The U.S. Food and Drug Administration gave the company a green light for the trial at a recent meeting.
Zynerba expects the results to support the filing of a New Drug Application for ZYN002 as a Fragile X treatment. At the moment, the United States has no approved therapies for the disease.
The company is looking at ZYN002 as a potential treatment for children and adolescents with Fragile X syndrome and developmental and epileptic encephalopathies. It is also developing it as a possible epilepsy therapy.
ZYN002 is administered as a gel through the skin once or twice a day. The gel is absorbed directly into the bloodstream, bypassing the liver and gastrointestinal tract. The formulation allows patients to use lower doses than a pill, and minimizes the risk of adverse events.
“The FDA meeting was an important milestone for us as we advance the development of ZYN002 for patients and their families suffering with the profound behavioral symptoms” of Fragile X syndrome, Armando Anido, the chairman and CEO of Zynerba, said in a press release.
“We are pleased with the outcome of the discussion and the guidance on trial design, and believe we now have a path forward to advance the development of ZYN002″ to a New Drug Application, he said.
The company wants to provide Fragile X “patients and their families an effective and well tolerated therapy to treat the complex behavioral symptoms” of the disease, he added.
The 14-week, double-blind trial will evaluate ZYN002’s ability to improve those behaviors. Caregivers will provide the behaviorial reports.
Zynerba anticipates enrolling about 200 children and adolescents with the disease in the U.S., Australia and New Zealand.
Patients will be randomly assigned to one of two doses of ZYN002 or a placebo. At the end of dosing, a 12-month extension study will be conducted, where all patients receive the treatment.
“We believe that ZYN002 may address core behavioral symptoms of FXS [Fragile X syndrome] and improve the quality of life for patients and their families,” said Liza Squires, Zynerba’s chief medical officer.
“We believe we have designed an efficient pivotal program that includes endpoints that measure clinically relevant and observable behaviors in patients with FXS, and if successful, positions us to bring the FXS community its first targeted treatment designed with patients’ symptoms in mind,” Squires added.
The FDA granted orphan drug status to ZYN002 as a potential treatment for Fragile X in 2016. The designation covers treatments for diseases affecting fewer than 200,000 Americans.