March 7, 2025

Research Triangle Park, N.C. – March 7, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort. The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two. DSMBs are independent groups of experts appointed to periodically review information from clinical trials. These reviews typically include risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant benefit of therapies.1 The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes two sequential, dose-level cohorts. Adult participants diagnosed with LGMD2I/R9 will be given a single intravenous infusion of AB-1003 or placebo. The trial was initiated in 2023. It will include up to 14 participants at six sites throughout the United States.2 Participants in the first cohort remain in the study until completion. Enrollment in cohort two is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com. “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.” AskBio has received rare pediatric disease designation (RPDD), orphan-drug designation (ODD) and fast track designation (FTD) for AB-1003 for the potential treatment of LGMD2I/R9 from the United States Food and Drug Administration (FDA).3-4 These designations serve as clear recognition of the significant unmet medical need in LGMD2I/R9, for which there is no approved therapy.5 “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, 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.”   AB-1003 is an investigational recombinant adeno-associative virus (AAV)-based gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. It is designed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion.2-4 About Limb-Girdle Muscular Dystrophy Type 2I/R9 LGMD2I/R9 is a rare form of LGMD caused ...read the news