Abeona Therapeutics (ABEO) Announces Successful Type B Meeting with U.S. FDA for ABO-102 AAV-based Gene Therapy in Sanfilippo Syndrome Type A

Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader in gene and cell therapy, today announced that the Company completed a successful Type B meeting with the U.S. Food and Drug Administration (FDA) regarding the pivotal trial to support filing and approval for its AAV-based gene therapy ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA). ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS IIIA.

Based on the Type B meeting with the FDA, the ongoing Transpher A study will serve as the pivotal study for ABO-102 and could potentially support a Biologics License Application (BLA) submission depending on the data set. In addition, Abeona also aligned with the FDA on the definition of the primary endpoint for the study, neurocognitive assessment using the raw score from the Bayley Scales of Infant and Toddler Development (BSITD) and the Kauffman Assessment Battery for Children (KABC-2), which are already part of the assessment plan in the Transpher A protocol. Abeona intends to work closely with the FDA through the regenerative medicine advanced therapy (RMAT) mechanism to assemble the most robust pivotal data package possible for the registration of ABO-102.

“We are grateful to the FDA for their guidance and collaborative exchange regarding the pivotal trial to support bringing ABO-102 to MPS IIIA patients who currently have no approved treatment,” said Michael Amoroso, Chief Executive Officer of Abeona. “From 2016 to-date we have treated 21 patients in the Transpher A trial. We are excited about the safety and magnitude of benefit seen with our investigational ABO-102 therapy in the younger children from the higher dose cohort reported earlier this year. We remain hopeful that if the more recently dosed children in cohort 3 display a similar treatment effect, we could have an evaluable data set in 2022. The patients we serve have tremendous unmet need and we remain fully focused on operational excellence with the intent of now delivering potentially two pivotal data packages—one for ABO-102 and one for EB-101—in 2022.”

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