- New data
consistent with positive topline results showing rapid and
clinically meaningful responses to efgartigimod and safety profile
comparable to placebo
-
Biologics License Application on track to be submitted to U.S. Food
and Drug Administration by end of 2020
October 5, 2020 Breda, the Netherlands /
Ghent, Belgium – argenx (Euronext & Nasdaq: ARGX), a
global immunology company committed to improving the lives of
people suffering from severe autoimmune diseases and cancer, today
announced the presentation of new data from the pivotal Phase 3
ADAPT trial evaluating efgartigimod for the treatment of patients
with generalized myasthenia gravis (gMG). The presentation took
place on Saturday, October 3, 2020 at the Myasthenia Gravis
Foundation of America (MGFA) 2020 Virtual Scientific Session.
argenx previously reported positive topline results from ADAPT in
May 2020.
“Myasthenia gravis can be a very debilitating
and potentially life-threatening chronic disease in patients
leading to impairments that affect a patient’s ability to complete
normal daily activities, including walking, swallowing, chewing
food, talking or breathing easily. Efgartigimod demonstrated in
ADAPT that it is well-tolerated and that patients can experience
clinically meaningful improvements in key measures of function and
strength following treatment, including, in some, the achievement
of minimal symptom expression. These exciting results suggest that
efgartigimod as a new potential therapy for gMG patients could have
a real impact on some of the daily limitations that patients face,”
commented 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.
Highlights of New Data Presented at MGFA
2020 Virtual Scientific Session
Magnitude of response: Substantial proportion
of efgartigimod-treated acetylcholine receptor-antibody positive
(AChR-Ab+) patients showed benefit at increasing thresholds on the
Myasthenia Gravis Activities of Daily Living (MG-ADL) and
Quantitative Myasthenia Gravis (QMG) scores compared to placebo
patients at week four (one week after first treatment cycle).
- At least half of efgartigimod-treated patients showed a
five-point or greater improvement on the MG-ADL score (55.6%) and a
six-point or greater improvement on the QMG score (50.0%)
- One third (33.9%) of efgartigimod-treated patients showed a
nine-point or greater improvement on the QMG score compared to zero
patients on placebo
Repeatability of response:
Similar proportion of efgartigimod-treated AChR-Ab+ patients were
MG-ADL responders in the first (67.7% efgartigimod versus 29.7%
placebo) and second (70.6% efgartigimod versus 25.6% placebo)
treatment cycles (p<0.0001 for both cycles)
- In efgartigimod-treated patients, mean change from cycle
baseline in total MG-ADL score at week four was 4.6 in cycle one
and 5.1 in cycle two
- 78.5% (51/65) of efgartigimod-treated patients were MG-ADL
responders across treatment cycles one and two
Clinical benefit in seronegative
patients: Inclusion of QMG score in responder analysis
showed further evidence of activity in patients where AChR
antibodies were not detected (AChR-Ab-)
- 52.6% (10/19) of efgartigimod-treated patients were responders
on the QMG score compared to 36.8% (7/19) of placebo patients
- Post-hoc analysis showed that 47.4% (9/19) of
efgartigimod-treated patients were responders on both the QMG and
the MG-ADL scores compared to 21.1% (4/19) of placebo patients
Key pharmacodynamic parameters:
Total IgG and pathogenic autoantibody levels were reduced in
efgartigimod-treated AChR-Ab+ patients throughout observation
period, supporting proposed mechanism of action
- Mean maximum reductions at week four were 61.3% for total IgG
and 57.6% for AChR-Ab
- Reductions similar across IgG subtypes and in overall
population (AChR-Ab+ and AChR-Ab-)
- No reduction in albumin levels
Key Topline Data Previously Reported
from ADAPT
Topline data from ADAPT were reported in May
2020. The trial met its primary endpoint showing 67.7% of
efgartigimod-treated AChR-Ab+ gMG patients were responders on the
MG-ADL score compared to 29.7% of placebo patients (p<0.0001).
Responders were defined by having at least a 2-point change on the
MG-ADL for at least four consecutive weeks. Efgartigimod was
demonstrated to be well-tolerated with a safety profile that was
comparable to placebo.
- 63.1% of AChR-Ab+ patients were responders to efgartigimod
compared with 14.1% on placebo on the QMG score (p<0.0001);
responder defined as having at least a three-point improvement for
at least four consecutive weeks.
- 40.0% of efgartigimod-treated AChR-Ab+ patients achieved
minimal symptom expression (MG-ADL scores of 0 (symptom free) or 1)
as a result of one treatment cycle, compared to 11.1% treated with
placebo (p<0.0001).
-
84.1% of patients who were MG-ADL responders (37/44) had an onset
of effect within the first two weeks
-
In AChR-Ab+ patients who met the primary endpoint, the majority
showed a sustained response, including 88.6% who achieved a
response for at least six weeks, 56.8% for at least eight weeks and
34.1% for at least 12 weeks
“These new data on the magnitude and
repeatability of response continue to support the potential of
efgartigimod as a meaningful treatment for gMG patients. ADAPT was
also a broad trial which included patients with acetylcholine
receptor antibodies present and those without. We were pleased to
show in this presentation proof of activity in the
antibody-negative patients who are often left out of clinical
trials,” commented Wim Parys, M.D., Chief Medical Officer of
argenx. “It is particularly gratifying to present these favorable
new data at the MGFA Virtual Scientific Session, a scientific
meeting solely focused on addressing unmet needs for people living
with gMG. We look forward to submitting our Biologics License
Application for efgartigimod to the U.S. Food and Drug
Administration before the end of the year with the goal to have
efgartigimod available to patients and physicians in
2021.”
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 Gravis (MG)
MG is a rare and chronic autoimmune disease
where IgG antibodies disrupt communication between nerves and
muscles, 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. Patients with confirmed AChR antibodies account for
80-90% of the total gMG population. There are approximately 65,000
people in the United States and 20,000 people in Japan living with
the disease.
About argenxargenx 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, and cusatuzumab in hematological cancers in collaboration
with Janssen. argenx is also advancing several earlier stage
experimental medicines within its therapeutic franchises. argenx
has offices in Belgium, the United States, and Japan. For more
information, visit www.argenx.com and follow us on LinkedIn at
https://www.linkedin.com/company/argenx/.
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,”
“estimates,” “anticipates,” “expects,” “intends,” “may,” “will” or
“should” and include statements argenx makes concerning the timing
of its BLA submission to the FDA and the availability of
efgartigimod to patients and physicians and the therapeutic
potential of its product candidates. 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
argenx’s expectations regarding its the inherent uncertainties
associated with competitive developments, preclinical and clinical
trial and product development activities and regulatory approval
requirements; 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.
For further information, please contact:
Beth DelGiacco, Vice President, Investor Relations (US)+1 518
424 4980bdelgiacco@argenx.com
Joke Comijn, Director Corporate Communications & Investor
Relations (EU)+32 (0)477 77 29 44+32 (0)9 310 34
19jcomijn@argenx.com
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