Three Clinical Trials Will Continue to Assess Effectiveness of BPN14770 in Fragile X

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by Patricia Inácio, PhD |

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Tetra Therapeutics has launched three pivotal Phase 2b/3 studies to continue to assess the safety and effectiveness of BPN14770, its investigational oral therapy for fragile X syndrome.

The results, if they’re in agreement with previous positive data from a Phase 2 trial, will support Tetra’s application for approval of BPN14770 with the U.S. Food and Drug Administration (FDA). The therapy has previously received orphan drug designation by the U.S. Food and Drug Administration for treating fragile X.

“We are excited to initiate these pivotal trials with BPN14770 in patients with fragile X syndrome, and are hopeful that the results of these studies will enable us to seek approval from FDA,” Chad Coberly, CEO of Tetra Therapeutics, said in a press release.

BPN14770 works by selectively binding to and partially suppressing the activity of phosphodiesterase 4D (PDE4D), an enzyme known to regulate a brain-signaling molecule involved in learning and memory called cyclic adenosine monophosphate (cAMP).

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Levels of cAMP are reduced in fragile X patients. By boosting them, BPN14770 may help promote nerve cell connections and improve cognitive function in fragile X patients.

The first two trials will randomly assign two separate groups of patients to a placebo or BPN14770 – one trial (NCT05163808) will recruit male adolescents, ages 12-17; while a second trial (NCT05358886) will recruit an adult male population, ages 18-45.

The trial for adolescents, underway at the Rush Medical Center, Chicago and Cincinatti Children’s Hospital Medical Center, Ohio is currently recruiting participants.

The studies’ main goal is to assess the effectiveness of BPN14770 in improving cognition after 13 weeks (around three months) of treatment, as measured by the computer-administered National Institutes of Health-Toolbox Cognitive Battery (NIH-Toolbox, a set of cognitive tests). Additional goals include the therapy’s safety and tolerability.

The trial for adolescents will also gather data on BPN14770’s pharmacokinetics, or its movement into, through, and out of the body.

Adolescents and adults who complete these trials will be able to enroll in an open-label extension study (NCT05367960) where they’ll receive BPN14770 only. The studies will be conducted over the next two years.

“We are anxious to get started with these studies that offer hope for people living with Fragile X Syndrome and their caregivers,” Elizabeth Berry-Kravis, MD, pediatric neurologist, Rush University Medical Center, said. Berry-Kravis is the trial’s principal investigator.

Data from a previous Phase 2 trial showed BPN14770 was effective at improving cognitive function, particularly language-related domains, and daily functioning in men with fragile X syndrome.

“Given our previous positive results with BPN14770 in the phase 2 study, we are hopeful these studies at a larger scale will show similar results. I find it exciting that we have a drug that potentially corrects a core deficit in FXS, a decrease in cAMP, that has been documented in patients as well as in the fly and mouse models of the disorder,” Berry-Kravis said.

Tetra also is evaluating BPN14770 as a potential treatment for Alzheimer’s disease.