Neurodon:
targeting cellular stress,
revolutionizing medicine

Pipeline

Targeted Disease
Cellular Discovery Lead Optimization IND-Enabling Clinical Trials

Diabetes Type 1 & 2

SERCA has been shown to be impaired in diabetic patients, leading to low ER calcium and thus impaired ability for the ER to produce insulin.

Neurodon’s first-in-class drug candidates target dysfunctional calcium homeostasis in pancreatic β-cells and have the potential to attenuate pancreatic β-cell death.

In preclinical studies, NRDN-101 and derivatives have been shown to:

  • Protect human pancreatic islet cells
  • Lower blood glucose
  • Increase insulin response
  • Lower HbA1c
  • Reduce fat and preserve muscle mass
  • Improve various metabolic parameters
  • Reduce plasma and liver cholesterol, trigylcerides, and fatty acids
  • Reverse hepatic steatosis
  • Reverse the diabetic phenotype in animal models for weeks after dosing is stopped

Alzheimer’s Disease

Neurodon’s compounds approach Alzheimer’s in a novel way. Instead of targeting amyloid plaques, a tactic that has not been successful to date in competitor therapies, we target cellular calcium homeostasis in hippocampal neurons, which is disrupted calcium is upstream from several pathological features, by addressing neuroinflammation, tau phosphorylation and amyloid aggregation.

In preclinical studies, NRDN-201 has shown to:
  • Improve learning, memory, and cognitive endpoints
  • Rescue cerebral function
  • Restore synaptic integrity
  • Re-establish mobility
  • Decrease neuroinflammation
  • Reduce amyloid plaque density

Parkinson’s Disease

Our novel approach to treating Parkinson’s lies in repairing cellular calcium homeostasis in dopaminergic neurons located in the substantia nigra pars compacta portion of the brain by accelerating SERCA’s activity for calcium transport.

In preclinical studies, NRDN-201 has shown to:

  • Prevent loss of dopaminergic neurons
  • Improve motor coordination
  • Reduce akinesia across several assessments

Duchenne Muscular Dystrophy

Neurodon’s compounds target calcium imbalance in muscle cells implicated in muscle weakening as well as cardiac and respiratory dysfunction in DMD patients by accelerating the activity of SERCA in skeletal muscle cells.

In preclinical studies, NRDN-301 has shown to:
  • Improve muscle contraction
  • Reduce necrosis
  • Increase SERCA activity in animal models of DMD
  • Reduce DMD-related muscle fibrosis
  • Reduce muscle cell degeneration

Rare Diseases

We have shown compelling efficacy in animal and cellular models of Huntington’s disease, ALS, Darier disease, and others. Therapies for these underrepresented rare and orphan diseases have potential for accelerated development to provide treatment options for patients.
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