*Editor's Note: This article is part of "Startup Series," a series of articles that profile multiple startup companies in the life science sector over the course of a few weeks. Click on the links below to access earlier articles in the series.

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Company Name: Ovid Therapeutics

Location: New York City

Year Founded: 2014

Focus: Orphan and rare diseases of the brain

In a pharmaceutical landscape where it takes billions of dollars and a decade to produce a single drug, companies tend to focus on diseases that cast a wide net, such as diabetes or cancer. Unfortunately, what gets lost in this process are the orphan and rare diseases that so often impact young children. Although the name implies that is doesn’t happen often, the parents and loved ones of these young children certainly don’t feel that way. A feeling that can be made even more frustrating by a lack of medicines and treatment options.

Ovid Therapeutics was founded in 2014 on a simple idea: take advantage of recent progress and discoveries in neurology—specifically orphan and rare disorders—to develop medicines that address underlying disease pathology and transform the lives of patients.

“At a time when many large pharmaceutical companies were exiting the field, frustrated with challenges associated with developing medicines to treat brain diseases, Ovid found opportunity,” Ovid CEO Jeremy Levin told Laboratory Equipment. “Many diseases—like monogenetic diseases, or a disease with a single genetic defect—were being overlooked.”

Ovid takes a unique approach in that the company is more than just its pipeline. It works with patient and advocacy communities to better understand the patient journey—including the natural history of the disease—to better define novel endpoints toward the ultimate goal of designing new medicines.

Of course, the pipeline is still extremely important. Ovid’s first—and main—product is OV101, the only clinically tested delta (δ)- Selective Extrasynaptic GABAA Receptor Agonist (SEGA). It services patients with both Angelman and Fragile X syndrome.

Both syndromes are autism spectrum disorders. Angelman syndrome is characterized by severe intellectual and developmental disability, sleep disturbance, seizures, jerky movements, frequent laughter or smiling and a usually happy demeanor. It is caused by deletion or inactivation of genes on the maternally inherited chromosome 15, while the paternal copy, which may be of normal sequence, is imprinted and therefore silenced.

Fragile X syndrome symptoms include mild to moderate intellectual disability, problems with social interactions, delayed speech, hyperactivity, seizures and well-defined physical features that may include a long and narrow face and large ears. The syndrome is caused by the expansion of the CGG triplet repeat within the Fragile X mental retardation 1 (FMR1) gene on the X chromosome. This results in a failure to express the fragile X mental retardation protein (FMRP), which is required for normal neural development.

Through its action as a SEGA, Ovid’s OV101 selectively regulates “tonic inhibition,” a critical mechanism that the brain uses to discriminate signal from noise.

In several autism spectrum disorders, including Angelman syndrome and Fragile X syndrome, patients lose tonic inhibition, and, it is believed, consequently cannot distinguish between the myriad of sensory inputs they receive in their daily lives. OV101 restores normal function when given to mice that have been genetically modified to lose tonic inhibition.

Currently, the oral OV101 drug for adults with Angelman syndrome is ready for Phase 2 clinical trials, while the adolescent version is ready for Phase 1 trails and the pediatric version is still in the pre-clinical phase. OV101 for Fragile X syndrome is Phase 1 ready.

Over the next two to five years, Ovid will look to broaden and advance development of its programs in both Angelman syndrome and Fragile X syndrome, with data results from both programs. The company is also exploring business development opportunities to broaden its rare neurological disease pipeline.

“Ovid believes neurology today is where immuno-oncology or hepatitis C were 8 to 10 years ago,” Levin said, “the beginning of an explosion of new therapies. With new progress and insights, neurology drug developers like Ovid can create treatments that are laser-focused and provide tangible benefits for patients.”

In August 2015, Ovid completed its Series B financing at a total of $75 million. The money has/will continue to support the company’s current pipeline development efforts, in terms of clinical trials in the near future. It will also allow Ovid to further broaden its pipeline through possible business development activities.