ARDA study data show potential for empasiprubart
to drive functional improvement and reduced risk of relapse for
multifocal motor neuropathy (MMN) patients
ADHERE+ data show durability of functional
improvements with VYVGART® Hytrulo (efgartigimod alfa and
hyaluronidase-qvfc), which is FDA approved for use in adults with
chronic inflammatory demyelinating polyneuropathy (CIDP)
June 25, 2024 – 4:30pm EDT
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 that new data from
across the company’s autoimmune pipeline were presented at the 2024
Peripheral Nerve Society (PNS) Annual Meeting in Montréal,
Quebec.
“argenx is on a mission to transform the
treatment of severe autoimmunity,” said Luc Truyen, M.D., Ph.D.,
Chief Medical Officer, argenx. “We have established MMN and CIDP as
autoantibody-mediated diseases, and by developing novel medicines
that precisely target disease biology, we are fulfilling our
mission to create truly transformative outcomes for patients. We
are excited to present data for our novel therapies that may offer
benefits beyond symptom management – to safely help patients regain
control of their lives without harsh side effects, residual
impairments, or treatments dependent on high frequency infusions.
argenx continues to build scientific and clinical evidence that
supports the advancement of our innovative pipeline and shows the
potential to expand therapeutic choices while reducing the risk of
relapse and accumulating disability.”
Phase 2 ARDA Data Support Empasiprubart
as Novel Targeted Treatment for MMNFor the first time at a
medical congress, argenx presented Cohort 1 data from the Phase 2
ARDA study, which support proof of concept for empasiprubart as a
potential new treatment option for MMN, a chronic, progressive
autoimmune disease with only one approved treatment option. ARDA is
the largest interventional study to-date in MMN.
- Improved function and
reduced risk of IVIg retreatment: Compared with placebo,
treatment with empasiprubart reduced the risk of IVIg retreatment
by 91% (HR: 0.09 [95% CI: 0.02–0.44]), with improvement of grip and
muscle strength, and improved patients’ ability to perform daily
activities.
- Favorable tolerability
profile: Empasiprubart was well-tolerated and most adverse
events were mild or moderate.
ADHERE Oral Presentation Highlights Consistent Sustained
Efficacy and Safety of VYVGART Hytrulo for Patients with
CIDP
argenx also presented new data from the pivotal
ADHERE and open-label extension ADHERE+ studies evaluating VYVGART
Hytrulo in patients with CIDP. The ADHERE data supported the recent
FDA approval of VYVGART Hytrulo as a safe and effective new
treatment option for CIDP, demonstrating sustained functional
benefit across all disease scores regardless of disease stage or
treatment history. ADHERE met its primary endpoint (p<0.0001)
demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the
risk of relapse versus placebo. Ninety-nine percent of trial
participants elected to participate in the ADHERE+ open-label
extension.
- Sustained functional improvements:
Demonstrated improvements in functional ability (mean aINCAT
scores) on VYVGART Hytrulo from Stage A baseline were maintained
through Stage B of ADHERE and to Week 24 of ADHERE+. Mean aINCAT
scores improved from Run-in Period baseline, indicating some
patients gain functional benefit on VYVGART Hytrulo compared to
baseline assessments while on prior treatment.
- Secondary endpoints show consistent improvements across
functional and strength measures: VYVGART Hytrulo-treated
patients demonstrated maintenance of functional benefit in Stage B,
whereas placebo patients experienced clinically meaningful
worsening across all disease scores, including aINCAT, I-RODS and
grip strength
- High retention and compliance in long-term extension
study: 98.9% of ADHERE+ participants demonstrated
treatment compliance with VYVGART Hytrulo and 86% were still
ongoing at Week 24 of the OLE.
- Favorable safety profile: Safety profile of
VYVGART Hytrulo was similar between ADHERE and ADHERE+ with no
increased rate of adverse events with increased exposure.
VYVGART Hytrulo was approved on June 21, 2024,
for the treatment of adult patients with CIDP by the U.S Food and
Drug Administration (FDA).
About Multifocal Motor
NeuropathyMultifocal 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 the presence of anti-GM1 IgM autoantibodies,
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 Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP)Chronic inflammatory demyelinating
polyneuropathy (CIDP) is a rare and serious autoimmune disease of
the peripheral nervous system. Although confirmation of disease
pathophysiology is still emerging, there is increasing evidence
that IgG antibodies play a key role in the damage to the peripheral
nerves. People with CIDP experience fatigue, muscle weakness and a
loss of feeling in their arms and legs that can get worse over time
or may come and go. These symptoms can significantly impair a
person's ability to function in their daily lives. Without
treatment, one-third of people living with CIDP will need a
wheelchair.
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 before
2:1 randomization into a double-blind treatment phase for 16-weeks.
Two sequential cohorts of 27 MMN patients receiving empasiprubart
or placebo were enrolled investigating two different dose levels of
empasiprubart. 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 clinical
efficacy scores (modified medical research council (mMRC)-14 sum
score, grip strength, MMN-RODS) as well as several patient-reported
quality of life outcome measures.
About ADHERE Trial Design
The ADHERE trial was a multicenter, randomized,
double-blind, placebo-controlled trial evaluating VYVGART® Hytrulo
(efgartigimod alfa and hyaluronidase-qvfc) for the treatment of
chronic inflammatory demyelinating polyneuropathy (CIDP). ADHERE
enrolled 322 adult patients with CIDP who were treatment naïve (not
on active treatment within the past six months or newly diagnosed)
or being treated with immunoglobulin therapy or corticosteroids.
The trial consisted of an open-label Stage A followed by a
randomized, placebo-controlled Stage B. In order to be eligible for
the trial, the diagnosis of CIDP was confirmed by an independent
panel of experts. Patients entered a run-in stage, where any
ongoing CIDP treatment was stopped and in order to be eligible for
Stage A had to demonstrate active disease, with clinically
meaningful worsening on at least one CIDP clinical assessment tool,
including INCAT, I-RODS, or mean grip strength. Treatment naïve
patients were able to skip the run-in period with proof of recent
worsening. To advance to Stage B, patients needed to demonstrate
evidence of clinical improvement (ECI) with VYVGART Hytrulo. ECI
was achieved through improvement of the INCAT score, or improvement
on I-RODS or mean grip strength if those scales had demonstrated
worsening during the run-in period. In Stage B, patients were
randomized to either VYVGART Hytrulo or placebo for up to 48 weeks.
The primary endpoint was measured once 88 total relapses or events
were achieved in Stage B and was based on the hazard ratio for the
time to first adjusted INCAT deterioration (i.e. relapse). After
Stage B, all patients had the option to roll-over to an open-label
extension study to receive VYVGART Hytrulo.
About Empasiprubart
Empasiprubart (ARGX-117) is a first-in-class
humanized monoclonal antibody that binds C2 and blocks activation
of both the classical and lectin pathways of the complement
cascade, leaving the alternative pathway intact for its
antimicrobial properties. By blocking complement activity upstream
of C3 and C5, empasiprubart has the potential to reduce tissue
inflammation and cellular damage, 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 Hytrulo (efgartigimod
alfa and hyaluronidase-qvfc)
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. It is the first-and-only
approved FcRn blocker administered by subcutaneous injection.
VYVGART Hytrulo is the proprietary name in the
U.S. for subcutaneous efgartigimod alfa and recombinant human
hyaluronidase PH20. It is marketed as VYVDURA® in Japan and VYVGART
SC in Europe.
See FDA-approved Important Safety Information
below and full Prescribing Information for VYVGART Hytrulo for
additional information.
What is VYVGART® HYTRULO (efgartigimod
alfa and hyaluronidase-qvfc)?VYVGART HYTRULO is a
prescription medicine used for the treatment of adult patients with
chronic inflammatory demyelinating polyneuropathy (CIDP).
IMPORTANT SAFETY INFORMATIONDo
not use VYVGART HYTRULO if you have a serious allergy to
efgartigimod alfa, hyaluronidase, or any of the other ingredients
in VYVGART HYTRULO. VYVGART HYTRULO can cause serious allergic
reactions and a decrease in blood pressure leading to fainting.
VYVGART HYTRULO may cause serious side
effects, including:
- Infection. VYVGART
HYTRULO may increase the risk of infection. The most common
infections for efgartigimod alfa-fcab-treated patients were urinary
tract and respiratory tract infections. Signs or symptoms of an
infection may include fever, chills, frequent and/or painful
urination, cough, pain and blockage of nasal passages/sinus,
wheezing, shortness of breath, fatigue, sore throat, excess phlegm,
nasal discharge, back pain, and/or chest pain.
- Allergic Reactions
(hypersensitivity reactions). VYVGART HYTRULO can cause
allergic reactions such as rashes, swelling under the skin, and
shortness of breath. Hives were also observed in patients treated
with VYVGART HYTRULO. Serious allergic reactions, such as trouble
breathing and decrease in blood pressure leading to fainting have
been reported with efgartigimod alfa-fcab.
- Infusion-Related
Reactions. VYVGART HYTRULO can cause infusion-related
reactions. The most frequent symptoms and signs reported with
efgartigimod alfa-fcab were high blood pressure, chills, shivering,
and chest, abdominal, and back pain.
Tell your doctor if you have signs or symptoms
of an infection, allergic reaction, or infusion-related reaction.
These can happen while you are receiving your VYVGART HYTRULO
treatment or afterward. Your doctor may need to pause or stop your
treatment. Contact your doctor immediately if you have signs or
symptoms of a serious allergic reaction.
Before taking VYVGART HYTRULO, tell your
doctor if you:
- take any medicines, including
prescription and non-prescription medicines, supplements, or herbal
medicines,
- have received or are scheduled to
receive a vaccine (immunization), or
- have any allergies or medical
conditions, including if you are pregnant or planning to become
pregnant, or are breastfeeding.
What are the common side effects of
VYVGART HYTRULO?The most common side effects in
efgartigimod-alfa-fcab-treated patients were respiratory tract
infection, headache, and urinary tract infection. Additional common
side effects with VYVGART HYTRULO are injection site reactions,
including rash, redness of the skin, itching sensation, bruising,
pain, and hives.
These are not all the possible side effects of
VYVGART HYTRULO. Call your doctor for medical advice about side
effects. You may report side effects to the US Food and Drug
Administration at 1-800-FDA-1088.
Please see the full Prescribing
Information for VYVGART HYTRULO and talk to your
doctor. 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 in the U.S.,
Japan, Israel, the EU, the UK, Canada and China. 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.
Contacts
Media: Ben Petokbpetok@argenx.com
Investors: Alexandra Roy
(US) aroy@argenx.com
Lynn Elton (EU) lelton@argenx.com
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 “aims,”
“continues,” “may,” or “potential,” and include statements argenx
makes regarding the potential for empasiprubart to drive functional
improvement and reduced risk of relapse for MMN patients; certain
results or previews of its clinical studies and potential
opportunities thereof; the potential benefits beyond symptom
management of its therapies; and its 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 the results of argenx's
clinical trials; expectations regarding the inherent uncertainties
associated with the development of novel drug therapies;
preclinical and clinical trial and product development activities
and regulatory approval requirements in products and product
candidates; the acceptance of argenx's products and product
candidates by patients as safe, effective and cost-effective; the
impact of governmental laws and regulations on our business;
disruptions caused on our reliance of third-party suppliers,
service providers and manufacturers; inflation and deflation and
the corresponding fluctuations in interest rates; and regional
instability and conflicts. 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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