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Zynerba reports $70M cash runway as it advances its flagship Zygel CBD Gel for Fragile X Syndrome

Last updated: 13:02 10 Mar 2020 EDT, First published: 09:27 10 Mar 2020 EDT

Zynerba Pharmaceuticals - Zynerba Pharmaceuticals Inc ended 2019 $70 million cash runway and as it advances its flagship Zygel CBD Gel to treat Fragile X Syndrome
Patients with Fragile X often have learning disabilities and other cognitive impairments

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) announced Tuesday that it had ended 2019 with more than $70 million in cash and cash equivalents, giving the company plenty of capital as it advances its flagship Zygel CBD Gel to treat Fragile X Syndrome (FXS). 

“Management believes that the cash runway is sufficient to fund operations and capital requirements beyond the expected NDA submission and potential approval of Zygel in FXS and into the second half of 2021,” the company said in a statement. 

Zynerba said enrollment is complete with 212 patients randomized into CONNECT-FX, a pivotal, multinational, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Zygel in treating common behavioral symptoms of FXS. 

READ: Zynerba to present data on its Zygel CBD Gel to treat autism spectrum disorder and Fragile X Syndrome

The company expects to report topline results late in the second quarter of 2020. If the results are positive, Zynerba intends to request a meeting with the US Food and Drug Administration to determine the acceptability of the data as a basis for a New Drug Application (NDA) and to seek advice on the preparation of the marketing authorization. 

Zynerba expects to submit its NDA for Zygel in FXS to the FDA in the second half of 2020, with potential approval by mid-year 2021.

Fragile X is a genetic condition caused by a mutation in the FMR1 gene. The mutation causes the X chromosome to appear constricted, or fragile, under a microscope, hence its name. Patients with Fragile X often have learning disabilities and other cognitive impairments. 

During the screening phase of CONNECT-FX, the company said caregivers of patients in the trial were informed that their participating child may have the opportunity to receive Zygel in an open-label extension trial following the child’s compliant completion of CONNECT-FX, regardless of their child’s perceived response or actual blinded drug assignment at randomization in CONNECT-FX. 

As of March 9, 97% of the 163 patients who have completed the 14-week blinded portion of the CONNECT-FX trial have enrolled in the open-label extension trial, said Zynerba, which recently won a US Patent to treat FXS with transdermal CBD.

Treating autism spectrum disorder

The company also is studying Zygel to treat autism spectrum disorder (ASD). Enrollment is complete in the Phase 2 BRIGHT trial assessing the safety, tolerability and efficacy of Zygel for the treatment of pediatric and adolescent patients with ASD. Zynerba expects to report topline results from this study in the second quarter of 2020.

Zynbera is also conducting the 14-week Phase 2 INSPIRE trial to evaluate the safety, tolerability and efficacy of Zygel in about 20 children and adolescents (ages six through 17) with genetically confirmed Deletion Syndrome (22q.). Zynerba now expects to report topline results from this study in the third quarter of 2020.

The company said it expects to meet with the FDA to discuss a pathway for Zygel in Developmental and Epileptic Encephalopathies (DEE) in the first half of 2020.

“The fourth quarter of 2019 capped off a year of strong execution by Zynerba,” said CEO Armando Anido.

“With a number of shots-on-goal in our clinical pipeline, each with near term milestones, our outlook is promising for the remainder of 2020 and beyond. We are positioned for major news events throughout this year and next, including the topline results from our pivotal CONNECT-FX trial of Zygel in patients with Fragile X syndrome which are expected late next quarter.

The company's stock recently traded up 7.2% to $3.71 a share.

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Contact the author: patrick@proactiveinvestors.com

Follow him on Twitter @PatrickMGraham

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