First patient dosed in LOPD gene therapy trial
Askbio's Phase 1/2 study to assess AB-1009's safety in adults now recruiting
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The first patient has been dosed in a Phase 1/2 clinical trial testing AB-1009, Askbio’s investigational gene therapy for late-onset Pompe disease (LOPD), the company said.
The trial, PROGRESS-GT LOPD (NCT07282847), primarily aims to assess the therapy’s safety in up to 12 adults with LOPD, aged 18 and older. Recruitment is ongoing at sites in California and in Pennsylvania, with additional study locations expected to open. The U.S. Food and Drug Administration (FDA) cleared the study to begin earlier this year.
“The initiation of PROGRESS-GT LOPD marks an important step in the development of AB-1009 and demonstrates our commitment to moving our clinical program forward with determination, to investigate a potential new treatment approach for people living with Pompe disease,” Canwen Jiang, MD, PhD, chief development officer and chief medical officer at Bayer subsidiary Askbio, said in a company press release. “Reaching this milestone reflects the momentum behind our gene therapy platform and our commitment to working toward improving quality of life for people with rare diseases.”
In Pompe disease, mutations in the GAA gene lead to either too little or poorly functioning acid alpha-glucosidase (GAA), an enzyme needed to break down the complex sugar glycogen. As a result, glycogen accumulates to toxic levels in cells, particularly muscle cells, driving progressive muscle damage and Pompe symptoms such as poor muscle tone, muscle weakness, and breathing issues. People with LOPD typically develop symptoms after age 1.
Need for new treatment options ‘critical’
Standard Pompe disease treatment relies on enzyme replacement therapy (ERT), which provides the body with a lab-made version of the missing enzyme. While ERT can help ease symptoms and slow disease progression, it requires regular, lifelong infusions that can be burdensome for patients. In addition, treatment responses may decline over time, potentially increasing patients’ treatment demands.
“Current treatment approaches may not fully meet the long-term needs of patients, and ongoing research into potential new options remains critical,” said Tahseen Mozaffar, MD, director of the UCI Health ALS & Neuromuscular Center and principal investigator of the AB-1009 clinical trial program.
AB-1009 aims to address the limitations of existing therapies by directly targeting the underlying cause of Pompe disease. The one-time gene therapy uses a harmless adeno-associated virus to deliver a functional copy of the GAA gene to cells. By enabling the body to produce its own GAA enzyme, the therapy may potentially ease glycogen accumulation and Pompe symptoms.
PROGRESS-GT LOPD participants will receive one of two doses of AB-1009 via a single intravenous (into-the-vein) infusion. The trial’s main goal is to assess the rate and severity of side effects over 52 weeks (one year) of follow-up.
The FDA has granted AB-1009 both orphan drug and fast track designations. Fast track status is intended to accelerate the development and review of therapies for serious conditions with unmet medical needs, while orphan drug designation provides incentives for treatments targeting rare diseases, or those affecting fewer than 200,000 people in the U.S.
