Cyclerion Therapeutics Presentation at Biomarkers in Alzheimer’s Disease Summit
The presentation detailed the application of translational biomarkers to guide clinical activities for IW-6463, the Company’s therapeutic candidate for CNS diseases. In preclinical studies, IW-6463 has demonstrated beneficial effects across four domains relevant to neurodegenerative diseases: cerebral blood flow, cellular bioenergetics, neuro-inflammation, and neuronal function. Cyclerion plans to develop IW-6463 in individuals with Alzheimer’s disease with vascular pathology (ADv), as well as in MELAS (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes), a genetically defined orphan disease. The Company is evaluating cerebrospinal fluid and plasma biomarkers and various imaging approaches to define optimal clinical endpoints.
“Accumulating preclinical and clinical data has deepened our understanding of the pathophysiological contributors to CNS diseases and of biomarkers that may be used to track the impact of therapeutic intervention. Our intent is to apply this to our development approach so that we may efficiently assess potential therapeutic benefits and determine how to optimally approach future clinical studies,” said
“Alzheimer’s Disease with vascular pathology represents a well-defined patient population with high unmet need. In preclinical studies, our lead CNS drug candidate, IW-6463, has shown beneficial effects across several distinct domains relevant to neurodegenerative diseases and we believe this molecule holds promise to improve the lives of patients with Alzheimer’s disease and other serious CNS conditions. We look forward to obtaining IW-6463 clinical pharmacology trial results later this summer and continuing to advance the program forward in clinical studies,” said Dr.
Cyclerion Therapeutics is a clinical-stage biopharmaceutical company harnessing the power of sGC pharmacology to discover, develop and commercialize breakthrough treatments for serious and orphan diseases. Lead programs include olinciguat in Phase 2 development for sickle cell disease and IW-6463 in early development for serious CNS disease. Additional programs include praliciguat, which the Company intends to out-license for further development and several late-stage research projects.
IW-6463, a CNS-penetrant sGC stimulator, is being developed as a symptomatic and potentially disease modifying therapy for neurodegenerative diseases. In preclinical studies, IW-6463 has been associated with increased cerebral blood flow, reduced markers of neuroinflammation, enhanced cognition, neuroprotection and enhanced cellular bioenergetics. Nitric oxide is one of several fundamental neurotransmitters, but it has yet to be leveraged for its full CNS therapeutic potential. sGC stimulators work synergistically with the nitric oxide naturally produced in the body to boost the positive effects of nitric oxide, even when the body is not producing enough. Extensive human genetic and preclinical data implicate nitric oxide signaling defects in neurodegenerative diseases. Evidence indicates that nitric oxide dysregulation leads to vascular contributions to neurodegenerative disease (e.g. endothelial cell damage, decreased blood flow and impaired vascular reactivity) and may also directly increase inflammation, neuronal dysfunction/loss and cognitive impairment. sGC is expressed widely throughout the CNS and CNS vasculature.
Forward Looking Statement
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Our forward-looking statements are based on current beliefs and expectations of our management team that involve risks, potential changes in circumstances, assumptions, and uncertainties, including statements about the progression of our clinical development programs and the business and operations of Cyclerion. We may, in some cases use terms such as “predicts,” “believes,” “potential,” “continue,” “anticipates,” “estimates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “likely,” “will,” “should” or other words that convey uncertainty of the future events or outcomes to identify these forward-looking statements. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks listed under the heading “Risk Factors” and elsewhere in our 2019 Form 10-K filed on
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Source: Cyclerion Therapeutics, Inc.