Ovid and Angelini Pharma Ink $222 Million Angelman Syndrome Partnership
New York-based Ovid Therapeutics and Rome-based Angelini Pharma announced a deal where Angelini will take over development, manufacturing and commercialization of OV101 (gaboxadol) for possible treatment of Angelman syndrome in the European Union, other countries in the European Economic Area, including Switzerland, Turkey and the UK, and Russia.
Angelman Syndrome is a genetic disorder that mostly affects the nervous system. It is marked by developmental delay, intellectual disability, severe speech impairment and movement and balance problems. Most children affected also have epilepsy and microcephaly (small head size). The development delay is typically noticeable between the age of six and 12 months.
Angelman syndrome have a collection of specific behaviors, including happy, excitable demeanor, frequent smile, laughter and hand-flapping movements. This led to it being called “happy puppet” syndrome, although that term is no longer used.
The syndrome affects about 1 in 12,000 to 20,000 people. It is caused by the loss of function of a gene called UBE3A. Normally people inherit one copy of the UBE3A genes from each parent. Then both copies of the gene are switched on in many of the body’s tissues. In some areas of the brain, only the copy inherited from the individual’s mother is active. This parent-specific gene activation is called genomic imprinting. If the copy of the UBE3A gene is missing because of a chromosomal change or a gene mutation, the person has no active copies of the gene in some parts of the brain. About 70% of Angelman syndrome cases happen when a small piece of the maternal chromosome 15 that contains the gene is deleted. About 11% of cases occur when there is a mutation in the maternal copy of the UBE3A gene.
In an even smaller percentage, the syndrome occurs when the patient inherits two copies of chromosome 15 from his or her father instead of one copy from each parent. This is called paternal uniparental disomy.
Under the terms of the deal, Angelini is paying Ovid $20 million up front. Ovid is also eligible to up to $212.5 million in various milestones in addition to double-digit royalties. Ovid will hold all U.S. and rest-of-world commercial rights to the drug.
“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,” said Jeremy Levin, chairman and chief executive officer of Ovid. “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. Finding the right partners to bring OV101 to the Angelman community as rapidly as possible is a core part of our global strategy. We believe this partnership with Angelini will help to maximize the potential commercial value of OV101 and achieve our strategic objectives in this important geography.”
The companies believe OV101 is the only delta-selective GABAA receptor agonist in development. It specifically targets the disruption of tonic inhibition, a central process of the brain that is believed to be the underlying cause of several neurodevelopmental disorders. In laboratory and animal studies, the drug selectively activates the delta-subunit of GABAA receptors, which are found in the extrasynaptic space. This appears to impact neuronal activity by modulating tonic inhibition.
The drug is being developed for Angelman syndrome and Fragile X syndrome, a genetic disorder marked by mild-to-moderate intellectual disability, and specific physical features, including long, narrow face, large ears, flexible fingers, and large testicles. Fragile X syndrome is caused by a mutation in the FMR1 gene, which is found on the X chromosome.