AceLink is advancing a small-molecule treatment option for Fabry disease that could provide a more convenient alternative to the current enzyme replacement therapy standard.
AceLink Therapeutics’ investigational GCS inhibitor AL01211 can safely lower levels of a key disease marker in patients with Fabry disease, according to an interim Phase II readout presented at the 2025 WORLD Symposium in San Diego that ran from February 3–7.
Fabry disease is a rare, genetic lysosomal storage disorder that develops when patients do not produce sufficient levels of alpha-galactosidase A, an enzyme that breaks down certain lipids. These fatty molecules build up in the blood vessels and various organs—including the heart, kidneys and brain—often leading to severe complications such as heart attack, stroke and kidney failure.
AceLink’s mid-stage study, conducted in China, enrolled 18 men with classic Fabry disease who had not yet received approved therapies. Interim data released over the weekend showed that a once-daily 30-mg dose of AL01211 can lower levels of globotriaosylceramide (GL3)—a toxic metabolite whose accumulation is a key marker of disease progression—by 50%.
Meanwhile, a 60-mg dose led to an even “faster and greater reduction of GL3 substrate levels,” according to AceLink’s news release.
Preliminary mid-stage results also suggest that AL01211 treatment “stabilizes” key symptoms of Fabry disease, such as proteinuria and kidney function, as measured by the estimated glomerular filtration rate. Patients on AL01211 saw “positive trends” in quality of life, symptom burden and pain reduction, the biotech noted.
Yan Ouyang, an investigator on the study, said in a statement that these findings point to AL01211’s “potential to address critical gaps in Fabry disease treatment.” AceLink has completed enrollment into the Phase II study, additional data from which are expected in the third quarter.
AL01211 is an orally available small-molecule inhibitor of glucosylceramide synthase, an enzyme that facilitates the first step in the formation of glycosphingolipids, a type of fatty molecule that accumulates in Fabry patients.
Whereas enzyme replacement therapy (ERT)—currently a popular option—restores patients’ ability to break down these lipids, AL01211 is designed to prevent their formation in the first place. AceLink’s oral candidate also provides a more convenient and accessible treatment alternative versus the standard intravenous infusion of ERT, which typically can typically take hours.
Many other biotechs are seeking to advance a treatment for Fabry. Sangamo Therapeutics, for example, announced in October 2024 that it had aligned with the FDA on an accelerated pathway for its gene therapy isaralgagene civaparvovec. The “successful” discussions with the regulator could speed up the treatment’s time-to-market by three years, the biotech said at the time.
Also in the Fabry space is uniQure, which is advancing the gene therapy AMT-191, designed to deliver a functional copy of the galactosidase alpha gene and induce the expression of the protein in the liver. The biotech completed enrollment into the first cohort of a Phase I/IIa trial of AMT-191 earlier this month.
