December 2, 2015
By Mark Terry, BioSpace.com Breaking News Staff
Founded in 2014, Ovid Therapeutics is a privately-held biotech company focused on rare and orphan diseases of the brain. The company’s lead product, acquired from H. Lundbeck A/S , is gaboxadol (OV101). OV101 is currently the only small molecule, highly selective extrasynaptic GABAA(A) receptor agonist against (SEGA) that has been tested in clinical trials. Its focus now is on two disorders—Angelman syndrome and Fragile X syndrome. Both are chromosomal disorders.
Angelman syndrome is a rare disorder that occurs in one in 15,000 live births. Characteristics include developmental delay, sleep disorders, lack of speech, seizures, as well as walking and balance disorders. It is often misdiagnosed as cerebral palsy or autism. Patients are noted for a happy demeanor, often smiling and frequent laughter. Genetically, the disorder is caused by a deletion in a gene on chromosome 15 called UBE3A. Typically, people inherit one copy of the UBE3A gene from each parent and both copies are turned on in many of the body’s tissues. In some areas of the brain, the copy inherited from the individual’s mother is silenced. This parent-specific gene activation silencing is caused by something called genomic imprinting.
Fragile X syndrome is a genetic condition that causes intellectual disability, behavioral and learning difficulties, sleep disruption, as well as some physical characteristics. The intellectual disabilities (previously called mental retardation), range from moderate to severe. Physical features may include large ears, long face, soft skin and large testicles. There are often also behavioral problems such as attention deficit disorder (ADD), attention deficit hyperactivity disorder (ADHD), autism, social anxiety, and others.
Fragile X syndrome occurs in about one in 4,000 males and one in 6,000 to 8,000 females. It is caused by mutations in the FMR1 gene. Almost all cases are caused by a mutation in which a DNA segment, known as the CGG triplet repeat, is expanded within the FMR1 gene. Normally, this DNA segment is repeated from five to 40 times. In individuals with Fragile X Syndrome, the CGG segment is repeated more than 200 times. The disorder is called Fragile X because, prior to DNA testing, cells were treated with a chemical called floxuradine (FUdR), which induced chromosome breakage that could be seen in the cytogenetics lab under a microscope. Chromosome breaks were scored, with a higher number of X chromosome breaks at the distal q end an indicator of Fragile X Syndrome.
The company’s chief executive, Jeremy Levin, has said, “My goal is very, very, very straightforward,” in an interview with CNBC. “To build a highly focused neurology company whose goal is, hopefully, to become one of the best neurology companies in the world over time.”
The company is focused on employing recent scientific understanding of molecular pathways in the brain to develop drugs for novel diseases. Levin anticipates that Ovid will be very active in business development as it aims to develop a deep and innovative pipeline.
Company Leadership
Jeremy Levin is the chairman of the board of directors and chief executive officer. Prior to leading Ovid, he acted as president and chief executive officer of Teva Pharmaceutical Industries Ltd. , and was also a member of the executive committees of Bristol-Myers Squibb and Novartis .
Matthew During is the founder of Ovid, and acts as director, president and chief scientific officer. His training took place at MIT, Harvard and Yale, and was on the faculty of Yale, and was on the faculty of Yale University for nine years as a professor of Neurosurgery. He was the principal investigator and sponsor for two gene therapy studies, including the first and only successful Phase II trial of a biological therapy for Parkinson’s disease. Prior to Ovid Therapeutics, During was founder of Neurologix and co-founder of NightstaRx, both gene therapy companies.
The company’s chief financial officer is Yaron Werber, who previously was a biotechnology analyst at several Wall Street firms, as well as managing director at Citi Research where he was the head of the Healthcare and Biotech Equity Research teams. He was also a senior biotech analyst and vice president of SG Cowen Securities.
Anna Kazanchyan is senior vice president of business development. Prior to joining Ovid, Kazanchyan was the founder and managing partner of Primary i-Research. Previously she was a senior biotech analyst at Wachovia Securities, and was a member of the biotech equity research teams at Goldman Sachs and Citigroup.
Claude Nicaise is the executive vice president of strategic product development and global regulatory affairs, and is joined by Michael Ciaraolo as vice president, chief intellectual property counsel.
Company Financing
Ovid’s initial Series A was completed in March 2015 for $5 million. In August 2015, Ovid completed a Series B financing round totaling $75 million. Its venture round was in March 2015 for $5.06 million. It was led by Fidelity Management and Research Company, and joined by Cowen Private Investments, Sanofi-Genzyme BioVentures, Tekla Capital Management, Sphera Global Healthcare Fund, Jennison Associates, Remile Group and Cormorant Asset Management, as well as others. Existing investors included DoubleLine Equity Healthcare Fund, LLC, which also participated in the Series B round.
Pipeline
OV101 is currently advancing towards Phase II studies in Angelman syndrome and Fragile X syndrome. OV102, which is in preclinical development, will advance into Phase I testing in 2016.
Market Competition
The company’s lead product, OV101 (previously known as gaboxadol), was originally studied by Lundbeck and Merck for insomnia. The drug completed Phase III development where it showed a good efficacy and safety profile for insomnia but was not filed for approval, as it did not meet the pre-specified commercial profile required to be successful in an increasing generic market for sleep drugs. However, it has previously been tested in more than 3,000 patients, so the drug’s safety profile was well understood.
Other companies working in the orphan neurology area include Lundbeck, Genzyme, Amicus Therapeutics and Jazz Pharmaceuticals .
Dollars and Deals
In March 2015, Ovid inked a worldwide licensing deal with H. Lundbeck A/S for gaboxadol for Angelman syndrome and Fragile X syndrome. It expects to start a Phase II trial with OV101 (gaboxadol) in Angelman syndrome this year, followed by a Phase II trial in Fragile X syndrome. In the deal, Ovid obtained exclusive worldwide development and commercialization rights for OV101. Lundbeck became a minority shareholder in Ovid, and will receive milestone payments and royalties on sales. Ovid is responsible for all future development and commercialization for OV101.
What to Look For
The company expects to start two clinical trials this year. The company indicates that it plans to target orphan diseases of the brain that are well understood, but not well served. Part of this focus, Levin told CNBC, came about after watching large drug companies move away from Alzheimer’s research after several large-scale disappointments. He points out that, “there are new ways of measuring brain deterioration, new assays, new mouse models that have been developed. All of this is really, really encouraging.”
Levin was also noted when he was at Bristol-Myers Squibb for building the company’s so-called “string of pearls” set of acquisitions. So expect Ovid to be on the lookout to acquire drugs that either may have failed clinical trials in some neurological areas, but may have potential in other diseases, or that are novel and are targeting new pathways that may be involved in brain diseases.
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