Cyclerion Stops Development of SCD Therapy After Phase 2 Trial Failure

Rare Daily Staff

Cyclerion Therapeutics reported that top-line results from its phase 2 study of olinciguat for the potential treatment of sickle cell diseases, while generally well-tolerated, did not demonstrate adequate activity to support further clinical development.

Cyclerion intends to complete its analysis of the study results and present or publish them in a future forum. Shares of the company fell 44 percent on the news.

Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein. In sickle cell disease, the red blood cells are misshapen, in a sickle shape instead of the disc shape. The abnormal shape causes the cells to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease.

The phase 2 STRONG-SCD study was a randomized, placebo-controlled, dose-ranging study of 70 participants designed to evaluate safety, tolerability, and pharmacokinetics of olinciguat, a sGC stimulator, compared to placebo, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period.

“While we are disappointed that we won’t be contributing a much-needed new treatment option for SCD,” said Peter Hecht, CEO of Cyclerion, “we are continuing to analyze the data to understand several potential biomarker signals, including inflammation, as we explore partnership options for this program.”

Cyclerion will turn its focus on the development of its lead CNS-penetrant sGC stimulator, IW-6463 for serious diseases of the central nervous system including mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS), and Alzheimer’s disease with vascular pathology.

Photo: Peter Hecht, CEO of Cyclerion