Results of Phase 2 Trial Testing OV101 in Fragile X Expected Later This Year

Results of Phase 2 Trial Testing OV101 in Fragile X Expected Later This Year
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Ovid Therapeutics is expecting to have data from a Phase 2 trial testing OV101 (gaboxadol) in teenagers and young adults with fragile X syndrome later this year.

The main goal of the randomized double-blind ROCKET study (NCT03697161) is to assess the safety and tolerability of OV101 in males with fragile X, ages 13 to 22.

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.

The experimental therapy previously received orphan drug and fast track designations by the U.S. Food and Drug Administration for the treatment of fragile X.

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.

The company also anticipates to announce new data from other trials assessing soticlestat (previously OV935/TAK935), Ovid’s lead therapeutic candidate for rare forms of epilepsy, later this year.

“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.

Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
Total Posts: 12
José is a science news writer with a PhD in Neuroscience from Universidade of Porto, in Portugal. He has also studied Biochemistry at Universidade do Porto and was a postdoctoral associate at Weill Cornell Medicine, in New York, and at The University of Western Ontario in London, Ontario, Canada. His work has ranged from the association of central cardiovascular and pain control to the neurobiological basis of hypertension, and the molecular pathways driving Alzheimer’s disease.
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Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns.
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