Our Science

NeuroPn Therapeutics is developing a novel and working on improved therapies for the treatment of neurodegenerative diseases with focus on painful peripheral neuropathy and Parkinson's disease.

Painful Peripheral Neurotheraphy

NeuroPn is developing a novel, potential disease modifying approach to treat peripheral neuropathic pain.

Painful peripheral neuropathy [PPN] is caused by an injury or inflammation of the nervous system. PPN is associated with diseases such as diabetes or use of drugs such as chemotherapeutic agents or antiviral drugs. Peripheral neuropathies may involve injured sensory, motor, or autonomic nervous system nerves. In the central nervous system, injury, stroke, or disease in the brain or spinal cord can also generate a state of chronic neuropathic pain. Pain associated with acute neuronal damage can also transition to chronic pain.

Peripheral neuropathic pain affects over 16 million Americans and is causally linked to a number of diseases including diabetes, cancer, shingles, infections such as HIV, chronic back pain, stroke, and multiple sclerosis . The most commonly prescribed drug classes for neuropathic pain such as antidepressants, serotonin-norepinephrine reuptake inhibitors, anticonvulsants and opioids, have limited efficacy and dose-limiting adverse effects. No single agent provides optimal balance of safety, tolerability, and efficacy. As a result, treatment of peripheral neuropathy continues to be characterized by a high rate of polypharmacy and switching from one agent to another.

All currently approved treatments for PPN modulate neuronal targets and provide only symptomatic relief.

NeuroPn Approach - NeuroPn is pursuing an enzyme target that has the potential to be a disease modifying approach to treat peripheral neuropathic pain.

  • NeuroPn is pursuing small molecule inhibitors of an enzyme target to address unmet medical needs and develop disease modifying therapeutics for peripheral neuropathic pain. Inhibition of the target enzyme stimulates endogenous GABAergic and opioid pathways to provide effective pain relief without narcotic effects. Enzyme inhibition reduces endoplasmic reticulum (ER) and oxidative stress, neuronal inflammation and neurodegeneration.
  • NeuroPn has identified a series of novel, orally active, potent enzyme inhibitors that are effective in animal models of acute and chronic peripheral neuropathic pain.
  • In animal models of nerve injury, NeuroPn compounds prevent neuronal damage and promote nerve healing.
  • Unlike opioids and other centrally acting agents, NeuroPn treatment is not associated with narcotic and other CNS adverse effects.
  • In chronic toxicology studies NeuroPn compounds exhibited clean safety profile with high therapeutic window.

NeuroPn compounds present a novel approach to treat painful peripheral neuropathy with highly discriminatory profile (improved efficacy, clean CNS safety profile and neuronal protection) vs marketed drugs.

Parkinson's Disease

NeuroPn's enzyme inhibition approach shows marked improvement in locomotor activity and significant inhibition of neuronal degeneration; differentiating from current satandard of care (L-DOPA).

Parkinsons’s disease (PD) is a chronic and progressive degenerative movement disorder that affects over 1.5 million Americans. Currently marketed dopamine replacement therapy can alleviate motor symptoms in PD, but there are no therapies that target the underlying neurodegenerative processes. Despite intense research, molecular mechanisms causing neuronal loss are not fully understood which has hampered the development of new drugs and disease modifying therapies.

Neuroinflammation and mitochondrial dysfunction with associated oxidative/endoplasmic reticulum (ER) stress all work synergistically to accelerate Parkinson’s disease (PD) progression.

NeuroPn’s target enzyme has been extensively validated in PD studies

  • The target is widely expressed in brain and it's levels are elevated in PD models and patients.
  • In PD and DLB patints enzyme levels correlate with synuclein deposition.
  • In neuronal cells, target inhibition prevents endoplasmic reticulum (ER) and oxidative stress and neuroinflammation a key contributor to loss of dopaminergic neurons.
  • Target modulation enhances axonal growth in primary sensory and cortical neuronal cell cultures.

NeuroPn enzyme inhibitors in multiple models of PD

  • Improved locomotor activity without hyperactivity typically seen with L-DOPA.
  • Prevented neuronal cell damage differentiating from L-DOPA.
  • In therapeutic models of PD with established disease NeuroPn compounds revered PD symptoms.
  • In chronic toxicology studies, NeuroPn compound exhibited a clean safety profile .

The preclinical studies conducted establish NeuroPn approach as a disease modifying approach with clear differentiation from L-DOPA.