Niagen Bioscience, Inc. (NASDAQ: NAGE) has announced the launch of its first pharmaceutical drug candidate, NB4168, through its wholly owned subsidiary NAD Pharmaceuticals Corp., targeting accelerated aging and rare genetic diseases.

The initial indication for NB4168 is Ataxia Telangiectasia (A-T), a rare genetic disease caused by mutations in the ATM gene. A-T presents in early childhood and is characterized by progressive loss of motor coordination, impaired immune function, increased susceptibility to infections, pulmonary complications, and elevated cancer risk. The condition affects approximately 1 in 40,000 people in the U.S. and 1 in 150,000 in Europe. There are currently no FDA-approved therapies for A-T.

NB4168 is a proprietary oral molecule derived from nicotinamide riboside (NR), an NAD+ precursor. According to the company, nonclinical pharmacokinetic studies have shown NB4168 produces higher blood exposure to the active compound compared with NR chloride. The molecule is covered by a composition-of-matter patent and is not available as a supplement or approved drug.

The program is described by the company as building on two published open-label clinical studies of NR chloride in A-T patients that reported improvements in neurological measures. Those studies were not conducted with NB4168 and were not registrational.

"NB4168 is the next step in our strategy to translate Niagen Bioscience's NAD+ leadership into pharmaceutical development," said Rob Fried, Chief Executive Officer. "There are two independent, published clinical studies investigating the impact of NR on A-T, both of which have shown statistically significant results."

Andrew Shao, Ph.D., Senior Vice President of Global Scientific and Regulatory Affairs, stated the program's objective is to generate "pharmacological, toxicological, and eventually clinical evidence" to determine whether NB4168 can benefit patients.

Niagen Bioscience holds a portfolio of more than 50 patents covering NR and other NAD+ precursors. The company's existing consumer products include Tru Niagen and Niagen Plus.