Clinical trials are crucial for smaller pharma companies. While the ultimate aim is to achieve final regulatory approval, each phase of the development process adds value to the company. Starting from research and discovery, moving through Investigational New Drug filings, and advancing through the various stages of clinical trial, pharma developers systematically build the foundation of a progressively stronger company.
Cardiol Therapeutics Inc. (Nasdaq: CRDL) (TSX: CRDL) has been able to masterfully navigate this path in its quest to develop superior treatments for problematic heart conditions. Cardiol has one drug candidate in two clinical trial programs, recurrent pericarditis and acute myocarditis, with another candidate in the pre-clinical stage. The company also has an Orphan Drug Designation (ODD) under its belt, granted by the United States Food and Drug Administration for the treatment of pericarditis.
Here we’ll take a look at Cardiol’s strategic advancements and what is on the horizon as it paves its path towards attaining regulatory approval.
Where Cardiol Stands Now
Cardiol wrapped up its Phase 2 study of recurrent pericarditis with highly anticipated full data results to be presented next month at the American Heart Association Scientific Sessions 2024. The study, called MAvERIC Phase 2, covered treatment of recurrent pericarditis with Cardiol’s drug candidate, CardiolRx™, and is the program operating under the umbrella of the FDA’s ODD. Sponsors of drugs developed with the ODD are incentivized with significant tax credits, the possibility of seven years of market exclusivity following approval, fast-track review, and fee reductions or exemptions. The program was created to encourage research and development of therapies for relatively rare conditions that might be otherwise overlooked.
On the heels of that success, Cardiol announced plans to expand the MAVERIC clinical development program and advance CardiolRx™ into a late-stage clinical trial, MAVERIC-2. The Phase 2/3 study is expected to enroll approximately 110 patients and will evaluate the impact of CardiolRx™ in recurrent pericarditis patients following cessation of interleukin-1 (IL-1) blocker therapy with drugs like rilonacept and anakinra. The new study provides the opportunity for Cardiol to expand the market potential for CardiolRx™ and potentially provide a path for an accelerated regulatory approval timeline.
Not only is MAVERIC-2 expected to be initiated in Q4, but it will run concurrently with another planned pivotal Phase 3 study in recurrent pericarditis – a second late-stage study called MAVERIC-3. Importantly, the new MAVERIC-2 Phase 2/3 study announced is expected to report data as early as one year ahead of the data from the planned MAVERIC-3 Phase 3 program. Phase 3 trials are generally the last step before filing for drug approval from the FDA.
On top of the advancing pericarditis studies, Cardiol offers two more opportunities in its pipeline. The company’s Phase 2 ARCHER trial for acute myocarditis reached full enrollment in September 2024, and topline results are expected in the first quarter of 2025. Cardiol believes there is a significant opportunity to develop an important new therapy for acute myocarditis that would also be eligible for designation as an ODD in the United States and the European Union.
The company is also engaged in pre-IND research on CRD-38, a novel subcutaneously administered drug formulation intended for use for heart failure. Heart failure represents a much larger market opportunity than both pericarditis and myocarditis, affecting more than 64 million globally. The chronic syndrome is estimated to exceed $30 billion of healthcare costs in the United States each year.
What Does All This Mean?
Nothing is guaranteed in terms of results, but the potential is high even when looking solely at Cardiol’s MAVERIC program. In this case there is a very clear comparison to make between Cardiol and another company that has been down this particular road with recurrent pericarditis.
A few years ago, Kiniksa Pharmaceuticals licensed rilonacept from Regeneron in the hopes it could treat recurrent pericarditis. It was granted Breakthrough Therapy designation in 2019, and ODD in 2020. In 2021, rilonacept (trade name ARCALYST) was approved by the FDA. Kiniksa’s market cap rose from $380 million in 2019 to $1.375 billion at the time of approval, and rilonacept remains its only approved drug. Kiniksa is currently valued in the neighborhood of $1.88 billion.
There are reasons to believe that, should the trials prove efficacy, CardiolRx™ could be a superior treatment to rilonacept for recurrent pericarditis. Kiniksa’s drug is the first approved to treat recurrent pericarditis but comes with significant cost to the patient and a batch of side effects. Cardiol anticipates lower prices and has seen no significant side effects for CardiolRx™.
Keep in mind, Cardiol offers two more opportunities in its pipeline. The company recently completed a financing for gross proceeds of $15,525,000, providing sufficient cash to fund the execution of the new Phase 2/3 MAVERIC-2 trial, as well as allow completion of the Phase 2 ARCHER trial in acute myocarditis, and complete IND-enabling studies for CRD-38.
Cardiol Therapeutics is currently valued in the neighborhood of $153 million. The company’s stock opened the year on the Nasdaq at $0.82/share and now trades around $1.92/share. Interested investors are encouraged to dive in and investigate the company’s science and potential.
