Long-term clinical trial and real-world data
illustrate VYVGART® drives consistent, repeatable and clinically
meaningful responses, including minimal symptom expression (MSE) in
generalized myasthenia gravis (gMG)
Patients treated with VYVGART experienced
consistent improvements on key quality of life measures based on
long-term gMG extension data
Data across multiple indications and dosing
schedules confirm favorable safety profile and no increase in
treatment-emergent adverse event rates with longer exposure
Amsterdam, the Netherlands – Nov. 1,
2023 – argenx SE (Euronext & Nasdaq: ARGX), a global
immunology company committed to improving the lives of people
suffering from severe autoimmune diseases, today announced 20
presentations of clinical trial and real-world data from studies of
VYVGART® and VYVGART Hytrulo® (VYVGART) in neuromuscular autoimmune
disease. The data presentations will be featured at the American
Association of Neuromuscular and Electrodiagnostic Medicine (AANEM)
Annual Meeting and the Myasthenia Gravis Foundation of America
(MGFA) Scientific Session, taking place in Phoenix, AZ from
November 1-4, 2023.
“As leaders in the field of FcRn inhibition, we continue to
generate deep and broad data from our clinical trials, including
long-term extension and real-world evidence studies,” said Luc
Truyen, M.D., Ph.D., Chief Medical Officer at argenx. "With VYVGART
for gMG, we continue to show favorable safety and consistent,
repeatable clinically meaningful responses, including the ability
of patients to achieve MSE, across three years of treatment. The
ADHERE study, the largest in the history of CIDP, further
demonstrates our commitment to the neuromuscular community. These
data characterize the broad impact our first-in-class FcRn blocker
can have on the lives of people living with CIDP, and our work to
establish its safety and efficacy in the treatment of CIDP
represents an exciting step forward for this community.”
Highlights from Data Presented at AANEM
and MGFA
- Consistent, Repeatable
Responses: Long-term clinical data from ADAPT+ and
ADAPT-SC+ and real-world data illustrate the ability of VYVGART to
provide consistent, repeatable, and clinically meaningful responses
across more than 19 cycles, including improvements in
quality-of-life measures, for anti-acetylcholine receptor (AChR)
antibody positive patients with gMG.
- Achievement of Minimal
Symptom Expression: Treatment with VYVGART resulted in
40.5% - 44.6% of patients achieving MSE in ADAPT and ADAPT+.
Patients achieving MSE reported quality of life measures comparable
to healthy populations. Similar results were demonstrated in
ADAPT-SC+ with 35.5%-40.7% of patients achieving MSE following
treatment with VYVGART Hytrulo.
- ADHERE Results:
Positive topline results from the ADHERE study of VYVGART Hytrulo
in chronic inflammatory demyelinating polyneuropathy (CIDP) were
first reported in July 2023 and are being presented again during
the conference. The full dataset from ADHERE will be presented at a
medical meeting in 2024.
- Vaccine Response:
Study participants were able to mount effective humoral immune
responses to polyvalent pneumococcal vaccine regardless of whether
administered during or after VYVGART administration.
- Seronegative
Population: Long-term treatment with VYVGART Hytrulo was
associated with consistent and repeatable improvements on MG-ADL
and MG-QoL15r scales in seronegative (anti-AChR antibody negative)
gMG patients.
- Favorable Safety
Profile: VYVGART shows consistency of safety across
multiple indications with TEAE rates that were comparable to
placebo, mostly mild to moderate in severity and did not increase
with longer exposure.
Additional data are being presented from case
studies of gMG patients with LRP4 antibodies, a meta-analysis of
quality-of-life outcomes of VYVGART and other gMG treatments, and
argenx-sponsored health economic outcomes research studies
demonstrating gMG patients facing social-determinants of health
challenges experience health inequities related to increased
utilization of acute care facilities, and delayed diagnosis and
access to treatment.
AANEM Poster Presentations (November 1-4,
2023)
# |
Title |
Lead Author |
Presentation |
143 |
Efficacy, Safety, And Tolerability Of Efgartigimod In Patients With
Chronic Inflammatory Demyelinating Polyneuropathy: Results From The
Adhere Trial |
Richard Lewis, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IINov. 39:30 AM - 10:00 AM
MST |
151 |
Long-Term Safety, Tolerability, and Efficacy of Subcutaneous
Efgartigimod PH20 in Participants With Generalized Myasthenia
Gravis: Interim Analysis of Anti-Acetylcholine Receptor
Autoantibody Seronegative Participants in the ADAPT-SC+ Study |
James F. Howard Jr., MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
270 |
Dose Selection and Clinical Development Of Efgartigimod PH20
Subcutaneous In Patients With Generalized Myasthenia Gravis |
Tuan Vu, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IINov. 39:30 AM - 10:00 AM
MST |
222 |
Long-Term Safety, Tolerability, and Efficacy of Subcutaneous
Efgartigimod PH20 in Patients With Generalized Myasthenia Gravis:
Interim Results of the ADAPT-SC+ Study |
Yuebing Li, MD, PhD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
152 |
Long-Term Safety, Tolerability, and Efficacy of Efgartigimod in
Patients With Generalized Myasthenia Gravis: Concluding Analyses
From the ADAPT+ Study |
James F. Howard Jr., MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
240 |
A Case of Treatment With Efgartigimod in a Patient With Generalized
Myasthenia Gravis and LRP4 Antibodies |
Eduardo De Sousa, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IINov. 39:30 AM - 10:00 AM
MST |
220 |
Overview Of The Safety Profile From Efgartigimod Clinical Trials In
Participants With DiverseImmunoglobulin G-Mediated Autoimmune
Diseases |
Kelly Gwathmey, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
139 |
Treatment-Related Inequities in Patients With Generalized
Myasthenia Gravis Facing Social Determinants of Health Challenges:
A Survey of Neurologists in the United States |
A. Gordon Smith, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
138 |
Diagnosis Inequities In Patients With Generalized Myasthenia Gravis
Facing Social Determinants Of Health Challenges: A Survey Of
Neurologists In The United States |
A. Gordon Smith, MD |
Poster Session INov. 2 6:00 PM - 6:30 PM
MSTPoster Session IIINov. 33:00 PM - 3:30 PM
MST |
MGFA Oral and Poster Presentations (November 1,
2023)
Title |
Lead Author |
Presentation |
Achievement of Minimal Symptom Expression in Acetylcholine-Receptor
Antibody-Positive Participants with Generalized Myasthenia Gravis
and Effect on Disease-Specific Measures in ADAPT/ADAPT+
Studies |
James F. Howard, MD |
Oral Presentation 10:07 AM - 10:14 AM
MST |
Subcutaneous Efgartigimod PH20 Treatment in Participants With
Generalized Myasthenia Gravis in ADAPT-SC+: Interim Analyses on
Quality of Life, Efficacy, Tolerability, and Long-Term Safety |
Tuan Vu, MD |
Oral Presentation 10:42 AM – 10:49 AM
MST |
Racial Disparities in Acute Care Utilization Outcomes Among Those
with Myasthenia Gravis |
Pushpa Narayanaswami, MD |
Oral Presentation 9:07 AM - 9:14 AM MST |
Humoral Immune Response to Polyvalent Pneumococcal Vaccine in
Healthy Participants Receiving Efgartigimod |
Antoine Azar, MD |
Poster Session9:35 AM - 10:05 AM MST |
Network Meta Analysis of Treatment Options in Generalized
Myasthenia Gravis: Impact on Health-Related Quality of Life |
Gil Wolfe, MD |
Poster Session9:35 AM - 10:05 AM MST |
Overview of the Safety Profile From Efgartigimod Clinical Trials in
Participants with Diverse IgG-mediated Autoimmune Diseases |
Kelly Gwathmey, MD |
Poster Session9:35 AM - 10:05 AM MST |
Treatment-Related Inequities in Patients With Generalized
Myasthenia Gravis Facing Social Determinants of Health Challenges:
A Survey of Neurologists in the United States |
A. Gordon Smith, MD |
Poster Session9:35 AM - 10:05 AM MST |
Diagnostic Inequities in Patients With Generalized Myasthenia
Gravis Facing Social Determinants of Health Challenges: A Survey of
Neurologists in the United States |
A. Gordon Smith, MD |
Poster Session9:35 AM - 10:05 AM MST |
Real-World Burden Associated With Social Determinants of Health
Challenges For Individuals Living with Generalized Myasthenia
Gravis in the United States |
Tom Hughes |
Poster Session9:35 AM - 10:05 AM MST |
Real-World Outcomes of Patients living with Generalized Myasthenia
Gravis Initiating Efgartigimod treatment in the United States |
Cynthia Qi |
Poster Session9:35 AM - 10:05 AM MST |
Association between patient support program participation and
access to efgartigimod treatment for generalized myasthenia
gravis |
Glenn Phillips |
Poster Session9:35 AM - 10:05 AM MST |
More information on the programs are available at AANEM and
MGFA.
See Important Safety Information below and full Prescribing
Information for VYVGART Hytrulo for additional information
Important Safety
Information
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).
IMPORTANT SAFETY
INFORMATION
InfectionVYVGART and VYVGART
HYTRULO may increase the risk of infection. The most common
infections observed in Study 1 were urinary tract infection (10% of
efgartigimod alfa-fcab-treated patients vs 5% of placebo-treated
patients) and respiratory tract infection (33% of efgartigimod
alfa-fcab-treated patients vs 29% of placebo-treated patients).
Patients on efgartigimod alfa-fcab vs placebo had below normal
levels for white blood cell counts (12% vs 5%, respectively),
lymphocyte counts (28% vs 19%, respectively), and neutrophil counts
(13% vs 6%, respectively). The majority of infections and
hematologic abnormalities were mild to moderate in severity. Delay
the administration of VYVGART or VYVGART HYTRULO in patients with
an active infection until the infection has resolved; monitor for
clinical signs and symptoms of infections. If serious infection
occurs, administer appropriate treatment and consider withholding
treatment with VYVGART or VYVGART HYTRULO until the infection has
resolved.
ImmunizationImmunization with
vaccines during treatment with VYVGART or VYVGART HYTRULO has not
been studied; the safety with live or live-attenuated vaccines and
the response to immunization with any vaccine are unknown. Because
VYVGART and VYVGART HYTRULO cause a reduction in immunoglobulin G
(IgG) levels, vaccination with live-attenuated or live vaccines is
not recommended during treatment with VYVGART or VYVGART HYTRULO.
Evaluate the need to administer age-appropriate vaccines according
to immunization guidelines before initiation of a new treatment
cycle with VYVGART or
VYVGART HYTRULO.
Hypersensitivity
ReactionsHypersensitivity reactions, including rash,
angioedema, and dyspnea were observed in patients treated with
VYVGART or VYVGART HYTRULO. Urticaria was also observed in patients
treated with VYVGART HYTRULO. In clinical trials, hypersensitivity
reactions were mild or moderate, occurred within 1 hour to 3 weeks
of administration, and did not lead to treatment discontinuation.
Monitor patients during and for one hour after VYVGART
administration, or for at least 30 minutes after VYVGART HYTRULO
administration, for clinical signs and symptoms of hypersensitivity
reactions. If a hypersensitivity reaction occurs during VYVGART or
VYVGART HYTRULO administration, discontinue use and institute
appropriate supportive measures if needed.
ADVERSE REACTIONSIn Study 1,
the most common (≥10%) adverse reactions in efgartigimod
alfa-fcab-treated patients were respiratory tract infection,
headache, and urinary tract infection. In Study 2, the most common
(≥10%) adverse reactions in VYVGART HYTRULO-treated patients were
injection site reactions and headache. Injection site reactions
occurred in 38% of VYVGART HYTRULO-treated patients, including
injection site rash, erythema, pruritus, bruising, pain, and
urticaria. In Study 2 and its open-label extension, all injection
site reactions were mild to moderate in severity and did not lead
to treatment discontinuation. The majority occurred within 24 hours
after administration and resolved spontaneously. Most injection
site reactions occurred during the first treatment cycle, and the
incidence decreased with each subsequent cycle.
USE IN SPECIFIC
POPULATIONSPregnancyAs VYVGART and
VYVGART HYTRULO are expected to reduce maternal IgG antibody
levels, reduction in passive protection to the newborn is
anticipated. Risks and benefits should be considered prior to
administering live or live attenuated vaccines to infants exposed
to VYVGART or VYVGART HYTRULO in utero.
LactationThere is no
information regarding the presence of efgartigimod alfa-fcab from
administration of VYVGART, or efgartigimod alfa or hyaluronidase
from administration of VYVGART HYTRULO, in human milk, the effects
on the breastfed infant, or the effects on milk production. The
developmental and health benefits of breastfeeding should be
considered along with the mother’s clinical need for VYVGART or
VYVGART HYTRULO, and any potential adverse effects on the breastfed
infant from VYVGART or VYVGART HYTRULO or from the underlying
maternal condition.
INDICATIONVYVGART® (efgartigimod alfa-fcab)
for intravenous infusion and VYVGART® HYTRULO (efgartigimod
alfa and hyaluronidase-qvfc) for subcutaneous injection are each
indicated for the treatment of generalized myasthenia gravis in
adult patients who are anti-acetylcholine receptor (AChR) antibody
positive.
Please see the
full Prescribing
Information for VYVGART and the
full Prescribing
Information for VYVGART HYTRULO.
About Generalized Myasthenia
Gravis 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.1
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 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 and China
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 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.
References1 Behin et al. New
Pathways and Therapeutics Targets in Autoimmune Myasthenia Gravis.
J Neuromusc Dis 5. 2018. 265-277.
For further information, please
contact:Media:Ben
Petokbpetok@argenx.com
Investors:Alexandra Roy
(US)ARoy@argenx.com
Forward Looking Statement
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
“believes,” “hope,” “estimates,” “anticipates,” “expects,”
“intends,” “may,” “will,” or “should” and include statements argenx
makes concerning the expected consistency, safety, tolerability,
efficacy and quality-of-life benefits of VYVGART for patients with
gMG and CIDP; VYVGART’s potential to achieve MSE for patients; and
argenx’s planned future presentations of additional study data. 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. 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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