Tetra Discovery Partners today announced the initiation of a Phase 2 study of BPN14770 as a potential treatment for Fragile X Syndrome, the most common genetic form of autism. A selective small molecule inhibitor of the phosphodiesterase type-4D (PDE4D) subtype, BPN14770 has shown the ability to improve the quality of connections between neurons and to improve multiple behavioral outcomes in the Fragile X mouse model. BPN14770 has also received Orphan Drug Designation from the U.S. Food and Drug Agency for the treatment of Fragile X Syndrome.
The study, a randomized, double-blind, placebo-controlled study including two 12-week crossover periods, is being conducted in 30 adult males (ages 18 – 45 years). Endpoints for the study include preliminary cognitive and behavioral assessments of the efficacy of BPN14770 by a variety of standard tests and determinations of the experimental drug’s safety and tolerability. The study will also gather pharmacokinetic and biomarker data on BPN14770. The study is being conducted at Rush University Medical Center, Chicago, Illinois by principal investigator Elizabeth M. Berry-Kravis, M.D., Ph.D. with financial support from the FRAXA Research Foundation. Additional information is available through clinicaltrials.gov (Identifier: NCT03569631).
“BPN14770 targets a basic biochemical change in how the connections between cells in the brain mature in patients with Fragile X Syndrome,” said Mark E. Gurney, Ph.D., Chairman and Chief Executive Officer of Tetra Discovery Partners. “We look forward to exploring the potential therapeutic benefit of BPN14770 in Fragile X patients with Dr. Berry-Kravis, a noted expert in this disorder.”
“Inhibition of PDE4 has been validated as a treatment strategy by many research groups in the Fragile X field,” said Michael Tranfaglia, M.D., Medical Director and Chief Scientific Officer of the FRAXA Research Foundation. “We are very pleased to support this clinical investigation of BPN14770 in patients with Fragile X Syndrome by Dr. Berry-Kravis, whose early research was instrumental to our understanding of biochemical changes underlying the condition.”