Trial Begins for Multi-Neurological Disease Therapy

(commonfund.nih.gov)

25 Aug. 2023. A clinical trial is underway testing a new type of therapy its developer says addresses damage to nerve cells associated with a number of neurological disorders. Nura Bio Inc. in South San Francisco says the trial, the company’s first, is evaluating its lead product code-named NB-4746, but the study is not yet registered with ClinicalTrials.gov.

Nura Bio is a five year-old company discovering treatments for neurological conditions associated with degenerating axons, the long fiber tails in nerve cells or neurons that transmit electrical signals to other neurons. The Nura Bio process seeks to prevent and repair damage to axons from Wallerian degeneration, where a key protein needed for healthy neurons called nicotinamide mononucleotide adenylyltransferase 2 or NMNAT2 is missing, causing neurons to lose their signaling ability. Another protein, coded by the Sterile Alpha and TIR Motif Containing 1 or SARM1 gene, is triggered by nerve cell injury, believed to encourage further damage to axons, and also associated with Wallerian degeneration.

The company’s technology is based on research into Wallerian degeneration by its scientific founders Marc Freeman at Oregon Health and Science University and Steven McKnight at University of Texas Southwestern Medical Center. Freeman’s lab investigates damage to axons resulting in malfunctioning neurons and supporting glial cells, while the McKnight lab studies the chemistry of molecules associated with neurodegenerative diseases, focusing on compounds that limit nerve cell degeneration. As reported by Science & Enterprise in July 2020, Nura Bio was formed by Bay area venture investors, which staked the company to $73 million in its first venture round.

Effective in central and peripheral nervous systems

Nura Bio says NB-4746 acts as a SARM1 inhibitor to protect against damage to axons from Wallerian degeneration associated with several neurological conditions: amyotrophic lateral sclerosis or ALS, multiple sclerosis, traumatic brain injury, and nerve damage from chemotherapy. The company says its preclinical studies show NB-4746 can be given as an oral drug and still be effective in brain cells, as well as with neurons elsewhere in the central and peripheral nervous system, as well as the eyes.

The clinical trial, says Nura Bio, is a phase 1 or early-stage study with healthy volunteers, randomized to receive single and multiple ascending doses of NB-4746 or a placebo. Participants will be assessed for safety and tolerability of NB-4746, as well as chemical activity in the body. Numbers of participants in the trial and study sites were not disclosed.

“The initiation of the phase 1 trial of NB-4746 marks Nura Bio’s transition to a clinical-stage company,” says the company’s chief scientist Shilpa Sambashivan in a Nura Bio statement. She also notes the company is working on other other similar therapies. “We also continue advancing additional molecules through our pre-clinical pipeline,”  adds Sambashivan, “with the goal of delivering novel and potentially life-changing neuroprotective medicines.”

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