15 September 2020 - - US-based biotechnology company CuraSen Therapeutics, Inc has dosed its first subjects in a multi-part, Phase 1 clinical study with CST-2032, the company's first proprietary compound intended for the treatment of neurodegenerative disease, the company said.

CST-2032 is a brain-permeant, selective adrenoceptor modulator designed to target key receptors in cortical and limbic regions of the brain that are no longer optimally innervated in patients with neurodegenerative disease.

CST-2032 is being developed to treat the debilitating symptoms of neurodegeneration in patients battling Parkinson's, Alzheimer's and related neurodegenerative diseases, and slow or reverse underlying pathology.

The company's initial studies in healthy volunteers and then patients will measure potential for drug impact on core disease symptoms, including dysfunction in cognition, arousal, memory and mood, as well as brain inflammation and metabolic health.

Neurons in the locus coeruleus, located in the brain stem and the sole source of noradrenaline (or norepinephrine) for the higher brain centers, decline very early in neurodegenerative diseases.

By activating specific receptors in the brain, CST-2032 is expected to restore healthy function to multiple cell types, including neurons, microglia, pericytes and astrocytes.

This heterocellular approach is expected to broadly improve cerebral integrity and function, and represents a unique treatment paradigm in the field.

CuraSen's early clinical development work included three clinical trials using established drugs in more than 100 healthy subjects and patients.

The goal was to guide optimization of the company's proprietary compounds, identify meaningful clinical biomarkers and endpoints, and establish exposure and dose levels for proof-of-concept efficacy studies.

The company explored multiple pharmacodynamic measurements for rapid and objective assessment of drug effect on brain function in these trials.

These measures included chemical biomarkers, neuroimaging, neurophysiology and autonomic function assessments of drug-target engagement, as well as more standard tests of arousal, mood and cognitive performance.

The Phase 1 trial will include up to 70 healthy volunteers and patients with a variety of neurodegenerative diseases, including Parkinson's Disease or Mild Cognitive Impairment.

The study will consist of single-ascending and multiple-ascending dose components in healthy volunteers using biomarker assessments, including neuroimaging and autonomic function tests.

A third component will evaluate these measures in patients after receiving CST-2032 treatment. The study is taking place in New Zealand and Belgium.

A Phase 2 study is anticipated to begin in 2021.

CuraSen is focused on the development of new treatments for a variety of neurodegenerative diseases, including Parkinson's Disease, Alzheimer's Disease and other related orphan conditions.