After a series of stumbles, Abeona Therapeutics is hoping a make-or-break meeting with the FDA will set them on the right path toward finally getting a gene therapy approved.
The cell and gene therapy company is trying to move forward after reorganizing its leadership team following years of management struggles. In a letter to stockholders today, recently promoted CEO Michael Amoroso said Abeona is committed to getting things back on track with the recent appointments of four new independent board members.
And the new leadership class has arrived just in time, too. Abeona will meet with the FDA in June to discuss whether its Sanfilippo syndrome type A gene therapy, named ABO-102, can move toward a regulatory filing in the U.S., Amoroso said.
Sanfilippo syndrome is a rare genetic metabolic disorder that causes serious problems in the brain and nervous system in children.
Abeona is trying to move an application for ABO-102 across the finish line, and the FDA meeting will center on whether a February data set can form the basis of that filing.
In the Transpher A study, neurocognitive development was preserved in patients who received ABO-102 treatment at a young age.
New York City- and Cleveland-based Abeona met with the EU Medicines Agency in 2020 for feedback on a path to marketing authorization in that region. Following the FDA feedback session, Abeona will refine its plan and analyze next steps with the European Medicines Agency, Amoroso wrote in the letter.
Abeona was trading nearly 13% higher on the news, at $1.68 per share as of 2:20 p.m. ET.
Another of Abeona's troubled therapies, the gene-corrected cell therapy dubbed EB-101, was also addressed in the shareholder letter. That treatment is aiming to correct recessive dystrophic epidermolysis bullosa, an incurable connective tissue disorder that causes the skin to become very fragile and blister easily.
EB-101 was slapped with a clinical hold on a phase 3 clinical trial by the FDA in September 2019, with the regulator seeking more information on the transport stability of EB-101 to clinical sites.
Abeona met with the FDA in late 2020 to discuss the resumption of testing for EB-101, with “very positive” results, according to the letter. The company and regulatory body aligned on proposed endpoints for the VIITAL(TM), study and it has finally gotten underway, Amoroso said in the letter.
Patient enrollment is expected to be completed in 2021, and top-line data are to follow in mid-2022, with a regulatory filing expected if the data are positive, the letter said.
Amoroso also tried to reassure stockholders of Abeona’s corporate governance given a recent executive bench shake-up.
The company's leadership troubles kicked off in November 2018 when CEO Carsten Thiel was ejected for “allegations of misconduct toward colleagues.” Then, in the first week of 2020, President and Chief Scientific Officer Tim Miller resigned from the company to pursue a new role at Forge Biologics.
Amoroso’s promotion from COO to CEO in March was paired with the appointment of four new independent board members, including two in April and two in March. Yesterday, Abeona also beefed up its executive bench with the hire of a new head of research and clinical development, Vishwas Seshadri, Ph.D., who comes from Bristol Myers Squibb’s Celgene.
Abeona is also working on ABO-101 in mucopolysaccharidosis type IIIB, a metabolic disorder like Sanfilippo syndrome.
The company entered into two strategic partnerships with Taysha Gene Therapies in 2020 for two pipeline candidates.
Amoroso said the focus for 2021 will be on completing the EB-101 study enrollment and "gaining clarity on a regulatory path for ABO-102."