Niagen Bioscience, Inc. (NASDAQ: NAGE), the global authority on NAD+ science, today 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 program 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 program, 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, Chief Executive Officer 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 characterized 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. (Riboldi et al., 2023; Tieve et al., 2015) and 1 in 150,000 people in Europe (Bhatt et al., 2015).
NB4168 is a distinct, proprietary molecule designed for oral pharmaceutical development. It is not commercially available as a supplement or approved drug 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+.
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 program 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 (Presterud et al., 2023; Veenhuis et al., 2021; Yang et al., 2021; Fang et al., 2016). 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 (Shoji et al., 2025; Okur et al., 2020; Okur et al., 2020; Fang et al., 2014). These studies were not conducted with NB4168 and were not registrational, but they support the rationale for advancing NB4168 in rare pediatric diseases.
“The NB4168 program is focused and stage-gated: a proprietary molecule, a rare pediatric disease with high unmet need, a mechanistic link to NAD+ biology, and measurable pharmacokinetic and pharmacodynamic endpoints,” said Andrew Shao, Ph.D., 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.”
Niagen Bioscience, Inc. is a publicly traded bioscience company focused on NAD+ science and healthy aging research. The Company’s product portfolio includes its flagship patented NR ingredient, Niagen®, Tru Niagen®, Niagen™ Plus and a pharmaceutical development effort focused on proprietary NAD+ precursors. Niagen Bioscience maintains a portfolio of over 50 patents protecting NR and other NAD+ precursors.
For additional information on the Pharmaceutical Program for Rare Disease, visit www.niagenbioscience.com/nadtheraputics.
About Niagen Bioscience
Niagen Bioscience, Inc. (NASDAQ: NAGE) is the global authority in healthy aging and NAD+ (nicotinamide adenine dinucleotide) science. As a trusted pioneer of NAD+ discoveries, Niagen Bioscience™ is dedicated to advancing healthspan through precision science and innovative NAD+-boosting solutions.
The Niagen Bioscience team, composed of world-renowned scientists, works with independent investigators from esteemed universities and research institutions around the globe to uncover the full potential of NAD+. A vital coenzyme found in every cell of the human body, NAD+ declines with age and exposure to everyday lifestyle stressors. NAD+ depletion is a key contributor to age-related changes in health and vitality.
Distinguished by state-of-the-art laboratories, rigorous scientific and quality protocols, and collaborations with leading research institutions worldwide, Niagen Bioscience sets the gold standard for research, quality, and innovation. There’s a better way to age.
At the heart of its clinically proven product portfolio is Niagen® (patented nicotinamide riboside, or NR), the most efficient, well-researched, and high-quality NAD+ booster available. Niagen powers the Company’s consumer supplement, Tru Niagen®, the number one NAD+ boosting oral supplement in the United States† (available at www.truniagen.com), and Niagen™ Plus, featuring pharmaceutical-grade intravenous (IV) and injectable Niagen products (www.niagenplus.com). Pharmaceutical-grade Niagen IV and injections are compounded and distributed by U.S. FDA-registered 503B outsourcing facilities and are available exclusively at clinics with a prescription. NAD Pharmaceuticals Corp., the Company’s wholly owned subsidiary focused on developing therapies for accelerated aging and rare genetic diseases, is conducting research on NB4168, a differentiated molecule.
Niagen Bioscience’s robust patent portfolio protects NR and other NAD+ precursors. Niagen Bioscience maintains a website at www.niagenbioscience.com, where copies of press releases, news, and financial information are regularly published.
†Based on revenue per largest U.S. e-commerce marketplace (Jan. 2025 – Dec. 2025)
Forward-Looking Statements
This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, and Section 21E of the Securities Exchange Act of 1934. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as “expects,” “anticipates,” “intends,” “estimates,” “plans,” “potential,” “possible,” “probable,” “believes,” “seeks,” “may,” “will,” “should,” “could,” “predicts,” “projects,” “continue,” “would” or the negative of such terms or other similar expressions.
Forward-looking statements are based on current expectations and assumptions and are subject to risks and uncertainties that could cause actual results to differ materially from those described. These risks and uncertainties include, but are not limited to, statements regarding Niagen Bioscience’s NB4168 pharmaceutical development program; planned preclinical, IND-enabling and clinical development activities; the potential timing of an IND submission or first-in-human study; the potential bioavailability, exposure, safety, tolerability, efficacy, pharmacodynamic or clinical profile of NB4168; and the Company’s ability to translate its NAD+ platform into pharmaceutical products; inflationary conditions and adverse economic conditions; our history of operating losses; the growth and profitability of our product sales; our ability to maintain and grow sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; risks of conducting business in China; including unanticipated developments in and risks related to the Company’s ability to secure adequate quantities of pharmaceutical-grade Niagen in a timely manner; the Company’s ability to obtain appropriate contracts and arrangements with U.S. FDA-registered 503B outsourcing facilities required to compound and distribute pharmaceutical-grade Niagen to clinics; the Company’s ability to remain on the U.S. FDA Bulk Drug Substances Nominated for Use in Compounding Under Section 503B of the Federal Food, Drug, and Cosmetic Act Category 1 list; the Company’s ability to maintain and enforce the Company’s existing intellectual property and obtain new patents; whether the potential benefits of NRC can be further supported; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA and other governmental authorities, including with respect to products seeking to compete in our market; mislabeling or other misleading marketing practices by competitors; economic and market instability, including as a result of tariffs or trade conflicts; and the risks and uncertainties associated with our business and financial condition in general, described in our filings with the Securities and Exchange Commission (SEC), including, without limitation, our most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q as filed with the SEC.
Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Niagen Bioscience undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.
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