Zynerba Pharmaceuticals, Inc. (NASDAQ: ZYNE) saw its stock lift ever so slightly on news that the company has positive results from its drug trials. Shares were up by two cents in pre-market trading to $7.15.
The company said that the topline results of this six-month Phase 2 evaluation of Zygel in 48 children and adolescents with various DEEs showed meaningful reductions in seizures and excellent tolerability. Patients experienced 44% to 58% monthly median reductions in focal impaired-awareness seizures (FIAS; previously known as complex partial seizures) and/or convulsive seizures (CS; focal to bilateral tonic-clonic seizures and generalized tonic-clonic seizures), the most common and debilitating seizure types, starting at month two and continuing through month six.
Zynerba also noted that enrollment was progressing in CONNECT-FX, a pivotal, multinational, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of Zygel in treating common behavioral symptoms of FXS in three through 17-year old patients with Fragile-X Syndrome. The company expects to report top-line results in the first half of 2020.
In addition to those results, Zynerba said it was conducting the Phase 2 BRIGHT trial to assess the safety, tolerability, and efficacy of Zygel for the treatment of child and adolescent patients with Autism Spectrum Disorder (ASD). The 14-week trial is designed to evaluate the efficacy and safety of Zygel in approximately 36 children and adolescents (ages four through 17) with ASD as confirmed by DSM-5 diagnostic criteria for ASD. The efficacy assessments include the Aberrant Behavior Checklist, Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism Impact Measure, and Clinical Global Impression – Severity and Improvement. Zynerba expects to report topline results from this study in the first half of 2020.
“The third quarter of 2019 was a remarkable period of progress and execution for Zynerba,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We announced compelling topline safety and efficacy results from our six month BELIEVE Phase 2 trial of Zygel™ in childhood epilepsies. In this study, patients experienced median reductions in their most common and debilitating seizures of 44% or more starting at month two and continuing through month six. In this medically fragile patient population, and consistent with our prior trials, Zygel was very well tolerated. Caregivers also reported important improvements in seizure intensity and duration in their children, and in socio-behavioral and cognitive impairments that are common in this population. Finally, we continued to progress towards full enrollment in our pivotal CONNECT-FX trial in children and adolescents with Fragile X syndrome, and expect to announce topline results in the first half of next year.”
For the quarter ending September 30, 2019, Zynerba reported an $8.3 million credit to research and development expenses for amounts expected to be received through the AOF for the period from January 1, 2018, through September 30, 2019. “Excluding the $8.3 million reduction in research and development expenses for amounts expected to be received through the AOF for the period from January 1, 2018, through September 30, 2019, research and development expenses increased by $1.8 million to $6.7 million for the three months ended September 30, 2019 from $4.9 million for the three months ended September 30, 2018. The increase was primarily related to an increase in clinical trial and manufacturing costs related to our Zygel program. Stock-based compensation included in the R&D costs were $0.6 million.”
General and administrative expenses for the third quarter of 2019 were $3.5 million, including stock-based compensation expense of $0.8 million. The net loss for the third quarter of 2019 was $1.9 million with a basic and diluted net loss per share of $(0.08).