Remedy Pharmaceuticals Reacquires Acute Stroke Drug from Biogen; Reports Significant Phase 3 Study Results

NEW YORK, May 29, 2024 -- Remedy Pharmaceuticals, a leader in stroke drug development, revealed that subgroup analyses from their Phase 3 CHARM clinical trial of CIRARA (IV glibenclamide) demonstrated significant improvements in functional outcomes on the modified Rankin Scale (mRS). These results were further confirmed through post hoc analyses.

The pivotal Phase 3 CHARM trial was prematurely terminated by Biogen, based in Cambridge, following a strategic shift in resource allocation, resulting in the program's return to Remedy.

The CHARM trial was conducted across multiple centers and countries, employing a double-blind, randomized, placebo-controlled design. The trial enrolled patients aged 18-85 who had experienced a large hemispheric infarction (LHI), identified by an Alberta Stroke Program Early CT Score (ASPECTS) between 1-5 or an ischemic core volume ranging from 80-300 ml as determined by CT perfusion (CTP) or MRI diffusion-weighted imaging. Eligible participants had to begin the study drug within 10 hours of last being known well and were randomly assigned to receive either CIRARA or a placebo. Out of 535 enrolled patients, 431 were aged 18-70 (forming the efficacy analysis population), while 81 were between 71-85 years old.

Despite neutral top-line results, pre-specified analyses indicated directionally improved functional outcomes for CIRARA in various subgroups, including patients who received rtPA, underwent endovascular thrombectomy (EVT), enrolled using CTP/MRI, had wake-up strokes, or were enrolled in the U.S. Specifically, a notable improvement was observed in patients with an NIH Stroke Scale (NIHSS) score ≤20 (n=274), with an Odds Ratio of 1.66 in favor of CIRARA (p=0.03). The EVT subset showed an Odds Ratio of 1.75, which, while not statistically significant, was encouraging.

Post hoc analyses further investigated the impact of stroke severity on outcomes, unveiling significant results:
- In the modified intention-to-treat (mITT) population with CTP/MRI lesion volume <125mL (n=118), the Odds Ratio was 2.15 in favor of CIRARA (p=0.04). Mortality in the CIRARA group was numerically lower at 14% compared to 22% in the placebo group, without an increase in severe disability. The proportion of patients achieving an mRS 0-3 was 36% for CIRARA versus 19% for placebo, marking an 89% improvement.
- In the mITT EVT population with CTP/MRI lesion volume <125mL (n=34), the Odds Ratio was 7.13 in favor of CIRARA (p=0.01). Mortality was significantly lower at 6% for CIRARA compared to 31% for placebo, with no increase in severe disability. A 100% increase was observed in the proportion of patients with mRS 0-3 for CIRARA at 62%, compared to 31% for placebo.

Dr. W. Taylor Kimberly, Chief of the Division of Neurocritical Care at Massachusetts General Hospital and Associate Professor of Neurology at Harvard Medical School, and co-principal investigator of the CHARM study, remarked on the promising nature of these findings, suggesting that CIRARA could complement existing treatments like tPA and endovascular therapy, potentially revolutionizing care for LHI patients.

Kevin Sheth, MD, Professor of Neurology and Neurosurgery at Yale and co-principal investigator of the study, highlighted the persistent high mortality and long-term disability rates in severe stroke cases. He emphasized that CIRARA could offer a significant breakthrough for thousands of LHI patients annually, improving their chances of survival and quality of life.

Sven Jacobson, CEO of Remedy Pharmaceuticals, expressed gratitude for Biogen’s investment and acknowledged the dedication of all participants, families, and clinical investigators involved in the CHARM study. He reiterated the company’s commitment to advancing medicine for those in critical need.

Data from the CHARM study was presented at the 10th European Stroke Organization Conference held in Basel, Switzerland, from May 15-17.

Remedy Pharmaceuticals, based in New York, is a clinical-stage pharmaceutical company focused on developing treatments for acute central nervous system diseases and injuries. They are planning a confirmatory Phase 3 study of CIRARA in LHI.

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