On May 11, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported its financial results for the first quarter of 2023 and provided an update on progress toward achieving key corporate objectives (Press release, Abeona Therapeutics, MAY 11, 2023, View Source [SID1234631474]).

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!

"We remain focused on completing our EB-101 Biologics License Application for recessive dystrophic epidermolysis bullosa (RDEB) and are making good progress towards this goal," said Vish Seshadri, Chief Executive Officer of Abeona. "We are also excited to be presenting encouraging results from animal proof-of-concept studies from our AAV ophthalmology program at the ASGCT (Free ASGCT Whitepaper) annual meeting next week."

First Quarter and Recent Portfolio Update

EB-101 for RDEB

● Abeona continues to make progress toward submitting a BLA for EB-101 to the U.S. Food and Drug Administration (FDA) in late second quarter to early third quarter of 2023. The Company has submitted a pre-BLA meeting request to the FDA in advance of the anticipated BLA submission. If the BLA is approved, Abeona anticipates being granted a Priority Review Voucher (PRV), which can be used to receive expedited review by the FDA of a subsequent marketing application for a different product or sold to another company.
● Abeona announced today in a separate press release that additional data from the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB was presented during an oral session at the International Societies for Investigative Dermatology (ISID) Meeting. The positive top-line efficacy and safety data from the VIITAL study was reported in November 2022.
● As part of its commercial planning, the Company continues to engage with stakeholders across the healthcare system, including public and private payors, and healthcare providers to better understand market access and pricing for EB-101.

Preclinical programs

● Abeona’s preclinical programs are investigating the use of novel adeno-associated virus (AAV) capsids in AAV-based therapies for serious genetic eye diseases. The Company has been granted pre-Investigational New Drug Application meetings for two of its programs to take place in the second quarter of 2023.
● Abeona will present new preclinical data at the 26th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) taking place from May 16-20, 2023 in Los Angeles, CA. The Company’s presentations will include data on three internally developed investigational preclinical gene therapy product candidates from its AAV ophthalmology program, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis (XLRS) and ABO-505 for autosomal dominant optic atrophy (ADOA).

First Quarter Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $40.7 million as of March 31, 2023, compared to $52.5 million as of December 31, 2022. Net cash used in operating activities was $11.7 million for the three months ended March 31, 2023. Abeona estimates that its current cash and cash equivalents, restricted cash and short-term investments are sufficient resources to fund operations into the third quarter of 2024.

"Our operating cash burn for the first quarter of 2023 and projected operating cash burn for the second quarter of 2023 include BLA submission and personnel costs, which we expect will be substantially lower in the second half of 2023," said Joe Vazzano, Chief Financial Officer of Abeona.

License and other revenues in the first quarter of 2023 were nil, compared to $0.3 million in the first quarter of 2022. Research and development expenses for the three months ended March 31, 2023 were $8.0 million, compared to $10.5 million for the same period of 2022. General and administrative expenses were $4.0 million for the three months ended March 31, 2023, compared to $4.2 million for the same period of 2022. Net loss was $9.1 million for the first quarter of 2023, or $0.54 loss per common share as compared to a net loss of $22.0 million, or $3.80 loss per common share, in the first quarter of 2022.

Portfolio Update Conference Call

The Company will host a conference call and webcast on Tuesday, May 23, 2023, at 8:30 a.m. ET, to discuss the first quarter and recent portfolio update, and its data presentations at the ISID and ASGCT (Free ASGCT Whitepaper) meetings. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 885338 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 www.abeonatherapeutics.com. The archived webcast replay will be available for 30 days following the call.