Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc, a leader in transdermal cannabinoid therapies for rare neuropsychiatric disorders, revealed that it is making an oral podium presentation at the International Society for Autism Research’s four-day annual meeting starting on May 11, 2022.
The meeting is being held both in-person and virtually from Austin, Texas. Zynerba said the presentation will be made available on its website on May 12, 2022, at 4:30 pm ET.
The Devon, Pennsylvania-based company’s flagship product Zygel (ZYN002), a patent-protected CBD skin gel, is being developed to treat a slew of rare neuropsychiatric disorders, including Fragile X syndrome (FXS), 22q11.2 deletion syndrome (22q) and autism spectrum disorder (ASD).
In February 2022, the European Commission granted orphan drug designation to Zygel, for the treatment of FXS, which causes mild to severe intellectual disability.
The presentation titled, "Long-Term Safety and Sustained Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Fragile X Syndrome (ZYN2-CL-017)," includes data from 240 patients.
The presentation focuses on the firm’s study, which included patients who entered the trial from two completed trials with Zygel: CONNECT-FX, a randomized, double-blind, placebo-controlled Phase 3 trial, and FAB-C, the initial open-label Phase 2 trial in FXS.
The effectiveness data was evaluated through 15 months of treatment and demonstrated improvements in both “ABC-CFXS Social Avoidance and Irritability subscales,” noted the company.
Zynerba said patients with complete methylation of their FMR1 gene, who completed CONNECT-FX, and matched the primary efficacy population in the ongoing confirmatory RECONNECT trial, achieved “clinically meaningful change in ABC-CFXS Social Avoidance and showed similar improvements in ABC-CFXS Irritability subscale scores.”
As a result, these finding support the design enhancements that were made for RECONNECT, said the company.
Significantly, Zygel was “well tolerated” in patients, with a median duration of exposure of 16 months (range 21 days to 38 months). Adverse events potentially related to Zygel were reported in 12.9% of patients. The most common reported in 6.7% of the patients was application site pain, which “resolved quickly and was mild” in the majority of cases, said the company.
"The interim results from this open-label extension trial support the long-term safety and effectiveness of Zygel in children and adolescents with Fragile X syndrome, with the greatest improvements seen in those with complete methylation of their FMR1 gene," said Zynerba CEO Armando Anido said in a statement.
"We are excited by the potential that Zygel may offer for patients with FXS," he added,
Zygel has been designated a Fast Track development program for treatment of behavioral symptoms of FXS.
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
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