Rilonacept

Rilonacept

Phase 3

Photo of scientist with petri dish and syringe

Rationale

Final data from an open-label Phase 2 clinical trial of rilonacept in a range of RP populations showed improvements in the study on clinically meaningful outcomes associated with the unmet need in RP: resolution of pericarditis episodes, reduction in recurrences of pericarditis episodes while on treatment, tapering and discontinuation of corticosteroids without pericarditis recurrence, and improved quality-of-life scores. Safety data from this study were consistent with the known safety profile of rilonacept.

Status: Phase 3

We are conducting a pivotal, global Phase 3 clinical trial in RP, named RHAPSODY.

RHAPSODY is a double-blind, placebo-controlled, randomized-withdrawal (RW) design study with open-label extension in subjects with symptomatic RP. After a run-in period with stabilization and tapering of background medications to monotherapy rilonacept, clinical responders will be randomized to continued rilonacept or placebo injected subcutaneously weekly during the 24-week RW period. The primary efficacy endpoint is time to first pericarditis recurrence in the RW period. Topline data are expected in the second half of 2020.

Unmet Need

We are not aware of any current therapies approved by the FDA for the treatment of RP.

  • We believe RP presents a significant clinical and humanistic burden and imposes a significant financial burden on the healthcare system.
  • A patient’s initial acute episode of pericarditis is typically treated with over-the-counter or prescription nonsteroidal anti-inflammatory drugs (NSAIDs) or colchicine, both of which are used off-label. Recurrent episodes are treated in a similar manner or by adding systemic corticosteroids, which are also used off-label.
  • We believe the greatest demand in the market is for a product that not only treats the symptoms of a pericarditis recurrence but also reduces the risk of future recurrences while on treatment.
  • Within the estimated diagnosed and treated RP population of 40,000, we believe that there are initially 14,000 patients who may benefit from rilonacept— ~8,000 refractory, which includes those not suitable for steroids, ~1,000 steroid dependent, and ~5,000 multiple relapsing.
  • Refractory and steroid-dependent patients have the highest recurrence burden, with 30% to 40% of patients experiencing greater than 2 episodes per year.
    • They also have a higher incidence of severe complications, such as cardiac tamponade and constrictive pericarditis, often use opioids to manage their pain, and deal with anxiety and depression due to the unpredictability and severity of recurrences.

Stethoscope over chest

Explore Our Pipeline

We are intent on developing an array of innovative therapies that answer the many and varied needs of patients.