Researchers at the University at Buffalo in collaboration with Tetra Therapeutics made a major advancement toward a breakthrough treatment for Fragile X syndrome, according to new results from a phase 2 clinical study.
Known as BPN14770, the treatment is purportedly being tested for its role in improving cognitive function among patients with Fragile X syndrome, a genetic condition associated with the development of autism.
The drug’s primary mechanism of action involves the inhibition of the enzyme phosphodiesterase‐4D.
The research team demonstrated that the drug is safe and well-tolerated, while drastically improving cognitive functions in the brain.
“In addition to being safe and well-tolerated, treatment with BPN14770 led to significant cognitive improvement, specifically in the language domains, and we also saw a clinically meaningful benefit in overall daily functioning,” the study reads.
“These findings validate our approach to treating this disease through a mechanism that addresses a core deficit in the disorder.”
The study was funded by the nonprofit organization FRAXA Research Foundation.