Full Press Release Details
argenx Highlights Data Showing
Patient Impact Across Multiple Immunology Programs at 2024 American Association of Neuromuscular & Electrodiagnostic Medicine Annual
Meeting and Myasthenia Gravis Foundation of America Scientific Sessions
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
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
| 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
| 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
| 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 # 144 Session I Session II |
| Combined Analyses of Participants With Anti-Acetylcholine Receptor Seronegative Generalized Myasthenia Gravis Treated With Efgartigimod Across Clinical Studies | Poster # 146 Session 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 # 165 Session I Session III |
| Efficacy and Safety of Subcutaneous Efgartigimod PH20SC in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE Trial Subgroup Analysis | Poster # 176 Session I Session II |
| Efficacy and Safety of Subcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: ADHERE/ADHERE+ Trial | Poster # 177 Session I Session III |
| Safety Profile of Intravenous Efgartigimod From Clinical Trials in Immunoglobulin G-Mediated Autoimmune Diseases | Poster # 180 Session I Session II |
| Safety Profile of Subcutaneous Efgartigimod PH20 From Clinical Trials in Immunoglobulin G-Mediated Autoimmune Diseases | Poster # 181 Session I Session III |
| Empasiprubart (ARGX-117) in Multifocal Motor Neuropathy: Initial Safety and Efficacy Data of the Phase 2 ARDA Study | Poster # 198 Session 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 # 212 Session 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 # 237 Session I Session II |
| Risk of Serious Infections and Malignancies in Adult Myasthenia Gravis Patients: A US Claims Database Study | Poster # 251 Session I Session II |
| Chronic Steroid Toxicity in Adults With Myasthenia Gravis in the United States Based on Electronic Health Records | Poster # 263 Session I Session II |
| Subcutaneous Efgartigimod PH20 in Chronic Inflammatory Demyelinating Polyneuropathy: Key Secondary Outcomes from the ADHERE Trial | Poster # 280 Session 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 # 293 Session I Session II |
| COVID-19 Vaccination Response in Participants Receiving Efgartigimod IV or Efgartigimod PH20 SC in ADAPT+ or ADAPT-SC+ | Poster # 298 Session I Session III |
| Steroid Use, Toxicity, and Monitoring in Patients with Chronic Inflammatory Demyelinating Polyneuropathy: A Survey of Neurologists in The United States | Poster # 306 Session I Session III |
| Clinical Outcomes, Disease Course, and Quality of Life in Patients With Multifocal Motor Neuropathy: iMMersioN, Study in Progress | Poster # 307 Session 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
(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 INFORMATION
Do 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
| 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
What are the common side effects
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.
Prescribing Information for VYVGART and talk to your doctor.
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 INFORMATION
Do 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. |
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. |