Treatment is being administered over 12 weeks to three groups of participants, each receiving OV101 orally on a different dosing schedule — one, two, or three times a day.
Secondary goals include assessing changes in participants’ behavior over the 12-week treatment period, using the Aberrant Behavior Checklist-Community scale adapted for fragile X.
OV101 is designed to be a selective and potent agonist (activator) of a particular subunit of GABAA receptors to restore tonic inhibition — the process by which the brain correctly identifies excitatory and inhibitory neural signals without being overloaded — and improve quality of life of people with fragile X and Angelman syndrome.
Also expected in 2020 are results of SKY ROCKET, which is evaluating the suitability of behavioral, sleep, and functioning scales in males with fragile X, ages 5 to 30. Investigators at Ovid are planning to use data from this trial to complement some of the goals being explored in ROCKET and as a reference for the benefits offered by standard therapies.
Ovid is also expecting top-line results from the Phase 3 NEPTUNE study (NCT04106557) to become available by mid-2020. This trial is investigating the safety and efficacy of OV101 in children with Angelman.
“We expect new late-stage data from our two lead programs and across multiple indications in 2020,” Jeremy Levin, chairman and CEO of Ovid, said in a press release. “We will report data from the Phase 2 ROCKET signal-finding trial in Fragile X syndrome, a program that has the potential to provide a clinical path forward in this poorly served area.”
“2020 is poised to be a pivotal year for our company and importantly, for the patients and families for which we strive every day to help,” Levin added.