Niagen Bioscience, Inc. · NASDAQ:NAGE
Niagen Bioscience — KCSA Strategic Communications Fireside Chat with Rob Fried (July 2026)
15 slides from Niagen Bioscience's conference presentation, Jul 14, 2026
Slide 1: Therapies for Rare Genetic Diseases & Aging-Related Disorders
Therapies for Rare Genetic Diseases & Aging-Related Disorders JULY 2026
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation and other written or oral statements made from time to time by representatives of our reliance on a single or limited number of third-party suppliers; risks of conducting business in Niagen Bioscience contain “forward-looking statements” within the meaning of
Slide 3: Pursuing Drug Development for the Treatment of Rare Genetic Diseases & Aging-Related Disorders
Pursuing Drug Development for the Treatment of Rare Genetic Diseases & Aging-Related Disorders • Global authority on nicotinamide adenine dinucleotide (NAD+) research for metabolic health with over 25 years of preclinical and clinical research backing its patent estate across NAD+ precursors • Scien
Slide 4: NB4168: A Differentiated Molecule for Therapeutic Development
NB4168: A Differentiated Molecule for Therapeutic Development Higher Therapeutic Index Hypothesis • Significantly improved bioavailability and superior PK profile compared to NR De - Risked Clinical Development Opportunity • Published “remarkable” preclinical and clinical results for NR in rare gene
Slide 5: Therapeutic Hypothesis for Improved Performance
Therapeutic Hypothesis for Improved Performance NB4168 reaches higher drug levels NB4168 Comparison of NR and NB4168 release Levels in Whole Rat Blood NR Active Moiety • NB4168 is not present in nature and not • NB4168 is believed to have improved bioavailability, potentially conferring available as
Slide 6: NB4168 Mechanism of Action
NB4168 Mechanism of Action Preclinical studies demonstrate that NB4168 is 10x more bioavailable compared to NR Only NR can enter cells directly. NR is then converted into NAD+ through an efficient two - step pathway. Figure 1. Chemical structures and biosynthetic pathway of NR, NMN, and NAD+. Two ma
Slide 7: NR Increases NAD+ Levels in the Brain
NR Increases NAD+ Levels in the Brain A B D NAD / ATP α C Figure 1. 31P - MRS analysis reveals NR - mediated increase in cerebral NAD (A - C) Exemplary data from one subject. The position of NAD + /NADH is indicated. (A) Spectroscopy voxel position in the occipital cortex. Spectra were acquired for
Slide 8: ...and in a Dose-Dependent Manner in the Blood
…and in a Dose-Dependent Manner in the Blood Whole blood levels of NAD+ over 56 days of placebo, 100, 300, or 1000mg of NR per day supplementation 1. Conze, et al. Nature scientific reports (2019) 8
Slide 9: Significant Role of NAD+ Depletion in Ataxia Telangiectasia
Significant Role of NAD+ Depletion in Ataxia Telangiectasia DNA NAD+ Mitochondrial A- T damage depletion dysfunction Immunodeficiency, ↑ PARP ↓ cellular & ↓ respiration Neurodegeneration, organelle NAD+ and Loss of motor ↑ NAD+ ↓ mitophagy function consumption What is it?: Rare, inherited pediatric
Slide 10: Two Independent Clinical Trials Demonstrate Promising Results in A-T
Two Independent Clinical Trials Demonstrate Promising Results in A-T Netherlands Open Label Trial 1 Norway Open Label Trial 2 Patients and Length: 24 A - T patients, Patients and Length: 13 A - T patients 4 months, followed by 2 - months w/o. over 2 years. Methods: measured A - T progression using M
Slide 11: FDA has Approved Prescription Drugs from OTC Supplement Ingredients: Niaspan® is a Precedent
FDA has Approved Prescription Drugs from OTC Supplement Ingredients: Niaspan® is a Precedent Niacin Rate of Admin. Effects Metabolism, TI/Safety* • Niacin undergoes extensive, saturable metabolism. • The metabolic profile of niacin is influenced by the rate of niacin administration. • Pathway 1 is r
Slide 12: Short Term Development Plans to Create Value for NB4168
Short Term Development Plans to Create Value for NB4168 Efficacy Studies Pharmacokinetic & Drug Metabolism Formulation Clinical & regulatory pharmacodynamic Studies Development strategy development studies c. Elegans models, Rat PK and tissue Hepatocyte comparison Development of a Consulting with Ke
Slide 13: Beyond A-T: A Platform, Not a Single Product
Beyond A-T: A Platform, Not a Single Product INITIAL FOCUS Neuro - DNA Repair Mitochondrial Healthy degenerative Disorders Disorders Aging Diseases • Ataxia telangiectasia • Friedrich’s ataxia • ALS • Sarcopenia • Cockayne syndrome • Leigh syndrome • Alzheimer’s disease • Age - related NAD+ decline
Slide 14: Rare Disease Biotechs Command Premium Valuations
Rare Disease Biotechs Command Premium Valuations MARKET LTM COMPANY TICKER ENT. VALUE LEAD RARE-DISEASE PROGRAM STATUS / NEAR-TERM CATALYST CAP REVENUE ($M) ($M) ($M) Relutrigine — SCN2A/SCN8A developmental epileptic Praxis Precision Med. PRAX 8,740 8,270 — NDA accepted; FDA priority review encephal
Slide 15: Rare Disease: A Track Record of Successful Exits
Rare Disease: A Track Record of Successful Exits Selected M&A Transactions / Rare Disease Therapeutics TRANSACTIONS Reviewed 5 AGGREGATE Deal Value $67.5B AVERAGE PREMIUM 79% MEDIAN Deal Size $7.3B DATE TARGET ACQUIRER SECTOR D E A L VA L U E LTM REV PREMIUM STRATEGIC RATIONALE Skyclarys for Friedre
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