– Plan to enroll patients across six sites throughout China –

– Topline results are expected in 2H 2024 –

(BUSINESS WIRE)–AceLink Therapeutics, Inc., a clinical-stage biopharmaceutical company developing the next generation of oral substrate reduction therapies (SRTs), announced today that the first patient has been dosed with AL01211 in its Phase 2 trial in China in patients with Fabry disease.

AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase (GCS) inhibitor being developed to treat glycosphingolipid storage diseases, including Fabry Disease and Type 1 Gaucher Disease. In Phase 1 trials, AL01211 was generally well tolerated without significant adverse events.

“We are delighted to initiate enrollment in our Phase 2 study for patients with Fabry disease at one of the most renowned academic centers in China,” said Dr. Pedro Huertas, M.D. Ph.D., Chief Medical Officer of AceLink Therapeutics. “Today’s achievement reflects the dedication and hard work of our talented team and the unwavering support from our investigators. We believe AL01211, as the best-in-class GCS inhibitor, will provide a meaningful therapeutic benefit for the disorders of glycosphingolipid metabolism. We look forward to providing updates as the trial progresses and remain committed to bringing this promising therapy one step closer to those patients who need it most.”

The 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 been previously treated. AceLink plans to enroll 18 patients across six sites throughout China. Dr. Nan Chen at Shanghai’s Ruijin Hospital is serving as the Principal Investigator leading the efforts. AceLink expects topline results in 2H 2024.

“Despite the advancements made over the years, a significant gap remains in the treatment landscape for Fabry disease. Enzyme replacement therapy necessitates frequent IV infusions, which is an inconvenience for patients. Long-term enzyme replacement treatment may slow down disease progression, but cardiac, renal, and other complications still develop in most patients,” said Dr. Nan Chen, Professor of the Department of Nephrology at Ruijin Hospital. “AceLink’s proprietary GCS inhibitor provides patients with a convenient, oral option that overcomes the challenges of current treatment modalities. I am excited to have dosed the first patient in the company’s Phase 2 program for AL01211 and am dedicated to maintaining the highest standards throughout the trial.”

About AL1211

AL01211 is a proprietary, non-brain penetrant GCS inhibitor with excellent potency (single-digit nanomolar IC₅₀), great selectivity, and other favorable drug properties that support once-daily oral administration. AL01211 offers a much-needed oral small molecule therapy as an alternative to enzyme replacement therapy for Fabry disease obviating frequent intravenous infusions. Phase 2 clinical studies of AL01211 in patients with Fabry disease are planned to start in 2023.

About GCS inhibitor

GCS catalyzes the first step in the synthesis of glycosphingolipids, a group of bioactive molecules that play important roles in various cellular processes and diseases. GCS inhibitors reduce the synthesis of glycosphingolipids, thereby exerting beneficial effects to diseases such as Fabry and Gaucher diseases, which are caused by the accumulation of these lipids.

About AceLink Therapeutics, Inc.

Founded in 2018, AceLink Therapeutics is an innovative biopharma startup focusing on developing safe and effective medicines to address genetic diseases with high unmet needs. The company’s initial focus is to develop novel therapeutics for Fabry disease. For more information, please visit www.acelinktherapeutics.com.