SPG601 granted FDA fast track designation for fragile X
Top-line data from Phase 2a trial expected in 1st quarter, Spinogenix says
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 will provide Spinogenix incentives such as more frequent communication with the FDA.
The FDA’s move “demonstrates [SPG601’s] potential to impact patients’ lives and brings us one step closer to providing a new therapeutic to combat FXS,” Stella Sarraf, PhD, founder and CEO of Spinogenix, said in a company press release.
The company said it has completed its Phase 2a trial (NCT06413537) testing SPG601 in 10 men with FXS, and plans to announce top-line data in the first quarter.
“We remain focused on developing SPG601 as a first-in-class treatment for FXS capable of restoring synapse function, and the completion of this trial and this designation will allow us to accelerate its development to offer a much-needed therapy that can improve patients’ quality of life,” Sarraf said.
Boosting synapse health
FXS is a genetic disorder marked by a range of neurological symptoms, including intellectual disability, language delays, motor problems, and behavioral issues, due to changes in brain function.
SPG601 aims to help normalize brain function by boosting the health of synapses, which are the regions of near contact where individual nerve cells communicate with each other. In FXS, the structure and function of synapses are significantly affected, contributing to the neurological and cognitive symptoms seen in the condition.
The therapy works to increase the activity of nerve proteins called large-conductance calcium-activated potassium channels, which are important for synapse function.
“The possibility that SPG601 could address underlying synaptic deficits central to this condition in people with FXS brings immense hope, and with this designation, we are even closer to making it available to patients sooner,” Erickson said.
The now-completed Phase 2a study investigated the safety, tolerability, pharmacological properties, and efficacy of SPG601 in men with FXS. Patients were given a single dose of either SPG601 or a placebo, taken as eight, 100 mg capsules. A week later, those given a placebo were given a single dose of SPG601, and vice versa.
The study’s main goals are to evaluate the effect of treatment on measures of overall function, as well as objective tests of brain function.
“I am thrilled that the FDA has granted Fast Track designation to SPG601 for FXS, highlighting its potential as a novel therapeutic option for a disease that, to this day, has no approved treatment,” said Craig Erickson, MD, Spinogenix’s chief medical advisor and principal investigator of the Phase 2 study.
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