NEW YORK—Ovid Therapeutics Inc. has announced disappointing topline results from the company’s Phase 3 NEPTUNE clinical trial of OV101 (gaboxadol) — a delta-selective GABAA receptor agonist — for the treatment of Angelman syndrome. The NEPTUNE study failed to achieve its primary endpoint.
“We are deeply disappointed with the outcome of the NEPTUNE trial, which did not achieve its primary endpoint,” said Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics. “Other than the ongoing ELARA study, we plan to pause our OV101 program in Angelman syndrome pending a full understanding of this outcome and discussions with regulators and investigators.”
The NEPTUNE trial enrolled and treated 97 patients diagnosed with Angelman syndrome between 4-12 years of age, and 7 patients diagnosed with Angelman syndrome aged 2-3 years, for safety and pharmacokinetic evaluation only. The study was designed to assess the effects of treatment with OV101 (once-daily oral dosing) versus placebo over 12 weeks.
The sole primary endpoint was change in overall score on the Clinical Global Impression-Improvement-Angelman syndrome (CGI-I-AS) scale. Secondary endpoints included sleep, communication, motor function, socialization, daily living skills and behavior domains.
Patients who were given OV101 showed a 0.7 point improvement in CGI-I-AS over baseline. Patients given placebo showed a 0.8 point improvement in CGI-I-AS (p=NS). The company is still evaluating secondary endpoints, although initial results have shown no difference between OV101 and placebo. But there is one bit of bright news here: OV101 was well-tolerated, and researchers observed no significant safety issues.
“NEPTUNE is our first study focused on the pediatric and adolescent population in Angelman syndrome, and we will fully assess all the data from this trial to understand this outcome and determine next steps — if any — for OV101 in this and other conditions, including Fragile X syndrome,” noted Amit Rakhit M.D., president and chief medical officer of Ovid Therapeutics. “We are sincerely grateful for the commitment and dedication of patients, families, investigators and employees to this program, and in particular, to those who participated in the NEPTUNE trial.”
Ovid Therapeutics plans to complete a full analysis of the NEPTUNE study results, and to discuss these results with the FDA to determine the program’s future. The company will continue to offer OV101 to patients enrolled in the open-label extension trial (ELARA), pending further analysis of the NEPTUNE study. Ovid expects to report data from the ELARA study in the first quarter of 2021.
“For now, the focus of our future development efforts will concentrate on the development of our other late-stage asset, OV935, in two rare epilepsies: Dravet and Lennox-Gastaut syndromes. With our partner Takeda, we plan to initiate pivotal trials in these conditions in the first half of 2021,” Levin added.