Gaboxadol in Combo Therapy for Fragile X Worth Pursuing: Healx

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by Patricia Inácio, PhD |

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Healx has entered into an exclusive licensing agreement with Ovid Therapeutics to develop and commercialize gaboxadol in a potential combination treatment for fragile X syndrome and other disorders.

The announcement follows Ovid’s decision to discontinue the development of gaboxadol (previously called OV101), despite it being deemed safe and well-tolerated in multiple clinical trials.

Healx will now investigate combining gaboxadol with other compounds, including its candidate therapy HLX-0201, in treating rare diseases, starting with fragile X syndrome.

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“Gaboxadol shows encouraging activity and a good safety profile in a range of conditions. We are keen to see its clinical value unlocked,” Jeremy Levin, PhD, chairman and CEO of Ovid Therapeutics, said in a press release.

“We’re impressed by the capabilities that Healx has developed and are optimistic that they can unlock effective drug combinations that will take advantage of the unique mechanism that underlies the effects we see when patients take gaboxadol,” he added.

HLX-0201 (sulindac), initially approved as a nonsteroidal anti-inflammatory drug (NSAID), showed an ability to modify several behaviors characteristic of fragile X syndrome in mouse models of the disease. The therapy’s safety, tolerability and efficacy is to be investigated in a Phase 2 study (NCT04823052) in boys and men with fragile X.

Trial enrollment is not yet open, but site and contact information is available here.

HLX-0201 was identified using Healx’s artificial intelligence (AI)-driven approach to therapy discovery. Its AI platform cross-analyzes disease gene expression profiles with the company’s therapeutic database to find connections and pathways between the two. Of note, gene expression is the process by which information in a gene is synthesized to create a protein.

“This agreement represents an exciting opportunity for Healx to advance more potential treatments for rare conditions towards the clinic, and, hopefully, deliver them to patients in need,” said Tim Guilliams, PhD, a Healx co-founder and its CEO.

A Phase 2 trial (NCT03697161) of gaboxadol in 23 fragile X patients, called ROCKET and sponsored by Ovid, showed that a 5 mg dose given once, twice, or three times each day for 12 weeks was well-tolerated. Data also indicated that gaboxadol significantly eased patients’ behavioral and functional symptoms.

In announcing its 2021 decision to stop work on gaboxadol, however, Ovid reported that combined efficacy results in a clinical trial in Angelman syndrome patients failed to meet expectations.

Under the terms of the agreement, Ovid will receive milestone payments associated with gaboxadol’s development if specific clinical, regulatory, and commercial goals are met. It is also eligible to receive tiered royalties according to net revenues associated with the marketing of therapies with gaboxadol.

Ovid will retain an option to partner with Healx in the co-development and co-commercialization of gaboxadol therapies, sharing net profits and losses.