Abeona Therapeutics(ABEO) GlobeNewswire News Room·2024-11-14 12:30Core Viewpoint - The FDA has accepted the Biologics License Application (BLA) resubmission for pz-cel, targeting a PDUFA action date of April 29, 2025, indicating significant progress towards potential commercialization of the therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2][3]. Company Progress - Abeona Therapeutics has made strides in commercial readiness for pz-cel, including discussions with payors and onboarding treatment centers [2][3]. - The company completed a Type A meeting with the FDA in August 2024, aligning on the BLA resubmission content and addressing Chemistry Manufacturing and Controls (CMC) issues noted in a previous Complete Response Letter [3]. - The Centers for Medicare and Medicaid Services (CMS) granted a product-specific procedure code for pz-cel and assigned it to a high reimbursement category, facilitating hospital billing and patient access [3]. - Abeona has expanded its manufacturing capacity with a new facility lease in Cleveland, Ohio, and received new patents related to pz-cel, with the '504 Patent expiring on January 3, 2037 [3][4]. Financial Results - As of September 30, 2024, the company reported cash and cash equivalents totaling 123.0 million as of June 30, 2024 [4]. - Research and development expenses for Q3 2024 were 7.1 million in Q3 2023, while general and administrative expenses rose to 4.2 million in the same period [6]. - The net loss for Q3 2024 was 11.8 million in Q3 2023 [6][10]. Pipeline and Partnerships - In July 2024, Abeona entered a non-exclusive agreement with Beacon Therapeutics to evaluate its AAV204 capsid for potential use in ophthalmology gene therapies [3]. - Ultragenyx participated in a pre-BLA meeting with the FDA regarding its partnered program UX111 AAV gene therapy for Sanfilippo syndrome type A, expected to be filed by the end of 2024 [3].