pipeline & programs
We have reported positive data from a Phase 2 clinical trial of our lead program, NB-001, a small-molecule mGluR activator for the potential treatment of the neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (“22q11DS”) in children.
NB-001 for 22q11DS
Nobias is developing NB-001 (fasoracetam) for the potential treatment of the neuropsychiatric symptoms associated with 22q11DS. NB-001 is a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs) and has been granted the FDA’s Rare Pediatric Disease (RPD) designation, recognizing its potential to address serious conditions affecting children. It would be a first-in-class treatment for patients with 22q11DS and certain comorbid neuropsychiatric conditions, including anxiety, attention-deficit/hyperactivity disorder (ADHD), and autism.
22q11DS, also known as DiGeorge syndrome, is a genetic disease in which part of chromosome 22 is missing, which can result in a variety of neuropsychiatric conditions and delayed cognitive development, as well as congenital disorders. 22q11DS is the most common human deletion syndrome, and second most common chromosomal abnormality after Down Syndrome. More than 1,600 children are born with 22q11DS every year in the United States, affecting 1 in every 2,148 births.
Illustration adapted with permission from McDonald-McGinn et al. 22q11.2 deletion syndrome. Nat Rev Dis Primers. November 2015.
Many people living with 22q11DS experience comorbid neuropsychiatric conditions that impact their mental health, including ADHD, anxiety, and autism. These conditions can have significant impacts on patients’ development and social wellbeing, as well as negatively affect their caregivers and families. Currently, there are no population-specific approved therapies for these conditions for people with 22q11DS, and many patients struggle with inadequate treatment options.
Nobias recently completed a multi-center, randomized, double blind, placebo-controlled Phase 2 trial of NB-001 in 22q11DS patients, partnering with pediatric medical centers across the U.S. and Canada. Results from the trial demonstrate the safety and tolerability of NB-001, as well as robust efficacy trends that support further evaluation in a registrational clinical trial.
The responder rate was 40-70% higher in patients treated with NB-001 versus placebo.
Topline data was announced at the 52nd Child Neurology Society (CNS) Annual Meeting and presented at the 13th Biennial International 22q11.2 Scientific Conference. Please see the study summary on ClinicalTrials.gov for more information or read our press release.
In addition to its potential to treat the unmet needs of children and adolescents living with 22q11DS, indication expansion opportunities for NB-001 include adults with 22q11DS and, more broadly, other patients living with conditions driven by genetic disruptions to glutamatergic signaling, such as co-morbid ADHD.
