Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) CEO Armando Anido said Wednesday that the third-quarter was a period of “progress and execution” for the company which has sufficient cash reserves as it heads into key trials.
The Devon, Pennsylvania-based company revealed that, as of September 30, it had cash and cash equivalents of $77.5 million.
Management believes the company's $77.5 million war chest will be “sufficient” to fund operations beyond the expected “New Drug Application submission and potential approval of Zygel in Fragile X syndrome and into the second half of 2021.”
In a statement accompanying the company’s latest numbers, Anido said: “The third quarter of 2019 was a remarkable period of progress and execution for Zynerba.”
In September, Zynerba shared positive top-line results from BELIEVE 1, an open-label, multidose Phase 2 clinical trial evaluating the potency and safety of Zygel, a patent-protected CBD skin gel, for children and adolescents with developmental and epileptic encephalopathies (DEE).
The trial also assessed the safety and efficacy of Zygel in various DEEs, including Dravet syndrome and Lennox-Gastaut syndrome that may be associated with severe cognitive impairment and behavioral disturbances.
“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,” said Anido.
“In this medically fragile patient population, and consistent with our prior trials, Zygel was very well tolerated,” he added.
After completing an analysis of the promising data, the company intends to seek a meeting with the US Food and Drug Administration, likely in the first half of next year, to discuss the clinical pathway to approval.
In a boost for the company, the US Patent and Trademark Office recently granted Zynerba a patent for treating Autism Spectrum Disorder, which is characterized by challenges with social skills and speech, with CBD.
Topline results from CONNECT-FX study in 2020
The company said it is on track to report top-line results from tests of its CBD gel treatment for children and adolescents with Fragile X syndrome, the most common form of inherited learning disability and attention deficit disorder.
Those results will evaluate the potency and safety of Zygel in children aged three to 17 with Fragile X syndrome.
“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 top-line results in the first half of next year,” said Anido.
For the quarter ended September 2019, the company posted a smaller loss of $1.9 million, or $0.08 a share, compared to a loss of $11.1 million, or $0.50 per share in the previous quarter.
In July 2019, the Australian government responded to an Advance Overseas Finding (AOF) application submitted by Zynerba that will allow certain R&D expenses linked to Zygel outside of Australia to be eligible for the Australian R&D tax incentive program.
The company said it recorded an $8.3 million credit for amounts expected to be received through the AOF for the period from 1 January 2018 through September this year.
For the three months ended September 30, R&D expenses rose by $1.8 million to $6.7 million, compared to $4.9 million for the same period in 2018. The company explained that the increase was related to clinical trial and manufacturing costs related to its Zygel program.
Zygel offers a potential goldmine of therapies for multiple conditions. In addition to Fragile X syndrome, Zygel is also in Phase 2 clinical development in patients with refractory epilepsy, Autism Spectrum Disorder and 22q11.2 Deletion Syndrome, a disorder caused by a small missing piece of the 22nd chromosome. This tiny missing portion can affect every system in the body.
The company said that the Phase 2 open-label trial of Zygel in Autism Spectrum Disorder is ongoing and data is expected in the first half of 2020.
Contact Uttara Choudhury at [email protected]
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