By Chris Wack

Abeona Therapeutics Inc. said it completed a successful Type B meeting with the U.S. Food and Drug Administration regarding a trial to support filing and approval for its AAV-based gene therapy ABO-102 for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA.

The company said ABO-102 is currently being evaluated in the single-arm Transpher A study in patients with MPS IIIA.

Abeona said that 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 submission depending on the data set.

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 and the Kauffman Assessment Battery for Children, which are already part of the assessment plan in the Transpher A protocol.

The company said it intends to work closely with the FDA through the regenerative medicine advanced therapy mechanism to assemble the most robust pivotal data package possible for the registration of ABO-102.

Abeona shares were up 9%, to $1.36, in premarket trading.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

July 28, 2021 08:01 ET (12:01 GMT)

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