RNS60 is Revalesio’s lead clinical pharmaceutical candidate, which offers a combination of three key features:
- Mechanism of Action – In contrast to therapeutics based on biologics or small molecules, RNS60 was not designed to target a single cellular protein. Instead, RNS60 disrupts inflammatory signals and thereby prevents a cascade of biochemical events that cause cell and tissue damage. These effects of RNS60 have a positive impact on cellular bioenergetics and survival.
- Efficacy – RNS60 has demonstrated significant results in pre-clinical models of amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), Alzheimer’s disease, and Parkinson’s disease. Early phase clinical studies are exploring the disease modifying effects of RNS60 in patients with ALS and MS.
- Safety – RNS60 has demonstrated an excellent safety profile in pre-clinical toxicology studies and has been well tolerated in clinical safety studies both by healthy volunteers and patients.
RNS60 is an experimental drug and currently not available for use under an expanded access program.
Areas of Focus
Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS) is a progressive degenerative disorder of large motor neurons in the brain and spinal cord. The degeneration of these neurons results in muscle wasting, progressive paralysis, and death. In the United States, ALS affects approximately 20,000 patients; the cause in most cases is unknown. Life expectancy for half of ALS patients is only three years from onset of symptoms.
Research has increasingly pointed to neuro-inflammation as one of the major drivers of ALS progression. There are only two FDA-approved drug for ALS, Rilutek (riluzole) and Radicava (edaravone), which have been shown to be associated with only modest effects on survival and function. No available treatment prevents, halts, or reverses the disease. The development of a safe and effective therapy to lower the inflammation associated with ALS and to boost the survival of neurons and muscle cells is necessary to address this unmet medical need.
RNS60 has shown promising effects in a mouse model of ALS and was safe and well tolerated in a clinical open label pilot study at Massachusetts General Hospital (MGH). A larger Phase II, placebo-controlled study that aims to enroll 142 ALS patients at 19 centers in Italy and at MGH is underway. This study is funded in part by a grant from the ALS Association in partnership with ALS Finding a Cure and the Northeast ALS Consortium (NEALS).
Multiple sclerosis (MS) is a chronic immune-mediated disease of the central nervous system (CNS) affecting up to 400,000 people in the United States and over 2.1 million people worldwide. It leads to the destruction of myelin, a protective coating around the extensions of neurons. Without intact myelin, neuronal function is impaired, leading to clinical symptoms including vision loss, pain, fatigue, and paralysis.
Excessive inflammation has long been associated with MS. Most available MS drugs target inflammation but have been limited either by lack of efficacy or by severe side effects. New research has identified maintenance and repair of myelin as a target to develop more effective treatments for MS.
RNS60 effectively blocks the disease progression of MS in preclinical models. It reduces neuronal inflammation and supports maturation and survival of oligodendrocytes (OL), the cells responsible for maintaining myelin in the CNS. These effects have been linked to a favorable modulation of how OLs produce energy and survive. RNS60 is currently being evaluated in a pilot study in MS patients.