AskBio Receives FDA Fast Track Designation for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)

Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced that the U.S. Food & Drug Administration (FDA) has granted Fast Track Designation for the LION-101 gene therapy program. LION-101 is a novel recombinant adeno-associated virus (rAAV) based vector being developed as a one-time intravenous infusion for the treatment of patients with Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).

The FDA recently cleared an Investigational New Drug (IND) application for LION-101, to authorize the start of a Phase 1/2 multicenter study which will evaluate the safety, tolerability, and efficacy of a single intravenous (IV) infusion of gene therapy, in adult and adolescent subjects with genotypically confirmed LGMD2I/R9. AskBio plans to initiate dosing for the LION-101 Phase 1/2 clinical study in the first half of 2022.

“The FDA Fast Track Designation for LION-101 is an important step for the development of this program and is a clear recognition of the profound burden faced by LGMD2i/R9 patients,” said Sheila Mikhail, Co-Founder & CEO, AskBio. “We look forward to initiating clinical trials with this novel therapy, and we hope to bring a new therapeutic option to patients and families in the LGMD2I/R9 community who live with this devastating disease.”

The FDA Fast Track Program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and fill an unmet medical need. The purpose of the Program is to get important new therapeutics to the patient earlier. Therapeutics that receive this designation receive a number of benefits that include more frequent meetings with the FDA to discuss development of the gene therapy and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.