- Submitted Biologics License Application (BLA) to U.S. Food and
Drug Administration (FDA) for efgartigimod in generalized
myasthenia gravis (gMG)
- Initiated 50-patient gMG bridging study of subcutaneous (SC)
efgartigimod based on FDA feedback
- Enrolled first 30 patients, necessary for go/no-go decision, in
ADHERE trial of SC efgartigimod for chronic inflammatory
demyelinating polyneuropathy (CIDP)
- Announced interim data from Phase 2 CULMINATE trial of
cusatuzumab in development with Janssen
Breda, the Netherlands – Jan. 8, 2021 – argenx
(Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe
autoimmune diseases and cancers, today announced its 2021 corporate
priorities and highlighted recent achievements from its late-stage
immunology pipeline driven by its FcRn antagonist, efgartigimod.
Additionally, the Company announced interim data from the Phase 2
CULMINATE trial of cusatuzumab in development with Cilag GmbH
International, an affiliate of Janssen, and provided financial
guidance for 2021.
argenx previously announced an exclusive license agreement with
Zai Lab Limited (“Zai Lab”) for the development and
commercialization of efgartigimod in Greater China and the
acceleration of efgartigimod development through Phase 2
proof-of-concept trials in new autoimmune indications. Zai Lab will
also contribute Chinese patients to argenx’s global Phase 3 trials
of efgartigimod. Under the terms of the agreement, argenx will
receive $175 million in collaboration payments comprised of upfront
Zai Lab equity, a guaranteed development cost-sharing payment, and
a milestone payment upon U.S. efgartigimod approval. argenx will
also be eligible for tiered royalties based on annual net sales of
efgartigimod in Greater China.
“We are excited to enter a new chapter for argenx as we look
toward commercialization and achieving our mission of reaching
patients with debilitating rare diseases. We’ve submitted a BLA to
the FDA for efgartigimod in gMG and expect to have global
efgartigimod trials ongoing this year in six indications and two
formulations. We hope to continue to demonstrate the broad
opportunity of our FcRn antagonist within autoimmune diseases in
2021 and beyond,” said Tim Van Hauwermeiren, Chief Executive
Officer of argenx. “In parallel, establishing global commercial
infrastructure within the U.S. and Japan continues to be a top
priority. Now through our collaboration with Zai Lab in China and
with the appointment of a general manager in Europe, we’ve
solidified and accelerated our capabilities to bring efgartigimod
and our future immunology candidates to patients worldwide.”
2021 Corporate Priorities and Recent
Progress
The Company will continue its transition to a fully integrated
immunology company by executing on three corporate priorities in
2021, including: preparation for the potential FDA approval and
U.S. commercial launch of efgartigimod for the treatment of
patients with gMG; the progression of its clinical-stage autoimmune
pipeline; and the continued growth of its broad and differentiated
pipeline through its Immunology Innovation Program.
- Preparation for potential FDA approval and global
commercial launch of efgartigimod for the treatment of patients
with gMG
- Submitted BLA to FDA for efgartigimod for treatment of gMG and
continued preparations for other global regulatory submissions
- On track to submit application to Japan’s Pharmaceuticals and
Medical Devices Agency (PMDA) in first half of 2021
- On track to submit to European Medicines Agency (EMA) in second
half of 2021
- Submission in China expected to occur shortly following
potential approval in U.S.
- Initiated bridging study for SC efgartigimod in gMG based on
association between total IgG reduction and clinical benefit, and
feedback from the FDA. The study is a registrational,
non-inferiority trial comparing the pharmacodynamic effect of
1000mg SC efgartigimod with 10mg/kg IV efgartigimod and is expected
to enroll approximately 50 patients.
- Commercial preparation activities are underway and on track for
potential 2021 launch, including continued engagement with key
stakeholders, commercial inventory build, milestone-based hiring of
field force around potential BLA acceptance and FDA approval, and
development of a patient services program.
- Appointed Anant Murthy, Ph.D., as General Manager of argenx
Europe. In this role, Dr. Murthy will establish the commercial
infrastructure for a European launch and lead market development
activities in advance of a potential European Medicines Agency
(EMA) approval of efgartigimod. Dr. Murthy brings ~20 years of
international experience to argenx, most recently as Head of Market
Access for EMEA and Canada and the General Manager of multiple
European countries for Alnylam Pharmaceuticals.
- Progress clinical-stage autoimmune pipeline, including
seven expected global trials of efgartigimod and Phase 1 trial of
first-in-class C2 antibody ARGX-117
- ADVANCE (IV) and ADVANCE SC trials ongoing evaluating IV and SC
efgartigimod in patients with primary immune thrombocytopenia
(ITP); global program expected to support registration of both
formulations
- ADDRESS registrational trial ongoing evaluating SC efgartigimod
in patients with pemphigus vulgaris (PV) and pemphigus foliaceous
(PF)
- ADHERE trial ongoing evaluating SC efgartigimod in chronic
inflammatory demyelinating polyneuropathy (CIDP);
- Completed enrollment of first 30 patients
- Decision whether to expand enrollment up to 130-140 patients
expected in first quarter of 2021
- Clinical trials in fifth and sixth indications of efgartigimod
to begin enrollment in 2021
- Ongoing Phase 1 healthy volunteer trial of IV and SC ARGX-117,
a first-in-class C2 antibody, to evaluate safety and tolerability
and establish dosing regimen
- Data expected in mid-2021, after which argenx plans to launch
Phase 2 proof-of-concept trials in severe autoimmune diseases,
including multifocal motor neuropathy (MMN)
- Continued investment in broad and differentiated
pipeline through Immunology Innovation Program
- Preclinical work ongoing in early-stage pipeline, including
continued progress on ARGX-118 and ARGX-119, and the optimization
of ARGX-120
- Commitment to expand pipeline at cadence of one new candidate
per year from Immunology Innovation Program
Interim
Data from Cusatuzumab Phase 2 CULMINATE
Trial Development
of cusatuzumab in acute myeloid leukemia (AML) remains ongoing as
part of a global collaboration and license agreement with Cilag
GmbH International, an affiliate of Janssen.
The Phase 2 CULMINATE trial (NCT04023526) is evaluating
cusatuzumab in combination with azacitidine in newly-diagnosed,
elderly AML patients who are ineligible for intensive chemotherapy.
A total of 103 patients were randomized to receive either 10mg/kg
(n=51) or 20mg/kg (n=52) cusatuzumab plus azacitidine as part of a
dose identification. The 20mg/kg dose has been selected for ongoing
and future trials.
A pre-planned interim analysis was conducted of the 52 patients
(46.2% adverse ELN risk classification) receiving 20mg/kg
cusatuzumab plus azacitidine treatment (intent-to-treat population
(ITT)). The results from the ITT analysis showed a complete
remission (CR) rate of 27% (14/52) and composite complete remission
(CRc), including CRs with incomplete hematologic recovery, rate of
40% (21/52). The 30-day mortality rate of the ITT population was
9.6% (5/52). In a cohort where patients received at least two
treatment cycles (20mg/kg cusatuzumab plus azacitidine), 42%
(14/33) achieved CR and 64% (21/33) achieved CRc.
Cusatuzumab was observed to be well-tolerated and the safety
profile was consistent with prior studies. Final results from the
CULMINATE trial will be presented in a peer-reviewed forum.
The decision to initiate additional studies in the development
of cusatuzumab, under the collaboration, will be determined
following review of data from the ongoing Phase 1b ELEVATE trial
(NCT04150887), which is evaluating cusatuzumab in combination with
venetoclax and azacitidine in newly-diagnosed, elderly patients
with AML who are ineligible for intensive chemotherapy.
Financial Guidance
As of December 31, 2020, argenx had approximately $2.0 billion
in cash, cash equivalents and current financial assets. This
preliminary cash balance does not include expenses or proceeds from
recently announced business development transactions, including the
purchase of a priority review voucher from Bayer HealthCare
Pharmaceuticals, Inc. and the exclusive license agreement with Zai
Lab for efgartigimod in Greater China.
Based on current plans to fund anticipated operating expenses
and capital expenditures, argenx expects its cash burn to increase
significantly in 2021, approximately doubling compared to 2020. The
increased spend will support the Company’s transition to an
integrated immunology company in 2021, including the build-out of
global commercial infrastructure and drug product inventory ahead
of the expected launch of efgartigimod in gMG in the U.S, the
advancement of its clinical-stage pipeline, including seven
expected global trials of efgartigimod, and the continued
investment in its Immunology Innovation Program.
J.P. Morgan Healthcare Conference Presentation and
Webcast
argenx CEO Tim Van Hauwermeiren, will present these updates at
the virtual 39th Annual J.P. Morgan Healthcare Conference on
Monday, January 11, 2020 at 8:20 a.m. ET, followed by a question
and answer session.
The live webcast of the presentation and question and answer
session that follows may be accessed on the homepage of the argenx
website at www.argenx.com. A replay of the webcast will be
available for 90 days on the argenx website.
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 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, 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/.
Media:
Kelsey Kirkkkirk@argenx.com
Investors:
Beth DelGiacco bdelgiacco@argenx.com
Joke Comijn (EU)jcomijn@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 its 2021 business and
financial outlook and related plans, including with respect to
financial guidance and cash burn; the therapeutic potential of its
product candidates; the intended results of its strategy; the
expected benefits of its collaborations, including with respect to
the exclusive license agreement with Zai Lab; its and its
collaboration partners’ clinical development and regulatory plans,
including the timing, design and outcome of ongoing and planned
clinical trials and preclinical activities and the timing and
outcome of regulatory filings and approvals; and the timing and
progress of commercialization activities. 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 the
effects of the COVID-19 pandemic, the inherent uncertainties
associated with 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.
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