroso worked at Celgene for six years in several commercial roles before serving as the organization’s Commercial Lead for CAR T-cell therapy programs. In this capacity, he helped Celgene develop an organizational model to commercialize cell therapies including specialized manufacturing and customer services for patients with lymphoma and myeloma. Before joining Celgene, Mr. Amoroso held various marketing and sales leadership positions over his 10-plus year tenure at Sanofi. Announcement • Oct 07
Abeona Therapeutics Inc. Announces Board Changes On September 17, 2020, Abeona Therapeutics Inc. had filed a Current Report on Form 8-K reporting that Steven H. Rouhandeh had notified the company of his resignation from the Board of Directors, effective September 30, 2020. On September 30, 2020, Mr. Rouhandeh rescinded his resignation from the Board, and the Board accepted his rescission of resignation. Also on September 30, 2020, the Board voted unanimously to elect Mr. Rouhandeh as Chairman of the Board. Mr. Rouhandeh will not be taking on any additional compensation as Chairman other than what he was already receiving as a non-employee director, which such compensation is disclosed in the company’s proxy statement filed on April 10, 2020. Announcement • Sep 30
Abeona Therapeutics Inc. Announces Management Changes Abeona Therapeutics Inc. announced changes in its executive leadership and Board of Directors. Joao Siffert, M.D. resigned from his positions as Head of Research and Development, and Chief Medical Officer, as well as member of the Board of Directors, on September 23, 2020. On September 27, 2020, Brian J.G. Pereira, M.D., Executive Chairman, notified the company of his resignation effective immediately. Dr. Pereira was not a member of any committees of the Board. Also on September 27, 2020, George Migausky resigned as a member of the Board and Chairman of the Audit Committee of the Board effective immediately. On September 28, 2020, Shawn Tomasello, Stephen Howell, M.D. and Stefano Buono each resigned as a member of the Board and from their respective positions as members of the several committees of the Board effective immediately. Ms. Tomasello was serving as a member of the Nominating and Corporate Governance Committee (the Nominating Committee) at the time of her resignation. Dr. Howell was serving as a member of both the Compensation Committee and the Nominating Committee at the time of his resignation. Mr. Buono was serving as Chairman of the Nominating Committee. Announcement • Sep 29
Abeona Therapeutics Inc. Announces Resignation of João Siffert as CEO Abeona Therapeutics Inc. announced changes in its executive leadership and Board of Directors. João Siffert, M.D. resigned from his positions as CEO on September 23, 2020. Is New 90 Day High Low • Sep 25
New 90-day low: €1.53 The company is down 41% from its price of €2.58 on 26 June 2020. The German market is up 3.0% over the last 90 days, indicating the company underperformed over that time. It also underperformed the Biotechs industry, which is up 2.0% over the same period. According to the Simply Wall St valuation model, the estimated intrinsic value of the company is per share. 
