Angelini Pharma to Develop OV101 for Angelman in Europe

Angelini Pharma has obtained development, manufacturing and commercialization rights to Ovid Therapeutics’ OV101 (gaboxadol), an investigational treatment for Angelman syndrome, in the European Union, Switzerland, Turkey, the U.K., and Russia.

The partnership is between Ovid and Angelini Pharma’s new affiliate, Angelini Pharma Rare Diseases AG. According to the license agreement, Ovid will receive an upfront payment of $20 million. Additional payments, depending on the achievement of several milestones, may sum up to $212.5 million, as well as double-digit royalties.

Ovid will retain the commercial rights for OV101 in the U.S. and rest of the world.

“We are excited to enter into a strategic collaboration with Angelini Pharma with the goal of bringing OV101, if approved, to the Angelman community in Europe as quickly as possible,” Jeremy Levin, chairman and CEO of Ovid Therapeutics, said in a press release.

OV101 is being developed for the treatment of Angelman syndrome and Fragile X syndrome, two disorders characterized by reduced tonic inhibition — a process that decreases excessive activity of neurons in the brain.

OV101 works to normalize tonic inhibition in the brain by activating specific receptors for the neurotransmitter GABA (gamma-aminobutyric acid) on the surface of nerve cells. By activating these receptors, OV101 is thought to increase tonic inhibition and, consequently, ease symptoms of these diseases.

Data from a Phase 1 clinical trial (NCT03109756) showed in 2017 that OV101 was generally safe and well-tolerated in people with Angelman or Fragile X syndrome.

Results from the Phase 2 STARS trial (NCT02996305) showed that a once-daily 15 mg dose of the therapy improved sleep, motor function, and communication skills in adult and adolescent Angelman patients. The results also indicated that OV101 can reduce anxiety.

OV101 is currently being evaluated in the Phase 3 NEPTUNE trial (NCT04106557). The trial is recruiting up to 95 children, ages 2–12, who will be given either OV101 or a placebo once daily at bedtime for 12 weeks. Dosing will be weight-based.

Results are expected by the end of this year. More information about recruitment is available here.

Children who complete the NEPTUNE trial, as well as those who took part in earlier studies, can choose to roll over into ELARA (NCT03882918), an open-label extension trial in which all participants will be given OV101.

“The new business unit Angelini Pharma Rare Diseases AG will contribute to the development, registration, production and, if approved, commercialization in Europe of OV101, Ovid Therapeutics’ very promising drug being evaluated in a Phase 3 clinical trial for the treatment of Angelman syndrome,” said Pierluigi Antonelli, the CEO of Angelini Pharma.

“As of now, there is no effective treatment for this rare genetic disease, characterized by severe psychomotor disability, which manifests itself from childhood. Delivering on our commitment makes us proud both from a scientific and social impact perspectives,” Antonelli said.

The therapy previously received orphan drug and fast track designations from the FDA for both conditions. The European Commission also has granted orphan drug designation to OV101 for Angelman syndrome. In June, OV101 received FDA’s rare pediatric disease designation.