FDA Decision Clears Way for Phase 2 Clinical Trial of BPN14770

The U.S. Food and Drug Administration (FDA) recently accepted the investigational new drug (IND) application for BPN14770, a small molecule therapeutic agent developed by Tetra Discovery Partners for the treatment of Fragile X syndrome and other autism spectrum disorders.

Tetra Discovery now plans to launch a Phase 2 clinical trial to evaluate BPN14770 in adults with Fragile X syndrome, in the second quarter of 2018. The study intends to evaluate the safety and tolerability of BPN14770 and to explore its potential benefits on behavioral, cognitive and biomarker measures.

“We are very pleased to receive clearance from FDA to initiate our planned Phase 2 study of BPN 14770 in adult males with Fragile X Syndrome,” Mark E. Gurney, PhD, said in a press release. Gurney is chairman and CEO of Tetra Discovery. “This study will be carried out at Rush University Medical Center in Chicago, under the direction of principal investigator, Elizabeth Berry-Kravis, MD, PhD, one of the foremost experts on Fragile X Syndrome and founder of the university’s comprehensive Fragile X Clinic and Research Program, which provides care to more than 600 patients with Fragile X Syndrome.”

Tetra Discovery also is planning to extend its evaluation of BPN14770 in Fragile X syndrome to pediatric patients in 2018. The company said it also is considering BPN14770 as a potential candidate for the treatment of memory cognitive problems associated with Alzheimer’s disease.

BPN14770 selectively inhibits the phosphodiesterase type-4D (PDE4D) enzyme, known to play key roles in memory formation and learning processes, but also in neuro-inflammation and traumatic brain injury.

BPN14770 was designed to enhance early and late stages of memory formation. Its unique mechanism of action potentially could improve cognitive and memory function in disorders like Fragile X syndrome, as preclinical studies in mouse models have shown that the small molecule can improve behavioral outcomes and the quality of connections between neurons.

The safety, tolerability and pharmacokinetic profile of BPN14770 already have been tested in two Phase 1 trials (NCT02840279, NCT02648672) and the company currently is testing the effects of BPN14770 (10 mg and 50 mg) in reversing cognitive impairment induced by a drug used to treat Alzheimer’s disease —  scopolamine — in healthy volunteers (NCT03030105).