Abeona Therapeutics® Reports Second Quarter 2024 Financial Results and Concludes Type A Meeting with FDA to Align on Upcoming Pz-cel BLA Resubmission

Core Insights - Abeona Therapeutics is making significant progress in addressing Chemistry Manufacturing and Controls (CMC) items noted in the Complete Response Letter (CRL) from the FDA, with plans to resubmit its Biologics License Application (BLA) for pz-cel in the second half of 2024 [1][2][3] Company Developments - The company completed a Type A meeting with the FDA on August 8, 2024, discussing the forthcoming BLA resubmission for pz-cel, an autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) [2] - Abeona has addressed nearly all deficiencies noted in the CRL, with ongoing validation for two remaining items related to sterility and identity assays [2] - The FDA did not request new clinical trials or data for pz-cel, indicating a focus on CMC requirements rather than clinical efficacy or safety [2] Financial Performance - As of June 30, 2024, the company reported cash, cash equivalents, short-term investments, and restricted cash totaling $123.0 million, up from $62.7 million as of March 31, 2024 [4] - Net cash used in operating activities for the second quarter of 2024 was $12.7 million, with research and development expenses at $9.2 million, compared to $8.5 million in the same period of 2023 [5][6] - The company reported a net income of $7.4 million for the second quarter of 2024, including a $24.9 million gain from the remeasurement of warrant liabilities, contrasting with a net loss of $16.7 million in the second quarter of 2023 [6] Commercial Strategy - Abeona is preparing for a potential U.S. commercial launch of pz-cel, engaging with treatment sites, payers, and building supply chain capabilities [3] - The company has also entered a non-exclusive agreement with Beacon Therapeutics to evaluate its AAV204 capsid for ophthalmology gene therapies [3] Market Position - Abeona's pz-cel is positioned as a first-in-class therapy for RDEB, with a fully integrated cGMP manufacturing facility ready to support commercial production upon FDA approval [8]