Ovid Therapeutics Inc. (NASDAQ:OVID) has a reported topline data from its third phase NEPTUNE clinical study of OV101 (gaboxadol) in Angelman syndrome treatment.
NEPTUNE study fails to achieve the primary endpoint
The NEPTUNE third phase study was a double-blind, placebo-controlled, and randomized trial that enrolled and treated 97 Angelman syndrome patients. The subjects were 4-12 years of age and included seven patients aged 2-3 years diagnosed with Angelman syndrome. The study evaluated the effects of OV101 treatment (once-daily, oral dosing) compared to placebo for 12 weeks. The study’s key primary endpoint was the overall score change on the Clinical Global Impression-Improvement-Angelman syndrome scale. The study’s secondary endpoints included communication, sleep, socialization, motor function, behavior domains, and daily living skills.
The study didn’t achieve its primary endpoint as patients that received OV101 only showed 0.7 point improvement over baseline in CGI-I-AS. On the other hand, those in the placebo group achieved 0.8 point improvement CGI-I-AS. The company continues to evaluate secondary endpoints, although preliminary results indicated no change between OV101 and placebo.
Interestingly OV101 showed favorable tolerability without any treatment-related safety issues reported. The company is planning to finalize full analysts of the NEPTUNE study results and discuss the data with the US FDA to ascertain its next steps of the program. Ovid plans to continue offering OV101 to patients recruited in the open-label extension study (ELARA) pending analysis of the NEPTUNE study results. Data from the ELARA will be available in Q1 2021.
Ovid halts OV101 study
Jeremy Levin, the company’s CEO and Chairman said that Ovid is disappointed that the NEPTUNE trial did not achieve its primary endpoint. He added that besides the ongoing ELARA trial, the company is planning to halt the OV101 program in Angelman syndrome, subject to the completion of a full analysis of the results and discussions with investigators and regulators.
Levin added that the focus now is on the development of OV935 in Lennox-Gastaut and Dravet syndromes, which are rare epilepsies. Ovid will initiate the pivotal trials on the conditions in 1H 2021 in partnership with Takeda.