Ovid looks to biomarkers in new rare disease deal
- Ovid Therapeutics is bolstering its focus on the genetic disorder Angelman syndrome, inking a deal with biotech NeuroPointDX to find biomarkers to support clinical development of OV101 (gaboxadol).
- The collaboration between the two companies aims to use NeuroPointDX's metabolomics platform technology to find markers that could predict which patients are most likely to respond to the treatment, and use this information to guide recruitment into clinical trials.
- The deal is the second signed this year for the clinical-stage rare disease drugmaker. Last month,Ovid hooked up with Japanese company Takeda to co-develop TAK-935, a candidate for rare childhood epilepsy.
The deal with NeuroPointDX further solidifies the company's footing in the Angelman space, where no Food and Drug Administration-approved treatments exist.
Ovid's gaboxadol is in a Phase 1 study in Angelman syndrome in adults, and in preclinical development in adolescent and pediatric disease.
"The metabolic profile of [Angelman] syndrome is not well understood, and we believe this biomarker study will produce critical data to fill this gap and inform us about the impact of OV101 as a potential treatment option," Ovid CEO Matthew During said in a Feb. 9 statement.
"This collaboration is the first step in Ovid’s broader rare neurological disorder biomarker strategy to identify molecular markers of treatment responders and guide enrollment of participants in our clinical trials."
Biomarkers could also prove useful for the development of companion diagnostics once a drug is on the market.
The two companies did not disclose any financial information for the deal.
Gaboxadol has a somewhat varied past. Originally developed as an analgesic and anti-anxiety medication, Danish drugmaker Lundbeck and Merck & Co. later investigated it as a treatment for insomnia, though the drug failed in a late-stage trial.
Lundbeck then licensed gaboxadol to Ovid in 2015. Most recently, OV101 received orphan drug designation for the treatment of Angelman syndrome. The agent targets the disruption of tonic inhibition, and is also in development with Ovid for Fragile X syndrome.
With no drugs particularly targeting Angelman syndrome, the disease is typically treated with medications aimed at some of its more severe symptoms, such as seizures. Physical and behavioral therapies are also commons treatments.
Angelman syndrome affects as few as 1 in 12,000 people in the U.S., and is most commonly caused by the loss of function of a single gene that codes for ubiquitin protein ligase E3A, an enzyme responsible for dismantling cell proteins. The disease is characterized by motor and mental disabilities.
- Ovid Therapeutics Statement
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