Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE) reported a net loss of $9 million for the third quarter ending September 30, 2020, with a basic and diluted net loss per share of $(0.31). This beat the Yahoo Finance average analyst estimate for a loss of ($0.43). Six analysts have a Hold rating on the company, while two give it a Buy rating. The company still does not have a revenue-producing drug, but Zynerba said it has enough money until that time comes.
Research and development expenses for the third quarter of 2020 were $5.8 million, including stock-based compensation of $0.5 million. General and administrative expenses for the third quarter of 2020 were $3.4 million, including stock-based compensation expense of $0.7 million. As of September 30, 2020, cash and cash equivalents were $64.3 million and the company said it believes that the current cash and cash equivalents are sufficient to fund operations and capital requirements until late in the fourth quarter of 2021.
“We made good clinical, operational and regulatory progress during the third quarter of 2020 including presenting new data from the pivotal CONNECT-FX and Phase 2 BRIGHT trials, and completing our discussions with the FDA to clarify our clinical path forward to late stage clinical trials in patients with certain developmental and epileptic encephalopathies,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “The fourth quarter of this year is another important period for Zynerba. In particular, we look forward to announcing the results of our fourth quarter meeting with the FDA to discuss our pivotal CONNECT-FX results in patients with a fully methylated FMR1 gene and to understand the regulatory path forward.”
Zynerba said it expects to announce the outcome of its fourth-quarter meeting with the U.S. Food and Drug Administration (FDA) to discuss the CONNECT-FX Trial and the Regulatory path forward for Zygel in pediatric patients in the fourth quarter of 2020. This past summer the company disclosed that its latest top-line results from the 14-week pivotal CONNECT-FX (Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X) trial failed to produce the necessary threshold for positive results. However, the company went back to the drawing board and in October said, “Although the CONNECT-FX full analysis set did not achieve statistical significance in its endpoints, building on current scientific evidence, a pre-planned ad hoc analysis of patients having at least 90% methylation (“full methylation” or FMet) of the impacted FMR1 gene, representing 80% of the overall study population, was performed. The results, including the achievement of statistical significance (p=0.020) in the primary endpoint of improvement at 12 weeks of treatment in the Social Avoidance subscale of the ABC-CFXS compared to placebo, suggesting that Zygel may have benefit in patients with full methylation of the FMR1 gene.”
The company also reviewed its autism study saying that patients receiving Zygel in this study achieved statistically significant caregiver-reported improvements compared to baseline across all subscales of the Autism Impact Measure, which was designed to measure change in frequency and impact of core ASD symptoms: Atypical behavior (p<0.001), Communication (p<0.001), Peer Interaction (p<0.001), Repetitive Behavior (p<0.001), and Social Reciprocity (p=0.0053). In addition, statistically significant improvements compared to baseline were observed at week 14 of treatment with Zygel in the Autism Parenting Stress Index (p<0.0001). Zynerba also measured notable improvements in behaviors utilizing the Qualitative Caregiver Behavioral Problems Survey after 14 weeks of study drug. Clinically meaningful improvements were observed by a majority of surveyed caregivers in behavioral, social, and emotional behavioral problems.