Rare Daily Staff

Ovid Therapeutics has found a potential buyer in Healx for its lead experimental therapy for Fragile X syndrome experimental therapy gaboxadol, a little more than one year after the compound failed in a late-stage trial.

Ovid said that AI-driven rare disease biotech Healx has secured an exclusive option to license rights to develop and commercialize gaboxadol. Under the agreement, Healx plans to investigate the compound as part of a potential combination therapy for Fragile X syndrome, as well as a treatment for other indications.

Fragile X syndrome affects roughly 1 in 4,000 males and 1 in 8,000 females and causes a range of developmental problems including behavioral problems and cognitive impairment. Currently, there are no effective treatments or cures for the condition, but Healx is launching an adaptive clinical study to evaluate the efficacy of several compounds against the disease, with the ultimate aim of bringing at least one novel and effective combination therapy for the condition to market in the coming years.

“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, co-founder and CEO at Healx.

Gaboxadol was previously tested as a monotherapy for Fragile X syndrome in a phase 2a clinical study by Ovid. Healx will now seek to find an optimal treatment for the condition by combining gaboxadol with other compounds, including HLX-0201, that have been identified by the company’s proprietary AI platform, Healnet. Alongside the initial focus on Fragile X syndrome, Healx plans to use its cutting-edge indication expansion technology to identify other conditions that could also be treated by gaboxadol.

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

Should Healx exercise its option, Ovid will receive milestone payments for specific clinical, regulatory, and commercial achievements associated with gaboxadol’s development. Additionally, Healx will pay Ovid tiered royalties on net sales associated with marketed therapies containing gaboxadol. Ovid will retain the option to become Healx’s co-development and co-commercialization partner and will share net profits and losses in lieu of the milestones and royalty payments. Ovid does not plan to conduct further trials with gaboxadol.

This collaboration with Ovid follows Healx’s indication expansion partnership with Japan-based Ono Pharmaceutical and Healx says are an important part of the company’s mission to deliver novel, effective treatments to rare disease patients in need.

Photo: Tim Guilliams, co-founder and CEO at Healx

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