Curative Therapeutics

Science

Developing treatments for some of the most challenging diseases of the 21st Century

Our science is focused on specific cellular pathways and targets that are responsible for the loss of cellular homeostasis in many diseases.

Disease conditions lead to the disruption of normal cellular homeostasis and when these conditions are prolonged or excessive, devastating pathological consequences result. Among these are initiation of stress pathways that lead to cell dysfunction encompassing impaired signaling, respiration, and protein processing.

By leveraging our internal research and the expertise of our SAB, who are recognized leaders in fields relating to cellular homeostasis and human disease, we have gained an intimate knowledge of precise methods to correct aberrant cellular homeostasis. We have made significant progress towards bringing medicines to patients with difficult to treat diseases by leveraging our science through structure-based drug design, pragmatic application of DMPK and physicochemical information, and translational methodology.

Diabetes
Type 1 & 2

Addressing beta cell death

There is extensive clinical and basic research evidence that supports cellular stress pathways as major contributors to β-cell dysfunction and loss in both Type 1 and Type 2 Diabetes. In obese and diabetic patients, β-cells are excessively taxed due to glucose fluctuation and insulin demand which causes a disruption of cellular homeostasis. These fluctuations lead to abnormal cell function and death. Neurodon’s novel class of compounds have shown the potential to provide first-in-class drug candidates to address β-cell death and provide a new treatment option to patients.

Our patented compounds have been shown to protect human pancreatic islet cells in diabetes models, lower blood glucose and increase insulin response in several animal models, and improve various metabolic parameters in ob/ob mice – reversing the diabetic phenotype.

Targeting cellular homeostasis

Alzheimer’s disease (AD) is a complex, multi-component process with no available treatments to-date that slow or halt disease progression. Current therapeutic strategies targeting amyloid plaques only address one of the many potential pathological drivers of Alzheimer’s disease.

Neurodon’s compounds, by targeting the cellular homeostasis that is disrupted in AD, have demonstrated the ability to treat the multitude of disease conditions in addition to amyloid plaque deposition such as neuroinflammation that is particularly implicit in cognitive decline.

Alzheimer’s
Disease

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Parkinson’s
Disease

Blocking the initiation of neuronal death

Parkinson’s disease (PD) is a degenerative CNS disorder of the central nervous system marked by the dysfunction and eventual loss of dopaminergic neurons that regulate movement. Despite a rapidly evolving understanding of its pathophysiology, current therapies only focus on treating symptoms of PD (tremors, bradykinesia, rigidity). A common theme that NDs share is one of neuronal insult followed by neuron death, which contributes to the decline and, in many instances, eventual death of patients. In PD this is seen with the substantial loss of dopaminergic neurons in patient brains.

Neurodon’s work combining novel molecules with cellular and animal models of neurodegeneration has shown that maintaining cellular homeostasis blocks initiation of neuronal death pathways in PD. We have demonstrated that treatment with our novel molecules improve coordination in a mouse model of PD, while maintaining brain cell mass in the substantia nigra pars compacta, the brain area affected by PD.

Developing diverse candidates

Targeting aberrant cellular homeostasis and specific stress pathways has allowed us to develop candidates for many rare diseases where such pathways contribute to disease pathogenesis. For example, we have shown compelling efficacy in animal and cellular models of muscular dystrophy, Huntington’s disease, ALS, and others. Therapies for these underrepresented diseases have potential for accelerated development to provide options for patients.

Rare
Diseases

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