Release Date: November 14, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Abeona Therapeutics Inc (ABEO, Financial) successfully resubmitted their Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to the FDA, which was accepted for review with a PDUFA date set for April 29, 2025.
- The FDA did not request any new clinical trials or data for the approval of pz-cel, indicating confidence in the existing clinical efficacy and safety data.
- Abeona Therapeutics Inc (ABEO) has secured new patents for pz-cel, extending intellectual property protection until 2040, which underscores the innovative nature of their technology.
- The company has made significant progress in commercial launch preparations, including building a strong commercialization team and engaging with key treatment centers across the US.
- Abeona Therapeutics Inc (ABEO) has a solid financial position with $110 million in cash and equivalents, providing sufficient resources to fund operations into 2026.
Negative Points
- The company received a Complete Response Letter (CRL) from the FDA in April, highlighting the need for additional CMC data, which delayed the approval process.
- There is a potential supply constraint at launch, as the current manufacturing capacity may not meet the anticipated demand for pz-cel.
- The company faces competition from existing treatments like Vyjuvek and Filsuvez, which could impact market penetration and adoption of pz-cel.
- Abeona Therapeutics Inc (ABEO) reported a net loss of $30.3 million for the third quarter of 2024, partly due to a $15.2 million loss from the remeasurement of warrant and derivative liabilities.
- The expansion of manufacturing capacity involves regulatory reviews and could take 18 to 24 months, potentially limiting the ability to scale up production quickly.
Q & A Highlights
Q: Given the CRL was primarily CMC related, which aspect do you find most concerning going forward?
A: Vishwas Seshadri, CEO, explained that the main focus is on identity testing and Stargardt, which were discussed comprehensively with the FDA. Brian Kevany, CTO, added that Stargardt is a significant topic for the FDA, but they are confident in the data and package submitted.
Q: How are you strategizing the commercial rollout considering existing treatments like Vyjuvek and Filsuvez?
A: Madhav Vasanthavada, CCO, stated that market research shows patients are motivated by pz-cel's wound healing profile. They plan to target high-volume EB centers and leverage patient advocacy and referrals to drive demand. Payer discussions have not shown pushback against pz-cel.
Q: How does the favorable Medicare reimbursement designation impact commercial reimbursement plans?
A: Madhav Vasanthavada noted that while the Medicare DRG 018 is specific to Medicare patients, it positively reflects on the technology's value to commercial payers. The ICD-10-PCS code will aid seamless hospital billing for commercially insured patients.
Q: Can you provide insight into how many patients you could treat commercially by the end of 2025?
A: Madhav Vasanthavada mentioned that they aim to treat up to 120 patients annually with current capacity, but plan to expand. Vishwas Seshadri added that they are working on increasing manufacturing capacity, with more clarity expected in future updates.
Q: How will you address potential concerns from surgeons about the surgical procedure for pz-cel?
A: Vishwas Seshadri assured that surgeons do not see the procedure as complex beyond typical plastic surgery. Madhav Vasanthavada added that cell therapy coordinators are familiar with such procedures, and ongoing trials are building experience.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
