FDA clears gene therapy AB-1009 for Phase 1/2 trial in LOPD
Enrollment underway at California site, with more locations expected
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The U.S. Food and Drug Administration (FDA) has cleared AskBio’s investigational new drug application for AB-1009, a potential one-time gene therapy being developed for late-onset Pompe disease (LOPD).
The clearance enables the therapy to proceed to a Phase 1/2 clinical trial (NCT07282847) in the U.S., which will primarily assess the safety of the therapy in up to 12 adults aged 18 and older with LOPD. Enrollment is underway at a site in California, with additional sites expected to open. The first patient is slated to be enrolled early this year.
AB-1009 has also received FDA orphan drug and fast track designations, which support the development and review of treatments for rare diseases — those affecting fewer than 200,000 people in the U.S. — and for serious conditions with unmet medical needs, AskBio said.
“These advancements in the AB-1009 program, particularly the recently granted regulatory designations, highlight the recognized need for late-onset Pompe treatments,” Mansuo Shannon, PhD, AskBio’s chief scientific officer, said in a company press release. “Today’s news demonstrates AskBio’s commitment to progressing early-stage assets into the clinic and adding those to our clinical portfolio.”
Targeting disease’s underlying cause
Pompe disease is caused by mutations in the GAA gene that result in either too little or poorly functioning acid alpha-glucosidase (GAA), an enzyme necessary for breaking down the complex sugar glycogen. As glycogen accumulates inside cells, particularly in muscle cells, it leads to progressive muscle weakness and other characteristic symptoms of Pompe. In LOPD, symptoms typically develop after age 1.
Enzyme replacement therapy, which provides a lab-made version of the missing enzyme, is the current standard treatment for Pompe disease. However, it requires lifelong infusions, and clinical response may decrease over time.
AB-1009 is an investigational gene therapy developed by AskBio, a subsidiary of Bayer. It is designed to target the disease’s underlying cause by delivering a functional copy of the GAA gene using a modified, harmless adeno-associated virus (AAV). This approach aims to restore enzyme production and reduce glycogen buildup, potentially easing disease symptoms.
“This investigational gene therapy is being studied for its potential to address the underlying genetic defect and to explore whether it can increase production of the deficient enzyme in patients with Pompe disease,” said Tahseen Mozaffar, MD, director of the UCI Health ALS & Neuromuscular Center and principal investigator of the AB-1009 clinical trial program. “Patients receiving gene therapy may reduce reliance on exogenous enzyme replacement.”
Participants in the Phase 1/2 study, dubbed PROGRESS-GT LOPD, will receive one of two doses of the investigational therapy administered via a single intravenous (into-the-vein) infusion. The trial’s main goal is to assess the rate of side effects over a 52-week follow-up period.
Askbio is advancing another gene therapy for LOPD, known as ACTUS-101, which is being evaluated in a separate Phase 1/2 clinical trial (NCT03533673).
