Results from AceLink’s Phase 2 AL01211 Study in Fabry Disease Presented at WORLD Symposium 2025
AceLink Therapeutics, a clinical-stage biotech firm focused on developing next-generation oral substrate reduction therapies (SRTs), presented interim results from its ongoing Phase 2 clinical study of AL01211 in treatment-naïve, classic male Fabry disease patients. These findings were featured in a late-breaking oral presentation at the 2025 WORLD Symposium held in San Diego, California.
The open-label study, which evaluates the safety, pharmacokinetics, pharmacodynamics, and treatment effects of AL01211 in patients who have not previously received approved Fabry disease therapies, has completed enrollment of 18 male patients across six sites in China as of December 2024. The trial is expected to report topline data in the third quarter of 2025.
Interim results presented at the 2025 WORLD Symposium in San Diego indicate that AL01211 is well tolerated and demonstrates promising safety and efficacy trends. In the study, patients treated with a 30 mg once-daily dose showed a significant 50% reduction in GL3 (globotriaosylceramide) levels, a key biomarker in Fabry disease. Those receiving a higher dose of 60 mg daily experienced a more rapid and greater reduction of GL3, suggesting dose-dependent efficacy. Additionally, preliminary clinical data suggests AL01211 may help stabilize Fabry disease symptoms, including improving eGFR (estimated glomerular filtration rate) and reducing proteinuria levels, which are important markers of kidney function. Other positive trends were observed in pain reduction, quality of life, and overall disease symptom assessment.
Dr. Yan Ouyang, an investigator from Ruijing Hospital and Professor Nan Chen’s team, presented the interim results at the symposium. Dr. Ouyang emphasized that the findings offer exciting potential for AL01211, noting the importance of addressing the unmet needs in Fabry disease treatment. “The 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 validating these findings in ongoing research and ultimately bringing this much-needed therapy to patients,” Dr. Ouyang stated.
Michael Babcock, Head of Research and Development at AceLink Therapeutics, also highlighted the significance of the study’s results. “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.”
AL01211 is a proprietary, non-brain-penetrant glucosylceramide synthase (GCS) inhibitor that has demonstrated high potency and selectivity, with a single-digit nanomolar IC50. Its favorable pharmacological properties support once-daily oral administration, making it a promising alternative to enzyme replacement therapy (ERT), which typically requires frequent intravenous infusions. The ability to provide an oral treatment option could represent a major breakthrough for Fabry disease patients, who currently rely on ERT as a primary treatment option.
GCS inhibitors, like AL01211, work by targeting the enzyme glucosylceramide synthase, which catalyzes the first step in the production of glycosphingolipids—bioactive molecules involved in a variety of cellular functions and associated with diseases such as Fabry and Gaucher disease. By inhibiting this enzyme, GCS inhibitors reduce the synthesis of glycosphingolipids, which helps prevent their accumulation in tissues, a hallmark of both diseases.
Founded in 2018, AceLink Therapeutics is focused on developing oral SRTs to address the unmet medical needs of patients with inherited disorders of glycosphingolipid metabolism. The company’s pipeline includes the Phase 2 program for Fabry disease as well as Type 1 Gaucher disease. By pioneering oral therapies for these diseases, AceLink aims to improve the quality of life for patients and offer a more accessible treatment option compared to current intravenous therapies.
As the Phase 2 trial continues, AceLink remains focused on advancing AL01211 and its other pipeline candidates to provide innovative solutions for patients affected by rare, debilitating diseases like Fabry disease.
