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 the U.S. Food and Drug Administration (FDA)
accepted the supplemental Biologics License Application (sBLA) for
rilonacept in recurrent pericarditis. The FDA granted priority
review to the application and assigned a Prescription Drug User Fee
Act (PDUFA) goal date of March 21, 2021. Rilonacept is a weekly,
subcutaneously-injected, recombinant dimeric fusion protein that
blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (IL-1β)
signaling. 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.
The regulatory submission was based on positive data from
RHAPSODY, a pivotal Phase 3 trial of rilonacept in recurrent
pericarditis. RHAPSODY met its prespecified primary and all major
secondary efficacy endpoints, showing that rilonacept treatment in
the trial improved clinically meaningful outcomes associated with
the significant unmet medical need in recurrent pericarditis.
RHAPSODY data were recently published in The New England Journal of
Medicine simultaneously with a late-breaking scientific
presentation at the American Heart Association’s Scientific
Sessions 2020.
“We are thrilled to receive the FDA’s acceptance of the sBLA
submission for rilonacept in recurrent pericarditis with priority
review, said Sanj K. Patel, Chief Executive Officer and
Chairman of the Board of Kiniksa. “Rilonacept has the
potential to become the first FDA-approved therapy for this painful
and debilitating autoinflammatory disease. We are committed to
bringing this potential treatment option to patients as soon as
possible.”
Rilonacept was discovered and developed by Regeneron
Pharmaceuticals, Inc. (Regeneron) and is approved by the FDA for
the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
under the brand name ARCALYST®. 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.
Based on positive RHAPSODY data, the Biologic License Application
(BLA) for CAPS transferred to Kiniksa. If approved by the FDA
for recurrent pericarditis, Kiniksa will take responsibility for
sales and distribution of rilonacept for all the approved
indications in the United States and evenly split profits with
Regeneron, as described in the Rilonacept License Agreement.
Kiniksa is obligated to pay regulatory milestones to Regeneron
of up to an aggregate of $27.5 million through the time of a
potential approval of rilonacept in recurrent pericarditis, of
which $7.5 million is expected to be paid in the fourth quarter of
2020.
Kiniksa continues to prepare for the potential commercial launch
of rilonacept in recurrent pericarditis. The company has been
generating evidence on disease burden, building disease awareness
with payers, physicians, and advocacy groups, and establishing core
capabilities such as distribution, patient services and data
management.
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. The primary
efficacy endpoint was time-to-first pericarditis-recurrence in the
randomized withdrawal period. The Clinical Endpoint Committee
adjudicated all suspected pericarditis recurrences for inclusion in
the primary efficacy endpoint analysis. Kiniksa will continue to
follow patients in the long-term extension treatment period for up
to 24 months. The co-principal investigators are Dr. Allan Klein of
Cleveland Clinic and Dr. Massimo Imazio of the University of
Torino, Italy. For more information, refer to ClinicalTrials.gov
Identifier: NCT03737110.
About Recurrent Pericarditis
Recurrent 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 the
Rilonacept License Agreement with
RegeneronIn 2017, Regeneron granted Kiniksa an exclusive
license to develop and commercialize rilonacept worldwide, aside
from Israel, Egypt, Turkey and select countries in the Middle East
and North Africa. In the United States and Japan, Kiniksa’s license
is initially for all indications other than those involving local
administration to the eye or ear, oncology, deficiency of the
interleukin1 receptor antagonist (DIRA) and CAPS. If Kiniksa is
successful in receiving marketing approval for rilonacept in the
United States for a new indication, the scope of the license
granted to Kiniksa will automatically expand to include DIRA, if
approved, and CAPS in the United States and Japan, and Kiniksa will
assume the sales and distribution of rilonacept in these additional
indications. Outside the United States and Japan, Kiniksa’s license
is for all indications other than local application to the eye or
ear, oncology, CAPS, DIRA and certain periodic fever syndromes.
Kiniksa made an upfront payment of $5.0 million to Regeneron and is
obligated to make regulatory milestone payments of up to $27.5
million in the aggregate. Thereafter, Kiniksa and Regeneron will
evenly split profits on sales of rilonacept after deducting certain
commercialization expenses subject to specified limits.
About RilonaceptRilonacept is a weekly,
subcutaneously-injected, recombinant dimeric fusion protein that
blocks IL-1α and IL-1β signaling. 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 DIRA is currently under FDA review
following the submission of an 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
forward-looking 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: our
belief that rilonacept has the potential to become the first
FDA-approved therapy for recurrent pericarditis; our urgency in
bringing this potential treatment option to patients suffering from
recurrent pericarditis; our expectation regarding the timing of
payments to Regeneron in connection with regulatory milestones; and
our 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 to the
satisfaction of applicable regulatory authorities; the potential
for applicable regulatory authorities to delay or deny approval of
the sBLA for rilonacept in recurrent pericarditis; 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) 430-8289 mragosa@kiniksa.com
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