Niagen Bioscience selects Evotec to advance Ataxia Telangiectasia Drug Candidate NB4168

GERMANY—Niagen Bioscience has selected Evotec as its contract research organization (CRO) to support the preclinical development of NB4168, an oral small-molecule candidate being developed for the rare genetic disease ataxia telangiectasia (A-T).

The collaboration also involves NAD Pharmaceuticals, a wholly owned subsidiary of Niagen Bioscience.

The companies will work together to generate pharmacological and bioanalytical data needed to advance NB4168 toward clinical development.

Targeting a rare genetic disease

Niagen Bioscience and NAD Pharmaceuticals focus on healthy aging as well as potential therapies for accelerated aging and rare genetic diseases.

The company describes itself as a global authority on NAD+, or nicotinamide adenine dinucleotide, a molecule involved in several essential cellular processes.

Niagen initially developed NB4168 as a potential treatment for A-T, a rare pediatric genetic disorder for which no approved therapies currently exist.

The disease typically emerges during early childhood and progressively affects multiple body systems.

As A-T advances, patients can experience worsening neurological disability, including progressive loss of motor coordination.

The disorder can also impair immune function, increasing susceptibility to infections, while pulmonary complications can further affect patients’ health.

In addition, people with A-T face a substantially higher risk of developing cancer.

Evotec to conduct preclinical studies

Under the new collaboration, Evotec will conduct pharmacokinetic (PK) and pharmacodynamic (PD) studies at its research campus in Verona, Italy.

These studies will help researchers understand how NB4168 behaves in the body and assess its biological effects.

Evotec will also work to further validate bioanalytical methods for characterizing NB4168.

The resulting data will support additional preclinical work and activities required to prepare the candidate for an Investigational New Drug (IND) application.

The collaboration is intended to provide Niagen with additional development and execution capabilities as it progresses NB4168 through the preclinical stage.

“Selecting Evotec adds streamlined drug development capabilities and executional scale,” 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.”

Integrated development support

Evotec said the partnership will leverage its integrated drug development capabilities to support the NB4168 programme.

“Our collaboration demonstrates the value of Evotec’s integrated development offering,” said Ashiq Khan, PhD, executive vice president and chief commercial officer at Evotec.

“NB4168 is a promising programme, supported by a defined biological rationale and a focused preclinical development plan.”

Khan added that Evotec will support Niagen in generating the data required to guide the next stage of development for the investigational candidate.

The PK, PD and bioanalytical studies will therefore form part of the broader preclinical programme as Niagen works to establish the evidence required before evaluating NB4168 in human clinical studies.