AceLink Therapeutics, Inc. has unveiled promising results from their Phase 1 trial of AL01211, a novel therapeutic for genetic diseases. The study demonstrated the medication’s safety, tolerability, and pharmacokinetic and pharmacodynamic effects in healthy volunteers. With the successful completion of this trial, AceLink is one step closer to providing transformative treatments for those living with genetic disorders.
AL01211 is an exciting, new potential treatment for Fabry disease that is currently undergoing clinical trials! This Phase 1 trial is a randomized, double-blind, placebo-controlled, dose escalation study of AL01211 in 69 healthy adults. As an oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi), AL01211 holds great promise for those suffering from Fabry disease.
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We are thrilled with the promising results of our first-in-human trial of AL01211, which demonstrated a clean safety profile with no significant or serious adverse events. Furthermore, AL01211 treatment led to a dose-dependent reduction of plasma GL1 and GL3, indicating successful inhibition of GCS. Our Phase 2 program in patients with Fabry disease is set to commence later this year, providing a potentially transformative therapy for those who need a more convenient and effective alternative to enzyme replacement therapy.
Data Highlights:
AL01211 is an exciting new treatment for Fabry disease, with promising results in preclinical studies and healthy volunteers. It has been found to have a dose-dependent pharmacokinetic and pharmacodynamic profile, with reductions in plasma GL1 and GL3 levels. It also shows excellent distribution in peripheral organs, with no blood-brain barrier penetration, making it an ideal candidate for life-long treatment in younger patients. AL01211 has been found to be safe and well-tolerated, with no significant or serious adverse events reported. Overall, AL01211 is a potential game-changer in the treatment of Fabry disease, offering a safer and more efficacious treatment option.
About AL01211
AL01211 is an innovative, non-brain penetrant GCS inhibitor with remarkable potency and selectivity, offering a much-needed oral small molecule therapy as an alternative to enzyme replacement therapy for Fabry disease. This powerful drug has the potential to provide significant relief to patients, with Phase II clinical studies set to start in 2023. AL01211 promises to revolutionize the treatment of this debilitating condition, with once-daily oral administration providing an effective and convenient solution.
About GCS inhibitor
GCS (glucosylceramide synthase) is an essential enzyme in the production of glycosphingolipids, a group of bioactive molecules that are involved in various cellular processes and diseases. By inhibiting GCS, it is possible to reduce the synthesis of glycosphingolipids and offer beneficial effects for diseases such as Fabry disease and Gaucher disease, which are caused by the accumulation of these lipids. Thus, GCS inhibitors can be used to improve the quality of life of those affected by these diseases.