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.
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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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By GlobalDataIn 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.”
