Our lead drug candidate, RNS60, is being developed to treat chronic neurodegenerative diseases driven by mitochondrial dysfunction, such as amyotrophic lateral sclerosis (ALS), and to improve recovery from neurological trauma, such as ischemic stroke.
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 is currently being evaluated 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.
ALS is a progressive degenerative disorder of large motor neurons in the brain and spinal cord, leading to muscle wasting, progressive paralysis, and death. In the U.S. alone, the number of people with ALS is estimated to be as high as 30,000. While about 10% of those with ALS have an inherited, familial form of ALS, the cause in most cases is unknown. Life expectancy for most is three to five years from the onset of symptoms.
There are very few FDA-approved drugs for ALS, and the development of safe and more effective therapies to treat ALS represents a clear and urgent unmet medical need.
Research has increasingly pointed to mitochondrial dysfunction and neuro-inflammation as major drivers of ALS progression. RNS60 has demonstrated the ability to address both of these problems in preclinical disease models of ALS and in other preclinical disease models. RNS60 protects motor neurons and oligodendrocytes by providing bioenergetic support while lowering inflammation.
RNS60 was shown to be well tolerated in an open-label pilot study in ALS at Massachusetts General Hospital. A larger Phase 2, placebo-controlled study of 148 people with ALS at approximately 20 centers in Italy, funded in part by a grant from the ALS Association in partnership with ALS Finding a Cure and Northeast ALS Consortium, is near completion.
Stroke is a leading cause of mortality and neurological disability worldwide—with more than 795,000 people in the U.S. reported to have a stroke each year. The majority (87%) have an acute ischemic stroke, in which blood supply to a part of the brain is interrupted. While stroke deaths have declined in recent years, someone in the U.S. still dies of a stroke every 4 minutes. Stroke is also responsible for serious long-term disability— reducing mobility in more than half of stroke survivors older than 65 years and costing the U.S. over 46 billion dollars a year.1
To date, stroke treatment has focused on reestablishing blood flow to salvage as many neurons in the ischemic area as possible. Currently, there are only two FDA-approved treatments available: intra-arterial recombinant tissue plasminogen activator (tPA) and endovascular intervention. Yet, even when these treatments are combined, only about 10% of survivors return to normal and only approximately half regain functional independence. There is clearly a significant unmet need for more effective neuroprotective stroke therapies.
Data from preclinical studies strongly suggest that RNS60 protects brain cells after a stroke, preserving viable brain tissue and enhancing functional recovery. Based on these findings, we have initiated a Phase 2 clinical study investigating the potential benefits of RNS60 in the treatment of stroke.
We have several discovery programs ongoing in other neurological conditions with unmet need, including traumatic brain injury (TBI), Alzheimer’s disease and Parkinson’s disease.