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.
Gaboxadol has previously been 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, D.Phil, MB BChir, Chairman and Chief Executive Officer 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.”
“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,” commented Dr. Tim Guilliams, co-founder and CEO at Healx. “We are excited to be deploying our innovative combination and indication expansion methods to identify new therapeutic opportunities for compounds like 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 announcement last year that it had entered an indication expansion partnership with Japan-based Ono Pharmaceutical Co. Ltd. Collaborations like those with Ono Pharmaceutical and Ovid Therapeutics are an important part of Healx’s mission to deliver novel, effective treatments to rare disease patients in need. The Healx team is open to exploring other opportunities with industry, patient, and academic groups. Interested parties can find out more about partnering here.
About Fragile X Syndrome
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.
About Healx
Healx is a mission-driven technology company pioneering the next wave of drug discovery in order to bring novel, effective treatments to rare disease patients around the world. There are 7,000 known rare diseases that affect 400 million people across the globe, but only 5% of those conditions have an approved treatment. By combining frontier AI technology with deep drug discovery and development expertise, Healx can accelerate the pace, increase the scale, and improve the chance of success of rare disease treatment development in order to meet this huge unmet need and have unprecedented patient impact.
Founded in Cambridge, UK, in 2014 by Dr. Tim Guilliams (a biochemical engineer and tech entrepreneur) and Dr. David Brown (co-inventor of Viagra and former Global Head of Drug Discovery at Roche), Healx has raised around $70 million to date, added nearly 20 projects to its risk-balanced therapeutic portfolio, and built a cross-functional team of over 100 tech and drug discovery experts. For more information, visit www.healx.io or follow on Twitter and LinkedIn.
About Ovid Therapeutics
Ovid Therapeutics Inc. is a New York-based biopharmaceutical company using its BoldMedicine® approach to develop medicines that transform the lives of patients with neurological disorders. Ovid seeks to couple deep CNS experience with emerging advances in genetics and the pathways of the brain to build a leading, next-generation neuroscience pipeline. Ovid’s current pipeline programs include: OV329, a small molecule GABA aminotransferase inhibitor for seizures associated with tuberous sclerosis complex and infantile spasms; OV350, a direct KCC2 activator for potential treatment of resistant epilepsies; OV882, a short hairpin RNA therapy approach for Angelman syndrome; OV815, a genetic therapy approach for KIF1A-associated neurological disorders; and other research targets. Additionally, Ovid maintains a significant financial interest in the future regulatory development and potential commercialization of soticlestat, which Takeda is responsible for advancing globally. Two Phase 3 trials for soticlestat in Dravet syndrome and Lennox-Gastaut syndrome are active. For more information on Ovid, please visit www.ovidrx.com.