Therapies for rare genetic diseases and aging-related disorders
As pioneers and leaders in NAD+ research, we are leveraging decades of science and clinical trials to target diseases with high unmet needs, initially focused on rare genetic diseases and aging-related disorders.
NB4168: a Differentiated Molecule
NB4168 is a distinct, proprietary molecule designed for oral pharmaceutical development and has robust coverage by Niagen Bioscience's patent portfolio, including a composition-of-matter patent.
After oral administration, NB4168 is designed to safely deliver significantly increased doses of NR to the bloodstream. NR enters cells directly where it is converted through the nicotinamide riboside kinase pathway into NAD+.
Published clinical evidence
Independent, peer-reviewed studies demonstrate significant clinical benefits of NAD+ therapeutics across multiple rare diseases.
Ataxia Telangiectasia: Two independent clinical studies showed significant improvement in motor function in patients with ataxia-telangiectasia (A-T) after nicotinamide riboside (NR) supplementation, alongside rising levels of nicotinamide adenine dinucleotide (NAD+) — the molecule our therapy is designed to restore.
Ataxia Telangiectasia
A rare pediatric disease caused by NAD+ depletion with no approved treatments
Ataxia Telangiectasia (A-T) affects 1 in 40,000 to 100,000 people worldwide, typically manifesting between ages 1-4 with progressive loss of motor function requiring wheelchair assistance by age 10. A-T is caused by mutations in the ATM gene, which normally coordinates DNA repair—without functioning ATM genes, cells become unstable and die, particularly in the cerebellum that coordinates movement.
The NAD+ Connection: A-T creates a devastating cycle where DNA damage leads to NAD+ depletion, causing mitochondrial dysfunction and neuroinflammation. An NAD+ booster such as NB4168 is designed to compensate for the lack of ATM genes to rescue these dying cells. This provides the therapeutic rationale for pursuing development of NB4168 as a treatment for A-T.
Compelling Opportunity: As a disease with no currently approved treatments, A-T provides an opportunity for an accelerated drug development pathway. Independent clinical trials have already demonstrated significant improvements with a composite scale known as the RmICARS score which measures a variety of motor functions. There are other clinical endpoints that have been accepted by regulators and new ones are being developed and validated. We expect that NB4168 will be eligible for priority review and orphan drug exclusivity.
Therapeutic potential
Independent research has observed significant clinical benefits using NAD+ precursors
DNA Repair:
NAD+ boosting may enhance DNA repair processes., protecting neurons from genomic instability and cellular damage
Antioxidant Activity:
NAD+ augmentation has been shown to reduce oxidative stress and protects dopaminergic neurons through improved cellular antioxidant capacity.
Neuroinflammation:
Clinical studies suggest that NR reduces pro-inflammatory cytokines, directly mitigating neuroinflammation in neurodegenerative conditions.
Protein Quality Control:
NAD+ enhancement may promote autophagy and clearance of misfolded proteins, addressing a key pathological mechanism in neurodegeneration.
Potential Future Indications
The examples provided are illustrative and do not represent a complete list of disease types within each category.