AskBio presents early data on LION-CS101 clinical trial of AB-1003

German pharma major Bayer’s (BAYN: DE) gene therapy subsidiary AskBio today announced initial safety data from the first cohort of participants from its Phase I/Phase II LION-CS101 clinical trial of investigational gene therapy AB-1003 in participants with limb-girdle muscular dystrophy (LGMD) 2I/R9 at the 30th Annual International Congress of the World Muscle Society, taking place in Vienna, Austria.

The presentation represents interim, blinded Cohort 1 safety data. Participants enrolled in Cohort 1 received a single intravenous infusion of AB-1003 or placebo and were followed for 52 weeks post-treatment during the main trial before entering a planned four-year long-term follow-up period. Safety assessments included adverse event monitoring, laboratory testing, physical exams, vital signs, electrocardiograms, and echocardiograms.

There were no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment. Commonly reported (>2 participants) treatment-emergent adverse events were mild-to-moderate in severity and included headaches, falls, and nausea. Three participants reported asymptomatic transient transaminase elevations without changes in bilirubin levels, which returned to baseline levels after adjusting corticosteroid treatment.