Tetra Discovery Partners today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for BPN14770 for the treatment of Fragile X Syndrome. BPN14770 is a selective small molecule inhibitor of the phosphodiesterase type-4D (PDE4D) subtype. Research conducted collaboratively with the FRAXA Research Foundation has shown BPN14770 to improve the quality of connections between neurons and to improve multiple behavioral outcomes in the Fragile X mouse model.
“We are very pleased that FDA has recognized the potential benefit of BPN14770 in the treatment of Fragile X Syndrome, a genetic disorder for which there is neither a cure nor any approved therapy,” said Mark E. Gurney, Ph.D., Chairman and Chief Executive Officer of Tetra Discovery Partners. “BPN14770, which has potential to improve cognitive and memory function in a variety of devastating brain disorders, addresses one of the biochemical dysfunctions involved in Fragile X Syndrome and possibly other autism spectrum disorders.”
Tetra Discovery Partners is preparing to initiate a Phase 2 clinical study of BPN14770 in Fragile X Syndrome with support from the FRAXA Research Foundation during Q2 2018.