Tetra Discovery Partners Initiates Phase 2 Trial of BPN14770 in Fragile X Syndrome

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).

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“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.”

About BPN14770

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 CNS disorders including Alzheimer’s disease and other dementias, Fragile X Syndrome, learning/developmental disabilities, and schizophrenia.

Preclinical animal models show that BPN14770 has the potential to promote the maturation of connections between neurons, which is impaired in patients with Fragile X Syndrome, and to protect connections between neurons which otherwise are lost in patients with Alzheimer’s disease. Tetra currently is conducting an investigational Phase 2 study of BPN14770 in adults with early Alzheimer’s disease and a second investigational Phase 2 study in adults with Fragile X Syndrome, an indication for which BPN14770 has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA). BPN14770 currently is approved for investigational use only by the U.S. Food and

Drug Administration and is not currently approved for marketing in any territory.

About Tetra Therapeutics

Tetra Therapeutics is a clinical stage biotechnology company developing a portfolio of therapeutic products that will bring clarity of thought to people suffering from Alzheimer’s disease, Fragile X Syndrome, traumatic brain injury, and other brain disorders. Tetra uses structure-guided drug design to discover mechanistically novel, allosteric inhibitors of phosphodiesterase 4 (PDE4), an enzyme family that plays key roles in memory formation, learning, neuroinflammation, and traumatic brain injury.  BPN14770 was developed through a cooperative research agreement with the Blueprint Neurotherapeutics Program of the National Institutes of Health with funding from the National Institute on Aging, National Institute on Neurological Disorders and Stroke, the National Institute of Mental Health, the FRAXA Research Foundation and the Alzheimer’s Drug Discovery Foundation. Tetra Therapeutics is headquartered in Grand Rapids, Michigan. For more information, please visit the company’s website at http://www.tetratherapeutics.com.

Forward looking Statements

Certain other statements made throughout this press release that are not historical facts contain forward‐looking statements regarding the company’s future plans, objectives and expected performance. Any such forward‐looking statements are based on assumptions that the company believes are reasonable, but are subject to a wide range of risks and uncertainties and, therefore, there can be no assurance that actual results may not differ materially from those expressed or implied by such forward‐looking statements.

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Tetra Therapeutics:
Mark Gurney, Ph.D., Chief Executive Officer
Tetra Therapeutics
info@tetratherapeutics.com

For Media:  
Joan Kureczka
Bioscribe, Inc.
Joan@bioscribe.com
+1 (415) 821-2413

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