AskBio Inc., a gene therapy subsidiary of Bayer AG, announced today the successful advancement of its Phase 1/Phase 2 LION-CS101 clinical trial for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with the dosing of the first participant in the second cohort. This milestone follows a comprehensive safety review by an independent Data Safety Monitoring Board (DSMB), which determined it was safe to proceed based on data from the first cohort.
The double-blind, randomized, placebo-controlled, dose-escalation trial is evaluating AB-1003, an investigational gene therapy for adult patients with genetically confirmed LGMD2I/R9. Initiated in 2023, the study will include up to 14 participants across six U.S. clinical sites, with participants receiving either a single intravenous infusion of AB-1003 or placebo.
"The burden of LGMD2I/R9 on patients and their families is profound," said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. "Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion."
Addressing a Significant Unmet Medical Need
LGMD2I/R9 is a rare form of limb-girdle muscular dystrophy caused by mutations in the FKRP gene, resulting in progressive weakness and wasting of arm and leg muscles. Symptoms typically begin around 11 years of age, with patients experiencing increasing difficulty with mobility over time. Many eventually require wheelchairs and may develop impaired heart and lung function.
The disease affects fewer than 5,000 people in the United States, and currently has no approved treatments. Management is limited to addressing individual symptoms as they appear.
The U.S. Food and Drug Administration (FDA) has granted AB-1003 rare pediatric disease designation (RPDD), orphan-drug designation (ODD), and fast track designation (FTD) for the treatment of LGMD2I/R9, recognizing the significant unmet medical need for these patients.
Innovative Gene Therapy Approach
AB-1003 is a recombinant adeno-associative virus (AAV)-based gene therapy designed to restore FKRP enzyme activity primarily inside muscle cells. The therapy is administered as a one-time intravenous infusion and aims to address the underlying genetic cause of the disease.
"The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. "We are encouraged by the DSMB's recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one and are excited to proceed with the second cohort."
Trial Design and Progress
The LION-CS101 trial includes two sequential, dose-level cohorts to evaluate the safety profile of AB-1003. Participants from the first cohort remain in the study until completion, while enrollment for the second cohort is ongoing.
The DSMB, an independent group of experts appointed to periodically review clinical trial information, conducted a thorough assessment of trial recruitment activity and safety reporting from the first cohort. Their review included risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant therapeutic benefits.
Patients and healthcare providers interested in learning more about the LION-CS101 clinical trial can visit clinicaltrials.gov (NCT05230459) or askbio.com for additional information.
About AskBio
AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company headquartered in Research Triangle Park, North Carolina. The company maintains a diverse clinical portfolio across neuromuscular, central nervous system, cardiovascular, and metabolic disease indications.
With over 900 employees across five countries, AskBio has established itself as an early innovator in the gene therapy field. The company's technology platform includes Pro10™, a proprietary cell line manufacturing process, and an extensive array of capsids and promoters. AskBio holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids.
As gene therapy continues to emerge as a promising approach for treating genetic disorders, the advancement of the LION-CS101 trial represents an important step forward in addressing the significant unmet needs of patients with LGMD2I/R9.
