Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) posted topline results from its Connect-FX clinical trial in Q2 2020. Zygel exhibited considerable improvement in the main endpoint in adolescents/ children with FM (Full Methylation) of FMR1 Gene in a PAHA (pre-planned Ad Hoc analysis). Though it did not meet the primary or secondary endpoints compared to placebo in the full analysis set.
Seeks a conference with FDA
Severely impacted patients showed improvement during a 12-week treatment using Zygel. In the Connect-FX trial, the drug is well tolerated and exhibited consistent safety profile similar to the data obtained other previous clinical trials involving Zygel. Zynerba seeks a meeting with the US FDA for a regulatory path.
Significant improvements in impactful FXS Behavior
Zynerba presented data from the Connect-FX trial at the 17th NFXF ICRR (International Conference Research Roundup). The drug reported significant improvements in FXS behavior. Results achieved from this clinical trial supported Pre-planned Ad Hoc Analysis in Patients with FMet.
Zynerba gathered qualitative data from the clinical relevance of FXS behaviors during Connect-FX to the caregivers. According to the results obtained from the Behavioral survey, caregivers noticed disruptive behaviors, socially avoidance behaviors, and anxiety as most challenging.
According to the caregiver global impression results, the FMet patients administered with Zygel showed an improvement in irritable and disruptive behaviors, social avoidance and isolation, and social interactions from the baseline to the 12th weeks.
Anido said the company achieved significant progress in the past few months to meet the target of developing vital innovative therapies to treat patients suffering from underserved neuropsychiatric disorders.
He also said the Zynerba is evaluating the data collected from patients with Fragile X Syndrome engaged in the Connect-FX trial. It already noted that patients, who are severely impacted, well responded to the treatment using Zygel CBD Gel. The company will discuss with the FDA in H2 to review the data collected from both studies and the next step.
Armando presents an overview of Zynerba
Armando Anido, CEO of Zynerba expects to present an overview of its business at the 40th ACGGC (Annual Cannacord Genuity Growth Conference) on August 12, 2020. It is a virtual conference.
Zynerba holds cash equivalents and cash of $77 million by the end of Q2 2020. The company raised $27.9 million in Q2 2020 by issuing 5.682 million shares.