The company has received fast track designation for its lead candidate, Zygel, which aims to treat patients with Fragile X syndrome.
Zynerba Pharmaceuticals (NASDAQ:ZYNE) received a bump on Monday (May 6) thanks to the US Food and Drug Administration (FDA) granting the company fast track designation for its drug, which is indicated to treat symptoms related to Fragile X syndrome (FXS).
The company’s lead drug candidate, Zygel (ZYN002 CBD gel), is being tested in young patients aged between three and 17 years with FXS. It is a pharmaceutically produced cannabidiol (CBD) gel that is applied directly to the skin and into the circulatory system, with a formula that enhances permeation.
Following Zynbera’s announcement, shares of the company rose 11.44 percent to close the trading session on Monday at US$12.76.
Armando Anido, chairman and CEO of Zynerba, said that the fast track designation awarded by the FDA reinforces the fact that there is an unmet clinical need for patients with FXS and their providers.
“We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome,” Anido said in a release.
According to the US National Library of Medicine, FXS is a genetic condition that leads to various developmental problems such as learning disabilities and cognitive impairment. Symptoms, which usually occur by the age of two, include delays in speech development and language skills. FXS is the result of a mutation in the Fragile X mental retardation gene (FMR1) and can cause attention deficit disorder (ADD), while one-third of FXS patients also fall on the autism spectrum.
Zynerba’s clinical trial, called “Clinical Study of Cannabidiol in Children and Adolescents with Fragile X (CONNECT-FX),” is enrolling patients in 20 clinical sites across the US. The objectives of the trial are to test how safe and effective Zygel is in improving certain symptoms of FXS. Roughly 204 patients total will participate in the trial, for which the company says it anticipates having topline data sometime in the second half of the year.
Additionally, a Phase 2 open label study testing the safety and efficacy of Zygel when applied as a transdermal gel on children with developmental and epileptic encephalopathy (BELIEVE 1) should also have topline results in Q3 2019, while a Phase 2 study of Zygel for autism spectrum disorder should have data sometime in the first half of next year.
In after hours trading on Monday, Zynerba jumped an additional 1.25 percent to reach US$12.92 as of 5:08 p.m. EDT. The company currently has a “strong buy” ranking on TipRanks.com based off five analysts, with an average price target of US$22.80, a high estimate of US$36 and a low of US$11.
Michael Higgins, an analyst with the firm Ladenburg, reiterated his “buy” position on the company on Monday and gave a price target of US$26.
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Securities Disclosure: I, Jocelyn Aspa, hold no direct investment interest in any company mentioned in this article.