Results from ADAPT showed treatment with
efgartigimod provided clinically meaningful improvements in
strength and quality of life measures in generalized myasthenia
gravis (gMG)
Efgartigimod is under U.S. Food and Drug
Administration (FDA) review with December 17, 2021 Prescription
Drug User Fee Act (PDUFA) target action date
Breda, the Netherlands —Jun. 16, 2021— argenx (Euronext &
Nasdaq: ARGX), today announced that The Lancet Neurology has
published pivotal trial results from the Phase 3 ADAPT trial of
efgartigimod, an FcRn antagonist, for the treatment of adults
living with generalized myasthenia gravis (gMG). Efgartigimod is
currently under review with the U.S. Food and Drug Administration
(FDA) for the treatment of gMG with a Prescription Drug User Fee
Act (PDUFA) target action date of December 17, 2021, and if
approved, would be the first-and-only approved FcRn antagonist.
“Myasthenia gravis can have a devastating impact on a person's
life and independence, potentially affecting one's ability to
swallow, speak, walk and even breathe. In addition, each patient
experiences the course of MG differently, which can make disease
management unpredictable,” said James F. Howard Jr., M.D.,
Professor of Neurology (Neuromuscular Disease), Medicine and Allied
Health, Department of Neurology, The University of North Carolina
at Chapel Hill School of Medicine and principal investigator for
the ADAPT trial. “In the ADAPT trial, we observed clinically
meaningful improvements in the first two weeks of dosing in a
majority of patients treated with efgartigimod. These results are
important for the MG community and I am hopeful efgartigimod will
provide a first-in-class targeted therapy that can be dosed in an
individual way for people living with this chronic autoimmune
disease.”
The ADAPT trial met its primary endpoint demonstrating
significantly more acetylcholine receptor-antibody positive
(AChR-Ab+) gMG patients were responders on the Myasthenia Gravis
Activities of Daily Living (MG-ADL) score following treatment with
efgartigimod compared with placebo (67.7% vs. 29.7%; p<0.0001).
Responders were defined as having at least a two-point improvement
sustained for four or more consecutive weeks on the MG-ADL score.
Additionally, 40% of patients treated with efgartigimod achieved
minimal symptom expression defined as MG-ADL scores of zero
(symptom free) or one, compared to 11.1% of patients who received
placebo. Among AChR-Ab+ responders, 84.1% showed clinically
meaningful improvement on the MG-ADL score within the first two
weeks of treatment. The safety profile of efgartigimod was
comparable to placebo.
After completing ADAPT, 90% of participants entered ADAPT-plus,
an ongoing three-year open-label extension study evaluating the
long-term safety and tolerability of efgartigimod. In total across
ADAPT and ADAPT-plus, at least 118 patients have been on
efgartigimod therapy for 12 months or more.
“The publication of the ADAPT results provides an exciting
opportunity to share these data with the clinical community as we
aim to introduce a new treatment option for gMG patients. gMG is a
chronic, debilitating and potentially life-threatening disease
where both the disease symptoms and side effects from current
therapies can cause significant impairment on a person’s life,”
said Wim Parys, M.D., Chief Medical Officer of argenx.
"Efgartigimod is currently under review with the FDA for the
treatment of gMG, and if approved, we look forward to bringing this
therapy to MG patients who are in great need of new treatment
options.”
Phase 3 ADAPT Trial The Phase 3 ADAPT trial was
a randomized, double-blind, placebo-controlled, multi-center,
global trial evaluating the safety and efficacy of efgartigimod in
patients with gMG. A total of 167 adult patients with gMG in North
America, Europe and Japan enrolled in the trial and were treated.
Patients were eligible to enroll in ADAPT 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 efgartigimod or placebo for a
total of 26 weeks. ADAPT was designed to enable an individualized
treatment approach with an initial treatment cycle followed by a
variable number of subsequent treatment cycles. The primary
endpoint was the number of AChR-Ab+ patients who achieved a
response on the MG-ADL score defined by at least a two-point
improvement for four or more consecutive weeks.
About Efgartigimod Efgartigimod is an
investigational antibody fragment designed to reduce
disease-causing immunoglobulin G (IgG) antibodies and block the IgG
recycling process. Efgartigimod binds to the neonatal Fc receptor
(FcRn), which is widely expressed throughout the body and plays a
central role in rescuing IgG antibodies from degradation. Blocking
FcRn reduces IgG antibody levels representing a logical potential
therapeutic approach for several autoimmune diseases known to be
driven by disease-causing IgG antibodies, including: myasthenia
gravis (MG), a chronic disease that causes muscle weakness;
pemphigus vulgaris (PV), a chronic disease characterized by severe
blistering of the skin; immune thrombocytopenia (ITP), a chronic
bruising and bleeding disease; and chronic inflammatory
demyelinating polyneuropathy (CIDP), a neurological disease leading
to impaired motor function.
About Myasthenia GravisMyasthenia gravis (MG)
is a rare and chronic autoimmune disease, often causing
debilitating and potentially life-threatening muscle weakness. More
than 85% of people with MG progress to generalized MG (gMG) within
18 months, where muscles throughout the body may be affected,
resulting in extreme fatigue and difficulties with facial
expression, speech, swallowing and mobility. In more
life-threatening cases, MG can affect the muscles responsible for
breathing. There are approximately 65,000 people in the United
States and 20,000 people in Japan living with the disease.
About argenx argenx is a global immunology
company committed to improving the lives of people suffering from
severe autoimmune diseases and cancer. 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 is
evaluating efgartigimod in multiple serious autoimmune diseases.
argenx is also advancing several earlier stage experimental
medicines within its therapeutic franchises. argenx has offices in
Belgium, the United States, Japan, and Switzerland. For more
information, visit www.argenx.com and follow us on LinkedIn at
https://www.linkedin.com/company/argenx/ and Twitter at
https://twitter.com/argenxglobal.
# # #
Media Contact:
Kelsey Kirkkkirk@argenx.com
Joke Comijn (EU)jcomijn@argenx.com
Investor Contacts:
Beth DelGiaccobdelgiacco@argenx.com
Michelle Greenblattmgreenblatt@argenx.com
Forward-looking Statements
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,”
“could,” “estimates,” “anticipates,” “expects,” “intends,” “plan,”
“may,” “will,” or “should” and include statements argenx makes
concerning the Prescription Drug User Fee Act (PDUFA) target action
date of December 17, 2021; the clinical and commercial potential of
efgartigimod and clinical studies of efgartigimod or its commercial
readiness; its hope that efgartigimod will provide a first-in-class
targeted therapy that can be dosed in an individual way; its
statements regarding the therapeutic potential of efgartigimod in
patients; the therapeutic potential of its product candidates; and
the intended results of its strategy. 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, including
regulatory approval requirements and process, the effects of the
COVID-19 pandemic, argenx’s expectations regarding its the inherent
uncertainties associated with competitive developments, preclinical
and clinical trial and product development activities; argenx’s
reliance on collaborations with third parties; estimating the
commercial potential of argenx’s product candidates; argenx’s
ability to obtain and maintain protection of intellectual property
for its technologies and drugs; argenx’s limited operating history;
and argenx’s ability to obtain additional funding for operations
and to complete the development and commercialization of its
product candidates. 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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