Niagen Bioscience, Inc. has announced the formal launch of the first drug candidate of NAD Pharmaceuticals Corp., its wholly owned subsidiary focused on developing therapies for accelerated aging and rare genetic diseases.
The programme will primarily focus on how NAD+, a coenzyme central to energy metabolism, DNA repair, mitochondrial function and cellular stress responses, modulates in rare diseases for which DNA misrepair and mitochondrial dysfunction are foundational underlying causes. This represents a strategic expansion of Niagen Bioscience’s NAD+ platform from cellular-health innovation into regulated drug development. A derivative of the NAD+ precursor, nicotinamide riboside (NR), NB4168 is the first publicly announced investigational pharmaceutical product candidate within the programme, with ataxia telangiectasia (A-T) as the initial indication.
“NB4168 is the next step in our strategy to translate Niagen Bioscience’s NAD+ leadership into pharmaceutical development,” said Rob Fried, CEO of Niagen Bioscience.
“There are two independent, published clinical studies investigating the impact of NR on A-T, both of which have shown statistically significant results, in addition to several non-clinical studies. We believe this body of work significantly de-risks the development pathway for NB4168, which we specifically developed for therapeutic applications.”
A-T is a rare genetic disease caused by mutations in the ATM gene. The disease typically presents in early childhood and is characterised by progressive loss of motor coordination, impaired immune function, increased susceptibility to infections, pulmonary complications, and a substantially elevated risk of cancer.
Children living with A-T often experience worsening neurological disability over time, with many requiring wheelchair assistance as the disease progresses. There are currently no FDA-approved therapies for A-T, and treatment is largely limited to supportive care. A-T impacts roughly 1 in 40,000 people in the U.S. and 1 in 150,000 people in Europe.
NB4168 is a distinct, proprietary molecule designed for oral pharmaceutical development. It is not commercially available as a supplement or approved drug and has 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+.
In nonclinical pharmacokinetic studies conducted to date, NB4168 has demonstrated substantially higher blood exposure to the active moiety compared with NR chloride, supporting its continued development as a more bioavailable pharmaceutical candidate. The programme builds on published NR research in A-T, where two independent open-label clinical studies and several preclinical studies of NR chloride reported improvements in neurological measures and related biomarkers. NR and NAD+ augmentation have also been studied in DNA-repair and accelerated-aging disorders including Werner syndrome, Cockayne syndrome and xeroderma pigmentosum group A. These studies were not conducted with NB4168 and were not registrational, but they support the rationale for advancing NB4168 in rare paediatric diseases.
“The NB4168 program is focused and stage-gated: a proprietary molecule, a rare paediatric disease with high unmet need, a mechanistic link to NAD+ biology, and measurable pharmacokinetic and pharmacodynamic endpoints,” said Andrew Shao, senior vice president, global scientific & regulatory affairs.
“Our objective is to generate the pharmacological, toxicological, and eventually clinical evidence needed to determine whether NB4168 can provide meaningful benefit to patients.”