Shots:

• The P-II study is a two-way crossover study assessing BPN14770 (25mg- bid) vs PBO in 30 adult male patients aged 18-45yrs. with FXS due to >200 CGG repeats in the FMR1 gene
• The study demonstrated benefits in oral reading recognition (+2.80)- picture vocabulary (+5.79)- cognition crystallized composite score (+5.29)- benefits were maintained up to 12wks. and was well tolerated
• BPN14770 is a novel therapy that selectively inhibits PDE4D to increase the levels of cAMP. In preclinical trials- it promotes the maturation of connections between neurons- which is impaired in patients with FXS and has received the US FDA’s ODD as well as approved for investigational use in the US

  Ref: Businesswire | Image: Tetra Therapeutics