AceLink’s AL01211 Granted FDA Orphan Drug Status for Fabry

Phase 2 trials planned for 2023 to test once-daily oral therapy in patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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AL01211, a once-daily oral therapy being developed by AceLink Therapeutics for Fabry disease, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA).

The FDA gives this designation to investigational treatments with the potential to improve care for rare diseases that affect fewer than 200,000 people in the U.S. The designation gives AceLink, as the therapy’s developer, certain incentives including tax credits, fee waivers, and the potential for seven years of market exclusivity if the FDA ultimately approves the treatment.

“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,” Marvin Garovoy, MD, AceLink’s chief medical officer, said in a company press release.

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AL01221 is designed to block the activity of GCS, or glucosylceramide synthase, which is an enzyme that facilitates the first step in the production of glycosphingolipids, a diverse group of biologically active fatty molecules. Fabry disease is caused by genetic mutations that result in an abnormal buildup of certain glycosphingolipids, particularly globotriaosylceramide (Gb3), inside cells.

By blocking the activity of GCS, AL01221 is designed to reduce this toxic buildup, ultimately slowing disease progression. According to AceLink, the investigational therapy has high potency against GCS and other pharmacological properties that are expected to allow for once-daily oral dosing. Of note, the therapy is unable to cross the blood-brain barrier, meaning it will not get into the brain or spinal cord if administered systemically.

“AL01211 was selected based on its unique properties, including high potency and inability to cross the blood-brain barrier, to more effectively treat the peripheral organs affected by Fabry disease,” Garovoy said.

AceLink is planning to conduct Phase 2 clinical testing of AL01221 in people diagnosed with Fabry disease who either have never been on enzyme replacement therapy, or are seeking an alternative for this current standard treatment. These studies are planned to begin next year.

“Our objective is to rapidly advance AL01211, so we have initiated discussions with potential commercial partners that are 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, PhD, CEO of AceLink.