FDA Grants Orphan Drug Designation for Tetra Discovery Partners’ BPN14770 for the Treatment of Fragile X Syndrome

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.

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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