The US Food and Drug Administration (FDA) has given clearance to AceLink Therapeutics to begin a Phase II clinical trial of AL1211 for the treatment of Fabry Disease, a rare inherited glycosphingolipid storage disorder.
The randomised, dose-blinded study will evaluate AL1211’s pharmacological activity and safety in male patients with classic Fabry disease.
Patients who are willing to discontinue enzyme replacement therapy and switch to substrate reduction therapy are being enrolled in the study.
AceLink Therapeutics founder and CEO Jerry Shen said: “FDA clearance for AL1211 marks an important clinical milestone for AceLink.
“We are committed to providing a differentiated oral therapy for patients with Fabry disease and Type I Gaucher disease, who are desperately in need of a more convenient alternative to enzyme replacement therapy, and ultimately a better quality of life.”
AL1211 is a non-brain penetrating oral inhibitor of glucosylceramide synthase that is also used to treat Type I Gaucher disease by improving organ function and reducing inflammation.
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In a Phase I study, healthy volunteers were administered once a day with one dose of up to 60mg of AL1211 or multiple doses of up to 30mg for 14 days.
AL1211 was found to be generally well-tolerated and safe.
In another Phase I trial, patients administered with a 30mg dose of AL1211 showed a 78% reduction in glucosylceramide.
Fabry disease causing the lipid Globotriasosylceramide was also reduced.
AceLink chief medical officer Pedro Huertas said: “Given the encouraging results from our Phase I study, we look forward to further investigating AL1211 in our Phase II programme as we strive to bring a novel treatment option to those patients who need it most.
“Our clinical team has diligently prepared for this moment and we are eager to enrol patients as rapidly as possible.”