Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc has posted second-quarter results with ample cash on hand, which gives the company a long runway to run pivotal trials of its flagship product Zygel (ZYN002), a patent-protected CBD skin gel, which is being developed to treat a slew of rare neuropsychiatric disorders, including Fragile X syndrome (FXS) and developmental and epileptic encephalopathies (DEE) programs.
The Devon, Pennsylvania-based company had $85.8 million in cash and equivalents as of June 30, 2021, compared to $59.2 million at the end of December 2020.
Management believes the company’s cash and equivalents at the end of June are “sufficient to fund operations and capital requirements well into the first half of 2024.”
READ: Zynerba Pharmaceuticals showcases Zygel study poster at annual SLEEP meeting
For the period ended June 30, 2021, Zynerba reported R&D expenses of $5.5 million, including stock-based compensation of $1 million and admin expenses of $4.4 million. The company’s net loss for the quarter was $10 million, or $0.25 per share.
“We continue to make progress across our portfolio, particularly in FXS in which our confirmatory pivotal Phase 3 trial, RECONNECT, is expected to start in the third quarter of 2021,” Zynerba CEO Armando Anido said in an earnings statement.
“Following a positive meeting with the FDA on our development program in autism spectrum disorder (ASD), we are evaluating and prioritizing our development options for ASD, 22q and developmental and epileptic encephalopathies and we expect to provide guidance on the path forward in each of these indications by the end of 2021,” he added.
RECONNECT, or a “Randomized, Double-Blind, Placebo-Controlled, Multiple-Center, Efficacy and Safety Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome,” is a Phase 3, 18-week trial planned for the third quarter of 2021. The trial is designed to confirm the positive results observed in a population of responders in the company’s previously conducted CONNECT-FX trial.
The RECONNECT trial will enroll around 200 children and adolescents, roughly 160 of whom, will have complete methylation of their FMR1 gene and 40 of whom will have partial methylation.
The company said that the primary endpoint for the trial will be the change in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale in patients, who have complete methylation of their FMR1 gene. All patients, including the cohort of partially methylated patients, will be included in a key secondary endpoint analysis. Zynerba believes that the results, if positive, will be sufficient to support the submission of a New Drug Application (NDA) for Zygel in patients with FXS.
In the first half of 2021, Zynerba discussed data supporting the potential effectiveness of Zygel in ASD, including the results of the Phase 2 BRIGHT trial, with the US Food and Drug Administration (FDA) to determine the regulatory path forward. “The guidance from the FDA included agreement on utilizing the irritability subscale of the Aberrant Behavior Checklist – Community (ABC-C) as the primary endpoint to support an indication for the treatment of irritability in ASD,” said the company. This is the same primary endpoint utilized in the previously completed BRIGHT open label Phase 2 trial.
During the quarter, the Zynerba presented data at the 2021 American Society of Clinical Psychopharmacology (ASCP) annual meeting demonstrating that Zygel may provide important clinical promise across a spectrum of endpoints, including, behavior, seizure reduction and sleep in children with ASD, children with developmental epileptic encephalopathies (DEE) with comorbid ASD, and children with FXS with comorbid ASD. The data is from two open-label Phase 2 trials and one double-blind placebo-controlled trial when added to standard of care in children and adolescents.
In addition, despite coronavirus (COVID-19) restrictions in Australia, Zynerba has resumed screening of patients for the 14-week open label Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q, a disorder caused by a missing piece of the 22nd chromosome. “Once enrollment is complete, a timeframe for disclosing topline results of the trial will be provided,” said the company.
Also this year, Zynerba is conducting an observational trial that will help finalize target syndrome selection in one or more DEE syndromes in 2021. DEE is a heterogeneous group of epilepsy syndromes that may be associated with severe cognitive impairment and behavioral disturbances, the company said, and as a result, it will pursue individual syndromes rather than considering DEE as a single disorder or condition.
During the quarter, Zynerba presented data at SLEEP 2021, showing that in an open-label Phase 2 trial with patients with DEE, treatment with Zygel was associated with “improved sleep in children with clinically significant sleep disorders at baseline.” Zynerba believes that because epilepsy and sleep disorders “co-occur in individuals with ASD,” improvements in sleep may result in “better seizure control and behavior in these medically fragile children with DEE.”
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
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