AceLink Therapeutics, a clinical-stage drug development company, announced today that AL01211, the company’s innovative oral, small molecule drug that is being developed for Fabry disease, was granted “Breakthrough Therapy designation” by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA).

Fabry disease is a rare hereditary lysosomal storage disease that affects the kidney, heart, peripheral nervous system and skin, and can cause a wide range of mild to severe signs and symptoms, and lead to life-threatening consequences like kidney failure, heart disease, and stroke. AL01211 is a novel selective oral glucosylceramide synthase (GCS) inhibitor being developed by AceLink Therapeutics, which can reduce the synthesis of pathogenic substrates for Fabry disease, and potentially improve patients’ symptoms, quality of life and prognosis. Compared with the existing enzyme replacement therapy that requires intravenous infusion every two weeks, AL01211 is a more convenient oral therapy that can efficiently penetrate disease-related tissues to reduce the deposition of pathogenic substrates. Therefore, AL01211 is expected to provide patients with a safer and more effective therapeutic option, making up for the limitations of existing treatments.

Two Phase 1 clinical trials of AL01211 in healthy subjects have been completed in Australia and China, both showing a positive safety profile, and consistent and robust pharmacokinetic and pharmacodynamic responses. Both studies demonstrated that AL01211 was generally safe and well tolerated, with no serious adverse events observed at any dose level tested. Additionally, an ongoing Phase 2 clinical study of AL01211 in patients with Fabry disease in China is showing that AL01211 is generally safe, and preliminary pharmacodynamic and efficacy data shows significant reductions in substrate levels, stabilization of organ function, and improvement in quality of life.

AceLink CEO Wen Chen commented, “The CDE Breakthrough Therapy designation for AL01211 is another major milestone for the company in innovative drug development and clinical research. It will help further accelerate the clinical development of the product, enabling Fabry disease patients to access more treatment options faster, and potentially improving their quality of life and reducing the burden of the disease.”

About AL01211

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 the frequent intravenous infusions required with enzyme replacement therapy.

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 disease, which are caused by the pathogenic 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, or contact [email protected].