The mitochondrion is the powerhouse of the cell, providing energy to every cell type in the body. Brain cells rank among the most energy-demanding cells in the body. The disruption of cellular bioenergetic activity has emerged as a common factor in multiple pathways leading to nerve cell death in diseases such as ALS, Parkinson's and other neurodegenerative disorders.
Ion channels, proteins that regulate the excitability of cells, represent an important class of drug targets. Drugs that modulate ion channels play an important role in treating a variety of neurological and cardiovascular conditions. Among ion channel targets, potassium channels play a key role in the pathophysiology of epilepsy and other unmet needs in diseases of the nervous system. Knopp has initiated an effort to synthesize novel potassium channel openers as candidates for further development in epilepsy, neuropathic pain, or both.
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.