Spinogenix’s Investigational New Drug Application for Novel Treatment for Fragile X Syndrome Cleared to Begin Phase II Trial


Phase IIa clinical trial of SPG60 for Fragile X syndrome offers hope for disease with no current FDA-approved treatment options.

neurodevelopmental disorder. Image Credit: Adobe Stock Images/Toon

Image Credit: Aobe Stock Images/Toon

Spinogenix announced that the FDA has given authorization to its Investigational New Drug (IND) application for SPG601, a medicine in development to treat patients with Fragile X syndrome (FXS). According to the company, the IND clearance has led to the launch of a Phase IIa clinical trial for SPG601 for FXS. The treatment is a novel small molecule designed to enhance synaptic function by activating BK channels, which is commonly scarce in patients with FXS. Currently, there are no treatments for FXS that are approved by the FDA.1

“The FDA approval of our US IND for SPG601 for FXS represents a significant milestone for the company as we look to expand our pipeline of game-changing therapeutics that aim to restore synaptic function,” said Stella Sarraf, PhD, founder, CEO, Spinogenix, in a press release. “Current treatments leave a critical gap in effective and patient-friendly solutions for neurodevelopmental conditions. Like many other conditions, loss of synaptic function remains a key driver of disease, and for that reason we are excited to launch our first U.S. trial to address this unmet need in FXS. The expansion of clinical programs with SPG601 represents an important step in our progress to bringing innovative treatments that offer new hope, and we look forward to dosing the first patient in the trial this year.”

According to the Centers for Disease Control and Prevention (CDC), FSX is the most commonly inherited intellectual disability. Typically, female patients tend to have milder symptoms than males, and while exact numbers are unknown, it is estimated that approximately one in every 7,000 males and about one in 11,000 females have been diagnosed with FXS.2

“Young children with FXS often take longer than their peers without FXS to reach early developmental milestones and to develop nonverbal communication (communication without words, typically through gestures, facial expressions, and body language),” reports the CDC.

Additional facts and statistics regarding FXS include:

  • It typically manifests in boys anywhere between 35 and 37 months of age. For girls, diagnosis is usually around 42 months of age.
  • Patients with FXS tend to have lower IQ scores as they reach adolescence and adulthood. Boys diagnosed with FXS have an average IQ of 55, which is considerably lower than the average of 100 globally.
  • Four out of 10 families have reported that they have taken their child to a physician close to 10 times before reaching an actual diagnosis of FXS.2

“Despite the considerable impact of FXS, there are currently no FDA-approved drugs available for those with the condition, said Craig Erickson, MD, chief medical advisor, Spinogenix, in the press release. “SPG601’s novel mechanism works directly at BK channels and BK channel function has been shown to be abnormal in many animal studies in the Fragile X field. We look forward to the first study of a BK channel modulator in humans with Fragile X and taking the first step to evaluate this important drug mechanism in Fragile X Syndrome.”

The upcoming clinical trial is expected to assess the neurophysiological and clinical outcomes of SPG601 in adult men with FXS.1


1. Spinogenix Announces U.S. FDA Approval of its Investigational New Drug Application for its Phase 2a Clinical Trial of SPG601 for Fragile X Syndrome. GlobeNewswire. April 15, 2024.Accessed April 15, 2024. https://www.globenewswire.com/news-release/2024/04/15/2862713/0/en/Spinogenix-Announces-U-S-FDA-Approval-of-its-Investigational-New-Drug-Application-for-its-Phase-2a-Clinical-Trial-of-SPG601-for-Fragile-X-Syndrome.html

2. Data and Statistics on Fragile X Syndrome. CDC. Acces15, 2024. https://www.cdc.gov/ncbddd/fxs/data.html#:~:text=FXS%20affects%20both%20males%20and,have%20milder%20symptoms%20than%20males.&text=The%20exact%20number%20of%20people,have%20been%20diagnosed%20with%20FXS.

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