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 diseases.”
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
- Early, sustained platelet
count increase: 38% of
VYVGART-treated participants reached a platelet count of 30x109/L
platelets at week 1 compared to 11.1% placebo
- Sustained response across
all subgroups: subgroup analyses
(including on prior ITP therapy, time since diagnosis, baseline
platelet count and age/region demographics) of patients who
achieved the primary endpoint all favored VYVGART over placebo
- International Working Group
(IWG)
responder
status: VYVGART resulted in higher
responses than placebo on analysis of IWG response criteria
- 51.2% (44/86) of VYVGART-treated
patients achieved an IWG response compared to 20% placebo
- IWG responders were defined as
having a platelet count of at least 30x109/L, a two-fold increase
in platelet count from baseline, and the absence of bleeding for
two separate, consecutive weekly visits
- Extent of disease
control: VYVGART-treated patients
experienced substantially more weeks with disease control, with 44%
sustaining response for at least 5-9 weeks (12% placebo), 28% for
at least 10-14 weeks (0% placebo), and 17% for at least 15-19 weeks
(0% placebo)
- Sustained platelet count response
was achieved in 90% (9/10) of VYVGART responders who switched from
weekly to every other week dosing (after surpassing platelet counts
of 100x109/L for three out of four consecutive visits); one placebo
patient switched to biweekly dosing but did not achieve a sustained
platelet count response
- Key pharmacodynamic
parameters: total IgG levels were reduced
in VYVGART-treated patients throughout observation period,
supporting proposed mechanism of action
- Mean IgG levels decreased steadily
over the first 4 weeks of treatment; baseline remained >60%
throughout the trial
- Consistent safety
profile: continuous weekly or biweekly dosing was
well-tolerated and did not result in any new safety signals from
those reported from previous trials
“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 DesignThe
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 17 and 24.
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:
- Infection. VYVGART may increase the risk
of infection. In a clinical study, the most common infections were
urinary tract and respiratory tract infections. More patients on
VYVGART vs placebo had below normal levels for white blood cell
counts, lymphocyte counts, and neutrophil counts. The majority of
infections and blood side effects were mild to moderate in
severity. Your health care provider should check you for infections
before starting treatment, during treatment, and after treatment
with VYVGART. Tell your health care provider if you have any
history of infections. Tell your health care provider right away if
you have signs or symptoms of an infection during treatment with
VYVGART such as 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.
- Undesirable immune reactions (hypersensitivity
reactions). VYVGART can cause the immune system to
have undesirable reactions such as rashes, swelling under the skin,
and shortness of breath. In clinical studies, the reactions
were mild or moderate and occurred within 1 hour to 3 weeks of
administration, and the reactions did not lead to VYVGART
discontinuation. Your health care provider should monitor you
during and after treatment and discontinue VYVGART if needed. Tell
your health care provider immediately about any undesirable
reactions.
Before taking VYVGART, tell your health care provider about all
of your medical conditions, including if you:
- Have a history of infection or you think you have an
infection.
- Have received or are scheduled to receive a vaccine
(immunization). Discuss with your health care provider whether you
need to receive age-appropriate immunizations before initiation of
a new treatment cycle with VYVGART. The use of vaccines during
VYVGART treatment has not been studied, and the safety with live or
live-attenuated vaccines is unknown. Administration of live or
live-attenuated vaccines is not recommended during treatment with
VYVGART.
- Are pregnant or plan to become pregnant and are breastfeeding
or plan to breastfeed.
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 ExpositionThe 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 options.
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.
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-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 medicines within its therapeutic franchises. For more
information, visit www.argenx.com and follow us on LinkedIn,
Twitter, and Instagram.
Media:
Kelsey Kirkkkirk@argenx.com
Investors:
Beth DelGiaccobdelgiacco@argenx.com
Forward Looking StatementsThe 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
potential of VYVGART® (efgartigimod alfa-fcab) for the
treatment of adult patients with ITP; the
intended results of its strategy and its collaboration partners’,
advancement of, and anticipated clinical development, data readouts
and regulatory milestones and plans, including the timing of
planned clinical trials and expected data readouts; and the timing
and outcome of regulatory filings and regulatory approvals. 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 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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