Full Press Release Details
argenx Highlights 2024 Strategic Priorities
billion in preliminary* full-year 2023 global net product sales
sBLA to FDA for VYVGART Hytrulo for CIDP with priority
review voucher (PRV); if approved, launch expected mid-2024
data from Phase 2 ARDA study establishing proof-of-concept for empasiprubart in MMN
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
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.
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
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.
Advance Current Pipeline through Upcoming Data
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.
argenx is on track to report topline data from
five additional proof-of-concept studies in 2024, including:
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.
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.
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
VYVGART SC is a subcutaneous combination of efgartigimod