Niagen Bioscience selects Evotec to advance NB4168 for rare disease
Niagen Bioscience partners with Evotec to conduct IND-enabling studies for NB4168, a novel therapy for Ataxia Telangiectasia. The deal utilizes Evotec's integrated platform to accelerate preclinical development, building on NB4168's superior bioavailability profile and existing orphan drug designations from the FDA and EMA.

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Niagen Bioscience, Inc. (NASDAQ: NAGE) has selected Evotec (NASDAQ: EVO) to lead preclinical development activities for NB4168, a proprietary oral small-molecule candidate targeting Ataxia Telangiectasia (A-T). The collaboration aims to generate the pharmacological evidence required to submit an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA), marking a critical step toward initiating human clinical trials for a rare pediatric disease with no approved therapies.
The agreement designates Evotec as a contract research organization to conduct pharmacokinetic and pharmacodynamic studies and validate bioanalytical methods for NB4168. Work will be executed in collaboration with NAD Pharmaceuticals Corp., Niagen Bioscience’s wholly owned subsidiary focused on therapies for accelerated aging and rare genetic diseases. The studies will take place at Evotec’s campus in Verona, Italy, utilizing the company’s INDiGO platform, which integrates active pharmaceutical ingredient development, drug product development, safety assessments, and regulatory services to streamline the path to clinical evaluation.
"Selecting Evotec adds streamlined drug development capabilities and executional scale to the NB4168 program," said Ozan Pamir, Chief Financial Officer of Niagen Bioscience. "This collaboration is an important step in building the pharmacological and bioanalytical evidence required to advance NB4168 toward clinical evaluation in patients with A-T."
NB4168 is designed to deliver significantly increased exposure to nicotinamide riboside (NR) compared to NR chloride, supporting its development as a more bioavailable pharmaceutical candidate. In nonclinical pharmacokinetic studies conducted to date, NB4168 demonstrated substantially higher blood exposure than NR chloride. Existing research on NR in A-T, including independent open-label clinical studies, has reported improvements in neurological measures and related biomarkers, providing a biological rationale for this approach.
Development Milestones and Regulatory Status
Niagen Bioscience has received both FDA Rare Pediatric Disease Designation and European Medicines Agency Orphan Medicinal Product Designation for NB4168 for the treatment of A-T. The company is currently advancing preclinical development activities with plans to submit an IND application to the FDA in anticipation of initiating human clinical studies.
| Regulatory Designation | Agency | Indication |
|---|---|---|
| Rare Pediatric Disease | U.S. Food and Drug Administration | Ataxia Telangiectasia |
| Orphan Medicinal Product | European Medicines Agency | Ataxia Telangiectasia |
Ataxia Telangiectasia is a rare genetic disease caused by mutations in the ATM gene, affecting roughly 1 in 40,000 people in the U.S. and 1 in 150,000 people in Europe. The condition is characterized by progressive loss of motor coordination, impaired immune function, and an elevated risk of cancer. There are currently no FDA-approved therapies for A-T, and treatment remains largely limited to supportive care.
Strategic Partnership Dynamics
Evotec brings deep drug development expertise, serving leading pharmaceutical and biotechnology companies across the life sciences industry. Dr. Ashiq Khan, Executive Vice President and Chief Commercial Officer of Evotec, stated that the collaboration demonstrates the value of Evotec’s integrated development offering in supporting innovative companies as they advance assets toward the clinic. He noted that NB4168 is a promising program supported by a defined biological rationale and a focused preclinical development plan.
The partnership allows Niagen Bioscience to leverage Evotec’s vertically integrated model, which is designed to accelerate timelines, reduce development risk, and improve cost efficiency. By consolidating ICH-directed activities required for early-phase clinical evaluation—including absorption, distribution, metabolism, excretion, and regulatory services—within a single platform, the companies aim to optimize the transition from preclinical data to first-in-human studies.
What is the projected timeline for Niagen Bioscience to submit the IND application to the FDA following the completion of Evotec's preclinical studies?
How might the success of NB4168 in treating Ataxia Telangiectasia influence investor sentiment toward other NAD-boosting therapies in the aging and rare disease sectors?
What are the potential regulatory or commercial risks associated with relying on a contract research organization like Evotec for critical IND-enabling activities?





























