logo-loader

Zynerba shares robust data from open label extension of Phase 2 FAB-C study on Fragile X patients at American Academy of Neurology

Published: 14:20 26 May 2020 EDT

Zynerba Pharmaceuticals - Zynerba shares robust data from open label extension of Phase 2 FAB-C study on Fragile X patients at American Academy of Neurology
With topline data from the CONNECT-FX trial on the way, investors are eyeing opportunities defined by marijuana-adjacent companies

Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) shared a poster depicting positive data amassed over two years from its Phase 2 FAB-C trial evaluating its flagship product Zygel (ZYN002), a patent-protected CBD skin gel, to treat children and adolescents with Fragile X syndrome.

In a statement on Tuesday, the Devon, Pennsylvania-based company said the poster was available at the American Academy of Neurology (AAN) science highlights virtual session. A copy of the poster is also available on the company website.

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.

The poster depicts “statistically significant improvements from baseline” that were observed at week 12 that were sustained in each subscale score of the Aberrant Behavior Checklist for Fragile X (ABC-CFXS) through two years for patients who participated in the open label extension.

“It’s very exciting to see that the observed early benefits of Zygel appear to be sustained for over two years in patients who enrolled in the open label extension of FAB-C,” Zynerba Chief Medical Officer Joseph M Palumbo said in a statement.

“Data suggests the potential for a sustained and measurable benefit for those patients who experience an early response.”

Separately, Zynerba is on track to report topline results from tests of its CBD gel treatment for children with Fragile X syndrome in the second quarter of 2020.

“It’s also reassuring to see these responses in the context of a strong tolerability profile. We look forward to the results of our pivotal CONNECT-FX study in children and adolescents with FXS late this quarter,” said Palumbo.

The 12-week treatment results of the Phase 2 FAB-C trial were initially announced in September 2017. At least 27 patients between 6-to-17 with Fragile X were enrolled in the open label FAB-C study. At the end of the 12-week study (Period 1), patients could enter an extension study (Period 2) which 13 patients did. Ten patients exceeded two years of therapy.

The company noted that it saw “statistically significant improvements from baseline” at week 12 in all six subscale scores of the ABC-CFXS in Period 1. Most significantly, these significant improvements were sustained through two years in subjects who entered Period 2 of the study.

The persistence of effect over the two-year period is as follows:

Zygel was well tolerated in the FAB-C trial over two years and no treatment-related adverse events occurred in more than one patient.

The authors of the poster concluded that:

  • In this post hoc analysis, the majority of patients who completed Period 1 met important criteria for therapeutic response (≥25% or ≥50% improvement from baseline in ABC-CFXS domains) at weeks 4, 8, and 12 of the Phase 2 FAB-C trial
  • This response was sustained or continued to improve through two years in patients who entered Period 2
  • Zygel was well tolerated through two years, and all AEs were mild or moderate and most were considered unrelated to treatment
  • Together, the data may suggest evidence of the clinical efficacy and favorable safety and tolerability of Zygel in children and adolescents with FXS when added to stable standard of care therapies

Contact the author Uttara Choudhury at uttara@proactiveinvestors.com

Follow her on Twitter: @UttaraProactive

ARway.ai announces multiple new SaaS developer contracts in both the United...

ARway.ai (CSE:ARWY, OTCQB:ARWYF) Chief Executive Officer Evan Gappelberg joined Steve Darling from Proactive to announce multiple new SaaS developer sign-ups for its augmented reality experience platform, focusing on AR indoor navigation. These partnerships represent significant milestones in...

1 hour, 2 minutes ago