On March 29, 2023 Abeona Therapeutics Inc. (Nasdaq: ABEO) reported financial results for the full year of 2022 and provided an update on progress toward achieving key corporate objectives (Press release, Abeona Therapeutics, MAR 29, 2023, View Source [SID1234629488]). The Company will host a conference call and webcast today, March 29, 2023, at 8:30 a.m. ET, to discuss its financial results and business update.

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"Since announcing positive topline results for the Phase 3 VIITAL study late last year, we have had multiple recent collaborative interactions with the FDA, and are continuing to work diligently toward completing and submitting our Biologics License Application for EB-101 in recessive dystrophic epidermolysis bullosa (RDEB)," said Vish Seshadri, Chief Executive Officer of Abeona. "We believe the VIITAL study results highlight the value that EB-101 could bring to RDEB patients as does the encouraging feedback we have received from our initial interactions with payors. In addition, we continue to make progress in our earlier stage programs that have the potential for treating serious eye diseases with new adeno-associated virus-based gene therapies, and we expect to keep advancing these toward an Investigational New Drug filing and clinical trials."

Fourth Quarter and Recent Operating Highlights, and Upcoming Events

EB-101 for recessive dystrophic epidermolysis bullosa (RDEB)

● In November 2022, Abeona announced positive topline results with both co-primary endpoints and all other endpoints met in the pivotal Phase 3 VIITAL study of investigational EB-101 in RDEB. Additional data from the VIITAL study has been accepted for an oral presentation at the International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Furthermore, durability of EB-101 clinical outcomes was published in the Orphanet Journal of Rare Diseases, demonstrating sustained wound healing with a mean follow up of 5.9 years and symptomatic relief based on long term follow up of the Phase 1/2a study.
● Abeona continues to make progress in the submission process for a Biologics License Application (BLA) for EB-101 to the U.S. Food and Drug Administration (FDA). The Company currently plans to submit a BLA for EB-101 in late second quarter to early third quarter of 2023. If the BLA is approved, Abeona may be eligible for 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.
● In the first quarter of 2023, as part of its commercial planning, the Company initiated discussions with stakeholders across the healthcare system, including public and private payors, and healthcare providers to better understand market access and potential 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 eye diseases, including ABO-504 for Stargardt disease, ABO-503 for X-linked retinoschisis (XLRS) and ABO-505 for autosomal dominant optic atrophy (ADOA).
● In 2022, Abeona evaluated the ability of its gene constructs and capsids to deliver and express the recombinant protein in target eye tissues and rescue mutant phenotypes in mouse disease models.
● The Company has started to submit pre-Investigational New Drug (IND) meeting requests to the FDA for these candidates and anticipates meetings to take place in the second quarter of 2023.
● The Company expects to present new preclinical data from these programs at a scientific congress in the second quarter of 2023.

Corporate highlights

● The Company enhanced its senior management team with three experienced biotechnology industry leaders. Abeona appointed Dmitriy Grachev, M.D., Ph.D., as Chief Medical Officer and Madhav Vasanthavada, Ph.D., M.B.A. as Vice President, Business Development. In addition, Amanda Moore, MSHS, was promoted to the role of Vice President, Program Leadership and Clinical Operations. Dr. Grachev has over 20 years of industry experience across multiple therapeutic areas including dermatology, ophthalmology, and oncology, and has multiple global drug approvals while leading clinical development programs at pharmaceutical and biotechnology organizations. Dr. Vasanthavada was most recently at Bristol Myers Squibb, where he led Global Marketing for the Global Car T Cell Therapy Franchise. Ms. Moore most recently served as Senior Director, Head of Program Leadership.
● In November 2022, the Company successfully completed a $35.0 million private placement financing with participation from new and existing institutional investors.

Full Year 2022 Financial Results

Cash, cash equivalents, restricted cash and short-term investments totaled $52.5 million as of December 31, 2022, compared to $50.9 million as of December 31, 2021. Net cash used in operating activities was $43.5 million for the full year of 2022, compared to $65.7 million in the full year of 2021. 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.

"With our existing cash resources, we believe we are funded through the anticipated timing for EB-101 BLA potential approval," said Joe Vazzano, Chief Financial Officer of Abeona.

License and other revenues for the year ended December 31, 2022 were $1.4 million, as compared to $3.0 million for the same period of 2021. The revenues in 2022 and in 2021 primarily represent clinical milestone payments under two licensing agreements with Taysha Gene Therapies for investigational AAV-based gene therapies for Rett syndrome and CLN1 disease (also known as infantile Batten disease), respectively.

Research and development expenses for the full year ended December 31, 2022 were $29.0 million, compared to $38.7 million for the full year ended December 31, 2021. General and administrative expenses were $17.3 million for the full year ended December 31, 2022, compared to $21.6 million for the year ended December 31, 2021. Net loss attributable to common shareholders for the full year ended December 31, 2022 was $43.5 million, or $5.53 loss per common share as compared to $84.9 million, or $21.57 loss per common share, for the full year of 2021

Conference Call Details

Abeona Therapeutics will host a conference call and webcast today, March 29, 2023, at 8:30 a.m. ET. To access the call, dial 888-506-0062 (U.S. toll-free) or 973-528-0011 (international) and Entry Code: 195981 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.