Cyclerion Sinks As Biopharma Shelves Sickle Cell Disease Study

Cyclerion Therapeutics Inc CYCN shares are pulling back sharply following a couple of clinical trial updates from the company.

What Happened: Cambridge, Massachusetts-based Cyclerion said its mid-stage investigational asset olinciguat did not show adequate activity to support further internal clinical development of it as a potential treatment for sickle cell disease.

Olinciguat, a vascular sGC stimulator, was found to be well tolerated across all dose ranges, top-line results from the Phase 2 STRONG-SCD study showed.

Separately, the company announced positive results from a Phase 1 translational pharmacology study of IW-6463, a soluble guanylate cyclase stimulator being developed for CNS disorders.

IW-6463, while found safe and generally well-tolerated, also resulted in meaningful improvements in certain neurophysiological and objective performance measures that are associated with age-related cognitive decline and neurodegenerative diseases, Cyclerion said.

Effects on cerebral blood flow and markers of bioenergetics were not observed in this study.

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What's Next: Cyclerion intends to complete analysis of the results of the STRONG-SCD olinciguat study and present or publish them in a future forum.

In the wake of the fiasco with the sickle cell disease indication, the company plans to focus on treatments for serious diseases of the central nervous system.

With the Phase 1 IW-6463 results supporting the investigational compound's ongoing development, Cyclerion said it will soon begin enrolling its Phase 2 clinical trial in mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes.

The company also said it will use the information gleaned from the translational pharmacology study and the previous Phase 1 study of IW-6463 to take up further clinical development activities, including initiation of a planned Phase 2 clinical trial in Alzheimer's disease with vascular pathology in 2021, as well as to explore other potential indications.

Cyclerion shares were slumping 45% to $3.88, their lowest level since early August.