Cyclerion Therapeutics Announces CY6463 Data Demonstrating Improved Cellular Energetics in Preclinical Models of Mitochondrial Disease
CY6463 alleviated mitochondrial dysfunction and reduced inflammation in preclinical models of mitochondrial complex 1 deficiency
Data provide support for ongoing CY6463 clinical studies in CNS diseases associated with mitochondrial dysfunction
“We’re excited to share results highlighting the beneficial impact of sGC stimulation in multiple preclinical models of mitochondrial disease,” said
Data will be presented by
- CY6463 significantly increased ATP levels in lymphoblasts derived from patients with mitochondrial complex 1 deficiency (Leber hereditary optic neuropathy or Leigh syndrome)
- CY6463 restored mitochondrial gene expression in lymphoblasts derived from patients with mitochondrial complex 1 deficiency (Leber hereditary optic neuropathy or Leigh syndrome)
- CY6463 significantly decreased the astrocytic marker, GFAP, linked to inflammation in a mouse model of mitochondrial complex 1 deficiency
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.
Cyclerion Therapeutics is a clinical-stage biopharmaceutical company on a mission to develop treatments that restore cognitive function. Cyclerion is advancing novel, first-in-class, CNS-penetrant, sGC stimulators that modulate a key node in a fundamental CNS signaling pathway. The multidimensional pharmacology elicited by the stimulation of sGC has the potential to impact a broad range of CNS diseases. The most advanced compound, CY6463, has shown rapid improvement in biomarkers associated with cognitive function and is currently in clinical development for Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes (MELAS), Cognitive Impairment Associated with Schizophrenia (CIAS) and Alzheimer's Disease with Vascular pathology (ADv). Cyclerion is also advancing CY3018, a next-generation sGC stimulator.
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Source: Cyclerion Therapeutics, Inc.