RNS60 is our lead clinical drug candidate to treat amyotrophic lateral sclerosis (ALS) and other neurological diseases. It can be administered by IV infusion or inhalation (nebulization). Based on preclinical efficacy and safety data, RNS60 has entered clinical evaluation1 in the US and in Europe. After demonstrating excellent safety and tolerability in Phase I safety studies as well as in pilot studies in people with ALS and multiple sclerosis (MS), RNS60 is currently being tested in a placebo-controlled, multicenter ALS Phase 2 study.

Unlike most therapeutics based on biologics or small molecules, RNS60 was not designed to target a single cellular protein. Instead, RNS60 acts on mitochondria and activates intracellular signaling pathways that have anti-inflammatory effects and promote cellular survival and differentiation.  Thereby, RNS60 protects neurons and oligodendrocytes, and helps the immune system restore homeostasis through modulation of immune cell activity in the central nervous system and throughout the body.

Amyotrophic Lateral Sclerosis

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 United States alone, the number of people with ALS (PALS) is estimated to be ~30,000. While about 10% of PALS have an inherited, familial form of ALS, the cause in most cases is unknown. Life expectancy for most PALS is three to five years from onset of symptoms.

There are very few FDA-approved drugs for ALS, and the development of safe and more effective therapies to treat ALS presents a clear unmet medical need.

Research has increasingly pointed to mitochondrial dysfunction and neuro-inflammation as major drivers of ALS progression. RNS60 has demonstrated to address both of these problems in a mouse model of ALS and other preclinical disease models: it protects motor neurons and their supporting cells (such as oligodendrocytes) by providing bioenergetic support, and it lowers inflammation.

RNS60 was safe and well tolerated in a clinical open label pilot study at Massachusetts General Hospital (MGH). A larger Phase 2, placebo-controlled study completed enrollment of 148 PALS at ~20 centers in Italy. This study was funded in part by a grant from the ALS Association in partnership with ALS Finding a Cure and the Northeast ALS Consortium (NEALS).

RNS60 has received FDA Orphan Drug status and Fast Track designation for ALS.

1 Revalesio does not currently sponsor expanded access for RNS60