Our lead drug candidate, RNS60, is being developed to improve recovery from neurological trauma, such as ischemic stroke, and to treat chronic neurodegenerative diseases driven by mitochondrial dysfunction, such as amyotrophic lateral sclerosis (ALS).
RNS60 uniquely activates intracellular signaling pathways that have anti-inflammatory effects and promote mitochondrial biogenesis, cell survival, and differentiation. In addition to protecting neurons and oligodendrocytes in the central nervous system, RNS60 modulates the activity of immune cells to restore cellular homeostasis throughout the body – without the undesirable side effects common to so many of today’s medicines.
RNS60 has recently been investigated in two placebo-controlled Phase 2 clinical trials, one in ALS and the other in ischemic stroke, and has been granted FDA Orphan Drug and Fast Track designations for ALS. In preclinical studies, RNS60 has demonstrated significant disease-modifying effects and has been well-tolerated. RNS60’s excellent tolerability has been further demonstrated in Phase 1 clinical trials. RNS60 can be administered by inhalation (nebulization) or IV infusion.