On May 15, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the first quarter of 2024 and recent corporate progress (Press release, Abeona Therapeutics, MAY 15, 2024, View Source [SID1234643313]).

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"We are grateful to our existing as well as new investors who have demonstrated their support through the recent financing, which has extended our cash runway into 2026, well beyond anticipated regulatory milestones," said Vish Seshadri, Chief Executive Officer of Abeona. "We now remain focused on working with the FDA to address the CMC deficiencies noted in the CRL and making the BLA resubmission to bring pz-cel to RDEB patients as soon as possible."

First Quarter and Recent Progress

Corporate highlights

● On May 7, 2024, Abeona closed a $75 million underwritten securities offering with participation from both new and existing investors.
● In January 2024, Abeona entered into a $50 million credit facility and received the first tranche of $20 million.

Pz-cel for RDEB

● In April 2024, Abeona received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding the Company’s Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB), based on the need for additional Chemistry Manufacturing and Controls (CMC) information. In the CRL, the FDA noted that certain additional information needed to satisfy CMC requirements must be resolved before the application can be approved. The information needed to satisfy the CMC requests in the CRL pertains to validation requirements for certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel. The Company anticipates completing the BLA resubmission in the second half of 2024.
● New pz-cel data will be presented at upcoming medical meetings. At the Society for Investigative Dermatology (SID) Annual Meeting, being held on May 15-18, 2024, new long-term safety data with up to 11 years of follow-up has been accepted as a late-breaking presentation.

U.S. commercial launch preparations for pz-cel

● Abeona continues to advance key commercial activities in preparation for a potential U.S. launch for pz-cel, including onboarding discussions with epidermolysis bullosa treatment sites, conducting medical and payer engagement, as well as building supply chain and enterprise capabilities to support the Company’s transition to a commercial stage company.

First Quarter Financial Results and Cash Runway Guidance

Cash, cash equivalents, restricted cash and short-term investments totaled $62.7 million as of March 31, 2024, compared to $52.6 million as of December 31, 2023. Net cash used in operating activities was $14.5 million for the three months ended March 31, 2024.

Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments, as well as the credit facility, combined with the net proceeds from the underwritten securities offering, are sufficient resources to fund operations into 2026, before accounting for any potential revenue from commercial sales of pz-cel, if approved, or proceeds from the sale of a Priority Review Voucher or PRV, if awarded by the FDA.

Research and development expenses for the three months ended March 31, 2024 were $7.2 million, compared to $8.0 million for the same period of 2023. General and administrative expenses were $7.1 million for the three months ended March 31, 2024, compared to $4.0 million for the same period of 2023. Net loss was $31.6 million for the first quarter of 2024, or $1.16 loss per common share, including a change in the fair value of warrant liabilities due to remeasurement of the Company’s issued stock purchase warrants. Net loss in the first quarter of 2023 was $9.1 million, or $0.54 loss per common share.

Conference Call Details

The Company will host a conference call and webcast on Wednesday, May 15, 2024, at 8:30 a.m. ET, to discuss the first quarter results. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 496484 five minutes prior to the start of the call. A live, listen-only webcast and archived replay of the call can be accessed on the Investors & Media section of Abeona’s website at View Source The archived webcast replay will be available for 30 days following the call.