Zynerba stock rises as CBD gel improves rare disorder symptoms; autism program deferred

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Zynerba Pharmaceuticals (NASDAQ: ZYNE) stock rose ~ 8% premarket June 22 after the company said its transdermal CBD gel Zygel helped improve behavior symptoms of 22q11.2 Deletion Syndrome in children / adolescents in a mid-stage study.

22q11.2 Deletion Syndrome (also known as DiGeorge syndrome) is a rare disorder which is caused when a small part of chromosome 22 is missing, where this deletion leads to several issues, such as problems with certain facial and skin features, delayed growth, learning disabilities and behavior problems, among other things.

“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,” said Tony Simon, professor emeritus at the University of California, Davis School of Medicine and the UC Davis MIND Institute.

The 14-week phase 2 trial, dubbed INSPIRE, evaluated Zygel (ZYN002) as an add-on therapy to the standard of care to treat behavioral symptoms of chromosome 22q11.2 deletion syndrome (22q) in children and adolescents (aged four to 15 years). The trial, enrolled 20 patients, while efficacy analyzes included 16 patients.

Zynerba said the patients showed statistically significant improvements at 14 weeks, compared to when treatment began, total score and all five subscales of anxiety, depression and mood scale. The patients also showed statistically significant improvements in aberrant behavior measures, such as social withdrawal, inappropriate speech, and irritability, among others.

The patients also showed improvement on the pediatric anxiety rating scale. Zygel was also well tolerated, the company noted in a June 22 press release.

The company said it will seek a meeting with the US Food and Drug Administration (FDA) to discuss the data and the regulatory path forward.

Pipeline update: Zynerba (ZYNE) said that it will defer the start of a phase 3 program in autism spectrum disorder (ASD), which was planned for H2 2022 due to the difficult financing market, even though the data for the program were compelling.

The company said it will prioritize its resources on Fragile X syndrome (FXS) and 22q.

As a result, the company believes that its $ 69.7M in cash and cash equivalents as of March 31 is sufficient to fund operations through the end of 2023 or early 2024, after the expected results from its confirmatory phase 3 trial, dubbed RECONNECT, of Zygel in patients with FXS.

ZYNE + 8.18% to $ 1.19 premarket June 22

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