– This first in human clinical study demonstrated that AL01211 was generally safe and well-tolerated at single doses up to 60 mg and at multiple doses up to 30 mg
– AL01211 displayed dose dependent pharmacokinetics and the ability to effectively reduce the disease biomarkers glucosylceramide and globotriaosylceramide in healthy volunteers
– Topline results from a Phase 2 study of AL01211 in patients with Fabry disease are expected in the second half of 2024
(BUSINESS WIRE)–AceLink Therapeutics, Inc., a clinical-stage biopharmaceutical company developing next generation oral substrate reduction therapies (SRTs), today announced that the findings from their Phase 1 study of AL01211 in healthy volunteers have been published in the peer-reviewed journal Clinical Pharmacology in Drug Development, a journal of the American College of Clinical Pharmacy.

AL01211 is a potent, oral, glucosylceramide synthase (GCS) inhibitor being developed for the treatment of Fabry disease and Type 1 Gaucher disease. In contrast to other GCS inhibitors in development, AL01211 has minimal central nervous system penetration (CNS), allowing therapeutic benefits to peripheral organs while avoiding the risk of CNS-associated adverse effects.

“These results highlight the transformative potential of AL01211 as a best-in-class GCS inhibitor,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink Therapeutics. “Our Phase 1 study provided the critical safety and biomarker data to support our ongoing Phase 2 clinical trial in patients with Fabry Disease. We expect to have topline results of the Phase 2 trial in the second half of 2024.”

In the published Phase 1 study, AL01211 was evaluated with a single ascending dose arm and a multiple ascending dose arm to determine the safety, pharmacokinetics (PK), and pharmacodynamic (PD) effects in healthy volunteers. Overall, AL01211 was generally safe and well-tolerated with no serious adverse events. At a 30 mg dose level, plasma glucosylceramide and globotriaosylceramide were reduced from baseline levels by 78% and 52%, respectively, thus supporting AL01211’s further clinical development.

“These data reinforce our commitment to provide more convenient and more effective therapeutic options to patients suffering from glycosphingolipid storage diseases such as Fabry disease,” said Michael Babcock, Ph.D., VP of Research and Early Development. “In our trial, AL01211 demonstrated dose-dependent PK and PD effects with a clear correlation between AL01211 exposure and reduction in disease biomarkers. The insights gained from this study have set us up nicely to test the therapeutic benefits of AL01211 in patients.”

In October 2023, AceLink Therapeutics dosed its first patient in a Phase 2 open-label study evaluating the safety, PK, and PD of AL01211 in males with classic Fabry disease who have not been previously treated. Topline results from this study are expected in the second half of 2024.

About AL1211

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.