Ovid pens deal to offload twice-failed former lead program, positioning Healx to write new chapter in fragile X

Ovid Therapeutics has found a potential buyer for its onetime lead program. A little more than one year after gaboxadol’s value-crushing phase 3 flop, Healx has stepped in to see whether it can write another chapter in the long, failure-strewn history of the neurological disease drug. 

New York-based Ovid took gaboxadol, also known as OV101, into a phase 3 clinical trial in patients with the rare genetic disorder Angelman syndrome only to report the drug had failed to beat placebo late in 2020. Ovid responded to the setback by turning its attention to other candidates, but Healx sees a future for the molecule in other indications.

British artificial-intelligence-enabled drug discovery shop Healx has secured an option to license gaboxadol in the belief it may work in fragile X syndrome. Ovid put the drug candidate through a phase 2a trial in the indication, emerging with an initial efficacy signal but ultimately opting against further development.

Healx is looking to improve on the initial efficacy signal seen in phase 2a. Under the agreement, Healx will explore the effects of combination therapies featuring gaboxadol. Healx has already found a possible partner for gaboxadol, the nonsteroidal anti-inflammatory drug HLX-0201, using its AI platform and will evaluate that cocktail and other combinations. 

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If Healx exercises its option, Ovid will receive undisclosed clinical, regulatory and commercial milestones, indicating the extent to which the perceived value of the drug candidate has fallen. Ovid will retain the option to co-develop and co-commercialize gaboxadol with Healx but has no plans to run further trials of the molecule. 

The deal creates the possibility of another rebirth for gaboxadol, a molecule that was in development at Lundbeck and Merck 15 years ago. Lundbeck and Merck were developing gaboxadol as a sleeping pill but called time on the program in 2007. The candidate then sat on the shelf until 2015, when Ovid picked up the rights and outlined plans to run midphase trials in Angelman and fragile X.