AB-1003 trial for LGMD proceeds after positive recommendation
The first participant in the second group testing the therapy has been dosed
Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9.
The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment of the second group, which is ongoing at several sites in the U.S., follows a positive recommendation from the trial’s data and safety monitoring board (DSMB) based on positive safety data from the first group of patients. The two groups differ in the gene therapy dose they receive.
DSMBs are independent groups of experts that periodically review information from clinical trials, including assessments of risk-benefit, monitoring for adverse events, and benefits of experimental therapies.
“The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101,” Canwen Jiang, MD, PhD, AskBio’s chief development officer and chief medical officer, said in a company press release. “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.”
LGMD is a type of muscular dystrophy marked by muscle wasting at the shoulders and the hips. LGMD 2I/R9 is caused by mutations in the FKRP gene, which codes for instructions to produce the fukutin-related protein that helps maintain muscle cell health.
What is AB-1003?
AB-1003 is designed to deliver a healthy version of the gene to cells via an engineered adeno-associated viral vector (AAV). It’s given as a single infusion into the bloodstream.
The LION-CS101 study expects to enroll up to 14 patients, ages 18-65, treated with the gene therapy in two sequential groups, or given a placebo. After treatment, patients are monitored for one year.
The trial started dosing patients in 2023 and should be finished in 2028.
“The burden of LGMD2I/R9 on patients and their families is profound,” said Nicholas Johnson, MD, principal trial investigator at the Virginia Commonwealth University. “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9. This advancement brings the LION-CS101 trial another step closer to completion.”
AB-1003 won orphan drug status by the U.S. Food and Drug Administration (FDA) and the European Commission, along with the FDA’s rare pediatric disease and fast track designations. These are meant to accelerate the development of therapies for rare and serious conditions that have the potential to provide significant benefits to patients.
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