Fragile X Syndrome Therapy Granted Orphan Drug Designation


BPN14770, in development for the treatment of Fragile X syndrome, was granted Orphan Drug Designation by the U.S. Food and Drug Administration this morning.

BPN14770, in development for the treatment of Fragile X syndrome, was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) this morning.

Earlier this month, the FDA accepted Tetra Discovery Partners’ Investigational New Drug (IND) Application for the drug. Historical research has shown BPN14770’s ability to improve the quality of connections between neurons. Additionally, the drug has exhibited the potential to improve multiple behavioral outcomes in mouse models of Fragile X.

“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,” said Mark E. Gurney, Ph.D., Chairman and Chief Executive Officer of Tetra Discovery Partners in a press release.

BPN14770 is a phosphodiesterase-4D (PDE4D) inhibitor intended to enhance early and late stages of memory formation. Its novel mechanism of action has demonstrated the potential to improve cognitive and memory function, not only in Fragile X syndrome, but also in other devastating neurological disorders like Alzheimer’s disease and other forms of dementia and schizophrenia.

BPN14770 has completed a trio of human Phase 1 clinical trials, enrolling 147 subjects in total, and showing exceptional safety, oral bioavailability, and preliminary cognitive benefit in elderly subjects.

The current standard of care for Fragile X syndrome, which occurs in an estimated 1 in 4,000 males and is half as common in females, is primarily the treatment of symptoms, such as seizures, mood swings or other neuropsychiatric issues. If approved, BPN14700 would be the first available treatment specifically for this indication.

“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,” Gurney said.

The drug is being prepared by Tetra Discovery Partners for the initiation of a Phase 2 trial in adults with the condition with support from the FRAXA Research Foundation in Q2 2018. Primary objectives include an evaluation of the safety and tolerability of BPN14770 in adult males with Fragile X Syndrome, as well as an exploration of potential benefits on behavioral, cognitive and biomarker measures.

This study will be carried out at Rush University Medical Center in Chicago, under the direction of principal investigator, Elizabeth Berry-Kravis, MD, PhD, one of the foremost experts on Fragile X Syndrome and founder of the university’s comprehensive Fragile X Clinic and Research Program, which provides care to more than 600 patients with Fragile X Syndrome.

Tetra intends to extend its evaluation of BPN14770 in Fragile X Syndrome to pediatric patients later this year.

For more from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter.

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