Full Press Release Details
argenx Announces 2021 Corporate Priorities
and Highlights Recent Achievements Across Immunology Pipeline
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
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.
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.
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
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.
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.
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/.
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