First patient dosed in Phase 2 study of oral therapy candidate AL01211

AceLink Therapeutics is recruiting classic Fabry disease patients at sites in China

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The first Fabry disease patient has been dosed in a Phase 2 clinical trial that’s testing the experimental oral therapy AL01211.

The Phase 2 study (NCT06114329) is expected to enroll 18 male patients with classic Fabry disease, ages 18 to 60, who have not received any prior disease-specific treatment. All participants in the trial will receive treatment with AL01211, with the main goals of evaluating the therapy’s safety profile and pharmacological properties.

Recruitment is ongoing at six sites in China, according to the therapy’s developer AceLink Therapeutics.

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“We are delighted to initiate enrollment in our Phase 2 study for patients with Fabry disease at one of the most renowned academic centers in China,” Pedro Huertas, MD, PhD, AceLink’s chief medical officer, said in a press release.

“Today’s achievement reflects the dedication and hard work of our talented team and the unwavering support from our investigators,” Huertas added.

The principle investigator for the study is Nan Chen, MD, at Ruijin Hospital in Shanghai.

“I am excited to have dosed the first patient in the company’s Phase 2 program for AL01211 and am dedicated to maintaining the highest standards throughout the trial,” Chen said.

Fabry disease is caused by mutations in the gene GLA. This gene provides instructions to produce an enzyme, alpha-galactosidase A (Gal A), which  normally is needed to break down certain fatty molecules such as globotriaosylceramide (Gb3 or Gl-3).

Without a working Gal A enzyme, these fatty molecules build up to toxic levels, ultimately leading to disease symptoms. In recent years, enzyme replacement therapies have become available that work to deliver a working version of the Gal A enzyme.

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“Despite the advancements made over the years, a significant gap remains in the treatment landscape for Fabry disease. Enzyme replacement therapy necessitates frequent IV [into-the-bloodstream] infusions, which is an inconvenience for patients. Long-term enzyme replacement treatment may slow down disease progression, but cardiac, renal, and other complications still develop in most patients,” Chen said.

AL01211 is designed to block the activity of glucosylceramide synthase (GCS), a protein that promotes the production of Gb3 and other related fatty molecules. By blocking this protein, AL01211 is expected to act as a substrate reduction therapy, blocking the toxic buildup of fatty molecules to lessen Fabry disease progression.

“AceLink’s proprietary GCS inhibitor provides patients with a convenient, oral option that overcomes the challenges of current treatment modalities,” Chen said.

AL01211 was generally well-tolerated in Phase 1 studies in healthy volunteers conducted in Australia (NCT04908462) and China (ChiCTR2200061431). The experimental treatment also has been granted orphan drug designation in the U.S., which is designed to incentivize the development of treatments for rare diseases.