Regulated information – Inside information
- Trial
met primary endpoint (p ˂0.0001)
-
Well-tolerated; safety profile comparable to placebo
-
Biologics License Application on track to be submitted to U.S. Food
and Drug Administration by end of 2020
-
Conference call scheduled for today, May 26, 2020 at 8:30 a.m. EDT
(2:30 p.m. CEST)
May 26, 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 positive topline data from the pivotal
ADAPT trial of efgartigimod. ADAPT met its primary endpoint defined
as percentage of responders on the Myasthenia Gravis Activities of
Daily Living (MG-ADL) score among acetylcholine receptor-antibody
positive (AChR-Ab+) generalized myasthenia gravis (gMG) patients.
Responders are defined as having at least a two-point improvement
on the MG-ADL score for at least four consecutive weeks. Based on
these results, argenx plans to submit a Biologics License
Application (BLA) to the U.S. Food and Drug Administration (FDA) by
the end of 2020.
Highlights of topline ADAPT
data
- 67.7% of AChR-Ab+ patients treated with efgartigimod achieved
the primary endpoint compared with 29.7% on placebo
(p<0.0001).
- 63.1% of AChR-Ab+ patients responded to efgartigimod compared
with 14.1% on placebo on the Quantitative Myasthenia Gravis (QMG)
score (p<0.0001); responder defined as having at least a
three-point improvement on the QMG score for at least four
consecutive weeks.
- 40.0% of efgartigimod-treated AChR-Ab+ patients achieved
minimal symptom expression defined as MG-ADL scores of 0 (symptom
free) or 1, compared to 11.1% treated with placebo.
- Efgartigimod was well-tolerated with a safety profile that was
comparable to placebo.
“The efgartigimod data showed rapid and robust
responses in people with gMG, as well as a favorable tolerability
profile,” 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.
“Patients with this devastating disease can experience chronic and
potentially life-threatening muscle weakness that has a major
impact on their quality of life, and more treatment options are
needed. These data are very encouraging as they show efgartigimod
has potential to make a meaningful impact on daily living
activities, and we are hopeful they will lead to a new treatment
being available for the gMG community.”
“With the ADAPT trial, we set out to evaluate
efgartigimod’s ability to redefine the treatment paradigm for
people living with gMG. The data showed that efgartigimod drove
fast and deep responses, including in a proportion of patients who
achieved minimal or no symptoms after treatment. In addition, we
saw responses that lasted beyond eight or 12 weeks, supporting our
plans to offer individualized dosing schedules that are purpose-fit
to the variability in disease course that gMG patients experience,”
commented Wim Parys, M.D., Chief Medical Officer of argenx. “Based
on these data, we intend to submit a BLA for efgartigimod to the
FDA before the end of the year, taking us one step closer to
potentially making efgartigimod available to patients in 2021. All
of us at argenx want to thank the patients and healthcare providers
who participated in the ADAPT trial. ADAPT is the first pivotal
trial of efgartigimod and these data further our confidence in its
broad opportunity in other severe, IgG-mediated autoimmune
diseases.”
Additional ADAPT results, including
secondary endpoints and prespecified analyses
- In the ADAPT trial, the secondary endpoints listed below also
demonstrated statistically significant differences in the
efgartigimod arm for AChR-Ab+ patients, unless otherwise noted,
compared to placebo:
- MG-ADL responders in the overall population, including both
AChR-Ab+ and AChR-antibody negative patients (p<0.0001).
- Time on trial in clinically meaningful improvement (MG-ADL
improvement ≥2) (p=0.0001).
- Fast onset of response on MG-ADL score (onset observed in first
two weeks) (p=0.0004).
- Time to qualify for retreatment endpoint did not meet
statistical significance.
- 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.
- Of AChR-Ab+ patients who received a second treatment cycle,
70.6% were MG-ADL responders compared to 25.6% of placebo patients.
- 90% of patients enrolled in the ADAPT trial continued to the
ADAPT-Plus open-label extension study.
- Percentage of efgartigimod responders on the MG-ADL score in
the AChR-antibody negative patient population was consistent with
the AChR-Ab+ patient population, but a greater placebo response was
observed in this cohort.
Detailed data from the ADAPT trial will be
submitted for presentation at a future medical meeting.
Phase 3 ADAPT Trial DesignThe
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. Enrolled patients had a
confirmed gMG diagnosis and an MG-ADL total score of five or
greater. 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
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 as part of
the primary trial. ADAPT was designed to enable an individualized
treatment approach with an initial treatment cycle followed by a
variable number of subsequent treatment cycles. Treatment cycles
consist of four infusions of efgartigimod (10mg/kg IV) or placebo
at weekly intervals. Retreatment with additional treatment cycles
was initiated according to clinical response. 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.
After the 26-week primary ADAPT trial, patients
were eligible to roll-over into an open-label extension, ADAPT
Plus.
About Efgartigimod
Efgartigimod is a first-in-class 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.
Conference Call
DetailsManagement will host a conference call and webcast
presentation today at 2:30 p.m. Central European Summer Time (CEST)
/ 8:30 a.m. Eastern Daylight Time (EDT). To participate in the
conference call, please select your phone number below and use the
confirmation code 6295982. The webcast may be accessed on the
Investors page of the argenx website at www.argenx.com or by
clicking here.
Dial-in numbers:Please dial in 5–10 minutes
prior to 2:30 p.m. CEST / 8:30 a.m. EDT using the number and
conference ID below.
Confirmation Code: 6295982
Belgium: +32 (0)2 793 3847Belgium: 0800 484
71France: +33 (0)1 7070 0781France: 0805 101 465Netherlands: +31
(0)2 0795 6614Netherlands: 0800 023 5015UK: +44 (0) 844 481 9752UK:
0800 279 6619US: +1646 741 3167US: 1877 870 9135
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 is translating 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/.
Contacts:
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
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 safety, tolerability and efficacy of efgartigimod
and the results of the ADAPT trial; the timing of planned
regulatory submissions with the FDA and, if approved, launch in the
U.S.; the therapeutic and commercial potential of efgartigimod; the
opportunity of efgartigimod in other severe, IgG-mediated
autoimmune diseases; 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 argenx’s expectations
regarding its the inherent uncertainties associated with
competitive developments, preclinical and clinical trial and
product development activities and regulatory approval
requirements; failure to demonstrate the safety, tolerability and
efficacy of argenx’s product candidates; final and quality
controlled verification of data and the related analyses; the
expense and uncertainty of obtaining regulatory approval, including
from the U.S. Food and Drug Administration and European Medicines
Agency; the possibility of having to conduct additional clinical
trials; 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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