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Cyclerion Therapeutics Announces Positive Topline Clinical Data for CY6463 in MELAS Patients at UMDF Mitochondrial Medicine 2022 Symposium
Data from an eight-patient, open-label study demonstrate improvements across multiple biomarkers of mitochondrial function, inflammation, cerebral blood flow,
About this update from Cyclerion Therapeutics, Inc.
[{"type":"text","content":"Data from an eight-patient, open-label study demonstrate improvements across multiple biomarkers of mitochondrial function, inflammation, cerebral blood flow, and functional connectivity CY6463 was well tolerated, with no reports of serious adverse events (SAEs) or treatment discontinuation due to adverse events (AEs); oral, once-daily administration provided expected CNS exposure Data support further development of CY6463 in CNS diseases with mitochondrial dysfunction CAMBRIDGE, Mass., June 10, 2022 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function, today announced positive topline data in its signal-seeking clinical study of CY6463, for the potential treatment of Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS). Chad Glasser, Pharm.D., Director of Clinical Research at Cyclerion Therapeutics, will present results from this clinical study today during the Clinical Trial Updates Panel at the United Mitochondrial Disease Foundation (UMDF) Mitochondrial Medicine 2022 Symposium, taking place June 8-11, 2022, in Phoenix, Arizona. CY6463 is a positive allosteric modulator of soluble guanylate cyclase (sGC), which amplifies endogenous NO signaling, a pathway that has been linked to mitochondrial biogenesis and function. In this open-label, single-arm study of the oral, once-daily sGC stimulator in eight MELAS patients, improvements were seen across a range of biomarkers, including mitochondrial disease-associated biomarkers such as lactate and GDF-15, a broad panel of inflammatory biomarkers, cerebral blood flow, and functional connectivity between neural networks. These positive effects after 29 days of dosing were supported by correlations across several endpoints and were more pronounced in patients with greater baseline disease burden. A return toward baseline levels after discontinuation of CY6463 dosing across several biomarkers was also observed. CY6463 was well tolerated with no adverse events leading to treatment discontinuation, and pharmacokinetics (PK) were consistent with the Phase 1 study in healthy volunteers. The positive data from this study further support the potential of CY6463, the first and only CNS-penetrant sGC stimulator in clinical development, to provide...