(BUSINESS WIRE)–AceLink Therapeutics, Inc., a clinical stage biopharmaceutical company, today announced the opening of the first clinical trial site in China for its Phase 2, open‑label study of the safety, pharmacokinetics (PK), pharmacodynamics (PD) and preliminary measures of physiological efficacy of AL01211 in males with classic Fabry disease who have not been previously treated with other Fabry disease therapies. AceLink is developing the next generation oral substrate reduction therapies (SRTs) to address significant unmet medical needs and improve the quality of life of patients with inherited disorders of glycosphingolipid metabolism.
AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease. It has completed a phase 1 clinical trial in healthy volunteers in Australia and a bridging phase 1 trial in China under IND approved by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). Both trials showed that AL01211 is generally well tolerated without significant AEs and that the expected plasma glycosphingolipids were reduced with increasing dose demonstrating a clear PK and PD correlation.
“The opening of our first clinical trial site in China demonstrates our ongoing commitment to Fabry Disease and is an important step in our efforts to provide these patients with a convenient, oral, therapeutic option,” said Dr. Pedro Huertas, M.D., Ph.D., Chief Medical Officer of AceLink Therapeutics. “We thank the patients in our ongoing clinical trials and their families, as well as our investigators and the broader Fabry community, for their ongoing trust and collaboration as we work to advance our investigational therapy in the clinic.”
The phase 2 study is now actively screening and enrolling patients across multiple sites in China. Dr. Nan Chen at Shanghai’s Ruijin Hospital is the Principal Investigator (PI) leading the efforts. In addition to Ruijin Hospital, five other sites are expected to open in Q3 of 2023. AceLink expects top line results in 2H 2024.
“The symptoms of Fabry disease are extremely burdensome, have a negative impact on the quality of life of both patients and their families,” said Dr. Nan Chen, Professor of the Department of Nephrology at Ruijin Hospital. “Even with advancements made in enzyme replacement therapies, the potential of immune reaction and the reoccurring need for IV infusion highlights a significant unmet need for an effective oral therapy. With this in mind, we are truly excited about our participation in this trial, as we strive to improve the daily lives of patients with Fabry disease.”
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.