Abeona Therapeutics(ABEO) - 2025 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of September 30, 2025, the company reported cash, cash equivalents, restricted cash, and short-term investments totaling $207.5 million, providing significant financial flexibility for operations over the next two years [13][14] - Research and development (R&D) spending for Q3 2025 was $4.2 million, a decrease from $8.9 million in Q3 2024, primarily due to costs capitalized into inventory [14] - Selling, general and administrative (SG&A) expenses increased to $19.3 million in Q3 2025 from $6.4 million in Q3 2024, reflecting the reclassification of R&D expenses and increased costs associated with the commercial launch [15] - The net loss for Q3 2025 was $5.2 million, or negative $0.10 per share, compared to a net loss of $30.3 million, or negative $0.63 per share, in Q3 2024 [15] Business Line Data and Key Metrics Changes - The company is focused on the commercial launch of ZEVASKYN, the first autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), with growing patient demand and an expanding network of qualified treatment centers (QTCs) [4][10] - The number of identified eligible patients at QTCs has more than doubled to approximately 30, up from over 12 previously reported [10] - The activation of Children's Hospital Colorado as a QTC brings the total to three, alongside Lurie Children's Hospital of Chicago and Lucille Packard Children's Hospital Stanford [11] Market Data and Key Metrics Changes - ZEVASKYN has received coverage decisions from all major commercial payers, covering over 80% of commercially insured lives, and has baseline coverage across all 51 state Medicaid programs effective October 1, 2025 [12] - A permanent product J-code for ZEVASKYN will be established by CMS effective January 1, 2026, simplifying claims and reimbursement processing [12] Company Strategy and Development Direction - The company aims to achieve its 2026 launch goals for ZEVASKYN despite a temporary delay in the first patient treatment, focusing on expanding treatment center networks and ensuring market access [4][5] - The company is also advancing its pipeline, with a gene therapy program for X-linked retinoschisis selected for the FDA Rare Disease Endpoint Advancement pilot program [17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to meet 2026 launch goals based on trends in patient demand and treatment center expansion, despite the delay in the first patient treatment [4][5] - The management team highlighted the importance of maintaining high-quality standards in manufacturing and the proactive measures taken to optimize release assays [6][7] Other Important Information - The company has strengthened its management team with the appointment of Dr. James A. Gao as Senior Vice President, Head of Clinical Development and Medical Affairs, bringing over 20 years of industry experience [17] Q&A Session Summary Question: Expected timeline for patients to receive treatment after ZEVASKYN Product Order Forms - Management indicated that many patients have already received prior authorizations and are scheduled for biopsies in November and early 2026, with treatment expected to follow if all paperwork is completed [20][22] Question: Impact of treatment delays on profitability timeline - Management stated that the timeline for achieving profitability remains unchanged, with expectations for profitability in the first half of 2026 [26][28] Question: Routine maintenance shutdown of the plant - Management confirmed a mandated FDA-required shutdown for general maintenance from mid-December to early January [29][30] Question: Status of biopsies collected before optimization pause - Management clarified that no biopsies were collected during the pause to ensure patient safety and product quality [31] Question: Lead time for ZEVASKYN Product Order Forms - Management expects the lead time to decrease as more patients progress through the treatment process, currently estimated at about three months [34] Question: Attrition rate among patients with ZEVASKYN Product Order Forms - Management expressed confidence in a high conversion rate for patients with ZPOFs, as these are motivated patients eager to proceed with treatment [40][41] Question: Prior authorization process for RDEB patients - Management described the prior authorization process as involving clinical and financial discussions, with most payers following inclusion/exclusion criteria from clinical trials [44][46]