Zynerba launches the Phase 2 trial of its Zygel CBD gel to treat 22q genetic condition

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) announced Wednesday that it has initiated the Phase 2 INSPIRE trial to assess how the company’s lead development candidate Zygel can treat the pediatric behavioral and emotional symptoms of 22q11.2 Deletion Syndrome.

The symptoms of 22q11.2 Deletion Syndrome can range from mild behavioural issues to serious physical and psychological complications. It is a disorder caused by a small missing piece of the 22nd chromosome that can affect every system in the body. Researchers say there can be up to 180 possible symptoms including congenital heart defects, a cleft palate and slow development linked to the condition. It is the second most common chromosomal disorder after Down syndrome.

Shares in Zygel were up slightly at $12.10 in premarket trading.

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Zygel was earlier referred to as ZYN002, a synthetic cannabidiol, which is a non-psychoactive cannabinoid, formulated as a permeation-enhanced gel for delivery through the skin.

Topline data in 2020

The INSPIRE trial will assess the safety, tolerability and potency of Zygel for the treatment of behavioral symptoms of 22q11.2 Deletion Syndrome. Topline data from this study is expected in the first half of 2020, said the company.  

“Children born with 22q often require surgeries to rectify acute physical concerns, like anomalies of the heart and palate; once corrected, there are a myriad of behavioral symptoms that need to be addressed,” said Zynerba Pharmaceuticals CEO Armando Anido.

Anido went on to explain that parents of children with 22q report significantly higher rates of “withdrawn behavior, affective disorders, pervasive developmental problems and anxiety” in their children compared to non-affected children.

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“There is also an increased risk of developing psychoses such as schizophrenia later in life, compared to the general population,” said Anido. “We are very excited to initiate the INSPIRE trial to assess the potential impact of Zygel in these children and adolescents, and look forward to presenting topline data in the first half of 2020.”

The 14-week INSPIRE trial is an open-label multi-dose Phase 2 clinical trial designed to evaluate the potency and safety of Zygel in 20 children and adolescents (ages six through 17) with genetically confirmed 22q.

Enrolled patients will receive weight-based doses of 250 mg daily or 500 mg daily of Zygel. The efficacy assessments include the Aberrant Behavior Checklist-Community, the Anxiety, Depression and Mood Scale, the Columbia Suicide Severity Rating scale, the Qualitative Caregiver Reported Behavioral Problem Survey, and Clinical Global Impression – Severity and Improvement standards.

Contact Uttara Choudhury at uttara@proactiveinvestors.com

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Story by ProactiveInvestors