NEW YORK and CLEVELAND, Dec. 22, 2021 (GLOBE NEWSWIRE) — Abeona Therapeutics Inc. (NASDAQ:ABEO), a fully-integrated leader in gene and cell therapy, today issued the following letter to shareholders.
Dear fellow shareholders,
We have never been closer to fulfilling our mission of providing novel gene and cell therapies to patients who currently have no approved treatment options as we continue to advance the EB-101 and ABO-102 pivotal studies toward completion to support two U.S. Biologics License Application (BLA) submissions. At the same time, we are continuing to make steady progress with other clinical and preclinical programs.
As we end the calendar year 2021, it is important to reflect on all that we at Abeona have achieved over the last 12 months, and it is even more crucial to look ahead at the opportunities awaiting us in 2022 and beyond. Here is a recap of our recent accomplishments, which have positioned Abeona on course for a potentially transformative year ahead.
EB-101 (Autologous, Gene-Corrected Cell Therapy) for RDEB
We are close to completing patient enrollment for the pivotal Phase 3 VIITAL™ study for our investigational product for recessive dystrophic epidermolysis bullosa (RDEB), EB-101. Under the study protocol, the enrollment target is approximately 35 randomized large chronic wounds and we have treated 80% of that target to date. To increase patient enrollment, we activated UMass Memorial Medical Center as the second clinical trial site in the VIITAL™ study. We remain confident in our ability to complete enrollment in the first quarter 2022, positioning us for topline data in third quarter 2022. We are optimistic about EB-101’s potential based on updated Phase 1/2a results presented at various medical congresses. On the manufacturing front, we have continued to prepare our cGMP commercial facility in Cleveland, Ohio for manufacturing EB-101 drug product to support our planned BLA filing. EB-101 study drug product for all our VIITAL study participants has been manufactured at our Cleveland facility and we have now completed submission of Module 3 for chemistry, manufacturing and control describing the in-house production of both retroviral vector and the final drug product to the Investigational New Drug application (IND).
ABO-102 (AAV-based Gene Therapy) for MPS IIIA
We have continued to gain clarity on a regulatory path for investigational ABO-102 as a potential treatment for Sanfilippo syndrome type A (MPS IIIA), having successfully completed a Type B meeting with the FDA where we aligned on the primary endpoint for registration. We are excited about important clinical data updates from the pivotal Transpher A study presented at multiple medical meetings and congresses on disease-specific biomarkers, preservation of neurocognitive development and anatomical development using brain MRI in children treated early in age at the therapeutic dose. We believe that the totality of data will provide a holistic view of the treatment effect of ABO-102 and contribute to a robust regulatory package in a disease where there are no approved treatments and other investigational products are at a very early stage. In a major step toward becoming non CDMO-dependent, we have completed manufacturing of six GMP lots of ABO-102. Additionally, in preparation for manufacturing commercial supply of ABO-102, we initiated construction of a 12,000 square foot commercial AAV manufacturing facility at our Cleveland site, which will also have the capacity to support additional AAV programs, including our pre-clinical ocular programs.
ABO-101 (AAV-based Gene Therapy) for MPS IIIB
We closed enrollment in our ABO-101 Transpher B study in Sanfilippo syndrome type B (MPS IIIB), and we are now following patients for safety and efficacy. We look forward to seeing two-year neurocognitive data and will subsequently determine next steps for the program.
Preclinical Pipeline
While our clinical programs are currently focused on rare diseases, we intend to address larger areas of unmet medical need in the future, and our preclinical programs are investigating novel AAV capsids in five undisclosed ophthalmic conditions each with estimated U.S. prevalence ranging from 5,000 to 15,000 patients. We have shared data from non-human primates that will help to determine optimal routes of administration and have made significant progress toward measuring efficacy in the preclinical setting. We have also generated appropriate mouse models, produced recombinant capsids, and started dosing mice in proof-of-concept studies that we hope will yield data beginning in mid-2022 to support pre-IND meetings with the FDA in the second half of 2022.
Well-positioned for 2022 Anticipated Milestones
With the settlement of the REGENXBIO dispute and the recent successful completion of a public offering of securities for approximately $17.5 million in gross proceeds, we expect to begin 2022 with cash and financial resources of more than $50 million (unaudited). We believe focusing on our milestones is the best way to create value for our shareholders, and our strengthened balance sheet is crucial in positioning us to further execute our operating plan and achieve important anticipated milestones. Our anticipated milestones in 2022 include:
EB-101
- Completion of patient enrollment for the EB-101 Phase 3 VIITAL™ study in the first quarter of 2022
- Top-line VIITAL™ study results in the third quarter of 2022
ABO-102
- Construct and operationalize the new AAV manufacturing facility in 2022
- Top-line results for MPS IIIA Transpher A study between the fourth quarter of 2022 and second quarter of 2023
ABO-101
- Two-year neurocognitive data from MPS IIIB Transpher B study in the second half of 2022
Preclinical Pipeline
- Generate proof-of-concept data assessing AAV capsids in multiple undisclosed eye indications beginning in the first half of 2022
- Pre-IND meeting with FDA in the second half of 2022
In summary, we have confidence in the potential transformative value that Abeona’s cell and gene therapies can deliver to patients. We are focused on our road map and making progress through disciplined execution. Our current leadership team is as strong as ever, especially with important recent additions geared to enhance our BLA readiness. I would like to thank our shareholders for supporting our efforts and sharing in our mission to bring gene and cell therapies to people impacted by serious diseases.
Sincerely,
Vish Seshadri, Ph.D., M.B.A.
Chief Executive Officer
Abeona Therapeutics Inc.
