UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM 6-K
REPORT OF FOREIGN PRIVATE ISSUER
PURSUANT TO RULE 13a-16 OR 15d-16
UNDER THE SECURITIES EXCHANGE ACT OF 1934
For the Month of January 2024
Commission File Number: 001-38097
ARGENX SE
(Translation of registrant’s name into English)
Laarderhoogtweg 25
1101 EB Amsterdam, the Netherlands
(Address of principal executive offices)
Indicate by check mark whether the registrant files or will file annual
reports under cover of Form 20-F or Form 40-F.
Form 20-F x
Form 40-F ¨
Indicate
by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1): ¨
Indicate
by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7): ¨
EXPLANATORY NOTE
On January 8, 2024, argenx SE (the “Company”)
issued a press release and a presentation the Company intends to use at the 42nd Annual J.P. Morgan Healthcare Conference on
January 8, 2024 at 9:00 a.m. PT, copies of which are attached hereto as Exhibits 99.1 and 99.2, respectively, and are incorporated
by reference herein.
The
information contained in this Current Report on Form 6-K, including Exhibit 99.1 and Exhibit 99.2, shall be deemed to
be incorporated by reference into the Company’s Registration Statements on Forms F-3
(File No. 333-258251) and S-8 (File Nos. 333-225375, 333-258253,
and 333-274721), and to be part thereof from the date on which this Current Report on Form 6-K is filed, to the extent not superseded
by documents or reports subsequently filed or furnished.
SIGNATURES
Pursuant to the requirements of the Securities
Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| |
ARGENX SE |
| |
|
|
| Date: January 8, 2024 |
By: |
/s/ Hemamalini (Malini)
Moorthy |
| |
|
Name: Hemamalini (Malini) Moorthy |
| |
|
Title: General Counsel |
Exhibit 99.1
argenx Highlights 2024 Strategic Priorities
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 Petok
bpetok@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.
Exhibit 99.2
| argenx
The Next Chapter
JP MORGAN HEALTHCARE CONFERENCE
JANUARY 8, 2024
1 |
| Forward Looking Statements
This presentation has been prepared by argenx SE (“argenx” or the “company”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be
construed as, a recommendation, promise or representation by the presenter or the company or any director, employee, agent, or adviser of the company. This presentation does not purport to be all-inclusive or to contain all of the information you may desire. Certain information contained in this presentation relates to or is based on studies, publications, surveys and other data obtained from
third-party sources and the company’s own internal estimates and research. While argenx believes these third-party studies, publications, surveys and other data to be reliable as of the date of this
presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition,
no independent source has evaluated the reasonableness or accuracy of argenx’s internal estimates or research and no reliance should be made on any information or statements made in this
presentation relating to or based on such internal estimates and research.
The financial results presented in this presentation 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 presentation. 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.
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 for innovation of its clinical programs; its pipeline; 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 ability of our clinical trials to reach their
endpoints, the ability to maintain, expand, and deliver on our pipeline; 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 presentation, including any forward-looking statements, except as may be required by law.
This presentation contains trademarks, trade names and service marks of other companies, which are the property of their respective owners.
2 |
| On a Journey to Transform Autoimmunity
Pioneering
novel target
biology
Leading
antibody
engineering
capabilities
Pipeline-in-a-product
opportunities
Creating optionality across and within molecules
3 |
| We are Hearing
Transformational
Stories with VYVGART
4
Mike, VYVGART Patient |
| PFS in development
15 indications
in development by 2025
ITP MAA filed
CIDP sBLA submitted
ARGX-109 $1.2B in gMG revenue in 2023
(Anti-IL-6)
ARGX-213
(Anti-FcRn)
ARGX-220
ARGX-121
Empasiprubart
POC established in MMN
Trials in DGF and DM
ARGX-119
Phase 1b/2a trials in
CMS and ALS
Our Innovation Horizons
5 |
| PFS in development
15 indications
in development by 2025
ITP MAA filed
CIDP sBLA submitted
ARGX-109 $1.2B in gMG revenue in 2023
(Anti-IL-6)
ARGX-213
(Anti-FcRn)
ARGX-220
ARGX-121
Empasiprubart
POC established in MMN
Trials in DGF and DM
ARGX-119
Phase 1b/2a trials in
CMS and ALS
VYVGART Opportunity Horizon
6 |
| Leadership in FcRn
Pioneering
FcRn
Unique
modulation
of FcRn
15 indications
by 2025*
Fc fragment and proprietary
ABDEG™ mutations
Transformational data
in gMG and CIDP
Generating key
learnings
*Indications in development 7 |
| Estimated 4,000 patient years of safety follow-up
between clinical trial and real-world experience
45% MSE
QoL comparable
to healthy
population*
78%
MG-ADL ≤4**
Meaningful
steroid
tapering by
at least
5mg/day
within first
6 months
Superior
cost/benefit
over IVIg***
Enables
significantly
faster
access to
treatment
VYVGART is Setting New Expectations in gMG
* Real world evidence
**Source: ADAPT and ADAPT+ clinical trial data
***Leading Health Technology Assessment agency
8 |
| 25% YoY increase
>2,300**
Prescribers in the US
PRESCRIBER EXPANSION
Strong Commercial Execution
21% 2023 CAGR
$1.2B*
Global Product Revenue
GROWTH
2023
Performance
Favorable payor policies
~90%
Access VYVGART after ≤2 Orals
BROAD PATIENT ACCESS
55% patients from orals
>6,000**
Global VYVGART Patients
EARLIER LINE PATIENTS
* Preliminary Financials. Unaudited and subject to change
** As of Q3 2023 Financial Results
9 |
| Innovation Builds Autoimmune Market Opportunities
10
17,000
6,000*
Growing
MG Market
gMG Patients
on VYVGART
Current
VYVGART TAM
Expanding
VYVGART TAM
Growing
VYVGART Share
US RoW argenx markets (exc. China) Global
31,000
Label-enabling trials in broader gMG populations
Phase 3b studies and externally sponsored research
Geographic expansion, including South Korea and Australia
Growing VYVGART share
US: VYVGART Hytrulo J-Code in effect; field force expansion
PFS development
Addition to China NRDL
Expanding VYVGART TAM
Growing MG market
Targeted biologics are expanding gMG market by providing
patients more treatment options
*As of Q3 2023 Financial Results Based on argenx market research |
| VYVGART Has Potential to Transform CIDP
11
67% Response rate demonstrates IgG
autoantibodies play significant role
in underlying CIDP biology
HR: 0.39
P = 0.000039 99%
Rollover of eligible patients
to open-label extension
Favorable safety and
tolerability profile consistent
with previous clinical trials
Stage B
sBLA submitted with priority review voucher
SET NEW
STANDARD FOR
HOW CIDP
TRIALS ARE RUN
SIGNIFICANT IMPACT
ON CIDP PATIENTS
99%
Study Compliance
Stage A
ESTABLISHED CIDP
AS IgG MEDIATED
61%
reduced risk
of relapse
11 |
| CONFIDENTIAL FOR INTERNAL USE ONLY. © 2023 argenx.
We Aim to Address the Unseen Suffering in CIDP
≤20% of patients achieve remission on current SOC
(CDAS=2)*
>50% of patients are dissatisfied with their symptom
burden**
>42K treated CIDP patients in US & ROW argenx markets
(ex-China)***
* Gorson KC, et al. 2010
** Mendoza M, et al. 2023
*** argenx market research
12
12 |
| Transforming the Patient Treatment Experience
13
Pre-filled Syringe Autoinjector
Single 30-90s injection
HCP administered
Increasingly convenient delivery
Preparing for self-administration
Approved June 2023 Ongoing in clinical trials Industrialization phase
Exclusive
FcRn license
to ENHANZE® High concentration formulation with low viscosity, no
back pressure |
| This is
Just the
Beginning
14
Generalized
Myasthenia
Gravis
Chronic Inflammatory
Demyelinating
Polyneuropathy
Expected 2024 PoC study readouts
ITP
Launched
2022
Anticipated
approval 2024
Japan submission
in review
TED
100K
IMNM
6K
Sjogren’s
330K
PC-POTS
500K
Beyond 2024 PoC studies
BP
52K
LN
40K
MN
80K
AAV
105K
AMR
8K
Potential 2024 Phase 3 Start
Phase 2 Proof of Concept
65K 24K 17K
2024 Phase 3 Start
ASyS
11K
DM
70K
#14 #15
*** argenx market research; US prevalence numbers (except Japan ITP) |
| Phase 2 Readouts Present Significant Commercial Opportunities
15
• Anti-Ro/Anti-La AutoAbs
• Passive transfer model evidence
• IgG reduction associated
with improvement
RCT - Phase 2
CRESS/ESSDAI
• Steroids/NSISTs
• Cholinergic agonists
• Artificial tears
• Anti-adrenergic receptor AutoAbs
• IVIG/PLEX effective
RCT - Phase 2
MaPS/COMPASS
• No approved
therapies
• Myositis AutoAbs
• Passive transfer model evidence (IMNM)
• AutoAb titer correlates with disease
activity
RCT - P2/P3
TIS
BIOLOGIC
RATIONALE
• Steroids
• IVIg
330K 500K
6K IMNM
11K ASyS
70K DM
Sjogren’s Syndrome PC-POTS Myositis (IMNM, ASyS, DM)
CLINICAL
FEASIBILITY
U.S.
COMMERICAL
OPPORTUNITY |
| PFS in development
15 indications
in development by 2025
ITP MAA filed
CIDP sBLA submitted
ARGX-109 $1.2B in gMG revenue in 2023
(Anti-IL-6)
ARGX-213
(Anti-FcRn)
ARGX-220
ARGX-121
Empasiprubart
POC established in MMN
Trials in DGF and DM
ARGX-119
Phase 1b/2a trials in
CMS and ALS
Pipeline Horizon
16 |
| Rewriting Immunology Textbook with Empasiprubart
Pioneering
complement
factor C2
Unique
sweeping
antibody
Ongoing
development in
3 indications
~80-day half-life supports
favorable dosing
Defining MMN as POC established in MMN
auto-IgM mediated
disease
17 |
| Empasiprubart has Potential to Transform MMN
18
HR (95% CI) = 0.09 (0.02; 0.44)
• 94% of treated patients rated their condition improved since starting therapy, including 55% who were much/very much improved
8/9 placebo patients had no change or worsened (Patient Global Impression of Change scale)
• Empasiprubart demonstrated improvement compared to baseline on 6/6 efficacy measurements
• Safety profile consistent with Phase 1 data
91%
reduction in need
for IVIg rescue with
empasiprubart
Cohort 2 is ongoing; results to inform dose for Phase 3 study initiation 18 |
| MMN Patients are Waiting
Clear opportunity for
empasiprubart…
…I’m not asking to be able
to run and jump like
I used to. I just want to be
able to stand like I used to.
“
”
IVIg only treatment
option
Patient journey
characterized by deep
frustration and anxiety
…to transform MMN
outcomes
ADDRESSABLE
MARKET
~10k patients
US + argenx ROW markets (ex China)*
*argenx market research; Arnold et al 2013; Park et al 2022 19 |
| ARGX-119: Enhancing Neuromuscular Junction
Pioneering
MuSK biology
at NMJ
Initial
development in
CMS and ALS
Agonistic
SIMPLE
Antibody™
Safety and tolerability data from extensive Phase 1 study support advancement into PoC studies
20 |
| CMS and ALS Trials to Start in 2024
21
ARGX-119 rescues early neonatal lethality and
disease relapse in adult DOK7 CMS mice
Nature, Oury et al. 2021
ARGX-119 preserves NMJ numbers and restores muscle
contraction in ALS patient derived NMJs on-a-chip Fidelity (%)
Biomaterials, Badu-Mensah et al. 2022;
Advanced Therapeutics, Guo et al. 2020
50
100
0
Frequency (Hz)
Healthy ALS ALS + ARGX-119
argenx internal data
mAb
mAb |
| PFS in development
15 indications
in development by 2025
ITP MAA filed
CIDP sBLA submitted
ARGX-109 $1.2B in gMG revenue in 2023
(Anti-IL-6)
ARGX-213
(Anti-FcRn)
ARGX-220
ARGX-121
Empasiprubart
POC established in MMN
Trials in DGF and DM
ARGX-119
Phase 1b/2a trials in
CMS and ALS
Immunology Innovation Horizon
22 |
| ARGX-109
(Anti-IL-6)
Pipeline Growth Driven By Immunology
Innovation Program
23
ARGX-213
ARGX-220
DISEASE
INSIGHT
ANTIBODY
ENGINEERING
CLINICAL
DEVELOPMENT
Continued Leadership in FcRn
Broad Immune System Targets
ARGX-121
First in class, novel targets
4 IND FILINGS BY END OF 2025
Strong Track Record with Repeatable Innovation Playbook
LEADING
TRANSLATIONAL
BIOLOGY LABS
TECHNOLOGY
KNOW-HOW |
| VYVGART
gMG
Decision on approval: Switzerland, Australia, Saudi Arabia, South Korea By Year End
Seronegative trial initiation By Year End
ITP Japan decision on approval 1Q 2024
VYVGART SC
gMG
Japan decision on approval By 1Q 2024
China decision on approval (Zai Lab) By Year End
CIDP
U.S. launch, if approved Mid-2024
Regulatory submissions Japan, Europe, China, Canada By Year End
MG, CIDP Update on PFS development 1H 2024
Efgartigimod
Primary Sjogren’s syndrome Proof of concept data 1H 2024
PC-POTS Proof of concept data 1H 2024
Myositis Proof of concept data 2H 2024
Empasiprubart MMN Full Phase 2 data 2024
ARGX-119 CMS, ALS Phase 1b/2a study initiations 2024
IIP Not Disclosed 4 INDs filed By End of 2025
24
Indication Milestone Timing
Strong Cadence of Milestones in 2024 |
| Q4 2023 Product Net Sales of $374 million
21
75
131
173
218
269
329
374
2022
Q1
2022
Q2
2022
Q3
2022
Q4
2023
Q1
2023
Q2
2023
Q3
2023
Q4*
($B) 2023
Product Net Sales(1) 1.2
Cash, cash equivalents and current
financial assets(1)
3.2
2024 Financial Guidance
(1) - Preliminary Financials. Unaudited and subject to change.
($B) 2024
Cash burn(2) ~ 0.5
Combined R&D and SG&A expenses < 2.0
*Preliminary Financials. Unaudited and subject to change
Preliminary 2023 Financial Results
(2) - Cash burn is equal to the decrease in our cash, cash equivalents and current financial assets
On Track To Be Sustainable
2023 CAGR: 21%
25 |
| 6
Leading to multiple
2024 Strategic Priorities
Committed to Driving Continued Growth
26
Broaden
leadership in
MG market
Launch CIDP
Advance PFS
Phase 2 data
readouts
Phase 3 initiations
INDs by 2025
4 |
| THANK YOU
27 |
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