- Collaboration will support pharmacokinetic, pharmacodynamic, and bioanalytical studies for Niagen Bioscience’s lead investigational candidate for rare pediatric diseases
- Evotec brings deep drug development expertise, serving leading pharmaceutical and biotechnology companies across the life sciences industry
LOS ANGELES--(BUSINESS WIRE)-- Niagen Bioscience, Inc. (NASDAQ: NAGE), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, today announced that it has selected Evotec (NASDAQ: EVO; Frankfurt Prime Standard: EVT), a global life science company specializing in drug discovery and development, as a contract research organization to support the advancement of NB4168. The work will be conducted in collaboration with NAD Pharmaceuticals Corp., Niagen Bioscience’s wholly owned subsidiary focused on developing therapies for accelerated aging and rare genetic diseases.
Under the collaboration, Evotec will conduct pharmacokinetic and pharmacodynamic studies and validate bioanalytical methods to further characterize NB4168 and support Niagen Bioscience’s preclinical and Investigational New Drug (IND)-enabling development activities. NB4168 is Niagen Bioscience’s proprietary oral small-molecule candidate initially developed for Ataxia Telangiectasia (A-T), a rare pediatric genetic disease with no approved treatments.
The studies will take place at Evotec’s state-of-the-art campus in Verona, Italy, which provides a streamlined path to the clinic through Evotec’s INDiGO platform. It integrates the ICH-directed activities required for early-phase clinical evaluation—including active pharmaceutical ingredient development, drug product development, safety, absorption, distribution, metabolism, and excretion, and regulatory and clinical services. This vertically integrated model is designed to accelerate timelines, reduce development risk and improve cost efficiency.
“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.”
In nonclinical pharmacokinetic studies conducted to date, NB4168 has demonstrated substantially higher blood exposure compared with NR chloride, supporting its continued development as a more bioavailable pharmaceutical candidate. Existing NR research in A-T, including 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).
“Our collaboration with Niagen Bioscience demonstrates the value of Evotec’s integrated development offering in supporting innovative companies as they advance assets toward the clinic,” said Dr. Ashiq Khan, EVP, Chief Commercial Officer of Evotec. “NB4168 is a promising program, supported by a defined biological rationale and a focused preclinical development plan. We look forward to helping generate the data needed to inform the next stage of development for this investigational candidate.”
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.
For further information, please follow this link to the full version of the press release from Niagen Bioscience.
