Cyclerion Updates Corporate Progress - Seite 2

The Company is tightly managing its spending. As of March 31, 2020, Cyclerion’s preliminary unaudited cash, cash equivalents and restricted cash balance was approximately $72 million. Cyclerion anticipates that this cash will fund its operations into Q2 2021, excluding net cash flows from potential business development activities.

Program Updates

Sickle Cell Disease (SCD)

Olinciguat is a once-daily oral sGC stimulator that primarily targets the vasculature and highly perfused organs such as the lungs and the kidney. Olinciguat has the potential to address multiple important clinical domains important in SCD by improving local blood flow, decreasing vascular inflammation, reducing anemia, and improving chronic symptoms.

The STRONG-SCD study is a randomized, placebo-controlled, dose-ranging study designed to evaluate safety, tolerability, and pharmacokinetics, as well as to explore effects on daily symptoms and biomarkers of disease activity when dosed over a 12-week treatment period. The study protocol was amended in late 2019 to add a higher-dose arm. Cyclerion recently closed enrollment of the study with a total of 70 participants randomized.

“We are excited to have closed enrollment for our SCD-STRONG study. We look forward to the top line results in Q3 2020 and to making a data-driven decision regarding advancement to the next phases of development,” said Chris Wright, M.D., Cyclerion’s Chief Medical Officer.

Central Nervous System (CNS)

Cyclerion is developing IW-6463, an oral, once-daily CNS-penetrant sGC stimulator for the treatment of serious neurodegenerative diseases. The nitric oxide pathway and sGC stimulation have long been known as central physiological regulators in the CNS, affecting cerebrovascular blood flow, neuroinflammation, neuronal function and cellular bioenergetics.

In January 2020, the Company reported encouraging Phase 1 healthy volunteer study results for IW-6463.  This potential new CNS medicine was well tolerated across the dose range assessed. Pharmacokinetic (PK) data from blood and cerebral spinal fluid (CSF), supported QD dosing and indicated the potential to reach pharmacologically active CNS exposures, based on preclinical data.

An ongoing translational pharmacology clinical study has enrolled 24 elderly subjects. The study will evaluate safety and biomarker measures of CNS activity.  Cyclerion expects top-line clinical results in mid-2020.

With supportive study results, the Company plans to direct further development towards serious CNS diseases with high unmet medical need where biological and/or genetic data suggest an important role for nitric oxide and cyclic guanosine monophosphate (cGMP) signaling.