Benzinga | Dec 17, 2020 06:33AM EST

Zynerba Pharma Offers Regulatory Update On Zygel In Fragile X Syndrome: Co. To Conduct Double Blind, Placebo-Controlled Pivotal Trial

Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided an update on its meeting with the U.S. Food and Drug Administration (FDA) regarding its Fragile X syndrome (FXS) program. The Company 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 believes that positive results from this confirmatory trial would be sufficient to support the submission of a New Drug Application (NDA) for Zygel(tm) in FXS.

"We believe that Zygel has the potential to meaningfully relieve the behavioral symptoms of the most impacted individuals with Fragile X syndrome. We are committed to bringing this important therapy to patients and their families within the Fragile X community," said Armando Anido, Chairman and Chief Executive Officer of Zynerba. "We are thankful for our ongoing constructive dialogue with the FDA on our path forward to NDA submission. Completing the development of Zygel in FXS and preparing for a successful launch will be the primary focus of the Company."

"The results reported in the analysis of children with a highly methylated FMR1 gene are a source of considerable hope for the patients and their families who are impacted by Fragile X syndrome," said Linda Sorensen, Executive Director of the National Fragile X Foundation (NFXF). "Zynerba has been an important partner to the families of children with Fragile X. We thank them for their continued dedication and commitment to bringing an FDA-approved treatment to our families and look forward to helping with the recruitment of patients for this study."

The Company's development plan for Zygel in other indications includes the following:

* Developmental and epileptic encephalopathies (DEE): Evaluation of potential target indications is ongoing, and Zynerba now expects to finalize target syndrome selection in 2021 in one or more DEE syndromes.

* 22q11.2 deletion syndrome (22q): 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. After recruitment has resumed, the Company will be able to provide a timeframe for completion of this trial.

* Autism spectrum disorder (ASD): In the first half of 2021, Zynerba intends to discuss data supporting the potential efficacy of Zygel in ASD, including the results of the Phase 2 BRIGHT trial in children and adolescents with moderate-to-severe ASD with the FDA to determine the regulatory path forward.

Financial Impact on Cash

The Company expects that its cash runway will extend into the beginning of 2023 as a result of these changes to its development plans.

Conference call information

Zynerba management will host a live conference call and webcast today at 8:30 am Eastern Time to discuss the recent FDA meeting and the regulatory pathway for Zygel in FXS. The call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345 (international) and referencing conference ID 4088027. To access the live webcast or the replay, visit the investor page of the Company's website at http://ir.zynerba.com/. The webcast will be recorded and available on the Company's website for 30 days.