Zynerba said it was “fully funded until 2024” and “on track to launch RECONNECT,” a confirmatory pivotal trial of Zygel in patients with fragile X in the third trimester
Company Said Positive Feedback From FDA Clarifies The Way Forward In Autism Spectrum Disorders
Zynerba Pharmaceuticals Inc released second quarter results with abundant cash flow, giving the company a long lead to conduct pivotal trials of its flagship Zygel (ZYN002), a patent-protected CBD skin gel, which is in the pipeline. being developed to treat a myriad of rare neuropsychiatric disorders, including fragile X syndrome (FXS) and developmental encephalopathies and epilepsy (DEE) programs.
The Devon, Pa.-Based company had $ 85.8 million in cash and cash equivalents as of June 30, 2021, up from $ 59.2 million at the end of December 2020.
Management believes that the company’s cash and cash equivalents at the end of June are “sufficient to finance operations and capital requirements until the first half of 2024”.
READ: Zynerba Pharmaceuticals presents Zygel study poster at SLEEP annual 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 administrative fees 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 where our Phase 3 confirmatory pivotal trial, RECONNECT, is expected to begin in the third quarter of 2021,” Zynerba CEO Armando Anido said in a statement. communicated.
“Following a positive meeting with the FDA on our development program in Autism Spectrum Disorders (ASD), we are evaluating and prioritizing our development options for ASD, 22q and Developmental and Epileptic encephalopathies and we plan to provide advice on the way forward in each of these indications by the end of 2021 ”, he added.
RECONNECT, or a “Randomized, Double-Blind, Placebo-Controlled, Multicenter, Efficacy and Safety Study of ZYN002 Given as a Transdermal Gel to Children and Adolescents with Fragile X Syndrome,” is a trial 18-week Phase 3 scheduled for Q3 2021. The trial is designed to confirm the positive results seen in a responder population in the Company’s previous CONNECT-FX trial.
The RECONNECT trial will recruit approximately 200 children and adolescents, of whom approximately 160 will have complete methylation of their FMR1 gene and 40 of them will have partial methylation.
The company said the primary endpoint of the trial will be modification of the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) social avoidance subscale in patients with complete methylation of their gene. FMR1. All patients, including the partially methylated patient cohort, will be included in a key secondary endpoint analysis. Zynerba believes 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 efficacy of Zygel in ASD, including the results of the phase 2 BRIGHT trial, with the United States Food and Drug Administration (FDA) to determine the regulatory route to follow. The FDA guidelines included agreement to use the Aberrant Behavior Checklist – Community (ABC-C) Irritability subscale as the primary endpoint to support an indication for the treatment of. irritability in ASDs, ”the company said. This is the same primary endpoint used in the previously completed BRIGHT open-label phase 2 trial.
During the quarter, Zynerba presented data at the 2021 American Society of Clinical Psychopharmacology (ASCP) annual meeting demonstrating that Zygel may offer significant clinical promise across a range of parameters, including behavior, reduction of seizures and sleep in children with ASD, children with developmental epileptic encephalopathies (DEE) with comorbid ASD, and children with SXF with comorbid ASD. The data come from two open-label phase 2 trials and one double-blind, placebo-controlled trial when added to standard care in children and adolescents.
Additionally, despite restrictions on coronavirus (COVID-19) in Australia, Zynerba has resumed patient screening for the 14-week open-label INSPIRE phase 2 trial in children and adolescents with genetically confirmed 22q, a disorder caused by a missing piece of the 22nd chromosome. “Once registration is complete, a deadline for the disclosure of the first results of the trial will be provided,” the company said.
Also this year, Zynerba is conducting an observational trial that will help finalize the selection of the target syndrome in one or more DEE syndromes in 2021. DEE is a heterogeneous group of epileptic syndromes that may be associated with severe cognitive and behavioral disorders, the company said, and therefore, it will pursue individual syndromes rather than seeing EED 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 EED, treatment with Zygel was associated with “improved sleep in children with sleep disturbances. clinically significant at baseline ”. Zynerba believes that because epilepsy and sleep disturbances “coexist in people with ASD”, improvements in sleep may result in “better seizure control and behavior in these medically fragile children with EED.”
Contact author Uttara Choudhury at [email protected]
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