Tetra Discovery Partners has commenced a Phase ll trial of BPN14770 for the treatment of Fragile X Syndrome, the most common genetic form of autism.
The randomised, double-blind, placebo-controlled trial includes two 12-week crossover periods.
A total of 30 adult males aged between 18 and 45 years have been enrolled for the trial at Rush University Medical Center in Chicago, Illinois.
The trial’s endpoints are preliminary cognitive and behavioural assessments of the efficacy of BPN14770 by several standard tests and determination of the drug’s safety and tolerability.
It also aims to collect pharmacokinetic and biomarker data on BPN14770.
To be conducted with financial support from the US’ FRAXA Research Foundation, the trial has appointed Elizabeth Berry-Kravis as its principal investigator.
Tetra Discovery Partners chairman and CEO Mark Gurney said: “BPN14770 targets a basic biochemical change in how the connections between cells in the brain mature in patients with Fragile X Syndrome.”
BPN14770 is an investigational therapeutic agent that selectively inhibits phosphodiesterase-4D (PDE4D) to improve early and late stages of memory formation.
It has the potential to improve cognitive and memory function in various devastating disorders such as Fragile X Syndrome, Alzheimer’s disease and other dementias, learning/developmental disabilities and schizophrenia.
In preclinical studies of Fragile X Syndrome, BPN14770 has demonstrated its ability to enhance behavioural results in the Fragile X mouse model and improves the quality of connections between neurons.
In addition, BPN14770 has completed three Phase l clinical trials that included 147 adult human subjects and has demonstrated improved safety, oral bioavailability, and preliminary cognitive benefit.