Tetra Discovery Partners announced that the U.S. Food and Drug Administration has accepted the Investigational New Drug Application (IND) for BPN14770, a novel, selective small molecule inhibitor of the phosphodiesterase type-4D (PDE4D) enzyme, which research suggests may be useful in the treatment of Fragile X Syndrome and possibly other autism spectrum disorders.
Tetra plans to initiate a U.S. Phase 2 trial of BPN14770 in adults with Fragile X Syndrome in 2Q 2018. Primary objectives for the study will be to evaluate the safety and tolerability of BPN14770 in adult males with Fragile X Syndrome and to explore potential benefits on behavioral, cognitive and biomarker measures. Tetra plans to extend its evaluation of BPN14770 in Fragile X Syndrome to pediatric patients later in 2018.
The company is also developing BPN14770 for the treatment of memory cognitive problems associated with Alzheimer’s disease.
“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,” said Mark E. Gurney, Ph.D., Chairman and Chief Executive Officer of Tetra Discovery Partners. “This study will be carried out at Rush University Medical Center in Chicago, under the direction of principal investigator, Elizabeth Berry-Kravis, MD, Ph.D, 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.”