Abeona Therapeutics(ABEO) - 2023 Q3 - Earnings Call Transcript

Financial Data and Key Metrics Changes - As of September 30, 2023, the company had cash, cash equivalents, restricted cash, and short-term investments totaling $54.1 million, an increase from $37.1 million as of June 30, 2023, primarily due to a $25 million gross proceeds from a registered direct offering in July 2023 [18] - Research and development expenses rose to $7.1 million for Q3 2023, compared to $5.5 million in Q3 2022, while general and administrative expenses increased to $4.2 million from $3.9 million in the same period [19] - The net loss attributable to common shareholders was $11.8 million for Q3 2023, or $0.48 loss per share, compared to a net loss of $6.4 million or $1 loss per share in Q3 2022 [19] Business Line Data and Key Metrics Changes - The company is focusing on the commercialization of pz-cel, an investigational therapy for recessive dystrophic epidermolysis bullosa (RDEB), with plans to supply up to 120 patient treatments in the first full year post-launch [10][12] - The company is onboarding 5 to 7 high-volume EB treatment centers to facilitate access to pz-cel upon approval [12] Market Data and Key Metrics Changes - The FDA's decision on the BLA review process for pz-cel is expected by late November 2023, with a potential approval in Q2 2024 if a priority review is granted [8][9] - The company anticipates a peak sales estimate of approximately $500 million over a five-year period, based on the assumption of treating around 500 patients annually [32] Company Strategy and Development Direction - The company aims to transition from a clinical-stage to a commercial-stage organization with the launch of pz-cel, leveraging the leadership team's previous experiences with autologous therapies [10][11] - The strategy includes engaging with payers to ensure broad access and favorable pricing for pz-cel, which is expected to provide significant value as a transformative therapy [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the potential impact of pz-cel on the RDEB community, highlighting strong clinical data and positive feedback from the medical community [20] - The company is preparing for a commercial launch while ensuring that its financial resources are sufficient to support operations into Q4 2024 [18] Other Important Information - The company has received rare pediatric disease designation from the FDA for pz-cel, which may allow it to obtain a priority review voucher valued at approximately $100 million upon approval [9] - The management team has been actively filling commercial roles to support the launch, including key account management and market access positions [17] Q&A Session Summary Question: Can you elaborate on patient identification efforts and involvement of prior trial participants? - Management indicated that they are identifying patients from previous studies and ongoing trials, which is encouraging for launch preparations [24][25] Question: How quickly can pz-cel be commercialized post-approval? - The estimated timeline for commercialization is about 1 to 2 months post-approval, focusing on establishing medical policies and treatment center readiness [29] Question: What assumptions underlie the peak sales estimate of $500 million? - The estimate is based on the ability to treat 500 patients annually over five years, with a pricing assumption in the seven-digit range [32] Question: Will the company reach out to community doctors for initial treatments? - The company plans to start with centers of excellence and may expand to community centers over time, ensuring education and awareness of pz-cel [35][36] Question: What is the potential for pz-cel to be used in younger patients? - Management is in discussions with the FDA regarding the potential to treat patients younger than six years old, based on ongoing studies [39][40] Question: Has the company started hiring for the sales team? - The company has successfully filled key commercial roles and does not anticipate a large sales team at launch due to existing interest from treatment centers [43][44] Question: What are the takeaways from Krystal Biotech's recent launch? - Management views the uptake of competing products positively, as it raises awareness and prepares the market for pz-cel [46][47]