Mitochondrial Discovery Program
Next Generation Compounds
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.
Drugs with the potential to improve or restore mitochondrial function show promise as treatments for those diseases. Dexpramipexole is undergoing research and development as a potential modulator of mitochondrial bioenergetics, based on results in preclinical models and evidence of metabolic effects in a clinical trial. Scientists at Knopp are making progress in characterizing the drug's mechanism of action, and have developed screening assays with the goal of discovering next generation compounds and advancing the pharmacology of dexpramipexole.
Publications of interest include:
- KNS-760704 [(6R)-4,5,6,7-tetrahydro-N6-propyl-2, 6-benzothiazole-diamine dihydrochloride monohydrate] for the treatment of amyotrophic lateral sclerosis
- Effects of dexpramipexole on brain mitochondrial conductances and cellular bioenergetic efficiency
- Creatinine as a Biomarker of Disease Subtype, Disease Progression, and Drug Response
in Patients with ALS in the Phase 3 Empower Study
- Learning from EMPOWER: Creatinine and other metabolic markers in ALS (video presentation)
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.