The National Institutes of Health (NIH) today announced three new projects being funded through its Blueprint Neurotherapeutics Network initiative, which was launched in 2011. Investigators at Cambridge, MA-based Sage Therapeutics, The Scripps Research Institute, and the University of Utah have won funds to develop drugs for Fragile X syndrome (FXS), nicotine addiction, and age-related macular degeneration, respectively.
NIH explained that its Blueprint initiative “uses a virtual pharma model to provide researchers with access to support and resources that have been traditionally available to large pharmaceutical companies.” In a statement, the National Institute of Neurological Disorders and Stroke’s Rebecca Farkas, Ph.D., program director at the Office of Translational Research, said NIH is “excited about the opportunity to apply cutting-edge science to the pursuit of novel treatments for these debilitating disorders.”
Through this initiative, NIH program staff form partnerships with awardee investigators in an effort to “bridge the funding gap between ground-breaking laboratory research and industry adoption,” the agency said. “NIH staff helps investigators work with veteran industry drug development consultants and contract research organization capabilities from the discovery stage through preliminary clinical trials.” NIH noted that awardees maintain sole ownership of the IP associated with their projects.
Investigators at Sage have won up to $10 million to develop a drug to treat anxiety and social deficits in patients with FXS. The firm is working to develop a positive allosteric modulator (PAM) targeting GABAA, which it said “may provide symptomatic and potentially disease-modifying therapeutic benefits to patients with FXS, with a focus on ameliorating anxiety and social deficits.”
In a statement, Sage interim CEO Kevin Starr noted that “the vast majority of patients with Fragile X suffer from debilitating fear, anxiety and social dysfunction, and there are no approved drugs for this devastating condition.” He added: “There is hope that in developing treatments for a monogenic form of autism like Fragile X, we may be able to make significant advances toward developing treatments for other forms of autism.”
Added Al Robichaud, Ph.D., CSO: “The hope is that this grant will enable us to accelerate our development of an innovative therapy designed to address the symptoms of Fragile X, and could potentially have a durable, disease-modifying effect, as well.”
The firm said it is planning to advance its FXS program toward a Phase I clinical trial within the next two years.
