Background

2N Pharma builds on a decade of research and development by co-founder and CSO Dr John Nieland and collaborators. To translate the scientific discoveries into treatment options for patients, 2N Pharma has developed novel small molecule therapeutic candidates which we have named Mitometin.

COMMON PATHOGENIC MECHANISMS AND OUR MEDICAL HYPOTHESIS

The essence of our medical hypothesis is the strong correlation – and we believe, causality – between dysregulated fatty acid oxidation and the development of neurological disorders.

Strongly upregulated lipid metabolism and deficient glucose metabolism are important, shared pathogenic mechanisms that underlie processes such as increased production of radical oxygen species and oxidative stress, inflammation, myelin sheath damage and demyelination, accumulation of misfolded proteins, neuronal loss and loss of muscle strength (Figure 1).

Figure 1.

We believe these mechanisms are related to prolonged upregulation of fatty acid β-oxidation. By targeting dysregulated lipid metabolism, cellular energy production is returned to homeostasis and the cascade of downstream pathological mechanisms will subside/recede.

Targeting dysregulated lipid metabolism as an approach to treat ALS, PD, and FTD

We believe CPT1 is a potential target for reversing metabolism disturbances because of its central role in fatty acid metabolism. Pharmaceutical downregulation of CPT1 activity inhibits fatty acid oxidation (Figure 2). When pyruvate dehydrogenase is uninhibited, glucose oxidation is activated, which helps restore energy homeostasis.

Figure 2.