Ovid Stops Development and Testing of OV101 for Fragile X

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by Vanda Pinto |

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OV101 work stops

Ovid Therapeutics announced it is discontinuing work to further develop or test OV101 (gaboxadol) as a potential treatment of fragile X syndrome.

Although OV101 was found to be safe and well tolerated in multiple clinical trials, its overall efficacy in these studies fell short, Ovid Therapeutics said in a press release.

The company now plans to shift its focus to the development of its early stage pipeline, which include OV882, a short hairpin RNA (shRNA) therapy for patients with Angelman syndrome.

“Ovid has always taken a disciplined approach to its investments,” Jeremy M. Levin, PhD, chairman and CEO of Ovid Therapeutics, said. “While we are disappointed in the outcome for OV101, our resources are now squarely focused on a very promising pipeline in rare neurological diseases, including Angelman syndrome.”

OV101 was developed to selectively activate the GABAA receptor and restore tonic inhibition — a process that occurs in the brain and allows the identification of inhibitory and excitatory signals — in people with fragile X and Angelman syndromes.

Several clinical trials were carried out by Ovid Therapeutics to test its safety and efficacy for both disorders.

Results from the ROCKET Phase 2 trial (NCT03697161), which enrolled 23 males with fragile X, ages 13 to 22, showed that a 5 mg dose of OV101, once, twice, or three times each day for 12 weeks was well-tolerated. Data also suggested that OV101 could significantly eased behavioral and functional symptoms.

Meanwhile, other clinical trials evaluated the safety and efficacy of OV101 for Angelman syndrome. Although the company did not provide specifics, it reported that the combined results regarding OV101’s efficacy failed to meet expectations.

“Ovid would like to thank everyone who participated in our OV101 clinical trials, including those with Angelman syndrome and Fragile X syndrome, families and investigators, to whom we remain committed to finding solutions for rare neurological disorders,” Levin said.

Ovid also announced recently completing an agreement with Takeda Therapeutics giving that company global rights to develop and commercialize soticlestat (TAK-935/OV935) for the treatment of developmental and epileptic encephalopathies.

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