Remedy Pharmaceuticals, Inc.

NEW YORK, Oct. 22, 2024 /PRNewswire/ -- Remedy Pharmaceuticals, a pioneer in stroke drug development, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Division (OOPD) has granted Orphan Drug Designation for CIRARA for the treatment of "large territory acute ischemic stroke," which includes large hemispheric infarctions (LHI). LHI is a particularly severe form of ischemic stroke caused by a blockage in the one of two large blood vessels in the brain: internal carotid artery or middle cerebral artery (MCA), which cuts off the blood supply to large areas of the brain, resulting in severe swelling, significant mortality, and high disability rates. "The Orphan Drug Designation for CIRARA underscores the uniqueness of large territory ischemic strokes in terms of mechanism and universally poor outcomes in contrast to smaller strokes," said Sven Jacobson, CEO of Remedy Pharmaceuticals. "This designation will accelerate our efforts to bring CIRARA to acute ischemic stroke patients at the highest risk of severe disability and death." The FDA's Orphan Drug Designation program provides orphan status to drugs or biologics intended to treat diseases or conditions that affect fewer than 200,000 people in the United States. Sponsors of medicines with Orphan Drug Designation may be eligible for various incentives, including tax credits for qualified clinical trials, prescription drug user fee exemptions, and seven years of marketing exclusivity upon FDA approval. About Large Hemispheric Infarction A large hemispheric infarction is a particularly severe type of ischemic stroke that affects a substantial portion of the brain. This occurs when there is a blockage in a major cerebral artery, such as the middle cerebral artery (MCA) or the internal carotid artery (ICA), both of which are responsible for supplying large regions of the brain with oxygen-rich blood. Treatment for LHI typically involves rapid revascularization strategies to restore blood flow, such as mechanical thrombectomy or intravenous thrombolysis (administering clot-dissolving drugs like tPA). However, the large scale of the infarction and the resultant edema leads to poorer outcomes compared to smaller strokes, with many patients suffering long-term disability or, in some cases, death. About Remedy Pharmaceuticals: New York-based Remedy Pharmaceuticals, Inc. is a privately held, clinical-stage pharmaceutical company focused on developing and bringing lifesaving treatments to people affected by acute central nervous system (CNS) diseases and injuries. Contact: Sven Jacobson CEO Remedy Pharmaceuticals 212.586.2226 [email protected] SOURCE Remedy Pharmaceuticals WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NEW YORK, Oct. 1, 2024 /PRNewswire/ -- Remedy Pharmaceuticals today announced additional promising data from the Phase 3 CHARM clinical trial of CIRARA (IV glyburide) in patients with Large Hemispheric Infarction (LHI), the most serious form of ischemic stroke, characterized by large-volume lesions that lead to malignant brain swelling, severe disability, and death. This latest analysis revealed that CIRARA demonstrated a significant, sustained effect at 12 months on the modified Rankin Scale (mRS) in patients with lesion volumes <125 mL. The odds ratio for CIRARA's effect across all <125 mL lesion volume patients was 2.22 (p=0.03), and in patients who also received endovascular thrombectomy (EVT), the odds ratio was even higher at 6.92 (p=0.02). The sustained effect of CIRARA over time is further exemplified by the fact that 78% of EVT patients treated with CIRARA were able to walk independently at 12 months, compared to only 51% in the placebo group—representing a 55% relative increase in this important outcome. In May 2024, Remedy announced that in the prespecified modified intent-to-treat (mITT) population with NIH Stroke Scale (NIHSS) ≤ 20 (n=274), CIRARA improved outcomes with an odds ratio of 1.8 (p=0.01) at Day 90. A post hoc analysis of patients with a lesion volume <125 mL (n=118) found an odds ratio of 2.2 in favor of CIRARA (p=0.04), while in the EVT population with lesion volumes <125 mL (n=34), the odds ratio was 7.1 (p=0.01). Further post hoc analysis showed that CIRARA significantly increased the likelihood of achieving an mRS score of 0-3 (indicating moderate disability or better) in patients with a lesion volume <125 mL (n=118), with an odds ratio of 4.1 (p<0.01). An mRS score of 0-3 includes the ability to ambulate independently—a highly desirable outcome. Among EVT patients with lesion volumes <125 mL (n=34), CIRARA's odds ratio for achieving an mRS score of 0-3 was an impressive 18.5 (p=0.03). "These latest findings are incredibly encouraging," said Sven Jacobson, CEO of Remedy Pharmaceuticals. "CIRARA continues to demonstrate its potential to dramatically improve outcomes for patients suffering from severe ischemic stroke. The ability to show sustained effects through one year and the profound impact on patients' ability to walk independently certainly strengthens our resolve as we move forward with a confirmatory Phase 3 study." About the CHARM Study The CHARM clinical trial was a multicenter, multinational, double-blind, randomized, placebo-controlled Phase 3 study involving patients aged 18-85 with LHI. Patients were eligible if the study drug was expected to start within 10 hours after they were last known well. A total of 535 patients were enrolled, including 431 patients aged 18-70 (the efficacy analysis population) and 81 patients aged 71-85. The study, which was sponsored by Cambridge-based Biogen, was terminated early after a strategic realignment of resources, leading to the return of the program to Remedy. About Remedy Pharmaceuticals: New York-based Remedy Pharmaceuticals, Inc. is a privately held, clinical-stage pharmaceutical company focused on developing and bringing lifesaving treatments to people affected by acute central nervous system (CNS) diseases and injuries. Remedy is planning a confirmatory Phase 3 study of CIRARA in LHI. Contact: Sven Jacobson CEO Remedy Pharmaceuticals 212.586.2226 [email protected] SOURCE Remedy Pharmaceuticals WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

NEW YORK, Sept. 3, 2024 /PRNewswire/ -- Remedy Pharmaceuticals today announced promising new data from the Phase 3 CHARM clinical trial of CIRARA (IV glyburide) in Large Hemispheric Infarction (LHI), the most serious form of ischemic stroke, characterized by large volume lesions that lead to malignant brain swelling, severe disability, and death. The analysis revealed significant and clinically meaningful improvements in patients' ability to ambulate independently following administration of CIRARA, particularly when used in combination with endovascular thrombectomy (EVT). The CHARM clinical trial was a multicenter, multinational, double-blind, randomized, placebo-controlled Phase 3 study involving patients aged 18-85 with LHI. Patients were eligible if the study drug was expected to start within 10 hours after they were last known well. A total of 535 patients were enrolled, including 431 patients aged 18-70 (the efficacy analysis population) and 81 patients aged 71-85. The study was terminated early by Cambridge-based Biogen after a strategic realignment of resources, leading to the return of the program to Remedy. In May 2024, Remedy conducted prespecified subgroup analyses, along with post hoc analyses examining the impact of lesion volume on outcomes, which revealed significant improvements in functional outcomes on the modified Rankin Scale (mRS) at 90 days. Specifically, in the prespecified modified Intent to Treat (mITT) population with NIH Stroke Scale (NIHSS) ≤ 20 (n=274), CIRARA improved outcomes with an Odds Ratio (OR) of 1.8, p=0.01. In a post hoc analysis of patients with CTP/MRI lesion volume <125 mL (n=118), the OR for improved 90-day outcome was 2.2 in favor of CIRARA (p=0.04). Additionally, in the EVT population with CTP/MRI lesion volume <125 mL (n=34), the OR was 7.1 in favor of CIRARA (p=0.01). Further post hoc analyses have now been performed and show that the OR for achieving an mRS score of 0-3 (moderate disability or better) in patients with a lesion volume < 125 mL (n=118) was 4.1 in favor of CIRARA (p<0.01). An mRS of 0-3 reflects no greater than moderate disability, which includes the ability to ambulate independently, a highly desirable outcome. In the mITT EVT population with a lesion volume <125 mL (n=34), the OR for achieving an mRS score of 0-3 was 18.5 in favor of CIRARA (p=0.03), indicating an even more dramatic improvement in functional outcomes, particularly in the ability to ambulate independently. "These new findings are incredibly encouraging and provide strong evidence that CIRARA could be a game-changer in the treatment of large hemispheric infarction," said Sven Jacobson, CEO of Remedy Pharmaceuticals. "The significant improvements in functional outcomes, and particularly the increased rates of independent ambulation, highlight the potential of CIRARA to revolutionize stroke care and offer new hope to patients and their families." About Remedy Pharmaceuticals: New York-based Remedy Pharmaceuticals, Inc. is a privately held, clinical-stage pharmaceutical company focused on developing and bringing lifesaving treatments to people affected by acute central nervous system (CNS) diseases and injuries. Remedy is planning a confirmatory Phase 3 study of CIRARA in LHI. Contact: Sven Jacobson CEO Remedy Pharmaceuticals 212.586.2226 [email protected] SOURCE Remedy Pharmaceuticals