Nobias Therapeutics is focused on developing innovative small-molecule therapeutics aimed at addressing rare pediatric diseases, leveraging their access to extensive genomic and health datasets. Their primary programs include NB-001, a small-molecule mGluR activator designed to treat neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS) in children, and NB-002, a small-molecule MEK inhibitor targeting complex lymphatic anomalies. The company has reported positive results from a Phase 2 clinical trial for NB-001, demonstrating its safety, tolerability, and efficacy trends, which suggest a significant responder rate compared to placebo. This program has received the FDA's Rare Pediatric Disease designation, highlighting its potential to fill a critical gap in treatment options for children affected by 22q11DS and related neuropsychiatric conditions.

In addition to NB-001, Nobias is advancing NB-002 for the treatment of complex lymphatic anomalies, which are rare and potentially life-threatening conditions caused by mutations in the Ras/MAPK pathway. Currently, there are no approved therapies for these conditions, which include Gorham Stout disease and kaposiform lymphangiomatosis. Nobias is conducting IND-enabling studies for NB-002, aiming to establish its clinical efficacy and safety through rigorous trials. The company’s approach includes evaluating various MEK inhibitors using zebrafish models to identify the most promising candidates for development, thereby addressing significant unmet medical needs in both pediatric and adult populations affected by these rare diseases.

United States

Nobias Therapeutics is a clinical-stage company developing novel small molecule therapeutics for rare diseases. Our unique access to rich genomic and health datasets and strong relationships with pediatric academic medical centers across North America allow us to rapidly advance drug discovery for genetically-defined rare diseases, with an initial focus on pediatric indications, but expansion opportunities in adults and non-rare indications. 

Nobias Therapeutics

Company Details