HARMONY BIOSCIENCES TO PRESENT RESULTS FROM BEHAVIORAL STUDY IN 22Q11.2 DELETION SYNDROME AT AMERICAN COLLEGE OF NEUROPSYCHOPHARMACOLOGY ANNUAL MEETING
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The INSPIRE trial evaluated ZYN002 as a potential treatment for anxiety and other irritable behavioral symptoms associated with 22q. Qualitative behavioral analyses of patients and their caregivers, both from INSPIRE and an independent panel, as well as prospective post-hoc analyses of the INSPIRE trial, informed the creation of a conceptual framework for the selection of patient-centered assessments for future clinical trials. This framework proposes endpoint measures to assess the treatment of behavioral symptoms associated with the condition, which currently lacks FDA-approved treatments and affects approximately 80,000 individuals in the
"Harmony is dedicated to finding effective treatments for children and their families living with 22q and other rare neuropsychiatric conditions that currently lack approved therapies," said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences. "Through our recent acquisition of Zynerba, we are making meaningful strides toward developing new treatments for individuals with the behavioral symptoms associated with 22q and Fragile X syndrome (FXS). The development of endpoint measures for future clinical trials is an important step forward in our work for 22q patients who have high unmet medical needs."
Poster: Anxious and Irritable Behaviors in Children with 22q11.2 Deletion Syndrome: A Qualitative Interview Study and Development of a Conceptual Framework
- Poster Session II: Tuesday, December 5, 5PM – 7PM (ET)
ZYN002 is being evaluated as an investigational treatment for 22q. It is not approved for commercial distribution by government regulatory bodies, including the
About the INSPIRE Trial
The 38-week INSPIRE trial was an open-label, Phase 2 clinical trial designed to evaluate the safety, tolerability and effectiveness of ZYN002 in children and adolescents (ages four through 15) with genetically confirmed 22q11.2 deletion syndrome. Enrolled patients received weight-based doses of 250 mg or 500 mg daily of ZYN002. Patients were allowed to increase the daily dose after six weeks of treatment to 500 mg and 750 mg if the investigator felt such an increase was appropriate. At the completion of the first 14-week period of treatment, patients who demonstrated an improvement in symptoms of irritability continued ZYN002 for an additional six months, for a total of 38 weeks of treatment.
About ZYN002
ZYN002 is the first-and-only pharmaceutically manufactured synthetic cannabidiol devoid of THC and formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. The product is manufactured through a synthetic process in a cGMP facility and is not extracted from the cannabis plant. ZYN002 does not contain THC, the compound that causes the euphoric effect of cannabis, and has the potential to be a nonscheduled product if approved. Cannabidiol, the active ingredient in ZYN002, has been granted orphan drug designation by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of FXS and for the treatment of 22q. Additionally, ZYN002 has received FDA Fast Track designation for the treatment of behavioral symptoms in patients with FXS.
About 22q11.2 Deletion Syndrome
22q11.2 deletion syndrome (22q) is a disorder caused by a small missing piece of the 22nd chromosome. The deletion occurs near the middle of the chromosome at a location designated q11.2. It is considered a mid-line condition, with physical symptoms including characteristic palate abnormalities, heart defects, immune dysfunction, and esophageal / GI issues, as well as debilitating neuropsychiatric and behavioral symptoms, including anxiety, social withdrawal, ADHD, cognitive impairment and autism spectrum disorder. It is estimated that 22q occurs in one in 4,000 live births, suggesting that there are approximately 80,000 people living with 22q in the
About Fragile X Syndrome
Fragile X syndrome (FXS) is a rare genetic disorder that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities and behavioral symptoms, such as social avoidance and irritability. There are approximately 80,000 people in the U.S. and approximately 121,000 people in the European Union and
FXS is caused by a mutation in FMR1, a gene which modulates a number of systems, including the endocannabinoid system, and most critically, codes for a protein called FMRP. The FMR1 mutation manifests as multiple repeats of a DNA segment, known as the CGG triplet repeat, resulting in deficiency or lack of FMRP. FMRP helps regulate the production of other proteins and plays a role in the development of synapses, which are critical for relaying nerve impulses, and in regulating synaptic plasticity. In people with full mutation of the FMR1 gene, the CGG segment is repeated more than 200 times, and in most cases causes the gene to not function. Methylation of the FMR1 gene also plays a role in determining functionality of the gene. In approximately
About Harmony Biosciences
At Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Established by Paragon Biosciences, LLC, in 2017 and headquartered in
Forward Looking Statement
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