Abeona Therapeutics(ABEO) - 2023 Q4 - Annual Report

Regulatory Submissions and Approvals - In September 2023, the company submitted a Biologics License Application (BLA) for pz-cel to the FDA, which was accepted for filing and granted priority review in November 2023, with a target action date set for May 25, 2024[10][27]. - Pz-cel has been granted multiple designations by the FDA, including Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy, which may provide benefits such as market exclusivity and expedited review[28][27]. - The FDA requires completion of preclinical laboratory tests and in vivo studies in accordance with current Good Laboratory Practice (GLP) regulations before marketing a biologic product candidate[59]. - An Investigational New Drug (IND) application must be submitted to the FDA, which becomes effective 30 days after receipt unless the FDA places the trial on clinical hold[62]. - The FDA may approve a Biologics License Application (BLA) based on a single Phase 3 clinical study under certain circumstances[68]. - The FDA may require a Risk Evaluation and Mitigation Strategy (REMS) for products to ensure safe use, which must be submitted with the BLA if deemed necessary[89]. - The FDA's Fast Track program allows for rolling reviews of BLAs, enabling earlier interactions and review of sections before the application is complete[94]. - Breakthrough therapy designation provides intensive guidance and may lead to priority review for products showing substantial improvement over existing therapies[95]. - Accelerated approval can be granted based on surrogate endpoints, but requires post-marketing studies to confirm clinical benefits[97]. - The FDA conducts inspections of manufacturing facilities and clinical sites to ensure compliance with regulatory standards before approving a BLA[87]. Clinical Development and Trials - The pivotal phase 3 VIITAL™ study demonstrated statistically significant improvements in wound healing and pain reduction for pz-cel in RDEB patients, with no serious treatment-related adverse events reported[25][24]. - Human clinical trials are conducted in three phases, with Phase 3 requiring two adequate and well-controlled trials for FDA approval[67][68]. - Post-approval clinical trials (Phase IV) may be required by the FDA to gain additional experience regarding long-term safety[70]. Manufacturing and Facilities - The company has established current Good Manufacturing Practice (cGMP) clinical-scale manufacturing capabilities in Cleveland, Ohio, which provide advantages in flexibility, scale, reliability, and reduced development risk[14]. - The company has established a cGMP manufacturing facility in Cleveland, Ohio, enhancing supply chain control and increasing supply capacity for clinical trials and commercial demand[38]. - The manufacturing facility has completed a 16,000+ square foot build-out, including a 6,000 square foot cGMP production facility and an additional 8,000 square feet of laboratory space[40]. - The company has produced three cGMP process validation lots for the retroviral vector used in pz-cel, supporting licensure and commercial manufacturing[41]. - The company has filed a Biologics License Application (BLA) to support commercial manufacturing of pz-cel from its Cleveland facility[44]. Market Potential and Financial Aspects - The incidence of RDEB is estimated to be between 0.2 to 3.05 per million births, with a potential prevalence of up to 3,850 patients in the U.S. who may benefit from COL7A1-mediated treatments like pz-cel[18]. - The annual cost of wound care for dystrophic epidermolysis bullosa (DEB) patients is identified to be 465% greater than the average annual healthcare cost, with wound care supplies potentially costing up to $996,000 per year[21]. - The company anticipates that healthcare reform measures may significantly affect the sales of its products, particularly regarding pricing and reimbursement policies[138]. - The company is exposed to risks related to coverage and reimbursement from third-party payors, which are increasingly reducing reimbursements for medical products[135][136]. Intellectual Property and Licensing - The company has licensed a patent family from Stanford University covering pz-cel, with patents expected to expire in early 2037, and additional applications pending in the U.S.[48]. - The AIM™ capsids licensed from UNC have a projected expiration date of November 6, 2036, with strong potential for delivering therapeutic transgenes[49]. - The company is actively seeking U.S. and international patent protection for various technologies, including AAV capsids and methods for treating genetic diseases[47]. Strategic Partnerships and Development - The company plans to leverage strategic partnerships and in-licensing to establish additional cell and gene therapy franchises and expand its intellectual property portfolio[15][16]. - The company aims to commercialize its assets either independently or through strategic partnerships, contingent upon FDA approval[12]. Competition and Market Environment - The company faces intense competition in the biotechnology sector, with competitors potentially having greater resources and experience[141][144]. - The company is focused on developing cell and gene therapies for life-threatening diseases, competing for skilled personnel in the pharmaceutical and biotechnology sectors[147]. Compliance and Regulatory Environment - The company is subject to significant regulatory scrutiny, including compliance with the federal False Claims Act and the Physician Payments Sunshine Act[132][134]. - Compliance with foreign regulatory requirements is critical, as failure can lead to fines, product recalls, and other legal consequences[120]. - The federal Anti-Kickback Statute prohibits remuneration for referrals or recommendations of pharmaceutical products, impacting marketing and distribution strategies[129]. - Manufacturers must comply with current Good Manufacturing Practice (cGMP) regulations and register their establishments with the FDA[75]. Workforce and Employment - The company had 84 full-time employees as of December 31, 2023, and maintains good relations with its personnel[148]. - The company has never experienced employment-related work stoppages, indicating a stable workforce[148]. - The company has contracts with scientific consultants and research organizations to support various aspects of drug development[148].