Ovid Therapeutics Rare Disease Drug Fails in Phase 3 Study

Ovid Therapeutics recently announced that the Phase 3 clinical trial of its rare disease drug for the treatment of Angelman syndrome did not meet primary endpoints. 

Specifically, OV101, or gaboxadol, elicited an 0.7 point improvement in Angelman syndrome over baseline, while placebo showed an 0.8 point improvement in Angelman syndrome. 

The company said it will continue to evaluate secondary endpoints.

But initial results show no difference between OV101 and placebo, so far, researchers stated.  

“We are deeply disappointed with the outcome of the NEPTUNE trial which did not achieve its primary endpoint,” Jeremy Levin, DPhil, MB, BChir, chairman and chief executive officer of Ovid Therapeutics, said in the announcement. 

“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,” he continued. 

The Phase 3 NEPTUNE study enrolled and treated 97 patients four to 12 years of age who were diagnosed with Angelman syndrome. Seven of the patients were two to three years of age to evaluate safety and pharmacokinetic evaluation only. 

Overall, OV101 was well-tolerated with no significant safety issues observed. Researchers stated they will complete a full analysis of the results of the NEPTUNE study and discuss the results with FDA to determine next steps.

Additionally, Ovid Therapeutics will continue to offer the investigational drug to patients enrolled in the open-label extension trial (ELARA) based on further analysis of the NEPTUNE study.

The company expects to report data from the ELARA study in the first quarter of 2021.

“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,” said Amit Rakhit MD, president and chief medical officer. 

“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,” Rakhit continued. 

Moving forward, Ovid Therapeutics will focus its future development efforts on its additional late-stage asset, OV935, intended for two rare epilepsies: Dravet and Lennox Gastaut syndrome.

Along with Takeda Pharmaceuticals, Ovid Therapeutics will also launch clinical trials for OV935 for use in infected patients. 

Dravet, Lennox Gastaut, and Angelman syndrome are three types of epileptic diseases that begin in infancy or early childhood and can elicit symptoms ranging from mild to severe.

Currently, there is no treatment for these rare diseases, which leaves room for biopharmaceutical companies to work together and develop potential drugs for use in infected patients.

In mid-June, Ovid Therapeutics and Columbia University Irvin Medical Center announced a strategic collaboration to advance genetic-based therapies for rare neurological conditions, complementary to Ovid’s current pipeline. 

Under the research collaboration, Columbia will combine its expertise in rare disease genetics with Ovid Therapeutics’ discovery, translational, and clinical development expertise in neurodevelopmental disorders and rare epilepsies.

This collaboration provides Ovid with the potential to expand its future drug development portfolio and impact individuals living with rare genetic neurological conditions.