Emalex Biosciences, a portfolio company of Paragon Biosciences, recently secured a Series D funding of $250 million for its upcoming Phase 3 Tourette syndrome trial. News of the successful financing round was revealed on Nov. 3 and follows a previous financing round of $35 million.
The round was led by Bain Capital Life Sciences and saw participation from several market leaders, including founder Paragon Biosciences, Fidelity Management & Research Company, Valor Equity Partners, and others.
Company executives reported that the Series D round will fund a Phase 3 clinical trial and the potential commercialization of a new class of drug for patients with Tourette syndrome. The Phase 3 clinical trial will pursue further research on Emalex’s first-in-class new drug, ecopipam, a novel dopamine-1 receptor antagonist. The study will be the largest for Tourette syndrome to ever be carried out in North America. Nearly 220 patients are expected to sign up across 90 sites worldwide.
The funding comes following positive Phase 2b clinical trial results (D1AMOND Study) that showed how ecopipam reduced motor and phonic tics in Tourette syndrome as compared to a placebo. Tourette syndrome is a neurological disorder characterized by motor and verbal tics, which are repetitive movements and vocalizations prompted by an irresistible urge to produce them. Symptoms can significantly interfere with communication, daily functioning, and quality of life.
The Phase 2b clinical trial involved 149 children and teens between the ages of 6 and 17 with Tourette’s. They were divided into two groups out of which 74 were treated with ecopipam, and 75 with a placebo. On average, the participants taking ecopipam improved their motor and vocal tic severity scores from 35 to 24, a decrease of 30%.
Headaches, fatigue, somnolence, insomnia, and restlessness were the most frequent adverse events reported by patients administered ecopipam in the study. Patients in the experiment showed no signs of harmful movement or metabolic side effects.
“The robust results of our ecopipam study are very encouraging, giving us momentum to continue our important work towards a safe and effective therapy for pediatric patients living with Tourette syndrome,” said Atul R. Mahableshwarkar M.D., Chief Medical Officer and Senior Vice President of Drug Development. “Behind the data is a strong team of scientists, clinicians, neurologists, psychiatrists, and industry experts determined to develop new treatment options for those with this disease, as well as dedicated patients and families who participated in the study.”
“Our results are exciting because they suggest ecopipam shows promise as a treatment for reducing the number, frequency, and severity of the tics young people experience with Tourette syndrome. That’s especially true because many people with the disease who are taking the medications currently available still have debilitating symptoms or experience weight gain or other side effects,” said study author Donald L. Gilbert, MD, of the Cincinnati Children’s Hospital Medical Center in Ohio, and a Fellow of the American Academy of Neurology.
“It’s important to note that a large number of patients with Tourette syndrome have co-morbidities like ADHD or OCD. During the Phase 2b study they were allowed to continue their medications for those conditions, and their Tourette symptoms measurably improved with ecopipam,” said Dr. Gilbert.
Emalex’s first-in-class development candidate ecopipam, a novel investigational compound that is being studied as a potential treatment for certain central nervous system disorders, blocks the actions of the neurotransmitter dopamine at the D1 receptor. Dopamine is a neurotransmitter in the central nervous system, and its receptors have been classified into two “families” based on their genetic structure: “D1” (including subtypes D1 and D5) and “D2” (including subtypes D2, D3, and D4). D1 receptor super-sensitivity may be a mechanism for the repetitive and compulsive behaviors associated with Tourette’s. Currently approved therapies for the treatment of Tourette’s act at D2 receptors. Ecopipam has been shown to be generally well tolerated in clinical trials conducted to date and has received Orphan Drug and Fast Track designation from the FDA for the treatment of patients with Tourette’s. Adverse events affecting primarily the central nervous system have been reported in clinical trials conducted to date, including headache, fatigue, somnolence, insomnia, restlessness, anxiety, depression, and rarely, suicidal ideation.
Emalex Biosciences was created by Paragon Biosciences to develop new treatments for central nervous system disorders. Emalex is in late-stage development of a new class of drug targeting the D1 receptor for patients with Tourette Syndrome and other conditions that have limited treatment options. Visit EmalexBiosciences.com to learn more.
Paragon Biosciences is a global life science leader that creates, builds and funds innovative biology-based companies in three key areas: cell and gene therapy, biology engineering and advanced biotechnology. Our portfolio companies use biology to accelerate scientific breakthroughs that solve some of society’s most challenging problems. Learn more at https://paragonbiosci.com/.
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