AceLink Therapeutics Presents Interim Results from a Phase 2 Trial of the GCS Inhibitor AL01211 in Treatment-Naïve, Classic Male Fabry Disease Patients at the WORLD Symposium 2025

AceLink Therapeutics, Inc., a clinical-stage biotech company developing next-generation oral substrate reduction therapies (SRTs), presented interim data from its ongoing Phase 2 clinical study of AL01211 in treatment-naïve, classic male Fabry disease patients. These findings were highlighted in a late-breaking oral platform presentation at the 2025 WORLD Symposium in San Diego, California.

The ongoing Phase 2 open-label study is evaluating the safety, pharmacokinetics, pharmacodynamics, and treatment effects of AL01211 in males with classic Fabry disease who have not received currently approved Fabry disease therapies. AceLink completed enrollment of 18 patients across six sites in China in December 2024. Topline data from this Phase 2 trial is expected in Q3 2025.

Interim results indicate that AL01211 is generally safe and well tolerated in classic male Fabry patients. Treatment with 30 mg once daily reduced GL3 levels by 50%, while a higher daily dose of 60 mg resulted in a faster and greater reduction of GL3 substrate levels. Preliminary clinical data suggest that AL01211 stabilizes Fabry disease symptoms, including eGFR and proteinuria levels, and shows positive trends in pain reduction, quality of life, and global disease symptom assessment.

Dr. Yan Ouyang, one of the investigators from our leading site Ruijing Hospital Professor Nan Chen's team, presented at the meeting. "The interim results from AL01211 treatment are showing promising safety and efficacy trends, underscoring its potential to address critical gaps in Fabry disease treatment. I look forward to further validating these findings in our ongoing research and ultimately bringing this much-needed therapy to patients," said Dr. Nan Chen.

Michael Babcock, Head of Research and Development at AceLink Therapeutics, stated: "With a robust pipeline and a commitment to advancing next-generation Substrate Reduction Therapies, AceLink Therapeutics is dedicated to transforming the treatment landscape for patients with Fabry disease and other glycosphingolipid-related disorders. We appreciate the invaluable support from the patient community and investigators as we strive to translate scientific discoveries into clinical solutions that benefit patients and address the challenges of rare disease treatments."

About AL01211

AL01211 is a proprietary, non-brain-penetrant GCS inhibitor with high potency (single-digit nanomolar IC50), excellent selectivity, and favorable drug properties that support once-daily oral administration. AL01211 represents a much-needed oral small molecule therapy as an alternative to enzyme replacement therapy (ERT), eliminating the need for frequent intravenous infusions.

About GCS Inhibitors

GCS catalyzes the first step in the synthesis of glycosphingolipids, a class of bioactive molecules involved in various cellular processes and diseases. GCS inhibitors reduce glycosphingolipid synthesis, offering therapeutic benefits for conditions such as Fabry and Gaucher disease, which are characterized by the accumulation of these lipids.

About AceLink Therapeutics, Inc.

Founded in 2018, AceLink Therapeutics is a clinical-stage pharmaceutical company developing the next generation of oral substrate reduction therapies (SRTs) to address significant unmet medical needs and improve the quality of life for patients with inherited disorders of glycosphingolipid metabolism. The company's pipeline includes a Phase 2 program for Fabry disease and Type 1 Gaucher disease.

For more information, please visit www.acelinktherapeutics.com.