Summary of Abeona Therapeutics (ABEO) 2025 Conference Call Company Overview - Abeona Therapeutics is a commercial-stage biotech company based in Cleveland, specializing in cell and gene therapy, particularly for patients with recessive dystrophic epidermolysis bullosa (RDEB) [4][5] - The company has launched its lead product, ZivaSkin, which is an autologous cell-based gene therapy aimed at treating large, chronic wounds associated with COL7 deficiency [4][5] Key Highlights - **Product Launch and Demand**: - ZivaSkin has shown strong initial demand with approximately 50 patients identified for treatment, even before a dedicated sales force was established [5][6] - The company has two qualified treatment centers (QTCs) operational, with plans to expand further [22][24] - **Patient Profile**: - The initial patient cohort includes severe cases with large, chronic wounds, many of whom are treatment naive or have previously tried other therapies [7][9][36] - An estimated 750 patients with severe and moderate wounds are eligible for ZivaSkin across 23 centers of excellence [9][10] - **Clinical Evidence**: - ZivaSkin's clinical trials have demonstrated unique efficacy in treating chronic wounds that have remained open for extended periods, with some patients experiencing wounds that have not healed for up to 21 years [12][13] - The therapy aims to provide durable wound healing and pain reduction after a single application [4][12] - **Manufacturing and Capacity**: - Initial manufacturing capacity is set at two patients per QTC per month, with plans to ramp up to 10 patients per month by mid-2026 [22][50] - The company is preparing for potential scalability in manufacturing to meet increasing demand [50][51] Market Potential and Financial Outlook - **Patient Demand and Market Potential**: - The company anticipates treating 10 to 14 patients in 2025, with a strong pipeline of referrals already in place [26][29] - The process from patient identification to treatment is expected to take about four months initially, with improvements anticipated as the process becomes more streamlined [30][32] - **Reimbursement Landscape**: - Positive trends in payer coverage have been observed, with all prior authorizations submitted so far being approved, some within 48 hours [41][42] - Approximately 60% of patients are commercially insured, with favorable coverage policies emerging from major payers like United Health [43][45] Additional Insights - **Patient Experience and Logistics**: - The treatment process involves multiple administrative steps, including insurance clearance and biopsy collection, which are expected to improve over time [17][18] - Patients are willing to travel significant distances to access treatment, indicating a strong commitment to receiving ZivaSkin [24][25] - **Future Developments**: - Abeona is also developing proprietary capsids for retinal inherited diseases, with clinical trials expected to start in late 2026 [55] - The company emphasizes the importance of execution in the current phase, with a strong balance sheet supporting its operations [54] Conclusion - Abeona Therapeutics is positioned for growth with its innovative ZivaSkin therapy, strong initial demand, and positive reimbursement trends. The company is focused on expanding its treatment capacity and enhancing patient access while preparing for future product developments in its pipeline.

Financial Data and Key Metrics Changes - As of June 30, 2025, the company reported unaudited cash, cash equivalents, short-term investments, and restricted cash of $225.9 million, a significant increase from $98.1 million as of December 31, 2024, providing over two years of operating capital without further capital infusion [15][16] - Net income for the second quarter of 2025 was $108.8 million, or $2.07 per basic share, compared to a net income of $7.4 million, or $0.19 per basic share, in the same quarter of 2024 [18] Business Line Data and Key Metrics Changes - The company has seen strong interest in ZevaSkin, with over 50 identified patients across two qualified treatment centers (QTCs) and expectations to treat 10 to 14 patients in 2025 [14][5] - Research and development expenses decreased to $5.9 million for the quarter ended June 30, 2025, from $9.2 million in the same quarter of 2024, primarily due to costs capitalized into inventory [17] Market Data and Key Metrics Changes - The company has achieved positive coverage with multiple national and regional payers, with 100% of prior authorization requests approved to date, indicating strong clinical acceptance among payers [11][12] - Approximately 60% of RDEB lives are covered by commercial plans, 30% by Medicaid, and the rest by Medicare, highlighting the market potential for ZevaSkin [10] Company Strategy and Development Direction - The company is focused on expanding its geographic footprint by activating additional QTCs to ease travel burdens for patients and increase demand for ZevaSkin [8][14] - The company plans to transition into a revenue-generating commercial entity and will provide high-level forward cost guidance alongside updates on commercialization progress [16] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch progress of ZevaSkin, citing positive feedback from the RDEB community and the potential to improve patient lives [4][14] - The company anticipates that the first ZevaSkin patient treatment will occur in 2025, leading to projected company-wide profitability in early 2026 [16] Other Important Information - The company has partnered with Deborah of America for a nationally broadcast webinar to share patient experiences, which has positively influenced community engagement [13] - The company is exploring opportunities for EU and Japan market entries, considering the feasibility of supplying from its Cleveland facility [77] Q&A Session Summary Question: How are identified patients defined? - Identified patients are those with severe wounds that have never healed, prioritized by physicians for treatment with ZevaSkin [24][25] Question: What is the treatment timeline for patients outside the two QTCs? - Patients have started the referral process and are going through initial consultations, with plans to activate additional treatment centers to facilitate their treatment [26][27] Question: How many cases does a center need to treat before adopting ZevaSkin as routine therapy? - There is no specific number of pre-treatments required; centers are confident in treating patients based on their experience with RDEB patients [33][36] Question: What are the payment mechanics for ZevaSkin? - Revenue is recognized after a patient has been treated with ZevaSkin, with hospitals able to secure payer agreements before placing orders [49][51] Question: What is the status of prior authorizations? - The company has seen a 100% approval rate for prior authorizations, with no pushback from payers regarding prior treatments before approving ZevaSkin [58][55] Question: What are the production capacity plans? - The company is on track to ramp up production capacity to treat 10 patients per month by mid-2026, with no anticipated FDA inspections affecting this timeline [64][68] Question: What are the expectations for the EU market? - The company is exploring the EU market but acknowledges that establishing a manufacturing footprint there would take significant time [77]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024 [19] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [20] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [20] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [21] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [5][6] - The first Qualified Treatment Center (QTC) was activated ahead of schedule, with Lurie Children's Hospital of Chicago being the first to offer ZivaSkin [8] - The company anticipates treating the first patient at Lurie in Q3 2025, with a goal of treating 10 to 14 patients by the end of 2025 [10][16] Market Data and Key Metrics Changes - The company has executed outcomes-based agreements with two payer contracting organizations to ensure access to ZivaSkin for patients [18] - The inbound interest from patients and families has been significant, with approximately 30 inquiries received in the first two weeks post-approval [12][13] Company Strategy and Development Direction - The company aims to achieve profitability driven by ZivaSkin revenues by early 2026, supported by the sale of a Priority Review Voucher (PRV) for $155 million [11][19] - The company plans to activate additional QTCs by the end of 2025, focusing on well-recognized institutions with experience in cell and gene therapy [14][15] - The company is also preparing to advance its ophthalmology pipeline, with plans to bring an asset into clinical trials in the second half of 2026 [45][46] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [12][16] - The management highlighted the importance of payer agreements to ensure timely access to ZivaSkin for patients [18] - The company is confident in its financial position, stating that the proceeds from the PRV sale will fund operations for over two years [19] Other Important Information - The company presented two late-breaking abstracts at the Society for Investigative Dermatology Annual Meeting, reinforcing ZivaSkin's efficacy and safety profile [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [19] Q&A Session Summary Question: What does site activation mean? - Site activation includes training staff and surgeons, allowing them to identify patients for ZivaSkin and begin the payer authorization process [25][27] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen RDEB patients treated at Lurie, with potential for more referrals from the local community [28][29] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [31][32] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be around two patients per month per site, with potential to increase as centers gain experience [41][42] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be approximately $152 million [58] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [78][80]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company had cash, cash equivalents, short-term investments, and restricted cash of $84.5 million, down from $98.1 million as of December 31, 2024, before accounting for the proceeds from the Priority Review Voucher (PRV) sale [17] - Research and development expenses increased to $9.9 million for Q1 2025 from $7.2 million in Q1 2024, primarily due to increased headcount for manufacturing scale-up [18] - General and administrative expenses rose to $9.8 million for Q1 2025 from $7.1 million in Q1 2024, also due to increased headcount related to the ZivaSkin launch [18] - The net loss for Q1 2025 was $12 million, or $0.24 loss per share, compared to a net loss of $31.6 million, or $1.16 loss per share, in Q1 2024 [19] Business Line Data and Key Metrics Changes - The company launched ZivaSkin, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), following FDA approval [4][5] - The first Qualified Treatment Center (QTC) activated was Lurie Children's Hospital of Chicago, which is expected to treat the first patient in Q3 2025 [6][8] Market Data and Key Metrics Changes - The company has received inquiries from approximately 30 patients and families across the country since ZivaSkin's approval, indicating strong demand [10] - The company is actively engaging with commercial payers and state Medicaid programs to expand access to ZivaSkin [16] Company Strategy and Development Direction - The company aims to treat 10 to 14 patients with ZivaSkin in 2025 and anticipates a robust start to 2026 [13] - The company has entered into an agreement to sell the PRV for $155 million, which will strengthen its balance sheet and fund operations for over two years [9][17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the initial launch of ZivaSkin and the positive response from patients and physicians [10][13] - The company is focused on ensuring that the value of ZivaSkin is recognized by payers and that patients have timely access to the therapy [16] Other Important Information - The company expects to achieve profitability driven by ZivaSkin revenues in early 2026 [17] - The company is on track to activate additional QTCs by the end of 2025, with ongoing discussions with recognized institutions [12] Q&A Session Summary Question: What does site activation mean? - Site activation means that staff and surgeons are trained and ready to identify patients for ZivaSkin treatment [22][25] Question: How many RDEB patients are eligible at Lurie Children's Hospital? - There are at least a couple of dozen patients treated at Lurie, with potential for more referrals from the local community [26] Question: What factors affect patient access to treatment? - Commercially insured patients are expected to have faster access compared to Medicaid patients, which may take longer [29] Question: What is the expected patient throughput at QTCs? - Initial throughput is expected to be two patients per month per site, with potential to ramp up to four patients per month at some centers [39] Question: What are the anticipated net proceeds from the PRV sale? - The net proceeds from the PRV sale are expected to be about $152 million [54] Question: How will billing and reimbursement work? - Revenue recognition will occur after patient treatment, with discussions already in place with insurance companies to ensure reimbursement [72][75]