Rare disease drug developer Ovid Therapeutics is slated to reveal Phase 3 data from its lead program, a potential treatment for Angelman syndrome, by year’s end. If the drug is approved, the privately held Italian company Angelini Pharma will carry it forward for that indication in the Europe Union and a handful of other countries.
On Monday New York-based Ovid (NASDAQ: OVID) announced the deal with Angelini, which nets it $20 million and makes it eligible for up to $212.5 million more tied to milestones, including royalties on net sales.
CEO Jeremy Levin says the company, founded in 2014, planned from its start to work with partners to advance the drug, gaboxadol, outside of the United States. In 2018 the drug candidate met the main goal of safety and tolerability in a Phase 2 study in adolescent and adult patients with the condition.
“We started two years ago with a question that was posed to us by several of the families in [the] Angelman [community] who said, ‘we are a global community … how are you going to help families all around the world?’” Levin told us.
Angelman syndrome, believed to impact about 1 in 12,000 to 1 in 20,000 people worldwide, is most often caused by a mutation in a gene that codes for UBE3A, a protein that plays a key role in nerve cell communication. Without the protein, a process called tonic inhibition—which controls the brain’s ability to appropriately interpret neural signals—doesn’t work as it should, which leads to symptoms such as seizures, impaired movement, and developmental delays.
Some with the condition are never able to walk or to speak. While those with the neurological disorder typically have normal lifespans, they are unable to live independently. No approved therapies currently exist for Angelman.
Ovid’s small molecule drug is designed to restore tonic inhibition by targeting a brain receptor involved in neurotransmission. (Read more here about the history of gaboxadol, which was once tested by Merck and Lundbeck as a sleeping pill.)
Angelini Pharma CEO Pierluigi Antonelli joined the family-owned firm in 2018 after extensive experience in Big Pharma, most recently as the Novartis (NYSE: [[ticker:NVS]) oncology head for Europe. Prior he was a senior vice president at Merck (NYSE: MRK); previously he served as president of the Bristol Myers Squibb (NYSE: BMY) unit in Italy unit.
Levin said his and Ovid Chief Medical Officer Amit Rakhit’s familiarity with Antonelli paved the way for negotiations, which took place entirely online due to travel restrictions imposed by the ongoing pandemic.
Although the company also met with representatives from other potential licensees, Levin said Antonelli was the right partner because of its deep pockets, its new commitment to rare disease drug development, and knowledge of the European marketplace. He also said Ovid was determined to retain rights to continue advancing the potential treatment in the US.
“We met with many companies, and the larger companies we met with all wanted global rights and we did not feel they could address our needs to be a producer and the lead in America,” he said. “Then we had a number of companies, really incredible companies all of them, with good names, and sizable, who said they would lend us their salesforces, but again I just couldn’t get comfortable with the commitment.”
The countries where Angelini gets development and commercialization rights to the drug candidate to treat patients with Angelman include those in the European Union, other countries in the European Economic Area, Switzerland, Turkey, the United Kingdom, and Russia.
Having teamed up with Angelini, Levin said the company will now seek partners to license the drug in Asia and elsewhere. If results from the Phase 3 trial, NEPTUNE, are positive, Ovid plans to combine that data, which is from pediatric patients, with that recorded in the Phase 2 study, and request the FDA consider the drug as a treatment for people of any age with the condition, Levin said.
Ovid is also in mid-stage testing of the drug as a potential treatment for fragile X syndrome, a rare genetic condition that causes intellectual disability.
Image: iStock/Max Isakov