Nobias Therapeutics

What Nobias Therapeutics does

Nobias Therapeutics is a clinical-stage biopharmaceutical company founded in 2020 and headquartered at 3675 Market Street, Philadelphia, PA. The company develops novel small-molecule therapeutics for rare diseases, with an initial focus on pediatric indications and stated expansion opportunities into adult and non-rare indications. Its pipeline is anchored by a single lead asset, NB-001 (fasoracetam), a first-in-class, non-stimulant activator of multiple metabotropic glutamate receptors (mGluRs) being advanced for the neuropsychiatric symptoms (ADHD, anxiety, autism) associated with 22q11.2 deletion syndrome (22q11DS) in children and adolescents. The company completed a multi-center, randomized, double-blind, placebo-controlled Phase 2 trial of NB-001 in 37 patients ages 6-17 across pediatric academic centers in the U.S. and Canada, reporting a 40-70% higher responder rate versus placebo and safety/tolerability that supported advancement toward registrational development.

The company's drug discovery approach leverages an AI-based deep phenotyping platform that analyzes clinical and genetic datasets to identify disease drivers and optimal therapeutic intervention points. This platform is supported by co-founder Dr. Hakon Hakonarson's directorship of the Center for Applied Genomics at Children's Hospital of Philadelphia (CHOP), which houses one of the world's largest pediatric biorepositories with 600,000 samples. Nobias joined the NVIDIA Inception Program for Startups in April 2024 to bolster AI capabilities.

Nobias is pre-revenue with no commercial products. Revenue is expected to derive from eventual commercialization of NB-001 and follow-on programs, supplemented potentially by licensing arrangements. The company has received $3.75M in disclosed funding from Domain Associates (July 2020). Operating geographies are North America (U.S. and Canada). The company's go-to-market approach for clinical development relies on partnerships with pediatric academic medical centers; commercial distribution upon approval would follow standard pharmaceutical channels. NB-001 has received FDA Rare Pediatric Disease and Orphan Drug designations (December 2024) and preliminary FDA alignment on registrational endpoints (June 2025).

Name

Nobias Therapeutics

Legal name

Nobias Therapeutics, Inc.

Website

https://nobiastx.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

1–10 employees

Short description

Nobias Therapeutics is a clinical-stage biopharmaceutical company developing NB-001 (fasoracetam), a first-in-class non-stimulant mGluR activator for neuropsychiatric symptoms in children with 22q11.2 deletion syndrome, using an AI-based deep phenotyping platform.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Medicinal Chemicals & Botanical Products (2833)

akta.pro primary industry

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN)

akta.pro secondary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Rare disease therapeutics
• Pediatric drug development
• Small molecule pharmaceuticals
• AI drug discovery
• Clinical-stage biotechnology

Headquarters

HQ city

Philadelphia

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Drug Development and Commercialization: Pre-revenue clinical-stage biotech company. Revenue will be generated through commercialization of novel small-molecule therapeutics for rare diseases upon regulatory approval. Initial focus on pediatric rare diseases with expansion opportunities in adults and non-rare indications.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Nobias Therapeutics is a clinical-stage biopharmaceutical company developing novel small-molecule therapeutics for rare pediatric diseases. Its lead asset NB-001 (fasoracetam) is a non-stimulant activator of multiple metabotropic glutamate receptors targeting the neuropsychiatric symptoms (anxiety, ADHD, autism) of 22q11.2 deletion syndrome in children. Drug discovery is supported by a proprietary AI-based deep phenotyping platform applied to large genomic and clinical datasets.

Product overview

Nobias Therapeutics is a clinical-stage biopharmaceutical company developing a single therapeutic pipeline anchored by its lead product NB-001 (fasoracetam), a small-molecule mGluR activator for 22q11.2 deletion syndrome. The company's drug discovery approach leverages AI-based deep phenotyping applied to genomic and health datasets to identify disease drivers and optimal intervention points for rare pediatric diseases.

Differentiator

Problem solved

Functional benefit

Brands

• NB-001: Lead program - a small-molecule mGluR activator (fasoracetam) for the potential treatment of neuropsychiatric symptoms associated with 22q11.2 deletion syndrome

Products and services

• NB-001 (fasoracetam) NB-001 is a first-in-class, non-stimulant small-molecule activator of multiple metabotropic glutamate receptors (mGluRs) being developed for the treatment of neuropsychiatric symptoms (anxiety, ADHD, autism) associated with 22q11.2 deletion syndrome (22q11DS) in pediatric patients aged 6-17.

Quantifiable outcome

• Responder rate was 40-70% higher in patients treated with NB-001 versus placebo
• +2 more outcomes

Named customers1 record

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Three partnerships are on record, tiered core.

• NVIDIAcoreTechnology or Integration · 16 April 2024Nobias Therapeutics joined the NVIDIA Inception Program for Startups, which supports cutting-edge AI and machine learning companies. This partnership provides access to NVIDIA's technology stack, expertise, and resources for advancing AI-based drug discovery capabilities.
• Pediatric Academic Medical CenterscoreStrategic or Co-development PartnerNobias has established strong relationships with pediatric academic medical centers across North America for conducting clinical trials. The company partnered with medical centers across the U.S. and Canada for its multi-center, randomized, double-blind, placebo-controlled Phase 2 trial of NB-001.
• Children's Hospital of Philadelphia (CHOP)coreStrategic or Co-development PartnerCo-founder Dr. Hakonarson serves as director of the Center for Applied Genomics (CAG) at CHOP, which hosts one of the world's largest pediatric biorepositories with 600,000 samples including more than 130,000 CHOP patients and family members. This provides unique genomic data access for drug discovery.

Scale indicators3 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Marinus Pharmaceuticals: Clinical-stage rare disease biotech developing therapeutics for rare neurological and neuropsychiatric disorders, with a similar model of small-molecule development for underserved pediatric rare populations.
• Zevra Therapeutics: Rare disease-focused company commercializing and developing therapies for rare neurological and metabolic conditions, comparable in targeting small-molecule drugs for underserved rare disease populations.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with multiple programs targeting rare genetic conditions, including pediatric rare diseases; directly comparable in regulatory strategy (orphan/pediatric designations) and rare disease commercial model.
• Travere Therapeutics: Rare disease biotech focused on small-molecule and biologic therapies for rare diseases, including pediatric rare disorders; comparable in pursuing FDA orphan/rare pediatric pathways and indication expansion strategy.
• Larimar Therapeutics: Clinical-stage rare disease biotech developing a single lead asset for a rare genetic disorder (Friedreich's ataxia), with comparable concentrated pipeline and orphan/rare pediatric regulatory strategy.
• Harmony Biosciences: Rare neurological disease company commercializing therapies for rare pediatric and adult CNS disorders; comparable in targeting underserved rare neuropsychiatric populations with novel mechanisms of action.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biotech with a broad rare disease pipeline including small-molecule and biologic therapies for rare pediatric disorders; comparable business model and regulatory orientation, though at significantly larger scale.

Emerging players

• Taysha Gene Therapies: Clinical-stage rare disease biotech focused on CNS and rare pediatric genetic disorders; comparable as an early-stage company targeting rare neurological/pediatric indications with limited assets and concentrated pipeline risk.
• Cerevel Therapeutics: Clinical-stage neuroscience company developing therapies for CNS disorders, including rare and genetically defined populations; comparable in pursuing novel receptor-targeted mechanisms for neuropsychiatric symptoms.
• Recursion Pharmaceuticals: AI-driven drug discovery company leveraging proprietary computational platforms to identify novel therapeutics, including for rare diseases; comparable to Nobias in combining AI-based discovery with rare disease therapeutic development.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/nobias-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds1 record

Investors2 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Nobias Therapeutics