Zevaskyn (prademagene zamikeracel) / Stanford University, Abeona Therap - LARVOL DELTA

Prademagene zamikeracel (Zevaskyn) for recessive dystrophic epidermolysis bullosa. (PubMed, Med Lett Drugs Ther) - No abstract available

Journal

Abeona Therapeutics Closes Sale of Rare Pediatric Disease Priority Review Voucher for $155 Million (The Brussels Times) - "Abeona Therapeutics Inc...announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million on June 27, 2025. Including net proceeds from the sale of the PRV, the Company reported that unaudited cash, cash equivalents, restricted cash and short-term investments as of June 30, 2025 were approximately $225 million....'The PRV proceeds, combined with our existing cash, provides Abeona with robust financial flexibility, ensuring over two years of operating capital for sustained growth without the need for further capital infusion and prior to accounting for ZEVASKYN sales. We anticipate the first ZEVASKYN patient treatment in Q3 2025, with profitability projected for early 2026'."

Commercial • Genetic Disorders

A Phase 3b Study for the Treatment of Dystrophic Epidermolysis Bullosa (DEB) in New and Previously EB-101 Treated Patients (clinicaltrials.gov) - P3 | N=12 | Active, not recruiting | Sponsor: Abeona Therapeutics, Inc | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2025 ➔ Sep 2025 | Trial primary completion date: Mar 2025 ➔ Sep 2025

Enrollment closed • Trial completion date • Trial primary completion date

Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial. (PubMed, Lancet) - P3 | "Prademagene zamikeracel improved wound healing and pain versus control and was well tolerated, supporting its potential to reduce wound burden in patients with large, chronic RDEB wounds."

Journal • P3 data • Dermatology • Pain • COL7A1

Prademagene Zamikeracel. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Absence of insertional oncogenesis and RCR in clinical and pre-clinical experience with prademagene zamikeracel, a retrovirally delivered cell-based gene therapy, in recessive dystrophic epidermolysis bullosa patients (SID 2025) - "In clinical trial subjects, no SCC was observed at 144 pz-cel-treated sites yet occurred exclusively in untreated areas. In SCC cases, RCR and proviral genome testing results were negative. In an ex-vivo non-clinical study, six RDEB samples were retrovirally transduced yielding 3, 203, 395 reads, 193, 317 inferred cells, and 184, 931 unique integration sites."

Gene therapy • Late-breaking abstract • Preclinical • Gene Therapies • COL7A1

Presence of large progenitor cell populations in pz-cel autologous gene-corrected epidermal sheets (SID 2025) - "Pz-cel (prademagene zamikeracel) was developed to address large, chronic wounds in RDEB patients... These results confirm the presence of a significant population of progenitor cells within the graft. The large proportion of these cells in the drug product may increase the likelihood of wound site repopulation by long-lasting, genetically corrected cells, consistent with the detection of Col7 at grafted sites at 24 months."

Clinical • Late-breaking abstract • Genetic Disorders • COL7A1 • ITGB1 • TFRC

Abeona Therapeutics Enters into Agreement to Sell Priority Review Voucher for $155 Million (GlobeNewswire) - "Abeona Therapeutics Inc...today announced it has entered into a definitive asset purchase agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $155 million upon the closing of the transaction. Abeona was awarded the PRV following the U.S. Food and Drug Administration (FDA) approval of ZEVASKYN (prademagene zamikeracel) on April 28, 2025....The transaction is subject to customary closing conditions, including expiration of the applicable waiting period under the Hart-Scott Rodino (HSR) Antitrust Improvements Act."

Commercial • Genetic Disorders

U.S. FDA Approves ZEVASKYN (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB) (GlobeNewswire) - "Abeona Therapeutics...today announced the U.S. Food and Drug Administration (FDA) has approved ZEVASKYN (pronounced as ‘ZEE-vah-skin’) (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease....The FDA approval of ZEVASKYN is based on the pivotal Phase 3 VIITAL study (NCT04227106)..."

FDA approval • Genetic Disorders

Abeona Therapeutics Reports Full Year 2024 Financial Results, Provides Pz-cel Regulatory Update and Commercial Launch Plans (GlobeNewswire) - "On March 14, 2025, Abeona received draft United States Prescribing Information (USPI) from the FDA to initiate discussion on the label for pz-cel. Discussions are also ongoing with the FDA on post-approval marketing requirements and commitments for pz-cel. If approved, the Company anticipates the first patient treatment with pz-cel in the third quarter of 2025...Obtained two additional patents from the United States Patent and Trademark Office for pz-cel, extending patent protection on the use of pz-cel for the treatment of RDEB to June 2037 and patent protection on the packaging and transport system for pz-cel to July 2040."

FDA event • Launch US • Patent • Genetic Disorders • Rare Diseases

Abeona Therapeutics Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (GlobeNewswire) - "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."

FDA filing • PDUFA • Genetic Disorders • Rare Diseases

Abeona Therapeutics Announces Favorable Medicare Reimbursement Decisions for Pz-cel (GlobeNewswire) - "Abeona Therapeutics Inc...announced that the Centers for Medicare and Medicaid Services (CMS) has granted a product-specific procedure code ICD-10-PCS (International Classification of Diseases, 10th Revision, Procedure Coding System) for prademagene zamikeracel (pz-cel), Abeona’s investigational autologous cell-based gene therapy currently in development for recessive dystrophic epidermolysis bullosa (RDEB). If pz-cel receives U.S. marketing approval, this code will allow for efficient and accurate documentation, billing, and analysis of inpatient hospital procedures using pz-cel. The code will go into effect on October 1, 2024."

Reimbursement • Genetic Disorders • Rare Diseases

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Abeona Therapeutics, Inc | Phase classification: P3b ➔ P3 | Trial completion date: Dec 2024 ➔ Jun 2025 | Trial primary completion date: Aug 2024 ➔ Mar 2025

Phase classification • Trial completion date • Trial primary completion date

Eicosapentaenoic Acid Level Predicts Long-Term Survival and Cardiovascular or Limb Event in Peripheral Arterial Disease. (PubMed, Ann Vasc Dis) - "In Cox stepwise multivariate analysis, lower EPA (hazard ratio [HR]: 0.996, 95% confidence interval [CI]: 0.993-1.000, p = 0.034), ankle brachial pressure index (ABI), body mass index, serum albumin, eGFR, age, CRP, D-dimer, critical limb ischemia, diabetes, cerebrovascular disease (CVD), and statin were related to ACD (p <0.05); lower EPA (HR: 0.997, 95% CI: 0.994-1.000, p = 0.038), ABI, serum albumin, eGFR, age, diabetes, coronary heart disease, CVD, and statin were related to MACEs (p <0.05); and lower EPA (HR: 0.988, 95% CI: 0.982-0.993, p <0.001), ABI, and low-density lipoprotein cholesterol were related to LEAEs (p <0.05). Low plasma EPA level was a significant risk factor for ACD, MACEs, and LEAEs in patients with PAD."

Journal • Cardiovascular • CNS Disorders • Coronary Artery Disease • Diabetes • Dyslipidemia • Heart Failure • Metabolic Disorders • Peripheral Arterial Disease • Vascular Neurology • CRP

Exploring the Financial Burden Associated with the Management of Dystrophic Epidermolysis Bullosa (DEB): A Systematic Literature Review (SLR) with Database Review of Future Disease-Modifying Therapies (DMTs) (ISPOR 2024) - "Nine clinical trials research 7 potential DMTs: advanced/gene therapies (EB-101, B-VEC, ALLO-ASC-SHEET, FCX-007), protein-replacement therapies (PTR-01, PTR-01-002), and a proteinase inhibitor (Polyphenon E). Previously, as there were no DMT options for patients with DEB treatment was symptomatic only. Future innovative DMTs should aim to prevent skin wounds, thereby decreasing the need for hospitalization and outpatient visits."

Review • Dermatology • Gene Therapies

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3b | N=12 | Recruiting | Sponsor: Abeona Therapeutics, Inc | Active, not recruiting ➔ Recruiting

Enrollment open

Abeona Therapeutics Announces Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission (GlobeNewswire) - "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....'With the constructive feedback from the FDA now in-hand, we are proceeding on a clear regulatory path leading to the planned BLA submission for EB-101 early this Fall.'"

BLA • Genetic Disorders

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3b | N=12 | Active, not recruiting | Sponsor: Abeona Therapeutics, Inc | Not yet recruiting ➔ Active, not recruiting

Enrollment closed

Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (clinicaltrials.gov) - P1/2 | N=12 | Completed | Sponsor: Stanford University | Active, not recruiting ➔ Completed

Trial completion • Gene Therapies

Results from VIITAL: A phase 3, randomized, intrapatient-controlled trial of an investigational collagen type VII gene-corrected autologous cell therapy, EB-101, for the treatment of recessive dystrophic epidermolysis bullosa (RDEB) (ISID 2023) - "EB-101 significantly improved wound healing and reduced pain, which translated to additional clinically meaningful benefits in terms of itch reduction and other quality of life improvements. These results demonstrate the favorable risk-benefit profile of EB-101."

Clinical • P3 data • Pain

EB-101 Treatment for New and Previously Treated Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3b | N=12 | Not yet recruiting | Sponsor: Abeona Therapeutics, Inc

New P3 trial

A Long-Term Extension Study for Participants Previously Treated With EB-101 for the Treatment of RDEB (clinicaltrials.gov) - P=N/A | N=22 | Enrolling by invitation | Sponsor: Abeona Therapeutics, Inc

New trial

Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) (clinicaltrials.gov) - P3 | N=11 | Completed | Sponsor: Abeona Therapeutics, Inc | Active, not recruiting ➔ Completed

Trial completion

"$ABEO based on the positive VIITAL topline data, plans to submit a BLA for EB-101 in Q2'23. Cash, cash equivalents $23.5M" (@BioStocks)

Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa. (PubMed, Orphanet J Rare Dis) - P1/2 | "Treatment with EB-101 appears safe and efficacious, and produces long-term improvements in wound healing, pain, and itch for RDEB patients. Results from the Phase 3 randomized controlled trial are forthcoming."

Journal • Dermatology • Gene Therapies • Genetic Disorders • Immunology • Infectious Disease • Oncology • Pain • Squamous Cell Carcinoma • Transplantation