Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc has announced “very encouraging” data from a trial of its Zygel CBD gel for the treatment of 22q11.2 Deletion Syndrome (22q).
The exploratory, open-label Phase 2 INSPIRE trial was designed for signal detection by assessing the safety, tolerability and efficacy of Zygel (also known as ZYN002) for the treatment of behavioral symptoms of 22q in children and adolescents. Zygel was administered to patients with 22q as add-on therapy to their standard of care and utilized a variety of efficacy assessments.
Following positive top-line results from the trial, the company said it will request a meeting with the US Food and Drug Administration (FDA) to discuss the data and the regulatory path forward.
“We believe the data from this Phase 2 trial are very encouraging and reinforce the potential of Zygel for the treatment of behavioral symptoms in children and adolescents with 22q, and we look forward to discussing the regulatory path forward with the FDA with these data in hand,” Armando Anido, chairman and CEO of Zynerba said in a statement.
“In the near term, we will focus our resources on completing the RECONNECT trial for children and adolescents with Fragile X syndrome and progressing 22q.”
READ: Zynerba Pharmaceuticals presents data on Zygel CBD gel at American Society of Clinical Psychopharmacology event this week
22q is chromosomal disorder caused by a small missing piece of the 22nd chromosome. Physical symptoms include characteristic palate abnormalities, heart defects, immune dysfunction, and esophageal/GI issues, as well as debilitating neuropsychiatric and behavioral challenges.
Key findings from the Phase 2 trial included:
- The total score and all five subscales of the Anxiety, Depression and Mood Scale (ADAMS) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
- All five subscales of the Aberrant Behavior Checklist – Community (ABC-C) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
- The Pediatric Anxiety Rating Scale (PARS – R) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
- The majority of patients showed clinically meaningful improvements at week 14 as demonstrated by the Clinical Global Impression – Improvement (CGI-I). 75% of patients were rated by the clinicians as ‘improved’, ‘much improved’ or ‘very much improved’ with nearly two-thirds (62.5%) of the patients being ‘much improved’ or ‘very much improved’;
- Zygel was shown to be well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials.
“I am encouraged with the results from the INSPIRE trial, particularly with the potential for real reductions in general anxiety, social withdrawal, and social avoidance in children and adolescents with chromosome 22q11.2 deletion syndrome,” said Tony J Simon, Ph.D. Professor Emeritus at the University of California, Davis School of Medicine and the UC Davis MIND Institute.
“I’m especially encouraged that ZYN002 is a unique, cannabidiol gel free of THC and manufactured to pharmaceutical specifications. Children and adolescents with 22q should avoid THC which may increase the likelihood of developing psychosis. I look forward to the further development of ZYN002 for this underserved population.”
Twenty patients with a mean age of 9.9 years were enrolled in the 14-week trial. The patients received weight-based doses of 250 mg or 500 mg daily of Zygel. Patients were allowed to increase the daily dose after six weeks of treatment to 500 mg and 750 mg if the investigator felt such increase was appropriate. At the completion of the trial, 13 patients entered into an extension study for up to six months.
Zynerba is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. It is committed to improving the lives of patients and their families living with severe, chronic health conditions, including Fragile X syndrome, 22q11.2 deletion syndrome and autism spectrum disorder.
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