argenx Initiates Second Cohort of Phase 2 ARDA Study of Empasiprubart in Multifocal Motor Neuropathy
20 Juin 2023 - 7:00AM
- Independent Data
Monitoring Committee recommended study continuation based on the
favorable safety profile observed in the first dose cohort
- Early efficacy
signals support proof-of-concept of empasiprubart in multifocal
motor neuropathy
Regulated
Information/Inside Information
Amsterdam, the Netherlands—June 20,
2023—argenx SE (Euronext & Nasdaq: ARGX), a global
immunology company committed to improving the lives of people
suffering from severe autoimmune diseases, today announced its plan
to advance to a second dose cohort with the Phase 2 ARDA study of
empasiprubart (ARGX-117) in multifocal motor neuropathy (MMN). The
decision follows a planned interim analysis of the first dose
cohort by an Independent Data Monitoring Committee (IDMC) meeting
held on June 19, 2023.
“We are encouraged by the favorable safety
profile and early efficacy signals from the ARDA study, as well as
the IDMC recommendation to advance the study to the next cohort.
Looking forward, we hope to build on the promising safety and
efficacy we observed in the first cohort while populating our PK/PD
model to understand the full potential of empasiprubart in MMN,”
commented Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx.
“People living with MMN continue to face significant burden
associated with this chronic autoimmune disease, including
progressive muscle weakness, which can lead to an inability to walk
or move one’s arms or hands. Our mission is to transform treatment
paradigms for autoimmune patients by changing expectations on what
well-controlled means. Based on these data, we are hopeful that we
can accomplish this in MMN and other autoimmune indications with
our first-in-class C2 inhibitor.”
The IDMC reviewed interim safety data from all
patients (n=22) currently enrolled in the first cohort of the ARDA
study, including nine patients who completed the full 16-week
treatment period. The IDMC confirmed a favorable safety and
tolerability profile of empasiprubart consistent with results from
the Phase 1 study and recommended advancing to the second cohort.
An early efficacy assessment of all 22 patients supports
proof-of-concept of empasiprubart in MMN. The data showed distinct
separation between treated patients and placebo based on a suite of
clinical outcome measures, including time to IVIg retreatment.
In total, the ARDA study is expected to enroll
48 patients across two cohorts. The study’s objective, in addition
to assessing safety and efficacy of empasiprubart, is to populate a
PK/PD model to inform the Phase 3 study dose selection. Based on
the IDMC recommendation to continue enrollment, argenx will aim to
build on the promising efficacy and safety observations from the
first cohort by evaluating a next dose level of empasiprubart.
Phase 2 ARDA Study Design
The Phase 2 ARDA study is a randomized,
double-blinded, placebo-controlled multicenter study to evaluate
the safety and tolerability, efficacy, pharmacokinetics,
pharmacodynamics, and immunogenicity of two dose regimens of
empasiprubart in adults with multifocal motor neuropathy (MMN). The
study consists of an IVIg dependency and monitoring period and two
16-week treatment cohorts of 24 MMN patients receiving
empasiprubart or placebo in a 2x1 randomization. The dosing for
Cohort 2 will be established after a planned interim analysis of
the first nine patients to complete the 16-week treatment period
from Cohort 1. The primary endpoint is safety and tolerability.
Additional endpoints include time to IVIg retreatment, biomarker
analyses of C2 levels, and changes in measurements on key clinical
efficacy scores (modified medical research council (mMRC)-14 sum
score, grip strength, MMN-RODS) as well as several patient-reported
quality of life outcome measures.
About
Empasiprubart
Empasiprubart (ARGX-117) is a first-in-class
humanized sweeping antibody that binds specifically to C2 thereby
blocking both the classical and lectin pathways of the complement
cascade. By blocking upstream complement activity, empasiprubart
has the potential to reduce tissue inflammation representing a
broad pipeline opportunity across multiple severe autoimmune
indications. In addition to multifocal motor neuropathy,
argenx is planning to evaluate empasiprubart in delayed graft
function following kidney transplant and dermatomyositis.
About
Multifocal
Motor
Neuropathy
Multifocal motor neuropathy (MMN) is a rare,
chronic autoimmune disease of the peripheral nervous system. The
disease is characterized by slowly progressive, asymmetric muscle
weakness mainly of the hands, forearms and lower legs. MMN is often
associated with anti-GM1 IgM autoimmunity, leading to activation of
the classical complement pathway, driving subsequent axon damage.
High-dose IV immunoglobulin (IVIg) is the only approved treatment
for MMN and patients typically experience disease progression
despite therapy, indicating an unmet need for efficacious and
better tolerated therapeutic options.
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, the EU and the UK. 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.
For further information, please
contact:
Media:Erin MurphyEMurphy@argenx.com
Investors:Alexandra Roy
(US)ARoy@argenx.com
Lynn Elton (EU)LElton@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,” “hope,” “estimates,” “anticipates,” “expects,”
“intends,” “may,” “will,” or “should” and include statements argenx
makes concerning the safety profile and efficacy signals from the
ARDA study; the prospects of empasiprubart as a treatment for MMN
and other indications; and the expected enrollment, objectives and
results of the ARDA study. 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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