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 reported fourth quarter and full-year 2020 financial results
and corporate and pipeline activity.
“Kiniksa delivered outstanding performance in 2020, with
encouraging clinical data across our pipeline as well as
Breakthrough Therapy designation or Orphan Drug designation for
rilonacept, mavrilimumab and vixarelimab,” said Sanj K. Patel,
Chief Executive Officer and Chairman of the Board of Kiniksa.
“Building on this momentum, we expect to launch rilonacept in
recurrent pericarditis, pending FDA approval, in the first half of
2021. We also anticipate final Phase 1 data for KPL-404, our
anti-CD40 program, in which data generated to-date support the
best-in-class potential of this molecule and the opportunity for
its evaluation in multiple devastating autoimmune diseases.
Further, we expect data from the fully-enrolled mavrilimumab Phase
2 clinical trial in severe COVID-19 in the first half of this year.
With these significant advances, it is important to highlight we
are well capitalized with cash reserves of approximately $323
million.”
Pipeline ActivityRilonacept (IL-1α and
IL-1β cytokine trap)
- Kiniksa is preparing for the commercial launch of rilonacept in
recurrent pericarditis in the first half of 2021, if approved by
the U.S. Food and Drug Administration (FDA).
- The FDA accepted the supplemental Biologics License Application
(sBLA) for rilonacept in recurrent pericarditis with priority
review and assigned a Prescription Drug User Fee Act (PDUFA) goal
date of March 21, 2021.
- Upon approval in recurrent pericarditis, Kiniksa will commence
sales and distribution of rilonacept for the approved indications
in the United States, including cryopyrin-associated periodic
syndromes (CAPS), and deficiency of IL-1 receptor antagonist
(DIRA), and evenly split profits with Regeneron
Pharmaceuticals, Inc. (Regeneron).
- Kiniksa is building commercial competencies in-house, including
sales operations, value and access, and sales and marketing teams.
In addition, the company is generating evidence on unmet need and
disease burden, building disease awareness with payers, physicians,
and advocacy groups, and establishing core capabilities such as
distribution, patient services and data management.
- Kiniksa achieved a regulatory milestone in the fourth quarter
of 2020, which triggered a $7.5 million payment to Regeneron. The
company is obligated to make a regulatory milestone payment of $20
million through the potential approval of rilonacept in recurrent
pericarditis.
Mavrilimumab (monoclonal antibody inhibitor targeting
GM-CSFRα)
- Kiniksa expects to provide next steps for mavrilimumab,
including for giant cell arteritis (GCA), in the first half of
2021.
- Kiniksa is conducting a Phase 2/3 clinical trial of
mavrilimumab in severe COVID-19 pneumonia and hyperinflammation.
- Kiniksa completed enrollment in the Phase 2 portion of the
clinical trial and expects data in the first half of 2021.
- Kiniksa is enrolling patients in the Phase 3 portion of the
clinical trial.
Vixarelimab (monoclonal antibody inhibitor of signaling
through OSMRβ)
- Kiniksa is conducting a Phase 2b dose-ranging clinical trial of
vixarelimab in prurigo nodularis.
- The Phase 2b clinical trial is expected to enroll approximately
180 patients experiencing severe pruritus. Patients will be
randomized to receive vixarelimab or placebo subcutaneously
once-monthly.
- The primary efficacy endpoint is the percent change from
baseline in the weekly-average Worst-Itch Numeric Rating Scale
(WI-NRS) at Week 16.
- Key secondary efficacy endpoints include the proportion of
patients achieving a greater-than-or-equal-to 4-point
weekly-average WI-NRS reduction at Week 16 and the proportion of
patients achieving a 0/1 score (clear/almost clear) on the prurigo
nodularis-investigator’s global assessment (PN-IGA) at Week
16.
KPL-404 (monoclonal antibody inhibitor of signaling
between CD40 and CD40L)
- Kiniksa expects final data and safety follow-up from all
cohorts of the single-ascending-dose Phase 1 clinical trial of
KPL-404 in the first half of 2021.
- Preliminary Phase 1 data showed full receptor occupancy through
Day 29 at the 3 mg/kg intravenous dose.
- This data corresponded with complete suppression of the T-cell
Dependent Antibody Response (TDAR) to the novel antigen keyhole
limpet hemocyanin (KLH) through Day 29.
- KPL-404 is a monoclonal antibody inhibitor designed to disrupt
CD40-CD40 ligand (CD40L) signaling, a well-known pathway that plays
a critical role in regulating B cell proliferation and T cell
activation as well as antibody production.
- Dysregulation of the CD40-CD40L pathway has been implicated in
a broad range of autoimmune diseases including rheumatoid
arthritis, Sjogren’s syndrome, Graves’ disease, systemic lupus
erythematosus and the prevention of solid organ transplant graft
rejection.
Financial Results
- Net loss for the fourth quarter of 2020 was $53.7 million,
compared to a net loss of $31.8 million for the fourth quarter of
2019. Net loss for the full-year 2020 was $161.4 million, compared
to a net loss of $161.9 million for the full-year 2019.
- Total operating expenses for the fourth quarter of 2020 were
$52.9 million, compared to $32.6 million for the fourth quarter of
2019. Total operating expenses for the full-year 2020 were $157.4
million, compared to $170.0 million for the full-year 2019.
- Non-cash, share-based compensation expense for the fourth
quarter of 2020 was $6.3 million, compared to $5.0 million for the
fourth quarter of 2019. Non-cash, share-based compensation expense
for the full-year 2020 was $20.9 million, compared to $15.1 million
for the full-year 2019.
- Total operating expenses for the fourth quarter and full-year
2020 included a $7.5 million payment made to Regeneron
related to our achievement of a regulatory milestone.
- As of December 31, 2020, the
company had cash, cash equivalents and short-term investments of
$323.5 million and no debt.
Financial Guidance
- Kiniksa expects that its cash, cash
equivalents and short-term investments will fund its current
operating plan into 2023.
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 achieved prospectively-defined primary
endpoints of efficacy and safety in over 550 patients with
rheumatoid arthritis through Phase 2b clinical studies
in Europe. 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-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 clinical data across our
pipeline along with breakthrough therapy and/or orphan drug
designations drove our performance in 2020; the potential
commercial launch of rilonacept in recurrent pericarditis in the
first half of the year, if approved by the FDA, and our plans to
commence sales and distribution of rilonacept for all other
approved indications in the United States at that time; expected
timing of data from clinical trials, including expected data from
the Phase 2 portion of the Phase 2/3 clinical trial of mavrilimumab
in severe COVID-19 pneumonia and hyperinflammation in the first
half of 2021 and final data and safety follow-up from all cohorts
of the single-ascending-dose Phase 1 clinical trial of KPL-404 in
healthy volunteers in the first half of 2021; expected timing of
next steps for mavrilimumab, including for giant cell arteritis
(GCA), 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 and KPL-404 potentially being a best-in-class
monoclonal antibody inhibitor; our belief 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;
potential undesirable side effects caused by our product
candidates; our inability to demonstrate safety and efficacy to the
satisfaction of applicable regulatory authorities ; potential for
applicable regulatory authorities to not accept our BLA filings or
to delay or deny approval of any of our product candidates,
including rilonacept, or to require additional trials to support
any such approval; delays, difficulty or inability to build
commercial competencies in-house and/or execute on commercial
launch activities as planned for rilonacept; 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
KINIKSA
PHARMACEUTICALS, LTD. |
CONSOLIDATED
STATEMENTS OF OPERATIONS |
(In
thousands, except share and per share amounts) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months
Ended |
|
Years
Ended |
|
|
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
|
|
|
|
|
|
2020 |
|
|
2019 |
|
|
2020 |
|
|
2019 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
37,398 |
|
|
$ |
22,886 |
|
|
$ |
112,042 |
|
|
$ |
135,001 |
|
|
|
Selling, general and administrative |
|
|
15,500 |
|
|
|
9,695 |
|
|
|
45,321 |
|
|
|
34,962 |
|
|
|
|
|
Total operating expenses |
|
|
52,898 |
|
|
|
32,581 |
|
|
|
157,363 |
|
|
|
169,963 |
|
Loss from operations |
|
|
(52,898 |
) |
|
|
(32,581 |
) |
|
|
(157,363 |
) |
|
|
(169,963 |
) |
Interest income |
|
|
30 |
|
|
|
1,130 |
|
|
|
1,134 |
|
|
|
6,049 |
|
Loss before (provision) benefit for income taxes |
|
|
(52,868 |
) |
|
|
(31,451 |
) |
|
|
(156,229 |
) |
|
|
(163,914 |
) |
(Provision) benefit for income taxes |
|
|
(789 |
) |
|
|
(346 |
) |
|
|
(5,152 |
) |
|
|
2,047 |
|
Net loss |
|
|
|
$ |
(53,657 |
) |
|
$ |
(31,797 |
) |
|
$ |
(161,381 |
) |
|
$ |
(161,867 |
) |
Net loss per share attributable to common shareholders —basic and
diluted |
|
$ |
(0.79 |
) |
|
$ |
(0.58 |
) |
|
$ |
(2.61 |
) |
|
$ |
(2.99 |
) |
Weighted average common shares outstanding—basic and diluted |
|
|
68,062,007 |
|
|
|
54,887,689 |
|
|
|
61,842,722 |
|
|
|
54,049,477 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
KINIKSA
PHARMACEUTICALS, LTD. |
|
SELECTED
CONSOLIDATED BALANCE SHEET DATA |
|
(In
thousands) |
|
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
As of |
|
|
|
|
|
|
|
|
|
|
December
31, |
|
December
31, |
|
|
|
|
|
|
|
|
|
|
2020 |
|
2019 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Cash, cash equivalents, and short-term investments |
|
$ |
323,482 |
|
|
$ |
233,380 |
|
|
|
Working capital |
|
|
301,403 |
|
|
|
213,797 |
|
|
|
Total assets |
|
|
|
349,464 |
|
|
|
254,534 |
|
|
|
Accumulated deficit |
|
|
(517,473 |
) |
|
|
(356,092 |
) |
|
|
Total shareholders' equity |
|
|
311,935 |
|
|
|
225,423 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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