Japanese Marketing Authorization Application (J-MAA) for efgartigimod accepted for review by
Japan’s Pharmaceuticals and Medical Device Agency (PMDA) for generalized myasthenia gravis
(gMG)
Management to host conference call today at 2:30 pm CEST (8:30 am ET)
May 14, 2021
Breda, the Netherlands – argenx (Euronext & Nasdaq: ARGX), a global immunology company
committed to improving the lives of people suffering from severe autoimmune diseases and cancer,
today reported financial results for the first quarter 2021 and provided a business update.
“We’ve had a strong start to 2021 with the acceptance for review of the BLA and J-MAA for
efgartigimod in gMG by the regulatory agencies in the U.S. and Japan. The submissions in China and
the EU are on track and we are well-positioned for a global launch of our first-in-class FcRn antagonist.
We are building an exceptional team with significant launch experience in neurology and rare disease
and hope to reach patients this year,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.
“Efgartigimod has the potential to help people living with gMG as well as several other severe
autoimmune diseases mediated by IgG autoantibodies. Our team is advancing registrational trials
across four indications with plans to start enrollment in two additional efgartigimod indications this
year. We are also broadening our reach within autoimmunity with our first-in-class C2 inhibitor, ARGX117, from which we will have Phase 1 data mid-year. To complement our clinical pipeline, we continue
to invest in our discovery capabilities through our Immunology Innovation Program and strategic
technology partnerships that position us well to generate long-term value for shareholders. We are
closer each day to building an integrated, innovative, global immunology organization with the goal
of impacting the lives of patients,” concluded Mr. Van Hauwermeiren.
FIRST QUARTER 2021 AND RECENT BUSINESS UPDATE
Commercial preparations on-track for global launch of IV efgartigimod for gMG, including regulatory
submissions, initial salesforce hires and key stakeholder engagement efforts
- Biologics License Application (BLA) for IV efgartigimod for treatment of gMG accepted for
review by U.S. Food and Drug Administration (FDA) with target action date of December 17,
2021 under Prescription Drug User Fee Act (PDUFA)
- J-MAA submitted to Japan’s PMDA and accepted for review with anticipated Japan
commercial launch in 2022
- MAA expected to be filed with European Medicines Agency (EMA) in second half of 2021
- Zai Lab Limited to discuss potential accelerated regulatory pathway for approval in China with National Medical Products Administration (NMPA)
- Commercial readiness activities on-track, including:

Continued build-out of global commercial organization, including hiring of U.S.
regional business directors during first quarter
o Launched pre-approval access (PAA) program in March 2021 in U.S., Canada and
Europe to open availability of efgartigimod to people living with gMG who meet the pre-approval access program criteria
Immunology pipeline advancing with five ongoing registrational trials of efgartigimod and initial upcoming data from second potential pipeline-in a product candidate, ARGX-117
- Enrollment ongoing in five registrational trials across four indications, including ADAPT-SC
(gMG), ADHERE (chronic inflammatory demyelinating polyneuropathy or CIDP), ADVANCE and ADVANCE-SC (primary immune thrombocytopenia or ITP), and ADDRESS (pemphigus)
o Go-forward decision confirmed in February 2021 in ADHERE trial evaluating
subcutaneous (SC) efgartigimod in CIDP based on evaluation of interim safety and
efficacy assessments that surpassed pre-defined threshold
o Enrollment in trials for fifth and sixth indications to begin in 2021
o Additional efgartigimod indications to be evaluated as part of collaboration with Zai Lab Limited
- Data expected mid-year from Phase 1 trial of C2 inhibitor, ARGX-117; Phase 2 dosing plan to be identified for indications, including multifocal motor neuropathy (MMN)
o Phase 2 trial of MMN on track to start by end of 2021
- Combination trials of cusatuzumab remain ongoing for treatment of acute myeloid leukemia (AML) as part of global collaboration and licensing agreement with Cilag GmbH International, an affiliate of Janssen
o Decision to initiate additional cusatuzumab studies under collaboration will be
determined following review of all available data
Immunology Innovation Program (IIP) continues to grow pipeline through wholly-owned development, partnered opportunities, asset-centric spinoff companies and the addition of strategic technology capabilities
- Preclinical work ongoing in early-stage pipeline, including ARGX-118, ARGX-119 and ARGX-120
- 15-20 discovery programs under evaluation at any point in time that have emerged from IIP
- Initiated collaboration and license agreement with Elektrofi to explore new subcutaneous formulations for current and future pipeline candidates, including efgartigimod
o Secured exclusivity for FcRn and one additional target
- Ongoing development of ARGX-112 (LEO Pharma), ARGX-114 (AgomAb), ARGX-115 (ABBV151, AbbVie) and ARGX-116 (Staten Biotech) by IIP collaboration partners
Strong balance sheet and expanded Board of Directors support transition into integrated, global immunology organization
- Completed public offering of 3,593,750 ordinary shares in February 2021 with gross proceeds of $1.15 billion
- Implemented transition agreement for Chief Financial Officer Eric Castaldi as part of evolution to commercial-stage company; recruitment efforts ongoing for U.S.-based successor
- Proposed resolutions presented during Annual General Meeting of Shareholders were approved, including:

o Appointment to Board of Directors of Yvonne Greenstreet, President and Chief
Operating Officer of Alnylam
o Re-appointment of Anthony Rosenberg to Board of Directors
o Approval of new remuneration policy
argenx to host virtual R&D Day on July 20, 2021 to share long-term corporate vision, disclose
additional potential efgartigimod indications and provide updates across immunology pipeline.

FIRST QUARTER 2021 FINANCIAL RESULTS (CONSOLIDATED)
Three Months Ended March 31,
(in thousands of $ except for shares and
EPS) 2021 2020 Variance
Revenue $ 158,155 $ 21,139 $ 137,017
Other operating income 9,260 4,672 4,588
Total operating income 167,415 25,811 141,604
Research and development expenses (122,328) (104,661) (17,666)
Selling, general and administrative expenses (56,253) (27,609) (28,644)
Total operating expenses (178,580) (132,270) (46,310)
Change in fair value on non-current financial
assets 11,152 0 11,152
Operating loss $ (13) $ (106,459) $ 106,446
Financial income/(expenses) (420) (3,591) 3,171
Exchange gain/(losses) (28,817) 22,985 (51,802)
Loss before taxes $ (29,249) $ (87,064) $ 57,815
Income taxes (11,184) (1,200) (9,984)
Loss for the period and total
comprehensive loss $ (40,433) $ (88,264) $ 47,831
Weighted average number of shares
outstanding 49,946,515 42,786,194
Basic and diluted profit/(loss) per share (in
$) (0.81) (2.06)
Net increase/(decrease) in cash, cash
equivalents and current financial assets
compared to year-end 2020 and 2019
910,903 (70,318)
Cash, cash equivalents and current financial
assets at the end of the period 2,907,355 1,430,343

DETAILS OF THE FINANCIAL RESULTS
As of January 1, 2021, the Company changed its functional and presentation currency from euro to U.S. dollars, which results in reporting its financial highlights in U.S. dollar as compared to euro in prior
periods. Historical financials have been converted at the average exchange rate of the related period.
Cash, cash equivalents and current financial assets totaled $2,907.4 million on March 31, 2021, compared to $1,996.5 million on December 31, 2020. The increase in cash, cash equivalents and current financial assets resulted primarily from (i) the closing of a global offering, which resulted in
the receipt of $1,092.1 million in net proceeds in February 2021, (ii) the net receipt of a $73.1 million non-creditable, non-refundable development cost-sharing payment in the form of newly issued Zai Lab shares received as part of the strategic collaboration for efgartigimod in Greater China, partially offset by (iii) the payment of $98.0 million related to the purchase of a priority review voucher from
Bayer HealthCare Pharmaceuticals and other net cash flows used in operating activities.
Total operating income increased by $141.6 million for the three months ended March 31, 2021 to
$167.4 million, compared to $25.8 million for the three months ended March 31, 2020. The increase
was primarily due to the closing of the strategic collaboration for efgartigimod with Zai Lab, resulting
in the recognition of $151.9 million in collaboration revenue.
Research and development expenses increased by $17.7 million for the three months ended March
31, 2021 to $122.3 million, compared to $104.7 million for the three months ended March 31, 2020.
The increase in the first three months of 2021 resulted primarily from higher external research and
development expenses, mainly related to the efgartigimod program in multiple indications and other
clinical and preclinical programs. Furthermore, the research and development personnel expenses
increased due to a planned increase in headcount and the increased costs of the share-based payment
compensation plans related to the grant of stock options.
Selling, general and administrative expenses totaled $56.3 million for the three months ended March
31, 2021, compared to $27.6 million for the three months ended March 31, 2020. The increase
resulted primarily from higher personnel expenses, including the costs of the share-based payment
compensation plans related to the grant of stock options, and consulting fees linked to the preparation
of a possible future commercialization of efgartigimod.
The increase in fair value on non-current financial assets amounted to $11.2 million for the three
months ended March 31, 2021, which is the result of the closing of a Series B financing round of
AgomAb Therapeutics, for which the Company maintains a profit share in exchange for granting the
license for the use of HGF-mimetic antibodies from the SIMPLE Antibody™ platform.
Exchange losses totaled $28.8 million for the three months ended March 31, 2021, compared to an
exchange gain of $23.0 million for the three months ended March 31, 2020. As a result of the change
in the Company’s functional and presentation currency, the exchange losses for the three months
ended March 31, 2021 are reflecting the unfavorable change in euro/U.S. dollar exchange rate, mainly attributable to unrealized exchange rate losses on cash, cash equivalents and current financial asset position in euro.

FINANCIAL GUIDANCE
Based on current plans to fund anticipated operating expenses and capital expenditures, argenx
continues to expect its 2021 cash burn to approximately double from 2020. The increased spend will support the Company’s transition to an integrated immunology company, 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.

EXPECTED 2021 FINANCIAL CALENDAR
- July 29, 2021: HY 2021 financial results and business update
- October 28, 2021: Q3 2021 financial results and business update

CONFERENCE CALL DETAILS
The first quarter 2021 financial results and business update will be discussed during a conference call and webcast presentation today at 2:30 pm CEST/8:30 am ET. A webcast of the live call may beaccessed on the Investors section of the argenx website at argenx.com/investors. A replay of the
webcast will be available on the argenx website.

tijd 09.22
De Bel 20 4.066,57 +34,32 +0,85% Argenx EUR 223,89 +7,40 vol. 5.298