Emalex Biosciences Announces FDA Fast Track Designation for Ecopipam in Tourette Syndrome

On September 6, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that biopharmaceutical company Emalex Biosciences (Emalex) announced that its drug Ecopipam had received FDA Fast Track designation for the treatment of Tourette syndrome.

Tourette Syndrome, also known as Tourette's Disorder or Gilles de la Tourette Syndrome, is a neurological disorder. Patients primarily present with motor tics or vocal abnormalities. Motor symptoms include frequent blinking, facial twitching, head jerking, shoulder shrugging, and tics of the arms and legs; vocal abnormalities manifest as the emission of rapid, meaningless sounds or noises.

Tourette syndrome typically manifests in childhood and is not caused by medications, other medical conditions, or neurological abnormalities. This syndrome is a complex and challenging condition to diagnose, and it is frequently comorbid with attention-deficit/hyperactivity disorder (ADHD), obsessive-compulsive disorder (OCD), anxiety, and depression.

Emalex Biosciences, Inc., headquartered in Chicago, Illinois, USA, is a biopharmaceutical company. The company is dedicated to developing innovative therapies for patients with neurological and psychiatric disorders, with its lead drug candidate, ecopipam, indicated for the treatment of conditions such as Tourette syndrome and stuttering.

FDA Grants Fast Track Designation, Aimed at Accelerating the Market Entry of Potential Innovative Therapies for Serious Diseases to Meet Patients’ Medical Needs. With this designation, ecopipam will benefit from FDA-facilitated development and clinical trial processes, as well as an expedited review process for new drug approval.

Ecopipam selectively blocks the action of dopamine on D1 receptors. Dopamine is a central neurotransmitter, and its receptors are classified into two major categories based on their genetic structure: D1 (including D1 and D5 subtypes) and D2 (including D2, D3, and D4 subtypes). The mechanism of repetitive and compulsive behaviors associated with D1 receptor hypersensitivity may be related to Tourette syndrome. Currently, FDA-approved therapies only target D2 dopamine receptors.

Phase II clinical studies in adult and pediatric patients with Tourette syndrome have demonstrated that ecopipam can reduce the excessive frequency of motor and vocal tics associated with Tourette syndrome, thereby alleviating patient symptoms. In early August this year, Emalex announced the initiation of a Phase IIb, double-blind clinical study in multiple research centers across the United States, Canada, and the European Union, targeting pediatric and adolescent patients with Tourette syndrome.

Clinical trial data for Ecopitham indicate that the drug is well tolerated in both pediatric and adult patients with Tourette syndrome. Adverse reactions primarily involve the central nervous system and gastrointestinal tract, manifesting as insomnia, emotional lability, nausea, and vomiting.

Dr. Atul Mahableshwarkar, Senior Vice President of Drug Development at Emalex Biosciences, stated, “Tourette syndrome is a serious condition with significant unmet medical needs. We are encouraged by the FDA’s granting of Fast Track designation to ecopipam, and Emalex will maintain close collaboration with the FDA throughout the new drug development process.”

(Compiled by Xu Xiaoxue)