AskBio, a Bayer subsidiary, announced the advancement of its Phase 1/Phase 2 LION-CS101 clinical trial for AB-1003, a gene therapy for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The first participant in the second cohort has been dosed following a positive safety review by the Data Safety Monitoring Board (DSMB) of the first cohort’s data. The trial is evaluating the safety of AB-1003 in adults with genetically confirmed LGMD2I/R9.
This progress is crucial because LGMD2I/R9 is a rare and debilitating disease with no currently approved treatments. The positive DSMB review and subsequent dosing of the second cohort signify that the therapy is demonstrating an acceptable safety profile, allowing the trial to proceed and potentially bringing a much-needed treatment option closer to reality for patients. This advancement offers hope to individuals and families affected by this disease, which manifests in childhood and progressively worsens, leading to significant mobility limitations and potential heart and lung complications.
The LION-CS101 trial is a double-blind, randomized, placebo-controlled study involving two dose-level cohorts. It will include up to 14 participants across six U.S. sites. AB-1003 is an investigational adeno-associated virus (AAV)-based gene therapy designed to restore FKRP enzyme activity within muscle cells via a single intravenous infusion. AskBio has secured Rare Pediatric Disease, Orphan Drug, and Fast Track designations from the FDA for AB-1003 for the treatment of LGMD2I/R9, underscoring the unmet medical need in this area.
The successful progression of the LION-CS101 trial strengthens the outlook for AB-1003 as a potential treatment for LGMD2I/R9. Positive results from this trial could pave the way for larger studies and eventual regulatory approval, offering a transformative therapeutic option for patients with this devastating disease and establishing AskBio’s leadership in gene therapy for neuromuscular disorders. Continued monitoring of safety and efficacy in the second cohort will be essential for determining the next steps in development.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
