Relay plans clinical development of Fabry disease treatment

Relay Therapeutics said it expects to start clinical development of its treatment candidate for Fabry disease in the second half of 2025.

The program in Fabry disease, together with other programs under development for another genetic disease and solid tumors, were disclosed in a June 6 event, “New Program & Platform.” A replay of the webcast and supporting materials are available on the company’s website.

“We are very excited to unveil the next set of innovative programs … which we believe will drive the next wave of the company’s growth,” Sanjiv Patel, MD, president and CEO of Relay, said in a company press release. “These new programs underscore the … expansion beyond precision oncology into genetic disease and beyond inhibitors to small molecule chaperones.”

Fabry disease is caused by mutations in the GLA gene, leading to reduced or absent activity of the alpha-galactosidase A (alpha-Gal A) enzyme that is responsible for breaking globotriaosylceramide (Gb3), a fatty molecule. This results in the toxic accumulation of Gb3 in blood cells and several tissues, causing a wide range of symptoms.

Potential treatment alone or with ERT

Using its Dynamo platform, Relay was able to create a non-inhibitory chaperone to stabilize the alpha-Gal A protein without inhibiting its activity, allowing greater clearance of Gb3 from tissues in people with Fabry. Chaperones are proteins that assist in the correct folding of larger proteins such as alpha-Gal A.

According to Relay, the molecule could be a potential chronic treatment for people with Fabry disease, either alone or in combination with enzyme replacement therapy (ERT), one of the mainstay treatments for the condition.

ERT works by delivering a functional alpha-Gal A enzyme to patients, reducing Gb3 toxic accumulation. This is expected to slow or prevent the progression of organ damage that characterizes the disease.

Dynamo works in three steps, the first involving understanding the target protein structure and identifying novel binding sites for new therapeutic agents that could modulate a protein’s behavior without affecting its function. Next, researchers transition to identify a chemical starting point to develop a lead compound, which then is optimized to obtain a molecule that has the most adequate characteristics regarding selectivity, potency, bioavailability, and drug-like properties.

In the eight years since Relay was founded, “our Dynamo platform has been very productive and we have made significant progress advancing our initial set of programs, including four that have entered the clinic,” Patel said.