AceLink Therapeutics Publishes Positive Phase 1 AL01211 Trial Results in Healthy Subjects

AceLink Therapeutics, a biopharmaceutical firm, has reported positive preliminary findings for AL01211, an oral medication being developed to treat Fabry and Type 1 Gaucher diseases. The drug, which inhibits glucosylceramide synthase (GCS), showed it was safe and well-tolerated in a Phase 1 clinical trial involving healthy individuals. It also showed a dose-dependent response in reducing glucosylceramide and globotriaosylceramide levels, which are biomarkers for the diseases it aims to treat. AL01211 is notable for its minimal penetration into the central nervous system, which could minimize side effects associated with this area.
The Phase 1 study, published in the Clinical Pharmacology in Drug Development journal, included both single and multiple dose assessments. The drug demonstrated a significant reduction in the targeted biomarkers at a dose of 30 mg, with no serious adverse events reported. The company's CEO, Jerry Shen, highlighted the potential of AL01211 as a leading GCS inhibitor and confirmed that the Phase 1 data will support the ongoing Phase 2 trial in Fabry disease patients, with results expected in 2024.
AceLink Therapeutics initiated a Phase 2 study in October 2023, focusing on the safety and efficacy of AL01211 in untreated male patients with classic Fabry disease. The company's VP of Research and Early Development, Michael Babcock, emphasized the drug's dose-dependent pharmacokinetic and pharmacodynamic effects and its potential to offer a more convenient oral treatment option for patients with glycosphingolipid storage diseases.
AL01211 is a proprietary compound with potent GCS inhibition properties, designed for once-daily oral dosing. GCS inhibitors are beneficial in treating diseases where glycosphingolipids accumulate abnormally. AceLink Therapeutics, established in 2018, is dedicated to creating innovative treatments for genetic diseases with significant unmet medical needs, with an initial focus on Fabry disease.

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