Anavex Life Sciences Corp. will receive funding for a phase 2 trial of its Anavex 2-73 drug from the International Rett Syndrome Foundation to study the rare neurological disorder, the company announced early Thursday.
The $600,000 grant, which will fund the majority of Anavex’s AVXL, -0.71% clinical trial, will be the fifth clinical trial for Rett syndrome funded by the foundation and underway this year. Other biotech companies with funded trials include Neuren Pharmaceuticals Ltd. NEU, +8.20%
Rett syndrome is a rare condition affecting about one in 10,000 girls that’s caused by a mutation on the X chromosome. Symptoms include motor and cognitive impairments, seizures and anxiety, and many of those affected die young.
Many in the Rett syndrome community are looking in the long term to gene therapy for a treatment. But having a drug that works across the broad spectrum of symptoms — as opposed to taking medications for each one — is a more near-term goal, said Gordy Rich, the Rett foundation’s chief operating officer.
Rich is also a Rett parent. The prospect of a more comprehensive medication for his 22-year-old daughter is “life-altering,” he said.
“When my daughter was diagnosed there was no known cause and no known cure,” Rich said. Five clinical trials being underway means “to all parents, this is really the most incredible time… We’ll have options for our girls that can improve their quality of life.”
Anavex 2-73, which was designated as an orphan drug for Rett syndrome last spring — which grants companies developing rare diseases various development and commercial incentives— may have that potential.
The 12-week trial — which will cost about $1 million total and enroll between 50 and 80 patients — will measure the drug’s effect on seizure reduction, cognitive impairment, mood disorder, autistic behavior and anxiety, Christopher Missling, president and chief executive officer of Anavex, told MarketWatch.
The drug was selected based on its results in mice, where it addressed a number of symptoms, Rich said.
“To us that’s very exciting, and we want to move as many of the compounds into clinical trials so we can get treatments on the path to a cure,” he said.
The trial is expected to begin this year, with data possibly coming out by the end of the year, Missling said.
Anavex’s relationship with the foundation will also assist in enrolling patients in the trial, which should help hasten trial results, he said.
Anavex 2-73 is also being tested in other areas, including Alzheimer’s disease, Parkinson’s disease, autism and more.