Abeona Therapeutics Reports Q2 2024 Results, Concludes FDA Meeting for Pz-cel BLA Resubmission

Abeona Therapeutics Inc. has announced its financial results for Q2 2024 alongside significant corporate advancements. Vish Seshadri, the CEO of Abeona, highlighted that the company has nearly completed the necessary data generation for the Chemistry Manufacturing and Controls (CMC) deliverables, as specified in the Complete Response Letter (CRL) received in April 2024. This puts the company on track to resubmit their Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) later this year. If approved, this will provide a new treatment option for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).

On August 8, 2024, Abeona had a Type A meeting with the U.S. Food and Drug Administration (FDA) to discuss the forthcoming resubmission of its BLA for pz-cel. During this meeting and in pre-meeting communications, Abeona presented data and reports addressing nearly all deficiencies noted in the CRL and gained preliminary FDA alignment pending formal review. Two remaining items related to sterility and identity assays are still being validated under protocols that include FDA feedback. Abeona anticipates resubmitting the BLA in the second half of 2024. Upon acceptance, the FDA will likely set a Prescription Drug User Fee Act (PDUFA) action date six months from the submission date.

In April 2024, the FDA issued a CRL requiring additional CMC information for the BLA. However, the CRL did not flag any issues related to clinical efficacy or safety and did not call for new clinical trials or data. In May 2024, long-term safety data for pz-cel, with follow-up periods extending to 11 years, were presented at the Society for Investigative Dermatology (SID) Annual Meeting. Additionally, in July 2024, data on wound healing post-pz-cel treatment were showcased at the Society for Pediatric Dermatology (SPD) Annual Meeting.

Abeona is also making headway in commercial preparations for a potential U.S. launch of pz-cel. This includes engaging with epidermolysis bullosa treatment sites, conducting medical and payer engagements, and building the necessary supply chain and enterprise capabilities to support the transition to a commercial-stage company.

In pipeline developments, Abeona announced in July 2024 a non-exclusive agreement with Beacon Therapeutics to evaluate Abeona’s AAV204 capsid for potential use in gene therapies for specific ophthalmology indications.

On the corporate front, Abeona closed a $75 million underwritten securities offering in May 2024, involving both new and existing investors. Financially, the company's cash, cash equivalents, short-term investments, and restricted cash totaled $123.0 million as of June 30, 2024, up from $62.7 million as of March 31, 2024. Net cash used in operating activities was $12.7 million for the second quarter of 2024.

Abeona estimates that its current financial resources, along with a $50 million credit facility, will fund operations into 2026, excluding potential revenues from pz-cel sales or the sale of a Priority Review Voucher (PRV), if awarded by the FDA. Research and development expenses were $9.2 million for Q2 2024, compared to $8.5 million for Q2 2023. General and administrative expenses rose to $8.6 million from $5.0 million the previous year, mainly due to costs related to commercial and launch preparations. Net income for Q2 2024 was $7.4 million, bolstered by a $24.9 million gain from the remeasurement of warrant liabilities. This contrasts with a net loss of $16.7 million in Q2 2023.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for severe diseases. The company’s portfolio includes prademagene zamikeracel (pz-cel), an autologous cell-based gene therapy for RDEB, and AAV-based gene therapies for ophthalmic diseases with high unmet medical needs. Abeona’s cGMP manufacturing facility supports both clinical and commercial production of its therapies.