ZYNE : Single Trial To Be Conducted In Patients With Fragile X Syndrome To Confirm Positive Results
(RTTNews) – Zynerba Pharmaceuticals Inc. (ZYNE), in an update on its meeting with the U.S. Food and Drug Administration regarding its Fragile X syndrome or FXS program, said that it plans to conduct a double-blind, placebo-controlled pivotal trial in patients with FXS who have a highly methylated FMR1 gene to confirm the positive results observed in this population of responders in the CONNECT-FX trial.
In the first half of 2021, Zynerba will review the trial design and protocol for the new trial through a Type C meeting with the FDA and expects to initiate the pivotal trial before the end of 2021.
Zynerba said it believes that positive results from this confirmatory trial would be sufficient to support the submission of a New Drug Application (NDA) for Zygel in FXS.
Zynerba expects to resume recruitment for the 14-week open label Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q once COVID-19-related restrictions in Australia are eased.
The company expects that its cash runway will extend into the beginning of 2023 as a result of the changes to its development plans.
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