Zynerba Pharmaceuticals Inc (NASDAQ:ZYNE) provided an update on Monday on 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 said that it has been notified that the US Food and Drug Administration (FDA) will meet with executives from the company via teleconference in the fourth quarter, to discuss the company’s pivotal CONNECT-FX data and the regulatory path forward in patients with Fragile X syndrome and a fully methylated FMR1 gene (FMet).
Zynerba said it also expects to disclose the outcome of the meeting in the fourth quarter of 2020.
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An analysis of its recent CONNECT-FX trial showed that Zygel achieved statistical significance in improving behavioral metrics among the most severely impacted patients — 80% of the total participants — after 12 weeks.
Fragile X is a genetic condition caused by a mutation in the FMR1 gene. The mutation causes the X chromosome to appear constricted, or fragile, under a microscope, hence its name. Patients with Fragile X often have learning disabilities and other cognitive impairments.
“The meeting with the FDA will be an important milestone for patients and their families who live with the debilitating behavioral impact of Fragile X,” Zynerba CEO Armando Anido said in the update.
“Our ongoing evaluation of the pivotal CONNECT-FX data continues to clarify the impact that Zygel achieved in the most severely impacted children and adolescents with FXS, as well as the excellent tolerability profile. We look forward to discussing the pivotal data and the regulatory path for potential approval in FMet patients with the FDA in the fourth quarter of this year.”
Bolstering its IP
Zynerba also revealed that the US Patent and Trademark Office has issued US Patent No 10,758,497, titled “Treatment of Fragile X Syndrome with Cannabidiol” which includes claims directed to a method of treating FXS comprising administering 250 mg or 500 mg of synthetic or purified cannabidiol in a pharmaceutically acceptable carrier to a person in need.
This new patent, which expires in 2038, is part of an expanding intellectual property portfolio covering Zygel.
Developmental and epileptic encephalopathies
Meanwhile, it said BELIEVE data has built on the strong safety and tolerability profile of Zygel, and provided evidence of its anti-seizure activity in children and adolescents suffering from developmental and epileptic encephalopathies, or DEEs.
“Zynerba has concluded its iterative meetings with the FDA utilizing their ‘Written Response Only’ format to discuss the clinical pathway for Zygel in DEE,” said the company. Zynerba said the FDA supports a development program which would “treat focal-impaired awareness and convulsive seizures.”
However, Zynerba said that due to the “heterogeneity of patients who fall under the DEE umbrella, the FDA suggests that Zynerba pursue individual syndromes rather than considering DEE patients as a single disorder or condition.”
The company is in the process of finalizing its evaluation of which epileptic syndromes it may pursue with Zygel.
“We appreciate our partnership with the FDA, and thank them for their input and support as we seek to advance the development of Zygel in certain rare epilepsy syndromes. We look forward to completing our target assessments and communicating our path forward around the end of this year, ” Anido concluded.
Contact the author Uttara Choudhury at [email protected]
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