FROM DISCOVERY
to drugs

Pipeline

pipeline

RNS60

Our lead product candidate, RNS60, is saline saturated with oxygen through a unique process. RNS60 is being developed as a potential treatment to improve recovery from neurological trauma, starting with ischemic stroke, and for chronic neurodegenerative diseases driven by mitochondrial dysfunction, such as amyotrophic lateral sclerosis (ALS). RNS60 has demonstrated promising efficacy and tolerability in two Phase 2 placebo-controlled clinical trials (in ischemic stroke and ALS) and also in multiple preclinical models.

RNS60 has the potential to address multiple neurological conditions

RNS60 can be administered by IV infusion or inhalation (nebulization) and uniquely activates intracellular signaling pathways, such as the Type 1A PI3K pathway, 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 appears to modulate the activity of immune cells to restore cellular homeostasis throughout the body—without the undesirable side effects commonly seen with so many of today’s medicines.

RNS60 preserves brain tissue

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the technology
behind RNS60

RNS60 FOR
ischemic stroke

The global impact of stroke is staggering. It’s the #1 cause of disability and the #2 cause of death worldwide, with an estimated global cost of $721 billion.1 Over 143 million years of healthy lives are lost each year to ischemic stroke-related death and disability.1 Caregivers are also affected, and can experience physical challenges in caring for their loved ones, as well as depression, anxiety, social isolation, and financial stress.2

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THE STROKE
BROCHURE (PDF)

There is a significant unmet need for more effective stroke therapies

The majority of people (87%) who have a stroke have an acute ischemic stroke, in which blood supply to a part of the brain is interrupted. Without oxygen, brain cells begin to die within minutes. Only ~20% of people having a stroke are eligible to receive currently available treatment options, including endovascular thrombectomy (clot removal) or thrombolytics (clot-busting drugs), leaving ~80% of people at risk for greater brain damage and long-term disability.3

In RESCUE, a Phase 2 clinical trial in stroke patients who were randomized within 24 hours after last known well and received endovascular thrombectomy, RNS60 demonstrated:4,*

  • 50% reduction (P<0.05) in loss of brain tissue (infarct growth post endovascular thrombectomy)
  • Clinically meaningful, numeric changes in multiple functional outcomes (modified Rankin Scale, Barthel Index, National Institute of Health Stroke Scale, EQ-5D-5L – EuroQoL Questionnaire)
  • Safety and tolerability

Revalesio is planning a global Phase 3 clinical trial (RESTORE), which could serve as the basis for approval, potentially impacting millions of lives worldwide.

LEARN MORE ABOUT
the results of
RESCUE

*Versus endovascular thrombectomy alone.

References: 1. World Stroke Organization (WSO): Global Stroke Fact Sheet 2022. 2. Omar O, et al. Cureus. 2021;13(9):e17948. doi: 10.7759/cures17948. 3. Anand SK, et al. J Neurosurg. 2021;51(1):E2. doi:10.3171/2021.4.FOCUS21117 4. Revalesio press release: https://revalesio.com/press-releases/revalesioannounces-positive- topline-data-from-phase-2-rescue-study-of-rns60-in-patients-with-acute-ischemic-stroke-in-late-breaking-oral-presentation-at-isc-2024/

RNS60
for ALS

Amyotrophic lateral sclerosis (ALS) is a progressive degenerative disorder that involves large motor neurons in the brain and spinal cord and can lead to muscle wasting, progressive paralysis, and death. In the US 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, 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 safer 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 problems in preclinical disease models of ALS and in other preclinical disease models. RNS60 is thought to protect 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. In a larger Phase 2, placebo-controlled study of 147 people with ALS at 22 centers in Italy, funded in part by a grant from the ALS Association in partnership with ALS Finding a Cure and Northeast ALS Consortium, RNS60 was also well tolerated and showed encouraging results on select outcomes of respiratory function. It has been granted FDA Orphan Drug and Fast Track designations for ALS.

LEARN MORE ABOUT
the ALS Phase 2
trial results

Discovery
programs

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.