Cyclerion Therapeutics to Discuss CY6463 MELAS Clinical Data at a Webinar Hosted by United Mitochondrial Disease Foundation
Company to discuss positive topline and additional clinical data for CY6463 in MELAS patients
Mitochondrial disease clinician-researcher,
Webcast scheduled for
“UMDF welcomes every opportunity to lift up the voices of MELAS patients and their loved ones, researchers and medical experts who know the most about this debilitating mitochondrial disease, which severely impacts multiple organs, including the CNS,” said
Mitochondrial disease clinician-researcher and investigator in the MELAS study,
“These highly encouraging data provide additional support for the therapeutic potential of CY6463 in MELAS,” said
For more information about the webinar, please visit the UMDF website. The live event can be accessed by visiting the investors' section of the Cyclerion website at https://ir.cyclerion.com/news-events/event-calendar. An archived replay will also be available on both the UMDF and Cyclerion websites.
CY6463 is the first CNS-penetrant sGC stimulator to be developed as a symptomatic and potentially disease-modifying therapy for serious CNS diseases. The nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) signaling pathway is a fundamental mechanism that precisely controls key aspects of physiology throughout the body. In the CNS, the NO-sGC-cGMP pathway regulates diverse and critical biological functions including neuronal function, neuroinflammation, cellular bioenergetics, and vascular dynamics. Although it has been successfully targeted with several drugs in the periphery, this mechanism has yet to be fully leveraged therapeutically in the CNS, where impaired NO-sGC-cGMP signaling is believed to play an important role in the pathogenesis of many neurodegenerative and neuropsychiatric diseases and other disorders associated with cognitive impairment. As an sGC stimulator, CY6463 acts as a positive allosteric modulator to sensitize the sGC enzyme to NO, increase the production of cGMP, and thereby amplify endogenous NO signaling. By compensating for deficient NO-sGC-cGMP signaling, CY6463 and other sGC stimulators may have broad therapeutic potential as a treatment to improve cognition and function in people with serious CNS diseases.
About the Study of CY6463 in MELAS
The Phase 2a study was an open-label, single-arm study of oral, once-daily CY6463 in eight adults with MELAS. The primary objective of the study was to assess the safety and tolerability of a 15 milligram, once-daily, oral dose of CY6463 over 29 days. The secondary objectives included pharmacokinetics and exploratory pharmacodynamic effects, with the goal of identifying which biomarkers to carry forward into additional studies.
Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) is one of the most complex orphan diseases affecting multiple organ systems, including the CNS, with different degrees of severity, and no approved therapies. MELAS is caused by some of the most common mitochondrial DNA mutations affecting the mitochondrial tRNA, and results in large clusters of familial cases of primary mitochondrial diseases (PMD). It is estimated that about 1 in 4,300 individuals has a mitochondrial disease, and ~80% of individuals with mitochondrial disease have CNS symptoms. The unmet need in MELAS is immense, symptoms can affect virtually any organ and cause intense fatigue, muscle weakness, and pain in addition to neurological manifestations. Life expectancy is estimated at ~17 years from onset of CNS symptoms. The disease impedes the individual’s ability to live independently, leads to social isolation, and overall reduced quality of life.
About Cyclerion Therapeutics
Cyclerion Therapeutics is a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function. Cyclerion’s lead molecule is CY6463, a novel, first-in-class, CNS-penetrant, sGC stimulator that modulates a key node in a fundamental CNS signaling network. The multidimensional pharmacology elicited by the stimulation of sGC has the potential to impact a broad range of CNS diseases. CY6463 has shown rapid improvement in biomarkers associated with cognitive function and is currently in clinical development for Alzheimer's Disease with Vascular pathology (ADv) and Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS) and Cognitive Impairment Associated with Schizophrenia (CIAS). Cyclerion is also advancing CY3018, a next generation sGC stimulator.
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. 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 2020 Form 10-K filed on February 25, 2021. Investors are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and Cyclerion undertakes no obligation to update these forward-looking statements, except as required by law.
Kendall Investor Relations
Source: Cyclerion Therapeutics, Inc.