Canadian biotechnology company Cardiol Therapeutics has announced topline results from its ARCHER Phase II clinical trial evaluating CardiolRx in patients with acute myocarditis.

CardiolRx, the company’s lead drug candidate, is a pharmaceutically manufactured cannabidiol oral solution currently in clinical development for the treatment of heart disease. Cardiol has received Investigational New Drug Application authorisation from the United States Food and Drug Administration (US FDA) to conduct clinical studies to evaluate the efficacy and safety of CardiolRx in two diseases affecting the heart: recurrent pericarditis and acute myocarditis.

ARCHER was a multi-national, randomized, double-blind, placebo-controlled Phase II trial designed to assess the safety, tolerability, and impact of CardiolRx on myocardial recovery in patients diagnosed with acute myocarditis. A total of 109 patients were enrolled across leading cardiovascular centers in the United States, France, Brazil, and Israel.

In the two primary endpoints—extracellular volume (ECV) and global longitudinal strain (GLS)—CardiolRx showed a notable improvement in ECV compared to placebo following 12 weeks of double-blind therapy, with no significant difference observed in GLS in a population that had preserved left ventricular (LV) function at baseline.

The reduction in ECV was associated with improvements over placebo in multiple pre-specified cardiac magnetic resonance imaging (CMR) endpoints, including a significant reduction in LV mass.

These results provide compelling clinical proof of concept for CardiolRx and strongly support advancing the clinical development of CardiolRx and CRD-38 in cardiomyopathies, heart failure, and myocarditis.

CRD-38 is a novel subcutaneously administered drug formulation developed by the company for use in heart failure—a leading cause of death and hospitalization in the developed world.  

Consistent with findings from Cardiol’s Phase II MAvERIC trial in recurrent pericarditis, CardiolRx was shown to be safe and well tolerated.

 “The results offer exciting new insights into the treatment of acute myocarditis and strongly support advancing the clinical development of this novel therapeutic approach for inflammatory cardiac conditions, including myocarditis and heart failure,” said Dr. Dennis M. McNamara, Chair of the ARCHER Steering Committee.

Dr. Leslie T. Cooper, Co-Chair of the ARCHER Steering Committee, noted that the findings reinforce their original hypothesis that pharmaceutically manufactured cannabidiol can attenuate myocardial inflammation and edema.

“ARCHER’s results provide sound rationale for advancing the clinical development of this novel therapy in conditions of the myocardium characterized by edema, fibrosis, and remodeling, including the growing challenge of immune checkpoint inhibitor-induced myocarditis which can be fatal,” he added.

David Elsley, President and CEO of Cardiol Therapeutics, stated that they’re now looking forward to integrating the ARCHER findings into their broader clinical development strategy and business development initiatives—supporting the continued advancement of CardiolRx and CRD-38 as potential treatments for inflammatory cardiac disorders.

The ARCHER findings have been submitted for presentation at an upcoming scientific meeting and will also be submitted for publication, the company said.

Acute myocarditis is an inflammatory condition of the heart muscle (myocardium) characterised by symptoms such as chest pain, shortness of breath at rest or during activity, fatigue, rapid or irregular heartbeat (arrhythmias), and light-headedness or the feeling one might faint. It is an important cause of acute and fulminant heart failure and is a leading cause of sudden cardiac death in people under 35 years of age.

Viral infection is the most common cause of myocarditis; however, it can also result from bacterial infection, commonly used drugs, and mRNA vaccines, as well as therapies used to treat several common cancers, including chemo-therapeutic agents and immune checkpoint inhibitors. There are no FDA-approved drug therapies for acute myocarditis. Patients hospitalised with the condition experience an average seven-day length of stay and a 4 – 6 percent risk of in-hospital mortality.

CardiolRx has received Orphan Drug Designation from the FDA for the treatment of pericarditis, including recurrent pericarditis. This inflammatory condition of the pericardium is associated with debilitating chest pain, shortness of breath, and fatigue, and results in physical limitations, reduced quality of life, emergency department visits, and hospitalisations.

Headquartered in Oakville, Ontario, Cardiol Therapeutics is a clinical-stage life sciences company focused on developing anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease.