Release Date: August 12, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Abeona Therapeutics Inc (ABEO, Financial) has made significant progress in addressing the complete response letter from the FDA, with nearly all issues resolved and alignment on the BLA resubmission for pz-cel.
- The company successfully completed a $175 million underwritten offering, strengthening its balance sheet and extending its cash runway into 2026.
- Positive feedback from healthcare professionals and payers on pz-cel's potential to address unmet needs in RDEB, with enthusiasm for its differentiated profile.
- Abeona Therapeutics Inc (ABEO) has a nonexclusive agreement with Beacon Therapeutics to evaluate its patented AAV204 capsid for gene therapies in ophthalmology, highlighting potential for future collaborations.
- The company reported a net income of $7.4 million for Q2 2024, including a significant gain from the remeasurement of warrant liabilities, indicating improved financial performance.
Negative Points
- Two outstanding items related to sterility and identity assays still require validation, which could delay the BLA resubmission timeline.
- Increased general and administrative expenses, primarily due to commercial and launch preparation costs, impacting overall financials.
- The company is not providing specific guidance on the exact timing of the BLA resubmission, creating uncertainty for investors.
- Potential competition from existing products like Vyjuvek and Filsuvez in the RDEB space, which could impact market penetration and sales of pz-cel.
- The PRV market has shown some signs of softening, which could affect the potential revenue from the sale of a Priority Review Voucher if awarded.
Q & A Highlights
Q: For the two remaining outstanding items related to sterility assays and identity assays, can you say more about what the FDA feedback was? Are they asking for a new experiment or replication?
A: Vishwas Seshadri, CEO: The FDA's feedback was primarily about the statistical approach for establishing comparability between the current gold standard and our developed method. It wasn't about developing a new experimental method. For the identity assay, we've started validation work and have alignment with the FDA on our approach.
Q: Did the FDA provide feedback on the retroviral replication assay? Is that one good to go?
A: Vishwas Seshadri, CEO: Yes, the retroviral replication assay is good to go. We have received confirmation from the FDA that the data we've generated addresses the need for the RCR assay.
Q: Will you need another formal meeting with the FDA? Any more granularity on when in the second half the resubmission will occur?
A: Vishwas Seshadri, CEO: We do not plan to have any more formal meetings with the FDA. We are not guiding exactly when in the second half the resubmission will occur, as it depends on the completion of validation runs. We remain on track for a second-half submission.
Q: What is the statutory timing with which the FDA would need to respond to the BLA resubmission once it is filed and to assign a PDUFA date?
A: Vishwas Seshadri, CEO: Upon resubmission, the FDA indicates their acceptance within two weeks and determines whether it's a Type 1 or Type 2 resubmission. We anticipate a Type 2, which would set a PDUFA date six months from resubmission.
Q: On the pricing and reimbursement, have you had discussions about funding both Vyjuvek and pz-cel?
A: Madhav Vasanthavada, Chief Commercial Officer: Payers understand the distinct profiles of Vyjuvek and pz-cel. We haven't heard objections to funding both, as the modalities are distinct. Payers recognize the clinical value of pz-cel for large and chronic wounds.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
