- Primary and all major secondary efficacy endpoints were highly statistically significant
- Rilonacept treatment resulted in a 96% reduction in risk of recurrent pericarditis events (primary efficacy endpoint: Hazard Ratio = 0.04, p<0.0001)
- Safety results consistent with FDA-approved label for CAPS
- sBLA submission expected later this year
- Conference call and webcast scheduled for 8:30 a.m. EDT today
HAMILTON, Bermuda, June 29, 2020 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (NASDAQ:KNSA) (“Kiniksa”), a biopharmaceutical company focused on discovering, acquiring, developing and commercializing therapeutic medicines for patients with significant unmet medical need, reported positive data from RHAPSODY, a pivotal Phase 3 trial of rilonacept, a weekly, subcutaneously-injected, recombinant fusion protein that blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β) signaling, in recurrent pericarditis. RHAPSODY met its prespecified primary and all major secondary efficacy endpoints, showing that rilonacept improved clinically meaningful outcomes associated with the unmet medical need in recurrent pericarditis, a painful and debilitating autoinflammatory disease. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to rilonacept for the treatment of recurrent pericarditis in 2019, and Kiniksa expects to submit a Supplemental Biologics License Application (sBLA) later this year.
“We are pleased to announce that RHAPSODY, our pivotal Phase 3 trial of rilonacept in recurrent pericarditis, met its primary and all major secondary efficacy endpoints,” said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. “Combined with a well-tolerated safety profile and a weekly dosing regimen, these data are an important step forward for patients. We believe rilonacept has the potential to be the first FDA-approved therapy for recurrent pericarditis. We are committed to submitting an sBLA to the FDA later this year and look forward to bringing this potential treatment option to patients as soon as possible.”
RHAPSODY is a global, randomized withdrawal design, pivotal Phase 3 clinical trial of rilonacept in recurrent pericarditis. The trial’s primary analysis population included 61 actively symptomatic recurrent pericarditis patients who were failing standard of care treatment, including nonsteroidal anti-inflammatory drugs (NSAIDs), colchicine, or corticosteroids, initiated rilonacept treatment during a run-in period, discontinued background medications, and achieved and maintained clinical response (11-point pain Numerical Rating Scale (NRS) ≤ 2.0 and C-reactive protein (CRP) ≤ 0.5 mg/dL) on rilonacept monotherapy. Clinical responders were randomized 1:1 to receive continued weekly rilonacept (n=30) or placebo (n=31) in a blinded manner in the randomized withdrawal period.
The primary efficacy endpoint of time-to-first adjudicated pericarditis recurrence in the randomized withdrawal period was highly statistically significant.
- Median [95% CI] time to pericarditis recurrence for rilonacept recipients in the randomized withdrawal period could not be estimated due to the low number of recurrences in the rilonacept treatment arm. The median time-to-recurrence for placebo recipients was 8.6 [4.0-11.7] weeks (Hazard Ratio = 0.04, p<0.0001).
- Rilonacept recipients experienced a 96% reduction in risk of recurrent pericarditis events.
All major secondary efficacy endpoints in the randomized withdrawal period were also highly statistically significant.
- 81% of rilonacept recipients maintained clinical response at Week 16 of the randomized withdrawal period, compared to 20% of placebo recipients (p=0.0002). Consistent results were observed at Week 8 and Week 24 and were also highly statistically significant (p<0.0001 and p=0.0022, respectively).
- The proportion of rilonacept recipients with absent or minimal pericarditis symptoms at Week 16 of the randomized withdrawal period was 81% compared to 25% for placebo recipients (p=0.0006). Consistent results were observed at Week 8 and Week 24 and were also highly statistically significant (p<0.0001 and p=0.0002, respectively).
Rilonacept was well-tolerated in the study, with adverse events consistent with the FDA-approved label for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS). The most common adverse events were injection site reactions.
“The RHAPSODY data provide hope for patients suffering from recurrent pericarditis,” said John F. Paolini, MD, PhD, Chief Medical Officer of Kiniksa. “In fact, rilonacept patients experienced no or minimal pericarditis pain for nearly 95% of study days through Week 16 compared to less than half of study days for placebo recipients, which was highly statistically significant. We believe that, by treating and preventing disease recurrence, rilonacept has the potential to be a transformational therapeutic advancement in the treatment of patients with recurrent pericarditis and to become the first FDA-approved therapy for this debilitating autoinflammatory disease.”
Additional analyses of the RHAPSODY trial results are ongoing, and Kiniksa plans to present the data at a future medical meeting or in a publication.
Rilonacept was discovered and developed by Regeneron Pharmaceuticals, Inc. (Regeneron) and is approved by the FDA under the brand name ARCALYST® for the treatment of CAPS. Kiniksa licensed rilonacept from Regeneron in 2017 for evaluation in diseases believed to be mediated by both IL-1α and IL-1β, including recurrent pericarditis. The FDA granted Breakthrough Therapy designation to rilonacept for recurrent pericarditis in 2019. Based on the Phase 3 RHAPSODY data announced today, the Biologic License Application (BLA) for CAPS will transfer to Kiniksa, and the company plans to submit an sBLA with the FDA in recurrent pericarditis later this year. Upon receipt of FDA approval for rilonacept in recurrent pericarditis, Kiniksa would assume the sales and distribution of rilonacept for the approved indications in the United States and will evenly split profits on sales with Regeneron.