Long-term and real-world data of VYVGART®
(efgartigimod alfa-fcab) and VYVGART® Hytrulo (efgartigimod alfa
and hyaluronidase-qvfc) demonstrate speed of onset, depth of
response, and durability of response
VYVGART demonstrates consistent, favorable safety
profile from follow-up safety data that totals >8,000 patient
years; no vaccinations required and no impact on human serum
albumin levels
Real-world data show more than 50 percent of gMG
patients demonstrate substantial and sustained reduction in steroid
use following VYVGART initiation
argenx continues to expand its reach in neurology
through pipeline programs, including empasiprubart advancing in MMN
and ARGX-119 in ALS and CMS
October 15, 2024 – 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 the presentation of
clinical and real-world data across its growing immunology pipeline
at the 2024 American Association of Neuromuscular &
Electrodiagnostic Medicine (AANEM) Annual Meeting and Myasthenia
Gravis Foundation of America (MGFA) Scientific Sessions in
Savannah, GA from October 15-18, 2024.
“VYVGART continues to deliver impactful benefits
to patients in terms of safety, speed of onset, depth of response,
and durability of response,” said Luc Truyen, M.D., Ph.D., Chief
Medical Officer, argenx. “The robust data we are showing at AANEM
and MGFA continue to confirm VYVGART as the leading innovative
biologic with an established ability to reduce steroid usage, drive
minimal symptom expression for gMG patients, and reduce CIDP
symptoms quickly. In addition to VYVGART, we are excited to
highlight our growing neurology pipeline, including empasiprubart
and ARGX-119, through which we can advance our mission of
delivering transformative outcomes for even more patients.”
VYVGART and VYVGART Hytrulo Demonstrate
Rapid, Deep and Sustained Responses in gMG and CIDP
The data presented at AANEM continue to
demonstrate the significant impact of VYVGART (including VYVGART
Hytrulo), the first-in-class neonatal Fc receptor (FcRn) blocker
for people living with generalized myasthenia gravis (gMG) and
chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART
is setting a new treatment standard in gMG and has shown rapid,
deep and sustained responses, enabling a majority of patients to
achieve minimal symptom expression (MSE) with a consistent and
favorable safety profile and more than 8,000 patient years of
safety data. Based on real-world data, more than half of patients
can reduce steroid use by >5mg/day following VYVGART initiation.
In CIDP, a majority of patients in the ADHERE trial responded to
VYVGART Hytrulo and experienced reduced risk of relapse versus
placebo and improvements in motor function and muscle strength,
regardless of prior CIDP treatment.
Highlights from VYVGART data presented
at AANEM and MGFA:
- Early Line Use and
Meaningful Steroid Reduction: VYVGART demonstrates
consistent improvement across gMG patient subtypes, including those
on mestinon alone, indicating its efficacy in early line use.
Real-world gMG data show that at one-year post VYVGART initiation,
55% of patients reduced corticosteroid use by ≥5 mg/day and 42% of
patients had achieved steroid doses of ≤5mg/day.
- Expansion to Seronegative
and Ocular MG: argenx is honoring its long-term commitment
to the broader MG community with two Phase 3 studies ongoing in
additional MG patient populations, including seronegative
(ADAPT-SERON) and ocular MG (ADAPT-OCULUS). Seronegative (AChR-)
gMG patients evaluated in VYVGART clinical studies experienced
consistent and clinically meaningful MG-ADL improvements, including
patients achieving MSE.
- Sustained Functional
Benefit in CIDP: VYVGART Hytrulo showed sustained
functional benefit in motor function and muscle strength,
regardless of prior treatment, which was maintained through ADHERE
and the open-label extension study (through week 24).
- Consistent, Favorable
Safety Profile: VYVGART’s consistent and favorable safety
profile has been established across multiple autoimmune diseases
with no increase in the incidence of adverse events with increased
exposure. The unique safety profile of VYVGART is further supported
by no black box warnings, no labs or immunoglobulin (Ig)
monitoring, and no vaccination requirements.
Advancing Immunology Pipeline Across Two
First-in-Class Opportunities to Reach New Patients
argenx will also highlight two additional
pipeline candidates, including Phase 2 ARDA data of empasiprubart
(anti-C2 inhibitor) for the treatment of multifocal motor
neuropathy (MMN), and clinical trial designs of ARGX-119
(muscle-specific kinase (MuSK) agonist) for the treatment of
congenital myasthenic syndromes (CMS) and amyotrophic lateral
sclerosis (ALS).
Cohort 1 data from the Phase 2 ARDA study will
be presented in a poster, showing treatment with empasiprubart for
MMN reduced the risk of IVIg retreatment by 91% (HR: 0.09 [95% CI:
0.02–0.44]) and demonstrated significant improvement in grip
strength in both hands as compared to placebo. Data from a Patient
Global Impression of Change scale show 94.4% of patients said they
improved on empasiprubart from the start of the study, compared to
11.1% of placebo patients. As part of its commitment to the MMN
community, argenx initiated the iMMersioN longitudinal study of
~150 patients to collect data on the impact of disease and burden
of current treatment options on clinical outcomes and quality of
life measures. A Phase 3 study of empasiprubart in MMN will start
by the end of 2024.
argenx posters included in MGFA
Scientific Sessions
- All MGFA posters to be presented on
Tuesday, October 15, 12:00 – 12:45pm ET
- Posters with an asterisk (*) will
also be presented in AANEM scientific program
Full Title |
Presentation Details |
Patterns of Efgartigimod Dosing in Clinical Practice in the United
States |
Poster # MG9 |
Real-World Reduction in Oral Glucocorticoid Utilization at 1-Year
Following Efgartigimod Initiation* |
Poster # MG31 / AANEM Poster # 262 |
Efficacy, Safety, and Pharmacodynamics of Efgartigimod PH20 SC
Across Bodyweight Quartiles: A Post hoc Analysis of the ADAPT-SC+
Trial |
Poster # MG32 |
Fixed Cycle and Every-Other-Week Dosing of Intravenous Efgartigimod
for Generalized Myasthenia Gravis: Part A of ADAPT NXT* |
Poster # MG33 /AANEM Poster # 182 |
Design of a Phase 3 Randomized, Double-Blinded, Placebo-Controlled
Study Evaluating the Efficacy and Safety of Subcutaneous
Efgartigimod PH20 Administered by Prefilled Syringe in Adults with
Ocular Myasthenia Gravis |
Poster # MG38 |
Phase 3 Trial Investigating Impact of Intravenous Efgartigimod in
Anti-Acetylcholine Receptor Antibody Negative Generalized
Myasthenia Gravis* |
Poster # MG39 / AANEM Poster # 178 |
Observed Efficacy of Efgartigimod in Generalized Myasthenia Gravis
Across Patient Subgroups in the ADAPT-SC+ Study |
Poster # MG68 |
Exploring the Impact of Non-Steroidal Immunosuppressive Drugs and
Steroids on the Development of Comorbidities in Patients with
Myasthenia Gravis in the National Veterans Affairs Health
Network |
Poster # MG86 |
Quality of Life of Patients with Symptomatic Ocular MG: Comparison
with the General Population |
Poster # MG87 |
Steroid Use, Toxicity, and Monitoring in Patients With Generalized
Myasthenia Gravis: A Survey Of Neurologists In The United
States* |
Poster # MG89 / AANEM Poster # 235 |
Comparative Risk-Benefit Profiles of Immunomodulatory Therapies for
Patients with Generalized Myasthenia Gravis |
Poster # MG98 |
argenx posters included in AANEM Scientific
Program
* Session Times:
- Session I: Wednesday, October 16, 6:15 - 6:45pm ET
- Session II: Thursday, October 17, 9:30 - 10am ET
- Session III: Thursday, October 17, 2:45 - 3:15pm ET
Full Title |
Presentation Details* |
Long-Term Safety and Efficacy of Efgartigimod PH20 SC in
Generalized Myasthenia Gravis: Interim Analysis of
Anti-Acetylcholine Receptor Antibody Seronegative Participants in
ADAPT-SC+ |
Poster # 144Session I & Session II |
Combined Analyses of Participants With Anti-Acetylcholine Receptor
Seronegative Generalized Myasthenia Gravis Treated With
Efgartigimod Across Clinical Studies |
Poster # 146Session I & Session II |
Safety, Tolerability, Pharmacokinetics, Immunogenicity, and
Efficacy of ARGX-119 in Participants With DOK7 Congenital
Myasthenic Syndromes: Phase 1b Study in Progress |
Poster # 165Session I & Session III |
Efficacy and Safety of Subcutaneous Efgartigimod PH20SC in Chronic
Inflammatory Demyelinating Polyneuropathy: ADHERE Trial Subgroup
Analysis |
Poster # 176Session I & Session II |
Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Chronic
Inflammatory Demyelinating Polyneuropathy: ADHERE/ADHERE+
Trial |
Poster # 177Session I & Session III |
Safety Profile of Intravenous Efgartigimod From Clinical Trials in
Immunoglobulin G–Mediated Autoimmune Diseases |
Poster # 180Session I & Session II |
Safety Profile of Subcutaneous Efgartigimod PH20 From Clinical
Trials in Immunoglobulin G–Mediated Autoimmune Diseases |
Poster # 181Session I & Session III |
Empasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Initial
Safety and Efficacy Data of the Phase 2 ARDA Study |
Poster # 198Session I & Session II |
Long-Term Safety, Tolerability, and Efficacy of Subcutaneous
Efgartigimod PH20 in Participants With Generalized Myasthenia
Gravis: Interim Results of the ADAPT-SC+ Study |
Poster # 212Session I & Session III |
Safety, Tolerability, Efficacy, Pharmacokinetics, and
Immunogenicity of ARGX-119 in Patients with Amyotrophic Lateral
Sclerosis: A Phase 2a Study in Progress |
Poster # 237Session I & Session II |
Risk of Serious Infections and Malignancies in Adult Myasthenia
Gravis Patients: A US Claims Database Study |
Poster # 251Session I & Session II |
Chronic Steroid Toxicity in Adults With Myasthenia Gravis in the
United States Based on Electronic Health Records |
Poster # 263Session I & Session II |
Subcutaneous Efgartigimod PH20 in Chronic Inflammatory
Demyelinating Polyneuropathy: Key Secondary Outcomes from the
ADHERE Trial |
Poster # 280Session I & Session III |
Empasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Baseline
Characteristics and MMN Confirmation Committee Outcome of the Phase
ARDA Study Cohort 1 |
Poster # 293Session I & Session II |
COVID-19 Vaccination Response in Participants Receiving
Efgartigimod IV or Efgartigimod PH20 SC in ADAPT+ or ADAPT-SC+ |
Poster # 298Session I & Session III |
Steroid Use, Toxicity, and Monitoring in Patients with Chronic
Inflammatory Demyelinating Polyneuropathy: A Survey of Neurologists
in The United States |
Poster # 306Session I & Session III |
Clinical Outcomes, Disease Course, and Quality of Life in Patients
With Multifocal Motor Neuropathy: iMMersioN, Study in Progress |
Poster # 307Session I & Session II |
More information on the data presented at the
2024 AANEM Annual Meeting can be found here.
See FDA-approved Important Safety Information
below, full Prescribing Information for VYVGART and full
Prescribing Information for VYVGART Hytrulo for additional
information.
Important Safety
Information
What is VYVGART® (efgartigimod
alfa-fcab)?VYVGART is a prescription medicine used to
treat a condition called generalized myasthenia gravis, which
causes muscles to tire and weaken easily throughout the body, in
adults who are positive for antibodies directed toward a protein
called acetylcholine receptor (anti-AChR antibody positive).
IMPORTANT SAFETY INFORMATIONDo
not use VYVGART if you have a serious allergy to efgartigimod alfa
or any of the other ingredients in VYVGART. VYVGART can cause
serious allergic reactions and a decrease in blood pressure leading
to fainting.
VYVGART may cause serious side effects,
including:
- Infection. VYVGART
may increase the risk of infection. The most common infections 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 can cause allergic
reactions such as rashes, swelling under the skin, and shortness of
breath. Serious allergic reactions, such as trouble breathing and
decrease in blood pressure leading to fainting have been reported
with VYVGART.
- Infusion-Related
Reactions. VYVGART can cause infusion-related reactions.
The most frequent symptoms and signs reported with VYVGART 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 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, 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?The most common side effects of VYVGART are
respiratory tract infection, headache, and urinary tract
infection.
These are not all the possible side effects of
VYVGART. 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 and
talk to your doctor.
What is VYVGART® HYTRULO (efgartigimod
alfa and hyaluronidase-qvfc)?VYVGART HYTRULO is a
prescription medicine used to treat a condition called generalized
myasthenia gravis, which causes muscles to tire and weaken easily
throughout the body, in adults who are positive for antibodies
directed toward a protein called acetylcholine receptor (anti-AChR
antibody positive).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 VYVGARTVYVGART 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 in the United
States, EU, China and Canada 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).
About VYVGART® HytruloVYVGART
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 may be marketed under different proprietary
names following approval in other regions.
About Generalized Myasthenia Gravis
(gMG)Generalized myasthenia gravis (gMG) is a rare and
chronic autoimmune disease where IgG autoantibodies disrupt
communication between nerves and muscles, causing debilitating and
potentially life-threatening muscle weakness. Approximately 85% of
people with MG progress to gMG within 24 months,1 where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population.
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.
About
EmpasiprubartEmpasiprubart (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, dermatomyositis and chronic inflammatory
demyelinating polyneuropathy.
About Multifocal Motor
NeuropathyMultifocal motor neuropathy (MMN) is a rare,
severe, 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 ARGX-119ARGX-119 is a
humanized agonistic monoclonal antibody (mAb) that targets and
activates muscle-specific kinase (MuSK) to promote maturation and
stabilization of the neuromuscular junction (NMJ). MuSK is a
receptor kinase that has a critical role in the structure and
function of the NMJ. ARGX-119 is being developed as a potential
therapy for patients with neuromuscular disease.
About Congenital Myasthenic Syndromes (CMS)
Congenital Myasthenic Syndromes (CMS) are a
heterogenous group of rare genetic disorders of the neuromuscular
junction (NMJ) that lead to muscle weakness. CMS cases are
classified into subtypes depending on the underlying genetic
mutation. While clinical features vary widely across and within
subtypes, the predominant manifestation of CMS is fatigable
weakness. Age of onset varies widely; while many patients are
diagnosed in infancy or early childhood, patients with milder
phenotypes may not present or be diagnosed until adulthood.
About Amyotrophic Lateral Sclerosis (ALS)
ALS is a neurodegenerative disorder of the brain
and spinal cord leading to deteriorating muscle function, weakness
and atrophy. The multisystemic nature can have impacts throughout
the body with specific signs and symptoms associated with lower
motor neuron and upper motor neuron loss. Life expectancy is
typically 2.5-5 years from diagnosis.
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,
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, X/Twitter, Instagram, Facebook,
and YouTube.
ContactsMedia: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 “aim,”
and “will,” and include statements argenx makes regarding the
anticipated timing of the initiation of the Phase 3 clinical trial
for empasiprubart in MMN 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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