Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) revealed positive top line results from the exploratory, open label Phase 2 INSPIRE trial. Based on the positive Phase 2 data, the company will request a meeting with the U.S. Food and Drug Administration to discuss the data and the regulatory path forward.
The Phase 2 trial was designed for signal detection by assessing the safety, tolerability and efficacy of Zygel (also known as ZYN002) for the treatment of behavioral symptoms of chromosome 22q11.2 deletion syndrome (22q) in children and adolescents. Zygel was administered to patients with 22q as add-on therapy to their standard of care and utilized a variety of efficacy assessments. Key findings from the trial include:
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The total score and all five subscales of the anxiety, depression and mood scale (ADAMS) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
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All five subscales of the aberrant behavior checklist – community (ABC-C) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
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The pediatric anxiety rating scale (PARS – R) showed statistically significant improvements at 14 weeks of treatment compared to baseline;
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The majority of patients showed clinically meaningful improvements at week 14 as demonstrated by the clinical global impression – improvement (CGI-I). 75% of patients were rated by the clinicians as “improved”, “much improved” or “very much improved” with nearly two-thirds (62.5%) of the patients being “much improved” or “very much improved”;
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Zygel was shown to be 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, both of which have been granted orphan drug designation by the FDA and both of which have no approved products. While the data from the company’s autism spectrum disorder (ASD) clinical development program to date are compelling, given the difficult financing market, the company will defer the start of the Phase 3 development program in ASD previously planned for the second half of 2022.
As a result, the company now believes its $69.7 million of cash and cash equivalents as of March 31, 2022 are sufficient to fund planned operations and capital requirements through the end of 2023 or early 2024, after the expected availability of top line results from its confirmatory pivotal Phase 3 RECONNECT trial of Zygel in patients with FXS.
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