$ZYNE Представили новые данные по гелю на конференции. Вероятность одобрения от FDA растет, на что и отреагировали котировки. Но однозначно что-то утверждать пока преждевременно. “We are excited to provide an update on the potential role of methylation status of the FMR1 gene as a predictive biomarker of preferential response to Zygel™ (ZYN002) in the treatment of the behavioral symptoms of FXS,” said Zynerba’s Chief Medical Officer, Joseph M. Palumbo, MD, FAPA, MACPsych. “These new data demonstrate that in patients diagnosed with FXS with a fully methylated FMR1 gene significantly more patients who received Zygel achieved a clinically meaningful improvement in their behavioral symptoms compared to patients who received placebo.” CONNECT-FX is a randomized, double-blind, multinational, 14-week pivotal study to evaluate the efficacy and safety of Zygel in children/adolescents aged 3 to 17 years. Although the CONNECT-FX full analysis set did not achieve statistical significance in its endpoints, building on current scientific evidence, a pre-planned ad hoc analysis of patients having at least 90% methylation (“full methylation” or FMet) of the impacted FMR1 gene, representing 80% of the overall study population, was performed. The results, including the achievement of statistical significance (p=0.020) in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo, suggest that Zygel may have benefit in patients with full methylation of the FMR1 gene.