FDA Agrees to Phase 2 Trial of HLX-0201 in Treating Fragile X

Yedida Y Bogachkov PhD avatar

by Yedida Y Bogachkov PhD |

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HLX-0201 fragile X trial | Fragile X News Today | announcment illustration

A Phase 2a clinical trial of HLX-0201 in treating behavioral symptoms in people with fragile X syndrome was given U.S. Food and Drug Administration (FDA) approval to open, the therapy’s developer, Healx, announced.

Recruitment for the trial (NCT04823052) should begin in the coming months, the company stated in a press release.

HLX-0201 (sulindac) was initially approved as a non-steroidal anti-inflammatory drug (NSAID). The therapy, identified using Healx’s artificial intelligence (AI)-driven approach to drug discovery, was then validated in preclinical models of fragile X. In these models, it showed an ability to modify several behaviors similar to those seen in patients, the company reported.

Healx’s AI-approach uses an omic-based drug matching method, where the gene expression profile of a disease is compared to the gene expression profiles in the company’s drug database to find connections and pathways between the two.

“Receiving this IND [investigational new drug] approval is a critical step towards delivering effective treatments to fragile X patients around the world, and is a significant milestone in the development of Healx’s clinical pipeline. It is also a strong validation of our AI-driven drug discovery methods,” said Anthony Hall, chief medical officer at Healx.

“We believe that HLX-0201 has strong potential to address the huge unmet needs for fragile X patients as part of a combination, and we are looking forward to launching the first phase of the study as soon as possible,” Hall added.

The double-blind and randomized Phase 2 trial will open testing HLX-0201 at two doses against placebo to evaluate the potential treatment’s safety, tolerability, and evidence of early efficacy, all given in an outpatient setting over 10 weeks. It aims to recruit 36 boys and men with fragile X, ages 13 to 40.

It may also test HLX-0201 in combination with other fragile X treatments. “By targeting different disease processes and pathways, a combination of drugs has the potential to improve a greater range of symptoms experienced by a patient,” Healx stated in the release.

Study site and contact information is not yet available.

HLX-0201 is one of several compounds Healx is developing to treat fragile X. The company also noted that preclinical studies in a model of fragile X syndrome indicated that some of its identified compounds work in combination at lower doses with the same effectiveness achieved at higher doses when given as a single therapy (monotherapy).

Healx, based in Cambridge, U.K., has been working closely with the FRAXA Research Foundation in the U.S. on this potential therapy and its planned trial.

“We are excited about the potential of HLX-0201: a proven drug with a long track record of effectiveness,” said Mike Tranfaglia, MD, medical director and chief scientific officer at FRAXA. “It never would have occurred to us to investigate the compound for fragile X without Healx’s innovative technology pointing us in the right direction, but the preclinical studies have shown remarkable effectiveness.”

“We also believe that combinations of available drugs can treat many rare diseases, and we are glad Healx is leading the way in devising optimal combinations to treat fragile X,” Tranfaglia concluded.

HLX-0201 has been designated an orphan drug in both the U.S. and the European Union, a status given to support the development and testing of therapies aiming to treat rare diseases.