News

KER-0193, an oral therapy being developed as a fragile X syndrome treatment, has been awarded orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). These designations — which follow news of positive Phase 1 trial data from a test in healthy volunteers and…

Long-term, daily treatment with the investigational cannabidiol (CBD) gel ZYN002 — also known as Zygel — led to clinically meaningful reductions in irritability and other behavioral symptoms in people with fragile X syndrome. That’s according to up to three years of data from an ongoing open-label extension…

KER-0193, Kaerus Bioscience’s oral treatment candidate for fragile X syndrome, was safe and well tolerated, and showed predictable pharmacological properties, in healthy volunteers, according to new data from a Phase 1 clinical trial. The therapy was also associated with changes in brain activity in regions known to be…

A single dose of Spinogenix’s investigational oral therapy SPG601 led to significant reductions in a measure of abnormal brain activity among men with fragile X syndrome, according to top-line results from a Phase 2a trial. Announced by Spinogenix in a company press release, the results from the…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to Spinogenix‘s experimental oral therapy SPG601 to treat fragile X syndrome (FXS). The designation aims to speed the development and review of new medicines that have the potential to improve medical care for serious conditions. It…

Researchers in Kansas and California will investigate people with the FMR1 premutation who are at risk of developing fragile X-associated tremor/ataxia syndrome (FXTAS), a condition related to fragile X syndrome that develops late in life. The five-year project, supported by a $3.1-million grant from the National Institutes of Health, will…

  A Phase 2 clinical trial to test MRM-3379, an experimental oral treatment for fragile X syndrome, is expected to launch in 2025. Mirum Pharmaceuticals announced the plan in a company press release reporting financial results and business updates. It didn’t provide details on the trial’s design…

With $1.6 million in pre-seed funding, researchers have launched a new biotech company called Aptadir Therapeutics that will develop RNA-based therapies to reactivate specific genes that are silenced in cancer and genetic diseases such as fragile X syndrome. The therapies will be based on a new class of RNA…

The nonprofit FRAXA Research Foundation has teamed up with Marvel Biosciences to test MB204, a drug that blocks adenosine A2A receptors, in a preclinical model of fragile X syndrome. As part of the collaboration, FRAXA will run the preclinical studies at its…

Tetra Therapeutics has amended the protocols of two ongoing late-stage clinical trials testing zatolmilast, its experimental oral therapy for fragile X syndrome, to improve access for patients and their families. The similarly designed studies are recruiting up to 300 male fragile X patients at 15 sites across the…