A US drugs firm has started a study on a potential new treatment for Fragile X Syndrome.
Tetra Discovery Partners announced a phase two trial on the treatment, known as BPN14770.
The drug can improve both behaviour and the quality of connections between neurons in mice with Fragile X, says Tetra.
Mark E Gurney, chairman of Tetra Discovery Partners: he says BPN14770 targets a basic biochemical change in how the connections between cells in the brain mature
Half of those with Fragile X also have autism
Fragile X is the most common genetic form of autism.
The genetic disorder causes inherited learning disabilities. It may affect around one in 4,000 males and one in 8,000 females.
It’s thought that around half of those with Fragile X also have autism.
The Rush University Medical Center in Chicago, Illinois, is conducting the study.
Drug ‘targets basic biochemical change’
Mark E Gurney is chairman and chief executive of Tetra. He said the drug “targets a basic biochemical change in how the connections between cells in the brain mature”.
Phase II trials focus on assessing how well a drug works, as well as continuing earlier safety assessments on a larger group of patients.
The BPN14770 trial is a randomized, double-blind, placebo-controlled study. This is where researchers give patients an inactive ingredient to compare against the drug itself.
In an attempt to prevent reporting bias, neither researchers nor patients know who is receiving the drug or the inactive ingredient.
The study includes two 12-week crossover periods. This is where researchers give patients the inactive ingredient then the drug, while those given BPN14770 change over to the placebo.
The work is being conducted on 30 adult males aged 18 to 45 years.
BPN14770 has completed three human phase one clinical trials, involving 147 subjects. Tetra says the drug has shown excellent safety and preliminary cognitive benefits in elderly people.
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Published: 23 July 2018
