AskBio Announces IND for LION-101 Cleared to Proceed by U.S. FDA
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, announced that the U.S. Food & Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LION-101 to proceed in a Phase 1/2 clinical study. 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).
LION-101 will be evaluated in a first-in-human Phase 1/2 multicenter study to evaluate a single intravenous (IV) infusion 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.
“In preclinical mouse models, LION-101 therapy demonstrated both dose-dependent efficacy and tolerability, providing a clear approach to study this novel AAV vector in first-in-human trials,” said Katherine High, MD, President, Therapeutics, AskBio. “Currently there are no approved therapies for LGMD2I/R9, and with limited treatment options that only address symptoms of the disease, the patient burden is profound. We are excited to begin first-in-human clinical trials with this novel therapy, and to hopefully bring a new therapeutic option to patients and families in the LGMD2I/R9 community living with this devastating disease.”
“Clearing of the LION-101 IND to proceed underscores our commitment to address unmet needs in musculoskeletal disorders, where very few or no treatments exist today, while we continue to advance our broad therapeutic pipeline of novel AAV gene therapies,” said Sheila Mikhail, CEO and Co-Founder, AskBio. “With this clearance, we have now advanced five of our pipeline programs into Phase 1/2 clinical development. We look forward to advancing these clinical programs to bring much needed gene therapy innovation and potential treatments to patients and their families who live with devastating conditions like LGMD2I/R9.”