Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a
biopharmaceutical company with a pipeline of assets designed to
modulate immunological pathways across a spectrum of diseases,
today announced that data from RHAPSODY, the pivotal Phase 3 trial
of rilonacept in recurrent pericarditis, were published in The New
England Journal of Medicine. Additionally, the RHAPSODY data were
presented at the late-breaking science session during the American
Heart Association (AHA) Scientific Sessions 2020. Kiniksa
previously reported positive top-line RHAPSODY results in June
2020. Rilonacept is a weekly, subcutaneously-injected, recombinant
fusion protein that blocks interleukin-1 alpha (IL-1α) and
interleukin-1 beta (IL-1β) signaling.
The manuscript entitled Phase 3 Trial of Interleukin-1 Trap
Rilonacept in Recurrent Pericarditis, is available on The New
England Journal of Medicine website with open access for seven
days.
The AHA presentation entitled RHAPSODY: Rilonacept an IL-1α and
IL-1β Trap Resolves Pericarditis Episodes and Reduces Risk of
Recurrence in a Phase 3 Trial of Patients with Recurrent
Pericarditis was presented virtually by Allan Klein, MD,
of Cleveland Clinic, co-principal investigator of the study
and compensated member of a 2019 Kiniksa scientific advisory
committee. Massimo Imazio, MD, of the University of Torino, Italy,
is co-principal investigator.
“Recurrent pericarditis is a debilitating autoinflammatory
disease with a clear unmet need,” said Dr. Klein. “Data were
reported which showed that patients treated with rilonacept
experienced a 96% reduction in risk of recurrent pericarditis
events. Furthermore, in acutely symptomatic patients who were
failing standard management, rilonacept treatment in the study
provided rapid and sustained reductions in pain and C-reactive
protein as soon as after the first dose. These data suggest that
targeted immunomodulation with rilonacept may signal a paradigm
shift in the management of patients with recurrent
pericarditis.”
In RHAPSODY, the primary efficacy endpoint of time-to-first
adjudicated pericarditis recurrence in the randomized withdrawal
period was highly statistically significant (Hazard Ratio = 0.04,
p<0.0001). Additionally, annualized incidence of pericarditis
recurrence decreased from 4.42 episodes per year prior to the study
to 0.15 episodes per year while on rilonacept treatment. All major
secondary endpoints were also highly statistically significant.
Rilonacept was well-tolerated in the study, with adverse events
consistent with the U.S. Food and Drug Administration
(FDA)-approved label for the treatment of Cryopyrin-Associated
Periodic Syndromes (CAPS). The most common adverse events were
injection site reactions and upper respiratory tract infections.
There were no drug-related serious adverse events.
About RHAPSODYRHAPSODY is the global,
randomized withdrawal design, pivotal Phase 3 clinical trial of
rilonacept in recurrent pericarditis. Eligible patients presented
at screening with at least a third pericarditis episode, defined as
at least 1 day with pericarditis pain of ≥ 4 on the 11-point
Numerical Rating Scale (NRS) and a C-reactive protein (CRP) value ≥
1 mg/dL within the 7-day period prior to first study drug
administration. Patients could be receiving concomitant
nonsteroidal anti-inflammatory drugs (NSAIDs) and/or colchicine
and/or oral corticosteroid treatment in any combination. The study
was comprised of 4 periods: a screening period; a single-blind
run-in period during which patients received a loading dose of
rilonacept 320 mg injected subcutaneously (SC) followed by 160 mg
SC weekly while background pericarditis medications were tapered
and discontinued; a double-blind, placebo-controlled randomized
withdrawal period during which clinical responders to rilonacept
were randomized 1:1 and received 160 mg SC weekly rilonacept or
placebo; and a long-term extension treatment period with up to 24
months of open-label rilonacept 160 mg SC weekly. For more
information, refer to ClinicalTrials.gov Identifier:
NCT03737110.
About Recurrent PericarditisRecurrent
pericarditis is a painful and debilitating autoinflammatory
cardiovascular disease that typically presents with chest pain and
is often associated with changes in electrical conduction and
sometimes buildup of fluid around the heart, called pericardial
effusion. Patients with pericarditis are deemed recurrent if they
have an additional episode after a symptom-free period of 4-6
weeks, and chronic if symptoms from any one episode last longer
than three months. Recurrent pericarditis symptoms impair qualify
of life, limit physical activities, and lead to frequent emergency
department visits and hospitalizations. There are currently no
FDA-approved treatments for recurrent pericarditis.
About RilonaceptRilonacept is a weekly,
subcutaneously-injected, recombinant fusion protein that blocks
IL-1α and IL-1β. Rilonacept was discovered and developed by
Regeneron and is approved by the FDA under the brand name
ARCALYST® for the treatment of CAPS, specifically Familial
Cold Autoinflammatory Syndrome and Muckle-Wells Syndrome.
Rilonacept for the treatment of deficiency of the interleukin1
receptor antagonist (DIRA) is currently under FDA review following
the submission of a Supplemental Biologics License Application
(sBLA) in June 2020. Rilonacept in recurrent pericarditis is
an investigational drug. The FDA granted Breakthrough Therapy
designation to rilonacept for the treatment of recurrent
pericarditis in 2019 and Orphan Drug designation to rilonacept for
the treatment of pericarditis in 2020.
Important information about ARCALYST® (rilonacept)
Injection IL-1 blockade may interfere with immune response
to infections. Serious, life-threatening infections have been
reported in patients taking ARCALYST. ARCALYST should be
discontinued if a patient develops a serious infection. Taking
ARCALYST with TNF inhibitors is not recommended because this may
increase the risk of serious infections.Patients should not receive
a live vaccine while taking ARCALYST. It is recommended that prior
to initiation of therapy with ARCALYST patients receive all
recommended vaccinations, as appropriate, including pneumococcal
vaccine and inactivated influenza vaccine. In the initial
development program for ARCALYST, six serious adverse reactions
were reported by four patients: Mycobacterium intracellular
infection, gastrointestinal bleeding and colitis, sinusitis and
bronchitis and Streptococcus pneumoniae meningitis. The most
commonly reported adverse reactions associated with ARCALYST were
injection site reaction and upper respiratory tract infection.
Patients should be monitored for changes in their lipid profiles
and provided with medical treatment if warranted. Treatment with
immunosuppressants, including ARCALYST, may result in an increase
in risk of malignancies. Hypersensitivity reactions associated with
ARCALYST administration in clinical studies have been rare. If a
hypersensitivity reaction occurs, administration of ARCALYST should
be discontinued and appropriate therapy initiated.
About KiniksaKiniksa is a
biopharmaceutical company focused on discovering, acquiring,
developing and commercializing therapeutic medicines for patients
suffering from debilitating diseases with significant unmet medical
need. Kiniksa’s clinical-stage product candidates, rilonacept,
mavrilimumab, vixarelimab and KPL-404, are based on strong biologic
rationale or validated mechanisms, target underserved conditions
and offer the potential for differentiation. These pipeline assets
are designed to modulate immunological pathways across a spectrum
of diseases. For more information, please
visit www.kiniksa.com.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. In some cases,
you can identify forward looking statements by terms such as “may,”
“will,” “should,” “expect,” “plan,” “anticipate,” “could,”
“intend,” “target,” “project,” “contemplate,” “believe,”
“estimate,” “predict,” “potential” or “continue” or the negative of
these terms or other similar expressions, although not all
forwardlooking statements contain these identifying words. All
statements contained in this press release that do not relate to
matters of historical fact should be considered forward-looking
statements, including without limitation, statements regarding: the
belief that the Phase 3 data suggest that targeted immunomodulation
with rilonacept may signal a paradigm shift in the management of
patients with recurrent pericarditis; and the belief that all of
our product candidates offer the potential for differentiation.
These forward-looking statements are based on management’s
current expectations. These statements are neither promises nor
guarantees, but involve known and unknown risks, uncertainties and
other important factors that may cause our actual results,
performance or achievements to be materially different from any
future results, performance or achievements expressed or implied by
the forward-looking statements, including without limitation, the
following: impact of additional data from us or other companies;
potential undesirable side effects caused by rilonacept; our
potential inability to demonstrate safety and efficacy of
rilonacept in recurrent pericarditis to the satisfaction of the FDA
or other applicable regulatory authorities; the potential for
applicable regulatory authorities to not accept sBLA filings or to
delay or deny approval of any of our product candidates; our
potential inability to demonstrate that the clinical data integrity
and quality of the biologic manufacturing processes and facilities
are sufficient for the FDA to approve rilonacept in recurrent
pericarditis; our reliance on third parties to manufacture our
product candidates, including our reliance on Regeneron to
manufacture the clinical and commercial supply of rilonacept; ; the
potential impact of the COVID-19 pandemic and measures taken in
response to the pandemic; and changes in our operating plan and
funding requirements.
These and other important factors discussed under the caption
“Risk Factors” in our Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (“SEC”) on
November 5, 2020 and our other reports subsequently filed with
the SEC could cause actual results to differ materially
from those indicated by the forward-looking statements made in this
press release. Any such forward-looking statements represent
management’s estimates as of the date of this press release. While
we may elect to update such forward-looking statements at some
point in the future, we disclaim any obligation to do so, even if
subsequent events cause our views to change. These forward-looking
statements should not be relied upon as representing our views as
of any date subsequent to the date of this press release.
ARCALYST® is a registered trademark of Regeneron
Pharmaceuticals, Inc.
Every Second Counts!™
Kiniksa Investor and Media
ContactMark Ragosa(781) 430-8289mragosa@kiniksa.com
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