FDA Accepts IND for Limb-Girdle Muscular Dystrophy Gene Therapy


Asklepios BioPharmaceuticals noted that it plans to initiate dosing for the LION-101 phase 1/2 clinical trial in the first half of 2022.

Katherine High, MD

Katherine High, MD

The FDA has cleared the investigational new drug (IND) application for Asklepios BioPharmaceutical’s LION-101, an investigational gene therapy for the treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). The agent will now proceed into a phase 1/2 clinical study.1

The phase 1/2 study will assess single intravenous infusions of LION-101, a novel recombinant adeno-associated virus (rAAV) based vector, among adult and adolescent individuals with genotypically confirmed LGMD2I/R9, with the trial expected to begin dosing in the first half of 2022. Further details from the study have not been released.

"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 clinical trials,” Katherine High, MD, president, Therapeutics, AskBio, said in a statement. “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 being 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.”

LGMD2I/R9 is a form of LGMD and is caused by mutations in the FKRP gene. Signs and symptoms for the disease often develop in late childhood and may include difficulty running and walking. Symptoms typically gradually worsen over time, with patients generally needing wheelchair assistance approximately 2 years after onset.

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Asklepios Biopharmaceutical, a wholly owned and independently operated subsidiary of Bayer AG acquired in 2020, is a fully integrated AAV gene therapy company with an array of clinical trials using AAV gene therapy across a range of late-onset Pompe disease, multiple system atrophy, and Parkinson disease (PD).

"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,” Sheila Mikhail, chief executive officer and co-founder, AskBio, said in a statement. “With this clearance we have now advanced 5 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.”

Despite the lack of approved therapies, there are a number of drug candidates in preclinical and clinical-stage therapy-development programs for LGMD.2 Sarepta Therapeutics has a multitude of gene therapies in clinical development for various forms of LGMD, including SRP-9003, SRP-9004, SRP-9005, SRP-6004, and SRP-9006. PTC Therapeutics is currently testing deflazacort (Emflaza), an FDA approved treatment for patients with Duchenne muscular dystrophy, in patients with LGMD2I/R9. Prednisone, which received approval for the treatment of rheumatoid arthritis in 2012, is also currently being evaluated in all forms of LGMD at Northwestern University.

1. AskBio announces IND for LION-101, a novel investigational AAV gene therapy for the treatment of Limb-Girdle muscular dystrophy type 2I/R9, cleared to proceed by US FDA. News release. Asklepios BioPharmaceutical. May 25, 2021. Accessed June 2, 2021. https://www.prnewswire.com/news-releases/askbio-announces-ind-for-lion-101-a-novel-investigational-aav-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2ir9-lgmd2ir9-cleared-to-proceed-by-us-fda-301298246.html
2. How MDA is supporting LGMD research and care in 2020. News release. MDA. August 10, 2020. Accessed June 2, 2021. https://strongly.mda.org/how-mda-is-supporting-lgmd-research-and-care-in-2020/
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