Submission package based on positive data from
the Phase 3 ADAPT-SC trial demonstrating noninferiority of
subcutaneous (SC) efgartigimod compared to intravenously
administered VYVGART® (efgartigimod alfa-fcab) based on total
immunoglobulin G (IgG) reduction at day 29
Amsterdam, the
Netherlands – September
21,
2022 – argenx SE
(Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases, today announced the submission of a Biologics
License Application (BLA) to the U.S. Food and Drug Administration
(FDA) for SC efgartigimod (1000mg efgartigimod-PH20) for the
treatment of generalized myasthenia gravis (gMG) in adult
patients.
SC efgartigimod is co-formulated with
recombinant human hyaluronidase PH20 (rHuPH20), Halozyme's ENHANZE®
drug delivery technology. ENHANZE facilitates the subcutaneous
injection delivery of biologics that are typically administered via
intravenous (IV) infusion.
“Our vision for the gMG program is to deliver
the broadest treatment offering for people living with this
debilitating, and often overlooked disease. Every individual
experiences gMG differently, which is why we’re excited about the
possibility of introducing multiple ways to meet the needs of
patients, including with route of administration and dosing
schedule,” said Tim Van Hauwermeiren, Chief Executive Officer of
argenx. “The submission of this BLA is the latest milestone in
honoring our commitment to the gMG patient community. We look
forward to working closely with the agency through the BLA review
process and to potentially bringing forth another first-in-class
option for gMG patients.”
The BLA submission package includes data from
the Phase 3 ADAPT-SC study evaluating the noninferiority of the
pharmacodynamic (PD) effect of SC efgartigimod as compared with
intravenously administered VYVGART in adult patients with gMG. The
majority of enrolled patients were positive for acetylcholine
receptor (AChR) antibodies, but the trial also included patients
where AChR antibodies were not detected.
ADAPT-SC met its primary endpoint (p< 0.0001)
of total IgG reduction from baseline at day 29 demonstrating
noninferiority of SC efgartigimod to VYVGART. Patients treated with
SC efgartigimod achieved mean total IgG reduction of 66.4% from
baseline at day 29, compared to 62.2% reduction with VYVGART.
Results were consistent across the overall population, including
those with AChR antibodies and patients where AChR antibodies were
not detected. Further, 69.1% of patients treated with SC
efgartigimod were responders on the Myasthenia Gravis Activities of
Daily Living (MG-ADL) score. Responders are defined as having at
least a two-point improvement on the MG-ADL score for at least four
consecutive weeks. 65.5% of patients treated with SC efgartigimod
were responders on the Quantitative Myasthenia Gravis (QMG) score.
Responders are defined as having at least a three-point improvement
on the QMG score for at least four consecutive weeks. Minimal
symptom expression (MSE), a measure of symptom-free status, was
achieved in 37% of SC efgartigimod-treated patients after one
treatment cycle. Onset of effect was also consistent with the Phase
3 ADAPT study.
The safety profile for SC efgartigimod was
consistent with the ADAPT study. It was generally well-tolerated;
the most frequent adverse event being injection site reactions
(ISRs), commonly observed with biologics administered
subcutaneously. All ISRs were mild to moderate and resolved over
time. After completing ADAPT-SC, 95% of participants entered
ADAPT-SC+, a three-year open-label extension study evaluating the
long-term safety and tolerability of SC efgartigimod.
Phase 3
ADAPT-SC Trial
Design
The Phase 3 ADAPT-SC trial was a multicenter,
randomized, open-label, parallel-group study evaluating the
noninferiority of the pharmacodynamic (PD) effect of SC
efgartigimod (1000mg efgartigimod-PH20) as compared with VYVGART
(10mg/kg) in patients with gMG. The pharmacodynamic effect as
measured by percent change from baseline in total IgG levels at day
29, one week after the last dose of IV or SC efgartigimod, served
as the primary endpoint in the ADAPT-SC trial. The correlation
between total IgG reduction and clinical benefit in gMG was
demonstrated in a Phase 2 trial and the Phase 3 ADAPT trial, which
served as the basis for approval of VYVGART in the U.S., Japan and
Europe. Safety, clinical efficacy, immunogenicity and
pharmacokinetics (PK) were also assessed.
A total of 110 adult patients with gMG in North
America, Europe and Japan enrolled in the ADAPT-SC trial and were
treated. Inclusion criteria of the trial were the same as the Phase
3 ADAPT trial of VYVGART; enrolled patients had a confirmed gMG
diagnosis and an MG-ADL total score of at least 5 with greater than
50% of the total score attributed to non-ocular symptoms, at
screening and baseline. Patients were on a stable dose of at least
one gMG treatment prior to randomization, including
acetylcholinesterase inhibitors, corticosteroids or nonsteroidal
immunosuppressive drugs, and were required to remain on that stable
dose throughout the primary trial. Patients were eligible to enroll
in ADAPT-SC regardless of antibody status, including patients with
AChR antibodies (AChR-Ab+) and patients where AChR antibodies were
not detected.
Patients were randomized in a 1:1 ratio to
receive SC efgartigimod or IV efgartigimod for one treatment cycle
consisting of four doses at weekly intervals. The total study
duration was approximately 12 weeks, including seven weeks of
follow-up after the treatment cycle.
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 Efgartigimod Efgartigimod
is an antibody fragment designed to reduce pathogenic
immunoglobulin G (IgG) antibodies by binding to the neonatal Fc
receptor and blocking the IgG recycling process. Efgartigimod is
being investigated in several autoimmune diseases known to be
mediated by disease-causing IgG antibodies, including neuromuscular
disorders, blood disorders, and skin blistering diseases, in both
an intravenous and subcutaneous (SC) formulation. SC efgartigimod
is co-formulated with recombinant human hyaluronidase PH20
(rHuPH20), Halozyme's ENHANZE® drug delivery technology.
About VYVGART
VYVGART® (efgartigimod alfa-fcab) 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).
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 months1, where muscles
throughout the body may be affected. Patients with confirmed AChR
antibodies account for approximately 85% of the total gMG
population1.
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-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 submission of the Biologics
License Application to the U.S. Food and Drug Administration for
Subcutaneous (SC) Efgartigimod for Treatment of Generalized
Myasthenia Gravis and the long-term safety and tolerability of SC
Efgartigimod. 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.
1 Behin et al. New Pathways and Therapeutics Targets in
Autoimmune Myasthenia Gravis. J Neuromusc Dis 5. 2018. 265-277
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