Niagen Bioscience Partners with Evotec to Advance NB4168 Development for Rare Genetic Disease
Niagen Bioscience, Inc. (NASDAQ: NAGE), a biotechnology company recognized for its leadership in NAD+ (nicotinamide adenine dinucleotide) science and healthy aging research, has announced a strategic collaboration with Evotec (NASDAQ: EVO; Frankfurt Prime Standard: EVT), a global drug discovery and development company, to accelerate the preclinical advancement of its investigational therapeutic candidate, NB4168.
The collaboration represents an important milestone in Niagen Bioscience’s efforts to develop innovative therapies for rare genetic disorders and age-related diseases through its wholly owned subsidiary, NAD Pharmaceuticals Corp. The subsidiary was established to focus on translating advances in NAD+ biology into pharmaceutical therapies targeting accelerated aging conditions and rare inherited diseases.
Under the agreement, Evotec will serve as the contract research organization (CRO) for NB4168, providing specialized expertise in pharmacology, bioanalysis, and drug development. The partnership is expected to generate critical data needed to support the candidate’s transition from preclinical development into human clinical testing.
Advancing a Novel Therapy for Ataxia Telangiectasia
NB4168 is Niagen Bioscience’s proprietary oral small-molecule therapeutic candidate currently being developed as a potential treatment for Ataxia Telangiectasia (A-T), a rare, inherited pediatric disorder for which no approved disease-modifying therapies currently exist.
Ataxia Telangiectasia is caused by mutations in the ATM gene, resulting in progressive neurological degeneration, immune dysfunction, increased cancer susceptibility, and premature aging. Children living with A-T often experience worsening coordination, impaired motor function, respiratory complications, and a significantly reduced life expectancy.
Despite advances in supportive medical care, treatment options remain limited, highlighting a significant unmet medical need.
Niagen Bioscience believes that restoring cellular NAD+ metabolism may offer a promising therapeutic strategy for addressing some of the underlying biological dysfunction associated with A-T.
Focus on NAD+ Biology
NAD+ is an essential coenzyme involved in hundreds of cellular processes, including:
- Energy metabolism
- DNA repair
- Cellular stress responses
- Mitochondrial function
- Cellular signaling
- Healthy aging mechanisms
Declining NAD+ levels have been associated with aging, neurodegenerative diseases, metabolic disorders, and several rare genetic conditions.
For years, Niagen Bioscience has focused on understanding how NAD+ biology can be leveraged to improve human health. While the company’s commercial business has centered on nutritional approaches to supporting NAD+ levels, the establishment of NAD Pharmaceuticals represents a broader effort to develop pharmaceutical-grade therapies targeting serious diseases.
NB4168 represents one of the company’s most advanced pharmaceutical development programs.
Evotec to Conduct Key Preclinical Studies
As part of the collaboration, Evotec will perform a series of critical pharmacokinetic (PK) and pharmacodynamic (PD) studies designed to better characterize NB4168 before it enters clinical development.
The company will also validate bioanalytical methods that are necessary for regulatory submissions and future clinical studies.
These activities are expected to provide valuable information regarding:
- Drug absorption
- Distribution throughout the body
- Metabolism
- Elimination
- Biological activity
- Dose optimization
- Exposure-response relationships
The resulting data will support Niagen Bioscience’s broader Investigational New Drug (IND)-enabling program, an essential step before seeking regulatory authorization to begin human clinical trials.
Leveraging Evotec’s Integrated Drug Development Platform
The research will be conducted at Evotec’s advanced drug development campus located in Verona, Italy, using the company’s integrated INDiGO® platform.
INDiGO is designed to streamline early-stage drug development by bringing together multiple scientific and regulatory capabilities within a single development framework.
The platform includes expertise in:
- Active pharmaceutical ingredient (API) development
- Drug formulation
- Bioanalytical testing
- Safety assessment
- Pharmacokinetic evaluation
- Absorption, distribution, metabolism, and excretion (ADME) studies
- Regulatory support
- Clinical development planning
This integrated approach enables biotechnology companies to coordinate multiple development activities more efficiently while reducing operational complexity.
By consolidating these services, the platform is intended to shorten development timelines, reduce overall project risk, and improve cost efficiency during the transition from laboratory research into clinical testing.
Building a Stronger Preclinical Data Package
One of the primary goals of the collaboration is to strengthen the scientific foundation supporting NB4168.
Comprehensive pharmacological characterization is increasingly important as regulatory agencies require robust evidence demonstrating that investigational therapies possess favorable safety profiles, predictable pharmacokinetics, and biologically meaningful activity before entering human studies.
The planned research will help determine how NB4168 behaves within biological systems while generating standardized analytical methods that can be used throughout future clinical development.
These data will ultimately support regulatory submissions and guide dose selection for first-in-human clinical trials.
Encouraging Early Pharmacokinetic Findings
According to Niagen Bioscience, previous nonclinical pharmacokinetic studies have already generated encouraging results for NB4168.
The investigational compound demonstrated substantially higher blood exposure compared with nicotinamide riboside chloride (NR chloride), suggesting improved bioavailability.
Higher systemic exposure may allow the drug candidate to deliver more consistent therapeutic concentrations while potentially improving clinical effectiveness.
Improved bioavailability represents an important consideration for oral therapies intended for chronic administration, particularly in rare diseases requiring long-term treatment.
These findings have reinforced the company’s confidence in continuing the development of NB4168.
Prior Research Supports the Biological Rationale
The development of NB4168 is supported by a growing body of scientific evidence exploring the potential role of nicotinamide riboside and NAD+ restoration in Ataxia Telangiectasia.
Several independent studies have suggested that increasing NAD+ availability may positively influence disease-related biological pathways.
Previous investigations include:
- Two independent open-label clinical studies involving NR chloride
- Multiple preclinical studies evaluating NAD+ restoration
Published research has reported improvements in several neurological measures along with favorable changes in biomarkers associated with disease progression.
Although these studies involved NR chloride rather than NB4168, the findings provide important scientific rationale supporting continued investigation of NAD+-based therapeutic strategies in A-T.
By developing a pharmaceutical candidate with improved pharmacokinetic properties, Niagen Bioscience aims to build upon these earlier observations while potentially delivering greater therapeutic benefit.
Executive Perspective
Ozan Pamir, Chief Financial Officer of Niagen Bioscience, emphasized that selecting Evotec provides the NB4168 program with significant drug development expertise and operational capabilities.
He explained that the collaboration represents an important step toward generating the pharmacological and bioanalytical evidence needed to support clinical evaluation of NB4168 in patients living with Ataxia Telangiectasia.
According to Pamir, establishing a strong preclinical data package is critical for advancing the investigational therapy through regulatory development and ultimately toward human studies.
Evotec Highlights Integrated Development Expertise
Dr. Ashiq Khan, Executive Vice President and Chief Commercial Officer of Evotec, noted that the collaboration demonstrates the value of the company’s integrated drug development platform in supporting innovative biotechnology companies.
He described NB4168 as a promising investigational program supported by a clear biological rationale and a focused preclinical development strategy.
Khan stated that Evotec looks forward to applying its multidisciplinary expertise to generate the scientific data necessary for the next stage of development.
Expanding Niagen Bioscience’s Pharmaceutical Pipeline
The partnership with Evotec reflects Niagen Bioscience’s broader strategy of expanding beyond nutritional science into pharmaceutical development.
Through NAD Pharmaceuticals, the company is building a pipeline focused on diseases where impaired NAD+ metabolism contributes to disease progression.
These efforts target conditions characterized by:
- Accelerated aging
- DNA repair deficiencies
- Mitochondrial dysfunction
- Neurodegeneration
- Rare inherited disorders
The collaboration also demonstrates the company’s commitment to partnering with experienced development organizations capable of accelerating complex drug development programs.
Addressing an Important Unmet Need
Ataxia Telangiectasia remains one of the most challenging rare pediatric disorders due to its progressive neurological decline and lack of approved therapies.
Families affected by the disease currently rely primarily on supportive care, with few options available to slow disease progression.
If successful, NB4168 could represent a meaningful advancement by targeting underlying biological mechanisms associated with the disorder rather than simply managing symptoms.
While the therapy remains in preclinical development, the ongoing collaboration with Evotec is expected to generate the evidence required for regulatory submissions and eventual clinical evaluation.
The collaboration between Niagen Bioscience and Evotec marks another significant step toward advancing NB4168 into human clinical studies. By combining Niagen Bioscience’s expertise in NAD+ biology with Evotec’s integrated drug development capabilities, the companies aim to accelerate the generation of high-quality preclinical data needed to support regulatory approval for clinical testing.
As research continues, the partnership underscores the growing interest in developing NAD+-based therapies for rare genetic diseases and highlights the potential of innovative drug development collaborations to bring new treatment options closer to patients living with serious, underserved conditions.
About NB4168
NB4168 is a distinct, proprietary small molecule designed for oral pharmaceutical development. It is not commercially available as a supplement or approved drug and is protected by Niagen Bioscience’s patent portfolio, including a composition-of-matter patent. After oral administration, NB4168 is designed to deliver significantly increased exposure to NR, which enters cells directly and is converted through the nicotinamide riboside kinase (NRK) pathway into NAD+. NAD+ supports biological processes including DNA repair, mitochondrial function, cellular energy production and stress responses—pathways that are disrupted in A-T.
Niagen Bioscience has received U.S. Food and Drug Administration Rare Pediatric Disease Designation and European Medicines Agency Orphan Medicinal Product Designation for NB4168 for the treatment of A-T. The Company is advancing preclinical development activities and plans to submit an IND application to the FDA in anticipation of initiating human clinical studies.
About Ataxia Telangiectasia (A-T)
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; Teive et al., 2015) and 1 in 150,000 people in Europe (Bhatt et al., 2015).
For additional information on Niagen Bioscience’s pharmaceutical program and NB4168, visit www.niagenbioscience.com/nad-pharmaceuticals.
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.