ABEONA THERAPEUTICS INC. Q4 FY2024 earnings call

• Vish Seshadri highlighted the PDUFA date for prademagene zamikeracel (pz-cel) on April 29, 2025, ongoing FDA review since resubmission in late October 2024, receipt of a marked-up draft USPI on March 14, 2025, and launch preparations with 5 U.S. treatment centers for pz-cel. Also mentioned the partnered program with Ultragenyx for Sanfilippo syndrome type A, where Ultragenyx submitted a BLA and received a priority review with a PDUFA date of August 18, 2025. • Madhav Vasanthavada discussed the commercial opportunity for pz-cel in the U.S., estimating approximately 1,500 treatment opportunities, patient enthusiasm based on clinical trial results (e.g., 100% of Phase 1/2a study participants would undergo pz-cel again), launch readiness with 5 treatment centers undergoing onboarding, market access focus including payer education and reimbursement strategies, and the Abeona Assist program for patient support. • Brian Kevany detailed the manufacturing capacity at the CGMP facility in Cleveland, with initial capacity of 4 treatments per month, ramping to 10 treatments per month by the first half of 2026, and a planned annual maintenance shutdown. • Joe Vazzano provided financial results: cash, cash equivalents, short-term investments, and restricted cash totaled $98.1 million as of December 31, 2024; R&D expenses were $34.4 million, general and administrative expenses were $29.9 million, net loss was $63.7 million, and revenue recognition occurs upon pz-cel administration.

No specific product segment financial performance with revenue contribution percentages provided in the transcript.

• Anticipate launching pz-cel in the third quarter of 2025 if approved by the FDA. • Plan to ramp manufacturing capacity from 4 treatments per month initially to 10 treatments per month by the first half of 2026. • Estimate sufficient cash resources to fund operations into 2026.

• Potential supply constraints during the manufacturing ramp-up phase. • Uncertainties related to the FDA approval process and any additional requirements that may arise.

Q: Considering discussions with the FDA, do you feel the FDA is satisfied with the CMC work that led to the CRL last year?

A: We addressed all the FDA's asks from the CRL, had multiple touch points with the FDA, and believe we've addressed all items, though the review is still ongoing.

Q: Of the five centers mentioned, have you had conversations about the percent of their patients that may be eligible for pz-cel over time?

A: These centers are large with patients, and we'll get more detailed post-approval, with at least a triple-digit number of eligible patients anticipated.

Q: On the label draft received from the FDA, is it in line with your vision for pz-cel?

A: The draft label is aligned with our vision, with details to be refined through back-and-forth discussions with the FDA.

Q: Do you anticipate a patient backlog until manufacturing capacity is maximized?

A: Yes, we expect a patient backlog as we ramp up manufacturing capacity to 10 treatments per month.

Q: What is the status of physician education needed for pz-cel launch?

A: Physician education is needed, targeting qualified treatment centers initially, with further outreach to other physicians and the community post-approval.

Q: What are the prospects for the PRV and recent trends in PRV resales?

A: We expect to be eligible for a PRV as we meet eligibility criteria, and our goal is to optimize pricing, with uncertainty in PRV sale trends but intent to maximize value.

Q: What are your plans for deploying pz-cel outside the U.S.?

A: There is interest in Europe and Asia, and we will explore deployment after U.S. approval, considering market access and reimbursement landscapes.

Q: What is the ultimate goal for the total number of treatment centers?

A: Anticipate up to 10 treatment centers, starting with 5 this year and ramping up gradually.

Q: What is the maximum service area that pz-cel can treat at one time?

A: Up to 480-centimeter square with 12 sheets, estimating approximately 2 treatment cycles per patient on average.