AceLink's Oral Fabry Disease Treatment Shows Promise in Phase II Study

AceLink Therapeutics has announced promising interim results from its Phase II study of AL01211, a novel oral treatment for Fabry disease. The data, presented at the 2025 WORLD Symposium in San Diego, demonstrate the potential of this small-molecule therapy to address critical gaps in Fabry disease treatment.

AL01211: A New Approach to Fabry Disease Management

AL01211 is an orally available small-molecule inhibitor of glucosylceramide synthase, an enzyme involved in the formation of glycosphingolipids. Unlike current enzyme replacement therapies (ERT), which restore patients' ability to break down these lipids, AL01211 aims to prevent their formation altogether.

The Phase II study, conducted in China, enrolled 18 men with classic Fabry disease who had not previously received approved therapies. Interim data showed that a once-daily 30-mg dose of AL01211 reduced levels of globotriaosylceramide (GL3) – a key marker of disease progression – by 50%. A higher 60-mg dose led to an even faster and greater reduction in GL3 substrate levels.

Dr. Yan Ouyang, an investigator on the study, stated, "These findings point to AL01211's potential to address critical gaps in Fabry disease treatment."

Clinical Benefits and Quality of Life Improvements

Preliminary results suggest that AL01211 treatment stabilizes key symptoms of Fabry disease, including proteinuria and kidney function. Patients also experienced positive trends in quality of life, symptom burden, and pain reduction.

The oral administration of AL01211 offers a more convenient alternative to the current standard of care, which typically involves hours-long intravenous infusions of enzyme replacement therapy.

Competitive Landscape in Fabry Disease Treatment

AceLink's progress comes amid a competitive field of biotechnology companies working on Fabry disease treatments. Sangamo Therapeutics recently announced an accelerated pathway for its gene therapy, isaralgagene civaparvovec, potentially reducing time-to-market by three years.

Similarly, uniQure is advancing AMT-191, a gene therapy designed to deliver a functional copy of the galactosidase alpha gene and induce protein expression in the liver. The company recently completed enrollment for the first cohort of a Phase I/IIa trial.

As AceLink completes enrollment in its Phase II study, additional data are expected in the third quarter of this year. These developments collectively signal promising advancements in the treatment landscape for Fabry disease, offering hope for improved patient outcomes and quality of life.