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 highlighted its corporate priorities and expected 2021
milestones. Sanj K. Patel, Chief Executive Officer and Chairman of
the Board of Kiniksa will provide further detail in a corporate
presentation at the Virtual 39th Annual J.P. Morgan Healthcare
Conference today, Monday, January 11, 2021 at 4:30 p.m. Eastern
Time.
“2020 was marked by significant progress across our entire
pipeline, setting the stage to build long-term value across our
portfolio,” said Sanj K. Patel, Chief Executive Officer and
Chairman of the Board of Kiniksa. “Moving forward, 2021 has the
potential to be a transformational year for Kiniksa with multiple
catalysts expected across our pipeline, notably with the potential
commercial launch of rilonacept in recurrent pericarditis in the
first half of the year. As we focus on our launch readiness
preparations, our commitment to bringing novel therapies to
patients with unmet need remains at the core of our goals.”
Expected 2021 Milestones
Rilonacept (IL-1α and IL-1β cytokine trap)
- Kiniksa’s Prescription Drug User
Fee Act (PDUFA) goal date for rilonacept in recurrent pericarditis
is March 21, 2021, as assigned by the U.S. Food and Drug
Administration (FDA) upon the acceptance of the supplemental
Biologics License Application (sBLA), with priority review. If
approved by the FDA, Kiniksa expects the potential commercial
launch of rilonacept in recurrent pericarditis in the first half of
2021 and consequently would evenly split profits on sales of all
approved indications in the United States, including
cryopyrin-associated periodic syndromes (CAPS) and deficiency of
IL-1 receptor antagonist (DIRA), with Regeneron
Pharmaceuticals, Inc. (Regeneron).
Mavrilimumab (monoclonal antibody inhibitor targeting
GM-CSFRα)
- Kiniksa plans to provide next steps for the development of
mavrilimumab, including for giant cell arteritis (GCA), in the
first half of 2021.
- Kiniksa is conducting a Phase 2/3 clinical trial in severe
COVID-19 pneumonia and hyperinflammation. The company expects to
provide data from the Phase 2 portion of the trial in the first
half of 2021.
Vixarelimab (monoclonal antibody inhibitor of signaling
through OSMRβ)
- Kiniksa is conducting a Phase 2b dose-ranging trial of
vixarelimab in prurigo nodularis. The Phase 2b trial is expected to
enroll approximately 180 patients experiencing severe pruritus.
Patients will be randomized to receive vixarelimab or placebo
subcutaneously (SC) once-monthly.
KPL-404 (monoclonal antibody inhibitor of signaling
between CD40 and CD40L)
- Full receptor occupancy through Day 29 shown in preliminary
Phase 1 data from the single-ascending dose clinical trial of
KPL-404 in healthy volunteers at the 3 mg/kg intravenous (IV) dose.
This corresponded with complete suppression of the T-cell Dependent
Antibody Response (TDAR) to keyhole limpet hemocyanin (KLH) through
Day 29.
- The data to-date support further evaluation in patients,
including potential monthly IV or SC administration. Kiniksa
expects final data and safety follow-up from all cohorts in the
first half of 2021.
Financial GuidanceKiniksa ended 2020 with
approximately $323 million in cash, cash equivalents and short-term
investments (unaudited). The company expects that these cash
reserves will fund its current operating plan into 2023.
Presentation at the Virtual
39th Annual J.P. Morgan
Healthcare ConferenceKiniksa will webcast its corporate
presentation at the Virtual 39th Annual J.P. Morgan Healthcare
Conference today, Monday, January 11, 2021 at 4:30 p.m. Eastern
Time. A live webcast of Kiniksa’s presentation will be accessible
through the Investors & Media section of the company’s website
(www.kiniksa.com). A replay of the webcast will be available on
Kiniksa’s website for 14 days following the conference.
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 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.
About RilonaceptRilonacept is a weekly,
subcutaneously-injected, recombinant dimeric fusion protein that
blocks interleukin-1 alpha (IL-1α) and interleukin-1 beta (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, and DIRA. 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 MavrilimumabMavrilimumab is an
investigational fully-human monoclonal antibody that targets
granulocyte macrophage colony stimulating factor receptor alpha
(GM-CSFRα). Mavrilimumab was dosed in over 550 patients with
rheumatoid arthritis through Phase 2b clinical studies
in Europe and achieved prospectively-defined primary
endpoints of efficacy and safety. Kiniksa’s lead indication for
mavrilimumab is GCA, a rare inflammatory disease of medium-to-large
arteries. Kiniksa is also evaluating mavrilimumab in COVID-19
pneumonia and hyperinflammation. The FDA granted Orphan Drug
designation to mavrilimumab for the treatment of GCA in 2020.
About VixarelimabVixarelimab is an
investigational fully-human monoclonal antibody that targets
oncostatin M receptor beta (OSMRβ), which mediates signaling of
interleukin-31 (IL-31) and oncostatin M (OSM), two key cytokines
implicated in pruritus, inflammation and fibrosis. Kiniksa believes
vixarelimab to be the only monoclonal antibody in development that
targets both pathways simultaneously. Kiniksa’s lead indication for
vixarelimab is prurigo nodularis, a chronic inflammatory skin
condition characterized by severely pruritic skin nodules. The FDA
granted Breakthrough Therapy designation to vixarelimab for the
treatment of pruritus associated with prurigo nodularis in
2020.
About KPL-404KPL-404 is an investigational
humanized monoclonal antibody that is designed to inhibit CD40-CD40
ligand (CD40L) interaction, a key T-cell co-stimulatory signal
critical for B-cell maturation and immunoglobulin class switching
and Type 1 immune responses. Kiniksa believes disrupting the
CD40-CD40L interaction is an attractive approach for multiple
autoimmune disease pathologies such as rheumatoid arthritis,
Sjogren’s syndrome, Graves’ disease, systemic lupus erythematosus
and solid organ transplant. Kiniksa owns or controls the
intellectual property related to KPL-404.
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 progress made across our
pipeline in 2020, has set the stage to build long-term value across
our portfolio; our belief that 2021 has the potential to be a
transformational year for Kiniksa; expected multiple catalysts
across our pipeline in 2021; the potential commercial launch of
rilonacept in recurrent pericarditis in the first half of the year,
if approved by the FDA; expected timing of data from clinical
trials, including expected data from the Phase 2 portion of the
adaptive design Phase 2/3 clinical trial of mavrilimumab in severe
COVID-19 pneumonia and hyperinflammation in the first half of 2021,
next steps for the development of mavrilimumab, including for giant
cell arteritis (GCA), in the first half of 2021, and final data
from the single-ascending-dose Phase 1 clinical trial of KPL-404 in
healthy volunteers in the first half of 2021; our belief that
KPL-404 has the potential to address a broad range of autoimmune
diseases; our beliefs about the mechanisms of action of our product
candidates and potential impact of their approach, including our
beliefs that vixarelimab is the only monoclonal antibody in
development that targets both interleukin-31 (IL-31) and oncostatin
M (OSM) pathways simultaneously; that KPL-404’s disruption of the
CD40-CD40L interaction is an attractive approach for multiple
autoimmune disease pathologies; our belief that all of our product
candidates offer the potential for differentiation; and expectation
about our cash reserves funding our current operating plan into
2023.
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: delays or difficulty in enrollment of patients in, and
activation or continuation of sites for, our clinical trials;
amendments to our clinical trial protocols initiated by us or
required by regulatory authorities; delays or difficulty in
completing our clinical trials, including as a result of the
COVID-19 pandemic; potential for changes between final data and any
preliminary, interim, top-line or other data from clinical trials
conducted by us or third parties; our inability to replicate in
later clinical trials the positive final data from our earlier
clinical trials or studies; impact of additional data from us or
other companies, including the potential for our data to produce
negative, inconclusive or commercially uncompetitive results;
impact of additional data from us or other companies; potential
undesirable side effects caused by our product candidates; our
inability to demonstrate safety and efficacy to the satisfaction of
applicable regulatory authorities or otherwise producing negative,
inconclusive or commercially uncompetitive results; potential for
applicable regulatory authorities to not accept our BLA or sBLA
filings or to delay or deny approval of any of our product
candidates or to require additional trials to support any such
approval; our reliance on third parties as the sole source of
supply of the drug substance and drug products used in our product
candidates and to manufacture our product candidates; drug
substance and/or drug product shortages; our reliance on third
parties to conduct research, clinical trials, and/or certain
regulatory activities for our product candidates; complications in
coordinating requirements, regulations and guidelines of regulatory
authorities across jurisdictions for our clinical trials; the
impact of the COVID-19 pandemic and measures taken in response to
the pandemic on our business and operations as well as the business
and operations of our manufacturers, CROs upon whom we rely to
conduct our clinical trials, and other third parties with whom we
conduct business or otherwise engage, including the FDA and other
regulatory authorities; changes in our operating plan and funding
requirements; and existing or new competition.
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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