Full Press Release Details
argenx to Present Pivotal ADVANCE Trial Data
During ASH Plenary Session Highlighting VYVGART (efgartigimod alfa-fcab) as Potential New Treatment Modality for Immune
First immune thrombocytopenia (ITP) plenary
selection in 15 years underscores significant unmet need in this rare, serious autoimmune bleeding disease
VYVGART showed rapid, clinically and statistically
significant improvements in platelet counts compared with placebo; safety profile consistent with previous trials
Topline data from ADVANCE-SC trial of subcutaneously
(SC) administered VYVGART for ITP expected in second half of 2023
Amsterdam, the Netherlands - December
10, 2022 - argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering
from severe autoimmune diseases, today announced that data from its Phase 3 ADVANCE trial will be presented during the plenary session
at the 64th American Society of Hematology (ASH) Annual Meeting & Exposition in New Orleans, LA (Sunday, December 11,
2022, 2-4pm CT). The ADVANCE study is the first of two registrational trials evaluating the efficacy, safety and tolerability of VYVGART
(efgartigimod alfa-fcab) for the treatment of adult patients with primary ITP.
"We are very excited to present our ITP
data during the plenary session at ASH, giving us the opportunity to highlight the potential of a new approach to treating this rare,
complex disease. People living with ITP need more treatment options with new mechanisms of action that target the underlying biology
of the disease, and we look forward to sharing our findings in helping to address this gap with the broader ITP community," said
Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. "ADVANCE is the second Phase 3 clinical trial in which VYVGART has demonstrated
a strong clinical benefit, underscoring our belief in the breadth of potential for this asset in a range of high-need IgG-mediated autoimmune
Topline data from ADVANCE were reported in May
2022. The trial met its primary endpoint demonstrating a significantly higher proportion (p=0.0316) of VYVGART-treated chronic ITP patients
achieved a sustained platelet response (17/78; 21.8%) compared to placebo (2/40; 5%). Sustained platelet response was defined as having
platelet counts greater than or equal to 50x109/L on at least four of the last six scheduled visits between weeks 19 and 24
of treatment. Key platelet-derived secondary endpoints (first two secondary endpoints) were also met. VYVGART was well-tolerated in this
24-week chronic dosing study and the observed safety and tolerability profile was consistent with previous clinical trials.
Highlights From ASH Plenary Session
"I am honored to deliver this oral presentation
on behalf of my co-investigators at ASH, one of the most prestigious hematology meetings, to provide my peers with additional details
on the promising results from the ADVANCE study," stated Catherine Broome, M.D., Associate Professor of Medicine at Georgetown
University Lombardi Comprehensive Cancer Center, and Principal Investigator in the ADVANCE trial. "Along with the previously
reported positive efficacy, safety and tolerability data from the trial, further analyses show efgartigimod demonstrated rapid and sustained
reduction in IgG autoantibodies, which correlated with platelet count response, as well as consistent improvement over placebo across
each evaluated weekly timepoint. The data generated to date give us optimism that this therapy could provide a new tool in the treatment
of ITP, and we look forward to seeing results from the subcutaneous study in 2023."
The Phase 3 ADVANCE IV trial is the first of
two registrational trials being conducted as part of the ongoing ITP development program. ADVANCE-SC is evaluating SC VYVGART for the
treatment of primary ITP. Topline data from the ADVANCE-SC study are expected in the second half of 2023.
Phase 3 ADVANCE Trial Design
The Phase 3 ADVANCE trial was a randomized, double-blind,
placebo-controlled, multicenter, global trial evaluating the efficacy and safety of VYVGART in adult patients with chronic or persistent
primary ITP. A total of 131 adult patients with primary ITP in North America, Europe and Japan enrolled
in the trial and received VYVGART or placebo for a total of 24 weeks as part of the primary trial. Enrolled patients had a confirmed
ITP diagnosis and a mean entry platelet count of less than 30x109/L. Patients were on a stable dose of at least one ITP treatment
prior to randomization and had received at least one prior therapy. Concomitant medications permitted included corticosteroids, nonsteroidal
immunosuppressive drugs, fostamatinib or TPO-RAs. If patients were on watch and wait' at baseline, they had to have received at least
2 prior treatments for ITP.
Patients were randomized in a 2:1 ratio to receive
VYVGART or placebo for a total of 24 weeks as part of the primary trial. Randomized patients received weekly infusions from weeks 1-4
and were eligible to adjust frequency to bi-weekly depending on platelet count. Administration frequency was fixed from study visits
16-24. The primary endpoint was measured by the proportion of patients with chronic ITP with a sustained
platelet count response defined as achieving platelet counts of greater than or equal to 50x109/L for at least four of the
last six scheduled visits between weeks 19 and 24. Patients who received rescue therapy at week 12 or later, or for whom dose and/or
frequency of concurrent ITP therapies increased at week 12 or later, were considered non-responders. Key
secondary endpoints included extent of disease control over 24-week treatment period, proportion of overall population with sustained
platelet count response, incidence and severity of WHO-classified bleeding events and an extended primary endpoint analysis between weeks
See the full Prescribing Information for
VYVGART in the U.S., which includes the below Important Safety Information. For more information related to VYVGART in Japan, visit argenx.jp.
Important Safety Information for VYVGART (efgartigimod
alfa-fcab) intravenous (IV) formulation (U.S. prescribing 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).
What is the most important information I should know about VYVGART?
VYVGART may cause serious side effects, including:
Before taking VYVGART, tell your health care provider about all of
your medical conditions, including if you:
Tell your health care provider about all the medicines you take, including
prescription and over-the-counter medicines, vitamins, and herbal supplements.
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.
About American Society of Hematology (ASH)
Annual Meeting and Exposition
The 64th ASH Annual Meeting and Exposition is
scheduled to take place December 10-13, 2022 at the Ernest N. Morial Convention Center in New Orleans, Louisiana. This in-person event
will be broadcast virtually. The ASH 2022 Annual Meeting abstracts are available at: https://www.hematology.org/meetings/annual-meeting/abstracts.
About Immune Thrombocytopenia (ITP)
Immune thrombocytopenia (ITP) is an autoimmune
disorder where immunoglobulin G (IgG) autoantibodies destroy platelets and reduce platelet production, which can lead to an increased
risk of excessive bleeding and bruising. In severe cases, frequent bleeding events can cause anemia or even brain hemorrhage in rare
cases. ITP is also associated with debilitating fatigue and significant impacts on mental health, including anxiety, fear and depression.
Many ITP patients are inadequately controlled on current therapies so there remains a significant unmet need for additional treatment
About VYVGART (efgartigimod alfa-fcab)
VYVGART is a human IgG1 antibody fragment that
binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating immunoglobulin G (IgG) autoantibodies. It is the
first and only approved FcRn blocker. VYVGART is approved in the United States and Europe 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). VYVGART is being studied in adults
with primary immune thrombocytopenia (ITP) and other IgG autoantibody-mediated diseases.
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-and- only approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan
and the EU. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental