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Zynerba's Cannabidiol Formulated Gel Shows Encouraging Action In Genetic Disease

  • Zynerba Pharmaceuticals Inc ZYNE announced positive top-line results from the Phase 2 INSPIRE trial of Zygel (Transdermal CBD gel) on pediatric behavioral and emotional symptoms of 22q11.2 deletion syndrome.
  • The total score and all five subscales of the Anxiety, Depression, and Mood Scale showed statistically significant improvements at 14 weeks of treatment compared to baseline.
  • All five subscales of the Aberrant Behavior Checklist – Community showed statistically significant improvements at 14 weeks.
  • The Pediatric Anxiety Rating Scale showed statistically significant improvements at 14 weeks of treatment.
  • The investigators rated 12 of 16 patients as “improved,” “much improved,” or “very much improved” at week 14, with 62.5% being “much improved” or “very much improved.”
  • Zygel was well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials.
  • In the near term, the company has decided to prioritize its resources on Fragile X syndrome (FXS) and 22q. 
  • Zynerba will defer the start of the Phase 3 program in autism spectrum disorder previously planned for 2H 2022, citing a difficult financing market.
  • Hence, the company expects its $69.7 million of cash, and cash equivalents will be sufficient to fund planned operations and capital requirements through the end of 2023 or early 2024.
  • Price Action: ZYNE shares are up 7.45% at $1.18 during the market session on the last check Wednesday.

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