Pericarditis is a rare autoinflammatory cardiovascular condition. It can lead to changes of the electrical conduction of the heart and a buildup of fluid surrounding the heart. Pericarditis is considered recurrent if episodes occur every 4 to 6 weeks. It is considered chronic if patients experience lingering symptoms from a single episode for longer than a 3 month period.
There are currently no approved treatments for this condition, and symptoms can severely impact patients’ quality of life.
Rhapsody was a Phase 3 clinical trial conducted by Kiniksa Pharmaceuticals. It aimed to investigate rilonacept in patients with recurrent pericarditis. This treatment is an IV injection which works to block IL-1a and IL-1B signaling. The treatment must be received once every week.
The principal investigators of this trial were Allan Klein from the Cleveland Clinic and Massimo Imazio from the University of Torino in Italy. They investigated the safety and efficacy of this therapy.
This trial included 61 patients who were not responding to standard treatment such as NSAIDs, corticosteroids, and colchicine. Patients were randomized to either receive rilonacept or a placebo weekly. Those who took rilonacept had a 96% decrease in their risk of experiencing another episode.
All of the trials secondary endpoints were met. At the end of week 16 81% of all patients on the treatment had maintained a clinical response versus just 20% of those taking placebo. Further, at this timepoint, 81% of those on the therapy had either no or minimal symptoms versus 25% of those taking placebo. At weeks 8 and 24, both of these findings had maintained consistency and statistical significance.
The treatment was found to be well tolerated for patients and AEs were consistent with the ones found for Cryopyrin-Associated Periodic Syndromes (CAPS), an indication Rilonacept is already approved for. The most common AE patients experienced was an injection site reaction.
This therapy could have meaningful benefits for patients. This is shown by the fact that those taking the drug in the Rhapsody trial experienced no or very small amounts of pain for a whopping 95% of the trial days until week 16. For those taking placebo, fewer than half of the patients experienced these benefits.
This therapy was first granted Breakthrough Therapy Designation by the FDA in 2019. A biologics licensing application should be submitted for this new indication by the end of the year.
The statistically significant results of this trial, for both primary and secondary endpoints, have provided researchers hope and excitement for the future of this therapy for pericarditis.
You can read more about this study and treatment here.