Cyclerion (CYCN) reports positive topline data for CY6463 in MELAS study; improvements in biomarkers

Cyclerion Therapeutics, Inc.

Open-label, eight-patient study showed improvements in lactate, GDF-15, inflammatory biomarkers, cerebral blood flow, and functional connectivity after 29 days.
CY6463 was well tolerated with no serious adverse events or treatment discontinuations; PK consistent with Phase 1.
75% of patients were on standard-of-care arginine or citrulline; effects more pronounced in patients with greater baseline disease burden.
Data support further development of CY6463 in CNS diseases with mitochondrial dysfunction, including schizophrenia and Alzheimer's disease.

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