Nat Med: BPN14770 improves the cognitive ability of patients with fragile X syndrome (FXS)

Fragile X syndrome (Fragile X syndrome, FXS) is a serious hereditary neurodevelopmental disorder, which is the most common X-linked mental retardation disease, which seriously affects patients' intelligence, behavior and daily functions.

Most male patients with FXS suffer from moderate to severe intellectual disability, with an average IQ between 40-45 and an average mental age of only 5-6 years.

BPN14770 is an allosteric inhibitor of PDE4D (phosphodiesterase 4D), which is selective for PKA-activated PDE4D dimer.

Clinical Results Forest Plot

The main outcome of the trial is the safety and tolerability of the drug, followed by the patient’s cognitive performance, the caregiver rating scale, and the physician rating scale.

Exploratory biomarker reveals that BPN14770 is better than placebo

The cognitive benefits of patients were also evaluated through oral reading recognition, picture vocabulary and cognitive comprehensive scores developed by the National Institutes of Health.