Positive Phase II Study of BPN14770 in patients with Fragile X syndrome

Shares of Japanese drugmaker Shionogi (TYO: 4507) rose 4.65% to 4,153 yen today, after it announced that BPN14770, a drug candidate for the treatment of brain disorders associated with cognitive and memory deficits, has shown positive top-line results from Phase III exploratory study in adult patients with Fragile X syndrome (FXS) conducted by Tetra Therapeutics, which is a wholly-owned subsidiary.

BPN14770 is a novel therapeutic agent that selectively inhibits phosphodiesterase-4D (PDE4D) to enhance early and late stages of memory formation. This unique mechanism of action has the potential to improve cognitive and memory function in devastating CNS disorders, including FXS, Alzheimer disease (AD) and other dementias, learning/developmental disabilities and schizophrenia. In the Phase II study in AD patients, BPN14770 showed a trend toward improvement in cognitive function. In pre-clinical studies, BPN14770 promoted the maturation of connections between neurons, which is impaired in patients with FXS.

Shionogi first partnered with Tetra Discovery on BPN14770 in December 2028, but in May this year, Shionogi took up its option to acquire full ownership of the US biotech firm in a transaction valued at $500 million.