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01/09 21:21
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Access earnings results, analyst expectations, report, slides, earnings call, and transcript.
The earnings call presents a positive outlook with reduced net loss, progress in market access, and strategic expansion plans. The Q&A section confirms no significant impact on profitability timelines despite delays, maintaining a positive sentiment. The company's progress in securing insurance coverage and expanding treatment centers suggests strong future growth potential. Despite some uncertainties in patient timelines and QTC partnerships, overall sentiment remains positive, supported by optimistic guidance and a clear path to profitability by 2026. The absence of a market cap suggests a neutral to positive reaction, likely around 2% to 8%.
Cash, cash equivalents, restricted cash, and short-term investments $207.5 million as of September 30, 2025. This robust cash position provides significant financial flexibility for the ZEVASKYN commercial launch. The current cash position is expected to fund operations for over 2 years without accounting for anticipated revenue from ZEVASKYN.
Research and Development (R&D) spending $4.2 million for the 3 months ended September 30, 2025, compared to $8.9 million for the same period of 2024. This reduction was primarily due to costs capitalized into inventory and the reclassification of selected costs to selling, general, and administrative expense (SG&A) following ZEVASKYN's FDA approval.
Selling, General, and Administrative (SG&A) expense $19.3 million for the 3 months ended September 30, 2025, compared to $6.4 million for the same period of 2024. This increase reflects the reclassification of R&D expenses, increased headcount, and professional costs associated with the commercial launch of ZEVASKYN.
Net loss $5.2 million for the third quarter of 2025 or negative $0.10 per basic and diluted common share, compared to a net loss of $30.3 million in the third quarter of 2024 or negative $0.63 per basic and diluted common share. The reduction in net loss reflects the changes in R&D and SG&A expenses.
ZEVASKYN launch: ZEVASKYN, the first autologous cell-based gene therapy for RDEB, is being scaled for commercial launch. Despite a delay in the first patient treatment to Q4 2025 due to assay optimization, patient demand and market access trends remain strong.
Pipeline updates: The AB0503 gene therapy program for X-linked retinoschisis has been selected for the FDA RDEA pilot program, enhancing communication with the FDA for endpoint development. Additionally, Dr. James A. Gow has been appointed as SVP, Head of Clinical Development and Medical Affairs.
Market access for ZEVASKYN: ZEVASKYN has achieved broad market access with coverage from major commercial payers (e.g., UnitedHealthcare, Cigna, Aetna) covering 80% of commercially insured lives. Medicaid baseline coverage is effective across all 51 states and Puerto Rico as of October 1, 2025.
Qualified Treatment Centers (QTCs): The network has expanded to three centers, including Children's Hospital Colorado, with ongoing discussions to further expand geographically.
Manufacturing and quality control: A batch of ZEVASKYN was rejected due to a false positive in a rapid sterility assay. The assay was optimized, validated, and regulatory submissions were completed, allowing biopsy collection to resume in November 2025.
Financial position: Cash and equivalents totaled $207.5 million as of September 30, 2025, sufficient to fund operations for over two years. R&D spending decreased due to reclassification of costs post-FDA approval, while SG&A expenses increased due to the commercial launch.
Strategic expansion: Efforts are underway to expand the QTC network and increase patient referrals from non-QTC sites. Promotional activities are driving awareness and patient identification.
Leadership enhancement: Dr. James A. Gow's appointment strengthens the management team, particularly in gene therapy and ophthalmology expertise.
Delay in First Patient Treatment: The first patient treatment for ZEVASKYN was delayed to the fourth quarter of 2025 due to the need to optimize a product release assay. This delay could impact the company's ability to meet its 2026 launch goals.
Manufacturing and Quality Control Issues: A full batch of drug product was rejected due to a false positive result in a rapid sterility assay, which was an FDA requirement. This issue led to a temporary pause in patient biopsy collection and required additional testing and regulatory submissions, potentially affecting operational timelines.
Regulatory Hurdles: The rapid sterility assay, added as an FDA requirement during the BLA review, caused complications in the manufacturing process, highlighting challenges in meeting regulatory standards.
Financial Risks: Increased SG&A expenses due to the commercial launch of ZEVASKYN and reclassification of R&D costs could strain financial resources, despite a robust cash position.
Dependence on Qualified Treatment Centers (QTCs): The company’s commercial success is heavily reliant on the activation and performance of QTCs. Currently, only three centers are activated, and further expansion is required to meet patient demand and geographic coverage.
Insurance and Financial Clearance Delays: Although insurance prior authorizations have been obtained for several patients, full financial clearance is still pending for some, which could delay patient treatments and revenue generation.
ZEVASKYN Commercial Launch: The company remains committed to achieving its 2026 launch goals despite a temporary delay in the first patient treatment. Patient demand is growing, and treatment center expansion is progressing. The first patient treatment is now anticipated in Q4 2025.
Qualified Treatment Centers (QTC) Expansion: The company has activated its third QTC, Children's Hospital Colorado, and is in discussions with additional EB centers to expand its geographic footprint. New centers will be announced as they are activated.
Patient Demand and Access: The number of identified eligible patients has doubled to approximately 30. Insurance prior authorizations have been obtained for several patients, and biopsies are expected to proceed in the coming months as financial clearance is achieved.
Market Access and Reimbursement: ZEVASKYN has received baseline coverage across all 51 state Medicaid programs and major commercial payers, covering over 80% of commercially insured lives. A permanent product J-code will be effective January 1, 2026, simplifying claims and reimbursement processing.
Pipeline Development: The AB0503 gene therapy program for X-linked retinoschisis has been selected for the FDA Rare Disease Endpoint Advancement pilot program, which aims to accelerate development and validation of novel efficacy endpoints.
The selected topic was not discussed during the call.
The earnings call presents a positive outlook with reduced net loss, progress in market access, and strategic expansion plans. The Q&A section confirms no significant impact on profitability timelines despite delays, maintaining a positive sentiment. The company's progress in securing insurance coverage and expanding treatment centers suggests strong future growth potential. Despite some uncertainties in patient timelines and QTC partnerships, overall sentiment remains positive, supported by optimistic guidance and a clear path to profitability by 2026. The absence of a market cap suggests a neutral to positive reaction, likely around 2% to 8%.
The earnings call indicates strong financial performance with a significant increase in net income driven by strategic asset sales. The Q&A section reveals positive developments, including successful patient advocacy, strong demand for ZEVASKYN, and effective partnerships. Despite some uncertainties in management responses, the overall sentiment is positive, with optimistic guidance and plans for expansion. The company's ability to meet production goals and achieve profitability in the near future further supports a positive stock price movement.
The earnings call reveals positive developments, such as the PRV sale securing funding for two years, launch preparations for ZEVASKYN, and improved financial metrics with reduced net loss. The partnership with Ultragenyx and revenue potential of pz-cel also add to the optimism. However, uncertainties in patient numbers and cash position projections slightly temper enthusiasm. Overall, the strong funding position and strategic partnerships suggest a positive stock price movement.
The earnings call reveals several positive aspects: the strategic partnership with Ultragenyx, a strong revenue potential for pz-cel, and a significant cash infusion from the PRV sale, which extends the funding runway. The reduced net loss and improved financial metrics further enhance the outlook. Despite some uncertainties in patient eligibility numbers and cash projections, the overall sentiment is positive, driven by strategic partnerships, financial improvements, and the upcoming launch of ZEVASKYN, indicating a likely stock price increase in the short term.
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