On November 14, 2024 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the third quarter ended September 30, 2024, and recent corporate updates (Press release, Abeona Therapeutics, NOV 14, 2024, View Source [SID1234648417]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"With the acceptance of our Biologics License Application (BLA) resubmission for pz-cel, we are ramping up our commercial readiness efforts, especially with respect to onboarding potential pz-cel treatment sites and continuing discussions with payors," said Vish Seshadri, Chief Executive Officer of Abeona.

Third Quarter and Recent Progress

Pz-cel for RDEB

Abeona completed a Type A meeting in August 2024 where it aligned with the FDA on the content for the resubmission of the Company’s BLA for pz-cel, its investigational first-in-class, autologous cell-based gene therapy currently in development for RDEB, including additional information to satisfy all Chemistry Manufacturing and Controls (CMC) requirements noted in the Complete Response Letter (CRL) issued in April 2024. The CRL required that certain CMC issues be addressed in the BLA resubmission, and did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA. The FDA did not request any new clinical trials or clinical data to support the approval of pz-cel.
Also in August 2024, the Centers for Medicare and Medicaid Services (CMS) granted a product-specific procedure code ICD-10-PCS (International Classification of Diseases, 10th Revision, Procedure Coding System) for pz-cel. Also, as part of the Inpatient Prospective Payment System (IPPS) Final Rule for fiscal year 2025, CMS assigned Medicare reimbursement of pz-cel to Pre-Major Diagnostic Category, Medicare Severity Diagnosis Related Group 018 (Pre-MDC MS-DRG 018), which is among the highest available inpatient hospital reimbursement levels for cell and gene therapies. The favorable Medicare decisions support efficient hospital billing, reimbursement and patient access.
In October 2024, Abeona resubmitted its BLA for pz-cel to the FDA, seeking approval of pz-cel as a potential new treatment for patients with RDEB.
Also in October 2024, Abeona entered into a lease agreement for additional facility space in Cleveland, Ohio to enable manufacturing capacity expansion beyond the current planned manufacturing footprint.
Also in October 2024, the United States Patent and Trademark Office issued a new patent (U.S. Patent No. 12,110,504) ("the ’504 Patent") and allowed the claims of a second patent (based on U.S. Patent Application No. 16/066,253) that is expected to issue in the coming weeks. Both patents are entitled "Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Using Genetically Corrected Autologous Keratinocytes," and include claims that cover the use of pz-cel for the treatment of RDEB. The ’504 Patent has an expiration date of January 3, 2037, subject to any applicable patent term extension.
In November 2024, the FDA accepted for review the resubmission of Abeona’s pz-cel BLA and set a PDUFA target action date of April 29, 2025.
In preparation for potential commercialization, Abeona continues to make progress on several key initiatives, including onboarding high-volume epidermolysis bullosa treatment centers in the U.S. for pz-cel treatment, engaging payers to ensure patient access, and educating key stakeholders.
In preparation for potential pz-cel launch, Abeona has hired and trained personnel to support commercialization, manufacturing, supply chain and quality.
Pipeline and partnered programs

In July 2024, Abeona announced a non-exclusive agreement with Beacon Therapeutics, under which Beacon Therapeutics will evaluate Abeona’s patented AAV204 capsid for its potential use in AAV gene therapies for select ophthalmology indications.
In October 2024, Ultragenyx participated in a successful pre-BLA meeting with the FDA during which Ultragenyx aligned on the details of its BLA for partnered program UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA) that is expected to be filed around the end of 2024.
Third Quarter Financial Results and Cash Runway Guidance

Cash, cash equivalents, short-term investments and restricted cash totaled $110.0 million as of September 30, 2024. As of June 30, 2024, cash, cash equivalents, short-term investments and restricted cash totaled $123.0 million.

Abeona estimates that its current cash and cash equivalents, short-term investments and restricted cash, as well as its credit facility, 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 (PRV), if awarded by the FDA.

Research and development expenses for the three months ended September 30, 2024 were $8.9 million, compared to $7.1 million for the same period of 2023. General and administrative expenses were $6.4 million for the three months ended September 30, 2024, compared to $4.2 million for the same period of 2023. The increase in general and administrative expenses is primarily due to commercial and launch preparation costs. Net loss for the third quarter of 2024 was $30.3 million, including a $15.2 million loss resulting from the quarterly remeasurement of the fair value of warrant and derivative liabilities. In the third quarter of 2023, net loss was $11.8 million, including a $1.1 million loss resulting from the quarterly remeasurement of the fair value of warrant liabilities.

Conference Call Details

The Company will host a conference call and webcast on Thursday, November 14, 2024, at 8:30 a.m. ET, to discuss the quarter results. To access the call, dial 877-545-0320 (U.S. toll-free) or 973-528-0002 (international) and Entry Code: 500590 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.