The designation accelerates the development and regulatory review of investigational medications for the treatment of serious conditions and that fill an unmet medical need. It also enables early and frequent communication with the FDA, often resulting in faster treatment approval.
“[FXS] continues to have a high unmet medical need and has no FDA-approved therapies available. Fast Track designation enables us to work closely with the FDA to accelerate our efforts to potentially provide an impactful therapy to people with [FXS],” Amit Rakhit, MD, chief medical officer and portfolio management officer at Ovid, said in a press release.
“This important designation by the FDA comes on the heels of several other clinical and regulatory milestones for our OV101 program in recent months, including positive Phase 1 data, orphan drug designation for [FXS], and Fast Track designation for Angelman syndrome (AS),” Rakhit said. “We are committed to exploring the potential of OV101 to become a transformative medicine and we look forward to announcing further progress this year.”
In November 2017, Ovid completed a Phase 1 pharmacological and safety trial (NCT03109756) in people with Fragile X or Angelman syndrome, which showed that a single 5 mg dose of OV101 has a similar safety and tolerability profile in adolescents and young adults. These data will inform dosing in future studies and support the development of OV101 as a treatment for adolescents with FXS.
The company now will start a Phase 2 clinical trial in 2018, assessing OV101 for the treatment of FXS males aged 13 to 22.
FXS is the leading known single-gene cause of intellectual disability and autism, affecting one in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females in the U.S. The syndrome results from mutations in the FMR1 gene, which lead to loss of FMRP, a key protein in the production and activation of GABAA receptors in the brain.
Both experimental models and studies in individuals with FXS showed that levels of the neurotransmitter GABA are reduced outside the synapse — the communication between nerve cells — and that GABA receptors are dysregulated. This ultimately leads to a reduction in the brain’s ability to filter neural signals and separate critical information from background noise, a process known as tonic inhibition.
OV101 selectively activates a specific subunit of GABAA receptors and is the first treatment candidate targeting tonic inhibition, Ovid said.
The active ingredient of OV101, gaboxadol, showed a positive safety profile in clinical tests with more than 4,000 patients. Preclinical studies suggested that OV101 improves symptoms of Fragile X and Angelman syndromes.
The company anticipates to release topline results from its Phase 2 STARS clinical study (NCT02996305), which assesses OV101 in the treatment of adults and adolescents with Angelman, in the second half of 2018. The trial is recruiting participants.