• BPN14770 has received orphan drug designation
• Also in trials for Alzheimer's and traumatic brain injury
• Ph I trials showed excellent safety and improved cognitive capability in elderly subjects

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.

BPN14770 is also in trials for Alzheimer's and traumatic brain injury.

“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.”

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 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 disorders including Fragile X Syndrome, Alzheimer’s disease and other dementias, learning/developmental disabilities and schizophrenia. In preclinical studies of Fragile X Syndrome, BPN14770 improves behavioral outcomes in the Fragile X mouse model and improves the quality of connections between neurons. BPN14770 has completed three human Phase 1 clinical trials enrolling 147 subjects and has shown excellent safety, oral bioavailability, and preliminary cognitive benefit in elderly subjects. Tetra Discovery has initiated a Phase 2 study of BPN14770 in adults with Fragile X Syndrome, an indication for which BPN14770 has received Orphan Drug Designation from the U.S. Food and Drug Administration. Preparations are also under way to initiate a Phase 2 trial of BPN14770 in patients with Alzheimer’s disease

A Ph I trial of BPN14770 that involved 45 elderly volunteers demonstrated a significant improvement measures of working memory.