AceLink Therapeutics Completes 6-Month Primary Phase of AL01211 Clinical Study in Fabry Disease Patients

AL01211, a novel oral GCS inhibitor, shows favorable safety and robust biomarker reduction in Phase 2 trial in Fabry patients

NEWARK, Calif.--(BUSINESS WIRE)--AceLink Therapeutics, a clinical-stage biopharmaceutical company developing best-in-class substrate reduction therapies for lysosomal storage disorders, today announced the successful completion of the 6-month primary treatment phase of its ongoing Phase 2 study of AL01211 in treatment-naive male patients with classic Fabry disease.

The study enrolled 18 adult males who had not received prior enzyme replacement therapy (ERT). Participants received once-daily oral AL01211 for 26 weeks. Primary objectives were to evaluate safety, tolerability, and biomarker response.

“We are very encouraged by the safety profile and glycolipid reductions observed in this treatment-naive Fabry population,” said Michael Babcock, PhD, Co-Founder and VP of Research and Early Development at AceLink Therapeutics. “The magnitude of substrate reduction reinforces our belief that AL01211 has the potential to provide a convenient and effective oral alternative to current therapies. It’s also encouraging that most patients are continuing into the long-term extension.”

“The successful completion of the 6-month main treatment period of this Phase 2 clinical trial is an important milestone,” said Wen Chen, acting CEO of AceLink. “We are looking forward to obtaining more clinical data and eventually being able to bring a safe and effective therapy to patients in urgent need. We are grateful to Fabry patients, their families and all investigators for their important contributions to the clinical research of rare disease drugs.”

AceLink is now preparing for regulatory discussions with the U.S. Food and Drug Administration (FDA) to explore a pathway toward accelerated approval. In parallel, the company is leveraging its Breakthrough Therapy Designation in China to engage with the Center for Drug Evaluation (CDE) on potential approval pathways. These efforts aim to accelerate patient access to AL01211 in both the U.S. and China, where significant unmet need remains.

About AL01211

AL01211 is a potent and selective glucosylceramide synthase (GCS) inhibitor designed to block the synthesis of glycosphingolipids that accumulate in Fabry disease. As an oral, once-daily therapy, it offers a non-invasive alternative to biweekly intravenous enzyme replacement therapies.

About Fabry Disease

Fabry disease is a rare, X-linked lysosomal storage disorder caused by pathogenic variants in the GLA gene, leading to α-galactosidase A deficiency and accumulation of globotriaosylceramide (Gb3) and lyso-Gb3, which drive progressive multi-organ damage including chronic kidney disease, cardiomyopathy/arrhythmias, and stroke.

About AceLink Therapeutics

AceLink Therapeutics is a clinical-stage biopharmaceutical company developing next-generation substrate reduction therapies for lysosomal storage disorders. The company combines deep expertise in glycosphingolipid metabolism with a focus on patient-centric drug design to deliver safe, effective, and accessible treatments for rare diseases.