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ADDRESS study did not meet primary or secondary endpoints
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Pemphigus deprioritized as efgartigimod indication
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Update on BALLAD study GO/NO GO decision
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Conference call scheduled for today, December 20, 2023, at 8:30am
ET (2:30pm CET)
Regulated Information – Inside
Information
December 20, 2023, 7:00am 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 announced topline results from
the ADDRESS study evaluating efgartigimod subcutaneous (SC)
(efgartigimod alfa and hyaluronidase-qvfc) in adults with pemphigus
vulgaris (PV) and pemphigus foliaceus (PF). The ADDRESS results
show the proportion of PV patients achieving the primary endpoint
of complete remission on a minimal dose of steroids (CRmin) was not
significantly different between efgartigimod SC and placebo.
argenx will not pursue additional development in
pemphigus and plans to prioritize clinical development of
efgartigimod in its ongoing severe autoimmune indications.
“We are disappointed by today’s results,
particularly for pemphigus patients who have seen little innovation
in this treatment space,” said Luc Truyen, M.D., Ph.D., Chief
Medical Officer at argenx. “At argenx, we are in the business of
transformation, providing new medicines that go beyond incremental
benefit and raise the bar for what patients can expect from a
treatment. While we will not move forward into pemphigus, our job
today is the same as yesterday – continue to be execution-focused
and data-driven, apply learnings across our ongoing development
programs, and pursue optimal development of efgartigimod,
empasiprubart and our earlier stage programs. 2023 was a remarkable
year of growth for argenx across the business and we are poised to
build on our success in 2024.
“We are grateful to the pemphigus community and
all involved in the ADDRESS study, including patients, healthcare
professionals, and our argenx teams,” continued Dr. Truyen.
ADDRESS Study Results
The Phase 3 ADDRESS study enrolled 222 adult
patients with newly diagnosed or relapsing moderate-to-severe PV
(n=190) or PF (n=32). Patients were randomized to efgartigimod SC
or placebo with both treatment groups receiving concomitant
steroids at a starting dose of 0.5mg/kg, which is a lower dose than
recommended by current treatment guidelines and was tapered
according to protocol upon achievement of complete remission.
- Consistent pharmacodynamic
(PD) effect of efgartigimod SC: Treatment with
efgartigimod SC led to total immunoglobulin G (IgG) and desmoglein
autoantibody (DSG-1 and DSG-3) reductions up to 75%. The observed
PD effect was consistent with previous clinical trials of
efgartigimod.
- Unexpected PD effect of
corticosteroids: There was a higher than expected response
to background treatment with corticosteroids, which showed a
reduction of DSG-1 and DSG-3 levels of up to 70% in the placebo arm
and correlated to sustained clinical benefit. The level of
autoantibody reduction driven by corticosteroids in both treatment
arms was sufficient for patients to achieve CRmin. The significant
PD effect of corticosteroids was specific to DSG-1 and DSG-3
autoantibodies, while the observed effect on total IgG reduction
was in line with the literature (up to 10%).
- Study did not meet primary
endpoint: Treatment with efgartigimod SC led to CRmin in
35.5% (44/124) of patients compared to 30.3% (20/66) with placebo
(p=0.5956). Secondary endpoints were also not met, including CRmin
in the overall pemphigus population (PV and PF), cumulative dose of
corticosteroids and time to disease control or complete
remission
- Consistent and favorable
safety profile: Efgartigimod SC was well-tolerated in
ADDRESS. The observed safety and tolerability profile was
consistent with other clinical trials and the confirmed safety
profile of VYVGART and VYVGART Hytrulo.
Update on BALLAD Studyargenx is
reviewing the BALLAD study in light of the ADDRESS results and the
comparable biology between pemphigus and bullous pemphigoid, and
has decided not to make a GO/NO GO decision at this time but rather
wait for learnings from all currently enrolled patients and
consider a new trial design for the path forward.
Conference Call Detailsargenx
will host a conference call today at 2:30 pm CET (8:30am ET) to
discuss the ADDRESS results. A webcast of the live call and replay
may be accessed on the Investors section of the argenx website.
Dial-in Numbers:Please dial in
15 minutes prior to the live call.
Belgium |
32 800 50 201 |
France |
33 800 943355 |
Netherlands |
31 20 795 1090 |
United Kingdom |
44 800 358 0970 |
United States |
1 888 415 4250 |
Japan |
81 3 4578 9081 |
Switzerland |
41 43 210 11 32 |
About the ADDRESS Study The
ADDRESS study was a randomized, double-blind, placebo-controlled,
multicenter, global trial evaluating the efficacy and safety of
efgartigimod SC (efgartigimod alfa and hyaluronidase-qvfc) in adult
patients with pemphigus. Enrolled patients had newly diagnosed or
relapsing moderate-to-severe pemphigus vulgaris (PV) or pemphigus
foliaceus (PF) with PDAI scores of ≥15. Patients were randomized in
a 2:1 ratio to receive efgartigimod SC or placebo for a total of 30
weeks as part of the primary trial. All patients were on
concomitant corticosteroids at a starting dose of 0.5mg/kg/day,
which could be tapered according to protocol upon achievement of
complete remission (PDAI = 0). The primary endpoint was measured by
the proportion of PV patients who achieved sustained complete
remission on a minimal dose of corticosteroids (CRmin) within 30
weeks. Key secondary endpoints included proportion of overall
population (PV and PF) who achieved CRmin, cumulative
corticosteroid dose, and time to disease control and complete
remission. At the end of the 30-week study, eligible patients
entered a double-blind 8-week follow-up as part of the ADDRESS
open-label extension study during which CRmin off treatment
(efgartigimod SC or placebo) was assessed.
About PemphigusPemphigus is a
rare group of chronic blistering autoimmune diseases that affect
the skin and mucous membranes, and are characterized by painful
blisters, erosions and acantholysis, or disruption of keratinocyte
adhesion. Blisters often break open, causing serious pain and
increased risk of infection. Pemphigus vulgaris and pemphigus
foliaceous are the most common forms of pemphigus.
About the BALLAD Study
The BALLAD study is a randomized, double-blind,
placebo-controlled, multicenter trial evaluating the efficacy and
safety of VYVGART Hytrulo (efgartigimod alfa and
hyaluronidase-qvfc) in adult patients with bullous pemphigoid (BP).
Enrolled patients have moderate to severe BP and are a more fragile
and older population than pemphigus patients. Patients were
randomized in a 1:1 ratio to receive VYVGART Hytrulo or placebo for
a total of 36 weeks as part of the primary trial. All patients are
on concomitant corticosteroids at a starting dose of 0.5 mg/kg/day,
which could be tapered according to protocol upon achievement of
complete remission (BPDAI= 0). The primary endpoint is measured at
36 weeks by the proportion of BP patients who achieved clinical
remission while receiving efgartigimod SC or placebo but off
corticosteroids therapy for at least 8 weeks. Key secondary
endpoints include cumulative dose of corticosteroids from baseline,
proportion of patients who achieve an Investigator Global
Assessment of BP (IGA-BP) score of 0 or 1, changes from baseline in
the BP Disease Area Index (BPDAI) activity score, proportion of
patients who are in CR on minimal corticosteroids for at least 8
weeks at week 36 and time to control of disease or complete
remission.
About Bullous Pemphigus
Bullous pemphigoid is a rare chronic
blistering autoimmune disease and is the most common form
of pemphigoid diseases. It is characterized by autoantibodies
against structural proteins of the dermal-epidermal junction and,
clinically, by tense blisters and erosions of skin or mucous
membranes close to the skin surface. The disease has a strong
impact on a person’s quality of life and is associated with a high
mortality.
About VYVGART Hytrulo
(efgartigimod SC)VYVGART Hytrulo is a subcutaneous combination of
efgartigimod alfa, a human IgG1 antibody fragment marketed for
intravenous use as VYVGART®, and recombinant human hyaluronidase
PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to
facilitate subcutaneous injection delivery of biologics. In binding
to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the
reduction of circulating IgG. VYVGART Hytrulo is the proprietary
name in the U.S. for subcutaneous efgartigimod alfa and recombinant
human hyaluronidase PH20. It is marketed in Europe as VYVGART and
may be marketed under different proprietary names following
approval in other regions.
About argenxargenx 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 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.
For further information, please contact:
Media:Ben Petokbpetok@argenx.com
Investors:Alexandra Roy (US)aroy@argenx.com
Lynn Elton (EU)lelton@argenx.com
This press release contains inside information within the
meaning of Article 7(1) of the EU Market Abuse Regulation
(Regulation 596/2014).
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,”
“anticipates,” “expects,” “intends,” “may,” “will,” “should,” or
“commitment” and include statements argenx makes concerning
argenx’s topline results from the ADDRESS study of efgartigimod SC
in adults with PV and PF, our plan to pursue optimal development of
efgartigimod, empasiprubart and our earlier stage programs, and our
goal of translating immunology breakthroughs into a world-class
portfolio of novel antibody-based medicines. 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 additional analyses of the dataset from the
ADDRESS and BALLAD studies, expectations regarding 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, 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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