Nobias Therapeutics
Nobias Therapeutics developed an AI-enabled deep phenotyping platform to identify disease drivers from genomic and electronic health record (EHR) datasets and apply those insights to small-molecule therapeutic development. The company advanced programs for neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (NB-001, an mGluR activator) and a MEK-directed program (NB-002), completed a randomized placebo-controlled Phase 2 pediatric trial for NB-001, and pursued regulatory interactions including orphan and rare pediatric disease designations. Substantially all assets have been acquired by Synaptiq Therapeutics (as noted on the site).
Industries
Nr. of Employees
small (1-50)
Nobias Therapeutics
Products
NB-001 (fasoracetam) — mGluR activator
Orally dosed small-molecule mGluR activator developed to treat neuropsychiatric symptoms associated with 22q11.2 deletion syndrome and related comorbid conditions (anxiety, ADHD, autism); advanced through a randomized Phase 2 pediatric crossover trial showing safety/tolerability and efficacy trends.
NB-002 (MEK pathway program)
MEK pathway-directed small-molecule program intended for diseases with MEK-relevant biology (listed indications include congenital lymphatic anomaly (CLA) with pulmonary involvement and other organ involvement); earlier-stage program in the portfolio.
NB-001 (fasoracetam)
NB-001 is a small-molecule mGluR activator developed for the potential treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS) in children. It is a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs) and has received FDA Rare Pediatric Disease designation.
NB-001 (fasoracetam) — mGluR activator
Orally dosed small-molecule mGluR activator developed to treat neuropsychiatric symptoms associated with 22q11.2 deletion syndrome and related comorbid conditions (anxiety, ADHD, autism); advanced through a randomized Phase 2 pediatric crossover trial showing safety/tolerability and efficacy trends.
NB-002 (MEK pathway program)
MEK pathway-directed small-molecule program intended for diseases with MEK-relevant biology (listed indications include congenital lymphatic anomaly (CLA) with pulmonary involvement and other organ involvement); earlier-stage program in the portfolio.
NB-001 (fasoracetam)
NB-001 is a small-molecule mGluR activator developed for the potential treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome (22q11DS) in children. It is a non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs) and has received FDA Rare Pediatric Disease designation.
Expertise Areas
- AI-driven phenotyping and biomarker discovery
- Clinical trial management for pediatric neuropsychiatric indications
- Genomic and EHR data integration for translational research
- Regulatory strategy for rare and pediatric indications
Key Technologies
- AI-based deep phenotyping
- Genomic data analysis
- Electronic health record (EHR) integration
- Randomized double-blind placebo-controlled crossover trial design
News & Updates
Participation and presentations at conferences including BIO International Convention, 22q11.2 scientific meetings, NORD symposiums, and other industry events (dates and locations listed on site).
Topline Phase 2 results reported: primary endpoint showing NB-001 is safe and well-tolerated; efficacy measures supportive of progression to registrational trial; trial was randomized, placebo-controlled crossover in pediatric 22q11DS.
Late-breaking abstract accepted for presentation at the 52nd Child Neurology Society Annual Meeting describing the randomized Phase 2 NB-001 study.
Site lists a news item titled 'FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Nobias Therapeutics for the Treatment of 22q11.2 Deletion Syndrome' dated December 5, 2024.
Site lists a news item titled 'Nobias Therapeutics Secures Preliminary FDA Alignment on Registrational Endpoints for NB-001' dated June 16, 2025.
Site lists a news item titled 'Nobias Therapeutics Joins NVIDIA Inception Program for Startups' dated April 16, 2024.
Participation and presentations at conferences including BIO International Convention, 22q11.2 scientific meetings, NORD symposiums, and other industry events (dates and locations listed on site).
Topline Phase 2 results reported: primary endpoint showing NB-001 is safe and well-tolerated; efficacy measures supportive of progression to registrational trial; trial was randomized, placebo-controlled crossover in pediatric 22q11DS.
Late-breaking abstract accepted for presentation at the 52nd Child Neurology Society Annual Meeting describing the randomized Phase 2 NB-001 study.
Site lists a news item titled 'FDA Grants Rare Pediatric Disease and Orphan Drug Designations to Nobias Therapeutics for the Treatment of 22q11.2 Deletion Syndrome' dated December 5, 2024.
Site lists a news item titled 'Nobias Therapeutics Secures Preliminary FDA Alignment on Registrational Endpoints for NB-001' dated June 16, 2025.
Site lists a news item titled 'Nobias Therapeutics Joins NVIDIA Inception Program for Startups' dated April 16, 2024.