AL01211, a novel, oral, non-brain penetrant GCS inhibitor, was safe and well-tolerated in a Phase 1 study –

In healthy volunteers, AL01211 displayed dose-dependent pharmacodynamic effects –

Phase 2 studies in patients with Fabry disease are planned to start in 2023 –

NEWARK, CA, USA I February 23, 2023 I AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today presented positive data from a Phase 1 trial evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL01211 in healthy volunteers.

AL01211 is a novel, oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi) being developed for the treatment of Fabry disease. The Phase 1 trial is a randomized, double-blind, placebo-controlled, dose escalation study of AL01211 in 69 healthy adult participants.

The data is being presented as a poster presentation during the 2023 WORLD Symposium, being held in Orlando, Florida, from February 22- 26, 2023.

“We are encouraged by the results of our first-in-human trial. AL01211 achieved a clean safety profile with no significant or serious adverse events,” said Jerry Shen, Ph.D., Chief Executive Officer and Founder of AceLink. “In addition, AL01211 treatment led to dose-dependent reduction of plasma GL1 and GL3, which is the pharmacodynamic biomarker of GCS inhibition. AL01211 has the potential to be a transformative therapy for Fabry patients who desperately need more convenient and more effective alternatives to enzyme replacement therapy. We look forward to commencing our Phase 2 program in patients with Fabry disease later this year.”

Data Highlights:

  • AL01211 was safe and well-tolerated, showing no significant or serious adverse events in healthy volunteers.
  • Treatment with AL01211 resulted in dose-dependent PK and PD (reduction of plasma GL1 and GL3) properties.
  • In preclinical studies, AL01211 was widely distributed in peripheral organs and lacked blood-brain barrier penetration. This makes AL01211 an excellent choice for treating diseases that primarily affect peripheral tissues.
  • The increased potency and low brain penetration make AL01211 a potentially safer and more efficacious molecule for treating Fabry disease patients, especially in younger patients seeking a convenient and life-long treatment option.

Details on the AceLink Word Congress Presentation

Abstract Title: Development of AL01211, an oral, non-brain penetrant glucosylceramide synthase inhibitor (GCSi), to treat Fabry disease
Authors: Michael Babcock, Jianhong Zheng, Li Li, Jessica Gail Shurr, Marvin Garovoy, Jerry Shen
Session Title: Translational Research – Poster Session IV
Session Date & Time: Thursday, February 23, 4:00 PM – 5:00 PM ET
Abstract Number: LB-08
Location: Hilton Orlando, Kiosk 8-A

About AL01211

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 enzyme replacement therapy for Fabry disease that require frequent intravenous infusions. Phase II clinical studies of AL01211 in patients with Fabry disease are planned to start in 2023.

About GCS inhibitor

GCS (glucosylceramide synthase) 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 production of glycosphingolipids, thereby exerting beneficial effects to diseases such as Fabry disease and Gaucher disease, which are caused by the 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

SOURCE: AceLink Therapeutics