Reported $1.2 billion in preliminary* full-year
2023 global net product sales
Submitted sBLA to FDA for VYVGART® Hytrulo for
CIDP with priority review voucher (PRV); if approved, launch
expected mid-2024
Reported positive data from Phase 2 ARDA study
establishing proof-of-concept for empasiprubart in MMN
Data from six Phase 2 proof-of-concept trials
expected by end of 2024
Nominated four new pipeline candidates with IND
filings expected by end of 2025
January 8, 2024, 7:00 AM
CET
Amsterdam, the Netherlands –
argenx SE (Euronext & Nasdaq: ARGX), a global immunology
company committed to improving the lives of people suffering from
severe autoimmune diseases, today reported preliminary financial
results for the full-year 2023, including global net product sales
(inclusive of both VYVGART® and VYVGART Hytrulo), and announced its
strategic priorities for 2024.
“In 2023, we reached more than 6,000 patients
globally, making VYVGART available to gMG patients around the
world,” said Tim Van Hauwermeiren, Chief Executive Officer of
argenx. “We continued to invest in and demonstrate the
sustainability of our business by successfully launching our
subcutaneous VYVGART product, and are poised for continued
expansion in gMG and beyond in 2024. argenx is delivering on its
promise to transform how the world understands autoimmunity. It is
with this commitment in mind that we submitted our sBLA for VYVGART
Hytrulo in CIDP and, if approved, expect to launch in the U.S. in
mid-2024. We will continue to be aggressive in advancing our
pipeline this year and expect to report efgartigimod data from six
Phase 2 studies in 2024, and to further develop empasiprubart in
MMN. Through sustained investment in our IIP, we expect to see
repeat value creation, and plan to submit four new INDs by the end
of 2025.”
“Two years ago, argenx’s key goal was to launch
efgartigimod in the U.S. Today, we have built a formidable global
commercial organization with product approvals in over 30 countries
worldwide and a vibrant pipeline of promising new therapeutics to
address immune-mediated diseases. We have forged important
partnerships that support our mission to advance the human
understanding of immunology to best benefit patients. argenx is
well positioned for sustained growth throughout 2024 and well into
the future.”
2024 Strategic Priorities
argenx will focus on three strategic priorities
in 2024 to drive sustainable long-term growth, including:
- Reach more patients with
VYVGART by building upon its strong commercial foundation
to address ongoing unmet patient need, broaden the MG opportunity,
and expand into CIDP
- Advance its extensive
pipeline through new data readouts, creating multiple
opportunities to demonstrate transformative clinical benefit
- Leverage its repeatable
innovation engine, driving pipeline growth through its
Immunology Innovation Program
Reach More Patients with
VYVGART
VYVGART (efgartigimod alfa fcab) is a
first-in-class antibody fragment targeting the neonatal Fc receptor
(FcRn) and is now approved in more than 30 countries globally.
VYVGART subcutaneous (SC) (efgartigimod alfa and
hyaluronidase-qvfc) is approved in the U.S. (as VYVGART Hytrulo)
and Europe, making VYVGART the only gMG treatment available as both
an IV and simple SC injection. argenx is planning to reach more
patients commercially in 2024 through its multi-dimensional
expansion efforts. argenx will work to reach patients earlier in
the MG treatment paradigm and improve the lives of new MG patient
populations through additional global regulatory approvals, and the
expansion of uses to treat additional autoimmune indications.
- Regulatory approval decisions of
VYVGART for gMG expected in Switzerland, Australia, Saudi Arabia
and South Korea by end of 2024
- Through strategic collaboration
with Zai Lab, VYVGART to be included on China’s 2023 National
Reimbursement Drug List (NRDL), starting in January 2024
- Decision on approval of VYVGART SC
for gMG in Japan expected in first quarter of 2024 and in China
through Zai Lab by end of 2024
- Decision on approval of VYVGART for
primary immune thrombocytopenia (ITP) in Japan expected in first
quarter of 2024
- Supplemental Biologics License
Application (sBLA) submitted to FDA for VYVGART Hytrulo for CIDP
with priority review voucher (PRV); if approved, launch expected
mid-2024
- Regulatory submissions of VYVGART
SC for CIDP in Japan, Europe, China and Canada expected in
2024
- Registrational studies to expand
VYVGART label into broader MG populations, including in
seronegative patients, to start in 2024
- Update on pre-filled syringe
development expected in first half of 2024; ongoing studies to
support potential approval in gMG and CIDP in 2024
Advance Current Pipeline through
Upcoming Data Readouts
argenx continues to demonstrate breadth and
depth within its immunology pipeline and is advancing multiple
pipeline-in-a-product candidates. With efgartigimod, argenx is
solidifying its leadership in FcRn and is on track to be approved
or in development in 15 autoimmune indications by 2025. Beyond
efgartigimod, argenx is advancing its earlier stage pipeline
programs, including empasiprubart (C2 inhibitor) with Phase 2
studies ongoing in multifocal motor neuropathy (MMN), delayed graft
function and dermatomyositis (DM). In addition, ARGX-119, a
muscle-specific kinase (MuSK) agonist, will initiate Phase 1b/2a
studies in congenital myasthenic syndrome and amyotrophic lateral
sclerosis in 2024.
Today, argenx reported positive clinical data
from the first cohort of the Phase 2 ARDA study of empasiprubart,
establishing proof-of-concept in MMN. After confirming IVIg
dependence, 27 patients were withdrawn from IVIg treatment and
randomized 2:1 to either empasiprubart or placebo for 16 weeks.
Patients were monitored for clinical deterioration that required
IVIg retreatment, which was the main efficacy endpoint of the
study.
- Empasiprubart demonstrated a 91%
reduction in the need for IVIg rescue compared to placebo [HR: 0.09
95% CI (0.02; 0.044)]
- According to the Patient Global
Impression of Change scale, 94% (17/18) of empasiprubart-treated
patients rated their condition as improved since study start,
including 55% (10/18) who were much or very much improved. Of
placebo patients, 89% (8/9) worsened or had no change.
- Empasiprubart demonstrated
improvement on all six efficacy measurements compared to
baseline
- Safety and tolerability profile
were consistent with Phase 1 results
- Cohort 2 is ongoing to determine
dose response ahead of a Phase 3 study start
argenx is on track to report topline data from
five additional proof-of-concept studies in 2024, including:
- Phase 2 RHO study evaluating
efgartigimod in primary Sjogren’s syndrome expected in first half
of 2024
- Phase 2 ALPHA study evaluating
efgartigimod in post-COVID-19 postural orthostatic tachycardia
syndrome (PC-POTS) expected in first half of 2024
- Seamless Phase 2/3 ALKIVIA study
evaluating efgartigimod across three myositis subsets
(immune-mediated necrotizing myopathy (IMNM), antisynthetase
syndrome (ASyS), and DM) expected in the second half of 2024
Leverage Repeatable Innovation Playbook
to Drive Long-Term Pipeline Growth
argenx continues to invest in its discovery
engine, the Immunology Innovation Program (IIP), to drive long-term
sustainable pipeline growth. Through the IIP, four new pipeline
candidates have been nominated, including: ARGX-213 targeting FcRn
and further solidifying argenx’s leadership in this new class of
medicine; ARGX-121 and ARGX-220, which are first-in-class targets
broadening argenx’s focus across the immune system; and ARGX-109,
targeting IL-6, which plays an important role in inflammation.
Preclinical work is ongoing in each candidate and argenx is on
track to file four investigational new drug (IND) applications by
the end of 2025.
Preliminary* Fourth Quarter and
Full-Year 2023 Financial Results
Today, argenx also announced preliminary* global
net VYVGART revenues for the fourth quarter and full-year 2023 of
approximately $374 million and $1.2 billion, respectively.
As of December 31, 2023, argenx had
approximately $3.2 billion in cash, cash equivalents and current
financial assets*. Based on its current operating plans, argenx
expects its combined R&D and SG&A expenses in 2024 to be
less than $2 billion. The projected 2024 cash burn will be
approximately $500 million. argenx expects its existing cash, cash
equivalents and current financial assets, together with anticipated
future product revenues, to fund the Company to profitability.
* - The preliminary selected financial results
are unaudited, subject to adjustment, and provided as an
approximation in advance of the Company’s announcement of complete
financial results in February 2024.
42nd
Annual J.P. Morgan Healthcare Conference Presentation and
Webcast
Mr. Van Hauwermeiren will highlight these
updates in a corporate presentation at the 42nd Annual J.P. Morgan
Healthcare Conference today, Monday, January 8, 2024, at 9:00 a.m.
PT. The live webcast of the presentation may be accessed under
Investors on the argenx website. A replay will be available for 30
days following the presentation.
Phase 2 ARDA Study Design
The Phase 2 ARDA study is a randomized,
double-blinded, placebo-controlled multicenter study to evaluate
the safety and tolerability, efficacy, pharmacokinetics,
pharmacodynamics, and immunogenicity of two dose regimens of
empasiprubart in adults with multifocal motor neuropathy (MMN). The
study consists of an IVIg dependency and monitoring period and two
16-week treatment cohorts of 24 MMN patients receiving
empasiprubart or placebo in a 2x1 randomization. The dosing for
Cohort 2 was established after a planned interim analysis of the
first nine patients to complete the 16-week treatment period from
Cohort 1. The primary endpoint is safety and tolerability.
Additional endpoints include time to IVIg retreatment, biomarker
analyses of C2 levels, and changes in measurements on key
functional scores (modified medical research council (mMRC)-10 sum
score, grip strength, MMN-RODS) as well as several patient-reported
quality of life outcome measures (fatigue severity score (FSS),
chronic acquired polyneuropathy patient-reported index (CAP-PRI),
and values of the patient global impression change (PGIC)
scale).
About Multifocal Motor
Neuropathy
Multifocal motor neuropathy (MMN) is a rare,
chronic autoimmune disease of the peripheral nervous system. The
disease is characterized by slowly progressive, asymmetric muscle
weakness mainly of the hands, forearms and lower legs. MMN is often
associated with anti-GM1 IgM autoimmunity, leading to activation of
the classical complement pathway, driving subsequent axon damage.
High-dose IVIg is the only approved treatment for MMN and patients
typically experience disease progression despite therapy,
indicating an unmet need for efficacious and better tolerated
therapeutic options.
About Empasiprubart
Empasiprubart (ARGX-117) is a first-in-class
humanized sweeping antibody that binds specifically to C2 thereby
blocking both the classical and lectin pathways of the complement
cascade. By blocking upstream complement activity, empasiprubart
has the potential to reduce tissue inflammation representing a
broad pipeline opportunity across multiple severe autoimmune
indications. In addition to multifocal motor neuropathy, argenx is
evaluating empasiprubart in delayed graft function following kidney
transplant and dermatomyositis.
About VYVGART and VYVGART
SC
VYVGART is a human IgG1 antibody fragment that
binds to the neonatal Fc receptor (FcRn), resulting in the
reduction of circulating IgG autoantibodies. It is the first
approved FcRn blocker globally for the treatment of adults with
generalized myasthenia gravis (gMG) who are anti-acetylcholine
receptor (AChR) antibody positive and in Japan for the treatment of
adults with gMG who do not have sufficient response to steroids or
non-steroidal immunosuppressive therapies (ISTs).
VYVGART SC is a subcutaneous combination of
efgartigimod alfa and recombinant human hyaluronidase PH20
(rHuPH20), Halozyme’s ENHANZE® drug delivery technology to
facilitate subcutaneous injection delivery of biologics. It is
marketed as VYVGART Hytrulo in the U.S. and VYVGART SC in Europe,
and may be marketed under different proprietary names following
approval in other regions.
About argenx
argenx is a global immunology company committed
to improving the lives of people suffering from severe autoimmune
diseases. Partnering with leading academic researchers through its
Immunology Innovation Program (IIP), argenx aims to translate
immunology breakthroughs into a world-class portfolio of novel
antibody-based medicines. argenx developed and is commercializing
the first approved neonatal Fc receptor (FcRn) blocker, globally in
the U.S., Japan, Israel, the EU, the UK, China and Canada. The
Company is evaluating efgartigimod in multiple serious autoimmune
diseases and advancing several earlier stage experimental medicines
within its therapeutic franchises. For more information, visit
www.argenx.com and follow us on LinkedIn, Twitter, and
Instagram.
Media:
Ben Petokbpetok@argenx.com
Investors:
Alexandra Roy (US)aroy@argenx.com
Lynn Elton (EU)lelton@argenx.com
Preliminary Financial
Results
The financial results presented in this press
release are preliminary, estimated, and unaudited. They are subject
to the completion and finalization of argenx’s financial and
accounting closing procedures. They reflect management’s estimates
based solely upon information available to management as of the
date of this press release. Further information learned during that
completion and finalization may alter the final results. In
addition, the preliminary estimates should not be viewed as a
substitute for full quarterly and annual financial statements
prepared in accordance with IFRS. There is a possibility that
argenx’s financial results for the quarter ended December 31, 2023,
and full year financial results for 2023 could vary materially from
these preliminary estimates. In addition to the completion of the
financial closing procedures, factors that could cause actual
results to differ from those described above are set forth below.
Accordingly, you should not place undue reliance upon this
preliminary information.
Additional information regarding the Company’s
fourth quarter 2023 financial results and full year financial
results for 2023 will be available in the Company’s annual report
and Form 20-F, which will be filed with the Netherlands Authority
for the Financial Markets and U.S. Securities and Exchange
Commission (the “SEC”), respectively.
Forward Looking Statements
The contents of this announcement include
statements that are, or may be deemed to be, “forward-looking
statements.” These forward-looking statements can be identified by
the use of forward-looking terminology, including the terms
“plans,” “aims,” “believes,” “continues,” “hope,” “estimates,”
“preliminary,” “anticipates,” “expects,” “intends,” “may,” “will,”
“should,” or “commitment” and include statements argenx makes
concerning its preliminary financial results for the full year
2023; its expansion efforts, including reaching more patients with
VYVGART within the MG treatment paradigm, through geographic
expansion and into new autoimmune indications, expanding into CIDP,
and the anticipated development of empasiprubart and ARGX-119; the
anticipated timing of its launch of SC efgartigimod for CIDP in the
U.S.; the initiation, timing, progress and results of its
anticipated clinical development, data readouts and regulatory
milestones and plans; its strategic priorities, including the
timing and outcome of regulatory filings and regulatory approvals;
its expectations of future profitability; the potential of its
innovative clinical programs; and the nomination of new development
candidates. By their nature, forward-looking statements involve
risks and uncertainties and readers are cautioned that any such
forward-looking statements are not guarantees of future
performance. argenx’s actual results may differ materially from
those predicted by the forward-looking statements as a result of
various important factors, including but not limited to argenx’s
ability to successfully execute its business and growth strategies,
the inherent uncertainties associated with development of novel
drug therapies, preclinical and clinical trial and product
development activities and regulatory approval requirements, the
acceptance of our products and product candidates by our patients
as safe, effective and cost-effective, volatile market conditions,
and the impact of governmental laws and regulations on our
business. A further list and description of these risks,
uncertainties and other risks can be found in argenx’s U.S.
Securities and Exchange Commission (SEC) filings and reports,
including in argenx’s most recent annual report on Form 20-F filed
with the SEC as well as subsequent filings and reports filed by
argenx with the SEC. Given these uncertainties, the reader is
advised not to place any undue reliance on such forward-looking
statements. These forward-looking statements speak only as of the
date of publication of this document. argenx undertakes no
obligation to publicly update or revise the information in this
press release, including any forward-looking statements, except as
may be required by law.
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