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AceLink Therapeutics Receives Orphan Drug Designation for its Novel GCS inhibitor AL01211 for the Treatment of Fabry Disease 
Time: 2022-09-13
Source: Viva Biotech
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[Abstract]:Receiving an FDA Orphan Drug Designation further validates AL01211 as a novel, once-daily orally administered GCS inhibitor as a potential treatment to improve the quality of life of patients suffering from Fabry disease.

Newark, CA, September 7, 2022 - AceLink Therapeutics, Inc. (AceLink), invested and incubated by Viva BioInnovator, an innovative biopharmaceutical company developing transformative therapies for genetic diseases, announced it has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for AL01211 as a treatment for Fabry Disease.

 

“Receiving an FDA Orphan Drug Designation further validates AL01211 as a novel, once-daily orally administered GCS inhibitor as a potential treatment to improve the quality of life of patients suffering from Fabry disease,” said Marvin Garovoy, M.D., Chief Medical Officer of AceLink Therapeutics. “AL01211 was selected based on its unique properties to more effectively treat peripheral organs affected by Fabry Disease by reducing the risk of off-target effects by preventing the crossing of the blood-brain barrier.”  

 

In an effort to expand future access to AL01211, AceLink Therapeutics plans to conduct Phase II clinical studies involving Fabry disease patients who are either naive to enzyme replacement therapy or are seeking an alternative to enzyme replacement therapy. “Our objective is to rapidly advance AL01211, so we have initiated discussions with potential commercial partners equally committed to ensuring that Fabry Disease patients around the world have access to a novel, once-a-day oral therapy with the potential to dramatically improve their quality of life,” said Jerry Shen, Ph.D., Chief Executive Officer of AceLink Therapeutics.


 
About AL01211 


AL01211 is a proprietary GCS inhibitor with excellent potency (single-digit nanomolar IC50) and great selectivity against a broad range of therapeutic targets.  It possesses favorable drug properties and is intended for once-daily oral administration.  Phase II clinical studies of AL01211 in patients with Fabry disease are planned to start soon.  

 


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.  A GCS inhibitor reduces the production of glycosphingolipids, thereby exerting beneficial effects to diseases such as Fabry Disease and Gaucher disease. 

 

 

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. 

Media contact: vivapr@vivabiotech.com
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