Release Date: March 20, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Abeona Therapeutics Inc (ABEO, Financial) is anticipating FDA approval for prademagene zamikeracel (pz-cel) for RDEB, with a PDUFA date set for April 29, 2025.
- The company has initiated label discussions with the FDA and received post-marketing commitments, indicating progress in the approval process.
- Abeona has identified a significant commercial opportunity for pz-cel in the US, estimating a potential revenue of over $2 billion.
- The company is preparing for a third-quarter 2025 launch of pz-cel, with five treatment centers in the US undergoing onboarding and activation.
- Abeona has a strong cash position with $98.1 million as of December 31, 2024, providing sufficient financial resources to fund operations into 2026.
Negative Points
- The launch of pz-cel is expected to be supply-constrained initially, with manufacturing capacity ramping up gradually.
- There is a potential backlog of patients due to limited initial manufacturing capacity, which could delay treatment for some patients.
- The company faces challenges in educating physicians and patients about pz-cel, as it is a new treatment option requiring extensive planning and coordination.
- Abeona's general and administrative expenses increased significantly in 2024 due to commercial launch preparation costs.
- The company has not yet finalized plans for international expansion, indicating potential delays in accessing markets outside the US.
Q & A Highlights
Q: Considering where you are in discussions with the FDA, do you feel that the agency is satisfied with the work you've done on the CMC side that resulted in the CRL last year?
A: Vishwas Seshadri, CEO: We have addressed all the asks from the FDA that came out from the CRL. We comprehensively addressed each of the 12 items and believe we've done that. We've had multiple informal meetings and a formal Type A meeting with the FDA, and based on the holistic picture, we feel like we're in a good place.
Q: Of the five centers you referenced, have you had conversations with them to get an early sense of the percent of their patients that may be eligible for this therapy over time?
A: Madhav Vasanthavada, Chief Commercial Officer: These centers are large institutions with patients who see pz-cel as a good option for moderate to severe RDEB patients. About 30% of the 750 patients are in seven centers of excellence, five of which we are in discussions with. We are looking at a triple-digit number of patients within these institutions.
Q: What is the top reason for patients wanting to seek this therapy?
A: Vishwas Seshadri, CEO: The key reason is the need for durable or longer-term closure of wounds. This treatment can minimize infections and reduce the need for bandage changes, significantly impacting quality of life. Squamous cell carcinoma is also a significant concern, and treating chronic wounds can help mitigate this risk.
Q: Is the draft label you received from the FDA in line with your vision for pz-cel?
A: Vishwas Seshadri, CEO: Yes, the draft label is consistent with our expectations. There will be routine refinements, but there are no big surprises in terms of the major items.
Q: Upon approval, do you expect a patient backlog until you reach maximum manufacturing capacity?
A: Madhav Vasanthavada, Chief Commercial Officer: We do anticipate a backlog. Early on, the launch will be supply-gated as we ramp up to 10 patients a month. We have received interest from physicians identifying patients, and we expect a queue of patients as we release manufacturing slots.
Q: Can you comment on the recent environment for PRV resales?
A: Vishwas Seshadri, CEO: Recent PRV sales have been north of $150 million. Our goal is to optimize pricing over speed, and we are not in a rush to sell the PRV immediately upon being granted.
Q: What is the ultimate goal for the total number of treatment centers you'd like to see one day?
A: Madhav Vasanthavada, Chief Commercial Officer: We don't anticipate going beyond 10 centers. Patients are used to traveling for specialized procedures, and we want to keep the number of centers tight to build experience and match our supply capacity.
Q: What is the maximum surface area that a patient can be treated with at one time, and what dictates that number?
A: Madhav Vasanthavada, Chief Commercial Officer: We can supply up to 12 sheets, each 40-centimeter square, totaling 480-centimeter square. Patients have, on average, 30% of their bodies wounded, requiring around 1,000-centimeter square coverage. We estimate about two treatments per patient based on this calculation.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
