Significant strides made in Nxera Pharma's oral compound for neurological conditions

By Liza Laws

- Last updated on GMT

© Getty Images
© Getty Images

Related tags Neurology Alzheimer's disease Parkinson's disease neurological

Neurocrine Biosciences Inc. has initiated its phase 1 clinical study, marking a significant stride in the evaluation of NBI-1117567, an investigational small molecule, oral compound developed by Nxera Pharma.

This first-in-human study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of NBI-1117567 in healthy adult participants.

The compound, a muscarinic M1 preferring (M1/M4) selective agonist, holds promise for addressing cognitive symptoms in patients with neurological and neuropsychiatric conditions.

Muscarinic receptors, pivotal in various bodily functions including brain activity, have long been targeted for treating cognitive and neuropsychological symptoms associated with diseases like Schizophrenia, Alzheimer’s, and Parkinson’s.

Previous attempts hindered by side effects

However, previous attempts at developing selective agonists have been hindered by side effects linked to the activation of other receptor subtypes. NBI-1117567, with its purported selectivity for M1 and M4 receptors, represents a breakthrough in this regard, potentially offering effective treatment options without the drawbacks of previous compounds.

The collaboration and licensing agreement between Nxera Pharma and Neurocrine Biosciences, established in November 2021, underpin this developmental leap. Neurocrine holds global development and commercialization rights to a range of muscarinic receptor agonists discovered by Nxera, while Nxera retains rights to develop M1 agonists in Japan. Under the terms of the agreement, Nxera stands to receive substantial R&D funding, development milestones, and royalties, contingent upon meeting specified criteria.

This development highlights the collaborative effort between pharmaceutical entities in advancing innovative therapies for addressing unmet medical needs in neurological and neuropsychiatric disorders. As the Phase 1 clinical study progresses, stakeholders anticipate further insights into the therapeutic potential of NBI-1117567 and its role in transforming treatment paradigms for cognitive symptoms associated with neurological conditions.

There's no single cause of mild cognitive impairment (MCI), although MCI may be due to early Alzheimer's disease. There's no single outcome for the disorder. Symptoms of MCI may remain stable for years. Or MCI may progress to Alzheimer's disease dementia or another type of dementia. In some cases, MCI may improve over time.

Mild cognitive impairment 

According to the Mayo Clinic MCI often involves the same types of brain changes seen in Alzheimer's disease or other forms of dementia. In MCI, those changes occur at a lesser degree. Some of these changes have been seen in autopsy studies of people with MCI.

These changes include clumps of beta-amyloid protein, called plaques, and tangles of tau proteins that are seen in Alzheimer's disease, microscopic clumps of a protein called Lewy bodies. These clumps are associated with Parkinson's disease, dementia with Lewy bodies, and some cases of Alzheimer's disease. Other changes can include small strokes or reduced blood flow through the brain's blood vessels.

Brain-imaging studies show that changes that may be associated with MCI include a decreased size of the hippocampus, increased size of the brain’s fluid filled spaces (ventricles) and a reduced use of glucose in key brain regions.

MCI risk factors 

The strongest risk factors for MCI are increasing age, having a form of a gene known as APOE e4. This gene also is linked to Alzheimer's disease. But having the gene doesn't guarantee that you'll have a decline in thinking and memory. Other medical conditions and lifestyle factors have been linked to an increased risk of changes in thinking, including diabetes, smoking, high blood pressure and cholesterol, obesity, depression, and obstructive sleep apnea.

Nxera Pharma (formerly Sosei Heptares) is a technology-powered biopharma company, in pursuit of new specialty medicines to improve the lives of patients with unmet needs in Japan and globally, while Neurocrine Biosciences is dedicated to providing breakthrough treatments for patients in need with rare and under-addressed diseases.

In addition to several products being commercialized in Japan, and the company is advancing an extensive pipeline of over 30 active programs from discovery through to late clinical stage internally and in partnership with leading pharma and biotech companies.

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