Zynerba Pharmaceuticals (NASDAQ:ZYNE) Inc has reported results for the fourth quarter and full-year that reveal ample cash on hand, giving the company scope to run pivotal trials of its flagship product Zygel (ZYN002).
In the financial year ended December 31, 2021, the Devon, Pennsylvania-based company increased its cash and equivalents to $67.8 million compared to $59.2 million at the end of December 2020.
Management believes the company's $67.8 million war chest will be “sufficient” to fund operations and capital requirements into the second half of 2023. The company's flagship Zygel will be in focus. The product is a patent-protected CBD skin gel, which is being developed to treat a slew of rare neuropsychiatric disorders, including Fragile X syndrome (FXS) and autism spectrum disorder (ASD) programs.
READ: Zynerba Pharmaceuticals says Zygel has received orphan drug designation in Europe
For the fourth quarter, Zynerba reported R&D expenses of $5 million, including stock-based compensation of $0.4 million and admin expenses of $3.8 million. The company’s net loss for the quarter was $8.8 million, or $0.22 per share.
“In 2022, we look to build on last year’s progress as we continue to enroll patients in our confirmatory pivotal Phase 3 trial in Fragile X syndrome, initiate a Phase 3 program in autism spectrum disorder and report topline results in our Phase 2 trial in 22q11.2 deletion syndrome,” Zynerba CEO Armando Anido said in an earnings statement.
“We are committed to achieving our goal of bringing the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome to market, as well as advancing Zygel in other rare and near rare neuropsychiatric indications,” he added.
In February 2022, the European Commission granted orphan drug designation to Zygel, for the treatment of FXS, which causes mild to severe intellectual disability. The company believes that based on the scientific advice of the European Medicines Agency, the successful completion of the current development program for Zygel in FXS will satisfy the requirements of a marketing authorization application in the European Union.
Zynerba said it continues to expect topline results from RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel in patients with FXS, in the second half of 2023. The company believes that 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.
In addition, in February 2022, Zynerba completed the enrollment 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. The firm said it expects topline data from INSPIRE mid-year 2022. A total of 20 patients have been enrolled in the INSPIRE trial.
Meanwhile, Zynerba plans to initiate the first of two Phase 3 trials in patients with ASD, a developmental disability caused by differences in the brain, in the second half of 2022. The company is finalizing the Phase 3 study protocol and will submit an Investigational New Drug application to the US Food and Drug Administration (FDA) before kicking off the pivotal program, said the company.
Zynerba reopted that R&D expenses totaled $21.4 million for the full year 2021, including stock-based compensation of $2.8 million, and general and admin expenses of $15.3 million.
Zygel is the first and only patent-protected cannabidiol clear gel, designed to provide controlled drug delivery into the bloodstream through the skin, according to the company. It is a potential goldmine of therapies for Fragile X syndrome and other conditions.
Contact the author Uttara Choudhury at uttara@proactiveinvestors.com
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