RARE Daily

Ovid Therapeutics Grants Angelini Pharma Exclusive European Rights to Experimental Angelman Syndrome Therapy

July 13, 2020

Ovid Therapeutics granted Angelini Pharma exclusive development, manufacturing, and commercialization rights to OV101 for the potential treatment of Angelman syndrome in the European Union, Switzerland, United Kingdom, Turkey, and Russia.

Photo: Ovid Chairman and CEO Jeremy Levin

Under the terms of the agreement, Ovid will receive an upfront payment of $20 million and is eligible to receive up to an additional $212.5 million in payments based on the achievement of development, manufacturing, and sales milestones for the initial indication in Angelman syndrome, as well as double-digit royalties on net sales if OV101 is successfully commercialized. Ovid will retain all U.S. and rest-of-world commercial rights to OV101.

Angelman syndrome is a rare genetic condition characterized by symptoms that include delayed development, intellectual disability, severe speech impairment, problems with movement and balance, seizures, sleep disorders, and anxiety. The most common cause of Angelman syndrome is the loss of function of a gene that codes for a protein that plays a critical role in nerve cell communication. This loss of function results in impaired tonic inhibition, a central physiological process of the brain that is thought to be the underlying cause of certain neurodevelopmental disorders.

OV101 (gaboxadol) is believed to be the first experimental drug to specifically target the disruption of tonic inhibition. OV101 has been demonstrated in laboratory studies and animal models to impact neuronal activity through tonic inhibition. Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and relieve several of the symptoms of these disorders.

In a mid-stage study, OV101 showed a favorable safety profile and that the experimental therapy was well tolerated in adults and adolescents with Angelman syndrome, but it met only one of several endpoints: the physician-rated clinical global impressions of improvement (CGI-I)—a measure commonly used in clinical trials that allows the physician to capture a constellation of clinical symptoms.

Although Ovid’s stock dropped significantly on the news, that endpoint was good enough to get the U.S. Food and Drug Administration’s nod to start a phase 3 trial with CGI-I as the primary endpoint due to the rare nature of Angelman syndrome, the lack of treatment options, the nonexistence of assessment instruments specific to Angelman syndrome, the heterogeneity of the disorder, and the lack of sensitivity or appropriateness of other validated measures.

The phase 3 trial was begun in the second half of 2019 and is ongoing with topline results expected in the fourth quarter of 2020.

Angelini Pharma, part of Italy-based Angelini Holding, has set up a new affiliate Angelini Pharma Rare Diseases that will be in charge of the new licensing agreement.

Ovid Chairman and CEO Jeremy Levin said the deal will bring OV101, if approved, as quickly as possible to the European Angelman community.

“Angelini Pharma is an ideal partner for Europe as they have deep regional knowledge, an established infrastructure with a history of successful product launches, and a commitment to improving the quality of life of the patient communities they serve,” he said.

Ovid is developing OV101 for the treatment of Angelman syndrome and Fragile X syndrome to potentially restore tonic inhibition and thereby address several core symptoms of these conditions. OV101 has received Rare Pediatric Disease designation from the FDA for the treatment of Angelman syndrome. The FDA has also granted Orphan Drug and Fast Track designations for OV101 for both the treatment of Angelman syndrome and Fragile X syndrome. In addition, the European Commission has granted orphan drug designation to OV101 for the treatment of Angelman syndrome.

Author: Rare Daily Staff

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