AskBio has released preliminary safety outcomes from the first cohort of subjects in its Phase I/Phase II LION-CS101 study, which is investigating the gene therapy AB-1003 for limb-girdle muscular dystrophy (LGMD) 2I/R9.
This data was presented at the 30th Annual International Congress of the World Muscle Society, held in Vienna, Austria.
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The presentation showcases interim, blinded safety data for Cohort 1.
Subjects in this cohort received a single intravenous infusion of either the gene therapy or placebo.
They were monitored for a period of 52 weeks following treatment as part of the main trial, prior to entering a planned long-term follow-up period for four years.
Safety evaluations comprised echocardiograms, electrocardiograms, laboratory tests, monitoring for adverse events, physical examinations and vital signs.
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By GlobalDataNo serious adverse events or dose-limiting toxicities were reported up to 52 weeks following treatment.
The commonly reported treatment-emergent adverse events, observed in more than two subjects, were mild to moderate in severity and included headaches, nausea and falls.
Additionally, three subjects experienced asymptomatic transient elevations in transaminase levels, with no changes in bilirubin levels.
These elevations returned to baseline after adjustments were made to their corticosteroid treatment.
AskBio chief development officer and chief medical officer Canwen Jiang said. “These initial safety data are encouraging and suggest an acceptable safety profile for AB-1003.
“We believe AAV-mediated gene therapy has the potential to restore FKRP function and stabilise disease progression, and we are excited to continue our clinical research efforts with the goal of developing an effective treatment for limb-girdle muscular dystrophy.”
The dose-escalation, placebo-controlled, randomised, double-blind LION-CS101 study is designed to assess the safety of AB-1003 in adult subjects aged 18–65 years with genetically confirmed LGMD2I/R9. It comprises two sequential cohorts.
This trial commenced in 2023 and involves up to 14 subjects across six locations in the US. Five individuals have been enrolled in the first cohort and continue to actively participate. Enrolment for Cohort 2 is currently in progress.
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