Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc has told investors it continues to expect topline data from its Phase 2 Zygel clinical trial for 22q11.2 deletion syndrome (22q) in children and adolescents in the middle of this year as the company revealed enrollment in the study was now complete.
The INSPIRE trial has 20 patients and will take 14 weeks. It is an open-label, multi-dose trial aimed at assessing the efficacy and safety of the transdermal cannabinoid therapy Zygel in children and adolescents (ages six through 17) with genetically-confirmed 22q.
The condition is the second most common chromosomal disorder after Down syndrome and is caused by a small missing piece of the 22nd chromosome.
Physical symptoms include palate abnormalities, heart defects, immune dysfunction, and esophageal/GI issues, as well as debilitating neuropsychiatric and behavioral challenges.
"The completion of enrollment in our INSPIRE trial is an important step forward in advancing Zygel as a potential new treatment option for patients suffering from 22q," said Armando Anido, the CEO of Zynerba in a statement.
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"Pending results from the INSPIRE trial and subsequent discussion with the FDA on the regulatory path forward, we plan to advance our clinical development program in 22q with Zygel. We are thankful for the combined efforts of our researchers, investigators and the patient and caregiver community."
Zygel in 22q has been designated orphan drug status by the US Food and drug administration (FDA).
The patients will receive weight-based doses of 250 miligrams (mg) daily or 500 mg daily of Zygel. Efficacy assessments include the Aberrant Behavior Checklist-Community (ABC-C), the Anxiety, Depression and Mood Scale (ADAMS), the Qualitative Caregiver Reported Behavioral Problem Survey, and Clinical Global Impression - Severity and Improvement.
Zygel is an investigational drug product which is also a potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS) and autism spectrum disorder (ASD).
It has been granted orphan drug designation by the FDA for the treatment of FXS and 22q.
The regulator has also designated Zygel a Fast Track development program for treatment of behavioral symptoms of FXS.
Contact the writer at giles@proactiveinvestors.com