Nospharma announces a new strategic partnership with the the FRAXA Research Foundation to conduct pre-clinical testing of NOS-01, a potential new treatment for Fragile X Syndrome. This partnership aims to validate NOS-01's efficacy using FRAXA's standardized preclinical testing platform to accelerate development for this Fragile X.
MONTREAL and PHILADELPHIA, Feb. 4, 2026 /PRNewswire-PRWeb/ -- Nospharma, a company specializing in the development of treatments for rare pediatric neurodevelopmental disorders announced today that it has entered a collaboration with the FRAXA Research Foundation to test its lead asset NOS-01 in a preclinical model (FMR1) of Fragile X Syndrome (FXS).
FXS is a one of the most common causes of inherited intellectual disability affecting approximately 1 in 4000 males and 1 in 8000 females. There are currently no approved drugs to treat FXS. The FRAXA Research Foundation is a non-profit organization that promotes awareness of Fragile X and supports independent pre-clinical testing of potential therapeutics using a battery of standardized tests in mouse models of FXS.
Through the FRAXA Drug Validation Initiative (FRAXA-DVI), scientists run dozens of experiments on compounds, evaluating their potential to correct Fragile X-related deficits. The FRAXA-DVI team is led by Dr. Patricia Cogram, Associate Professor of Genetics at Fundacion Ciencia y Vida, Santiago, Chile, and Dr. Robert Deacon who has over forty years of experience in rodent behavior as the head of the rodent behavior unit at Oxford University.
FRAXA will test Nospharma's lead asset NOS-01 in the first half of 2026. FRAXA previously supported preclinical work in FXS with compounds developed by Tetra Therapeutics (acquired by Shionogi in 2020) and Kaerus (acquired by Servier in 2025).
"We are very interested in testing NOS-01 in our independent laboratories for two primary reasons", said Dr. Mike Tranfaglia, the Co-Founder and Medical Director of FRAXA. "First, Nospharma has shown the two components of NOS-01 improve neuroplasticity and neurovascular coupling in FXS animal models. Second, the two components of NOS-01 have previously been tested in humans which is expected to accelerate the timeline to a proof-of-concept Phase 2 clinical trial".
About Nospharma
Nospharma is an emerging pharmaceutical company primarily focused on the development and commercialization of a proprietary drug candidate, NOS-01, to treat neurodevelopmental disorders with intellectual disability including Fragile X Syndrome, SynGAP1 Syndrome, GRIN Disorder, and Phelan-McDermid Syndrome. The technology behind NOS-01 was originally developed at McGill University by Dr. Derek Bowie, a former NIH researcher, whose research into glutamatergic receptors was recently published in Nature and highlighted in the The Scientist. Dr. Bowie is the Chief Scientific Officer and co-founder of Nospharma.
For more information, please visit the Company's website, https://www.nospharma.ca.
About Fragile X Syndrome (FXS)
Fragile X Syndrome (FXS) is a genetic condition that causes intellectual disability, behavioral and learning challenges. Behavioral characteristics can include ADHD, autism and autistic behaviors, social anxiety, stereotypic movements, poor eye contact, sensory disorders and increased risk for aggression. FXS is the leading known genetic cause of autism, accounting for about 2-6% of cases. FXS represents an unmet medical need and a rare disease, as defined by the Orphan Drug Act. According to the National Institutes of Health (NIH), approximately one in 4,000 males and one in 8,000 females have FXS. Approximately 80,000 Americans and a similar number of Western Europeans are affected by FXS. The FDA has not approved any drugs specifically for the treatment of FXS or its symptoms.
About FRAXA
FRAXA is a nonprofit organization headquartered in Massachusetts, USA, dedicated to supporting research and raising awareness for the development of treatments for Fragile X Syndrome.
For more information, visit https://www.fraxa.org
Media Contact
Anmol Nagpal, Nospharma, 1 5148352714, [email protected], nospharma.ca
SOURCE Nospharma
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