Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc has reported financial results for the second quarter ended June 30, 2022, and provided an overview of its recent operational highlights and a pipeline update on its Zygel cannabidiol (CBD) skin gel.
"During the second quarter, we were pleased to announce positive topline results from our phase 2 trial of Zygel in patients with 22q,” said Armando Anido, chairman and chief executive officer of Zynerba in a statement.
“In addition to further progressing 22q, we are focused on completing the phase 3 RECONNECT trial for children and adolescents with Fragile X syndrome, with topline results expected in the second half of 2023,” he added.
Cash and cash equivalents were $62.5 million as of June 30,2022, which the company said is sufficient to fund operations and capital requirements through the end of 2023 or into early 2024, after the expected availability of results from the Fragile X syndrome (FXS) trial.
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"With a cash runway extending past expected availability of topline results from our RECONNECT trial, we remain well-positioned on achieving our goal of bringing the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome to market," Anido said.
Zynerba said it believes the results from RECONNECT, if positive, will be sufficient to support the submission of a New Drug Application (NDA) for Zygel in patients with FXS.
The company presented data at the 18th NFXF International Fragile X Conference in July and at the American Society of Clinical Psychopharmacology Annual Meeting in June demonstrating that in the long-term safety and efficacy study of Zygel in children and adolescents with FXS, improvement was seen in Social Avoidance in the full population, with the greatest improvement in patients with complete methylation of the FMR1 gene.
Patients with complete methylation, who match the primary efficacy population in the ongoing RECONNECT trial, achieved and maintained clinically meaningful change in Social Avoidance, supporting one of the key design enhancements for RECONNECT.
Zygel was well-tolerated during long-term administration up to 38 months of exposure.
In June, Zynerba announced positive topline results from the exploratory, open-label phase 2 INSPIRE trial of Zygel in children and adolescents with 22q. The trial achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments. Zygel was shown to be well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials.
The company plans to meet with the US Food and Drug Administration (FDA) to discuss the data and the regulatory path forward. It plans to move forward in 22q as an orphan indication and has previously received orphan drug designation from the FDA for cannabidiol, the active ingredient in Zygel.
Given the difficult financing market, the company is concentrating its resources on FXS and 22q for the near term and has deferred the start of the phase 3 development program of Zygel in Autism Spectrum Disorder (ASD).
Second Quarter 2022 results
The company reported research and development expenses of $5.4 million for the second quarter of 2022, while general and administrative expenses were $3.7 million.
The net loss for the period was $9.9 million.
Zynerba is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. The company is committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, 22q11.2 deletion syndrome and autism spectrum disorder.
Contact the author at jon.hopkins@proactiveinvestors.com