argenx Reports Full Year 2020 Financial Results and Provides Fourth Quarter Business Update

– Biologics License Application (BLA) for IV efgartigimod accepted for review by U.S. Food and Drug Administration (FDA) for generalized myasthenia gravis (gMG)– Pre-approval access program opened in U.S. and Europe for eligible gMG patients            – Management to host conference call today at 2:30 pm CEST (8:30 am ET) –March 4, 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 full year 2020 and provided a fourth quarter business update.“2020 was an exceptional year for argenx, marked by execution across the organization and highlighted by the positive results of our pivotal Phase 3 ADAPT trial. We have started 2021 on strong footing with the FDA’s acceptance for review of the BLA for efgartigimod, bringing us one step closer to offering a new therapy option to people living with gMG.  In preparation for our first commercial launch, we remain committed to a series of educational and engagement efforts with patients, physicians and payors on FcRn as a target and the unmet disease burden that gMG patients face,” said Tim Van Hauwermeiren, Chief Executive Officer of argenx.“With proof-of-concept established for efgartigimod in four indications and enrollment on track to begin in the fifth and sixth indications this year, we are building out a broad development plan with our FcRn antagonist. In addition, we expect Phase 1 healthy volunteer data mid-year from our C2 antagonist ARGX-117, our second program in severe autoimmunity, solidifying our commitment to continued pipeline expansion with antibody-based medicines that have potential in multiple autoimmune indications,” concluded Mr. Van Hauwermeiren. FOURTH QUARTER 2020 AND RECENT BUSINESS UPDATEBLA for efgartigimod accepted for review by FDA; on track with commercial and regulatory preparations in the U.S., Japan, the EU and China.BLA for IV efgartigimod for treatment of gMG accepted for review by FDA with action date set for December 17, 2021 under Prescription Drug User Fee Act (PDUFA)Japanese Marketing Authorization Application (J-MAA) expected to be filed with Pharmaceuticals and Medical Devices Agency (PMDA) in first half of 2021 with anticipated Japan commercial launch in 2022MAA expected to be filed with European Medicines Agency (EMA) in second half of 2021Zai Lab Limited to discuss potential accelerated regulatory pathway for approval in China with National Medical Products Administration (NMPA)Commercial readiness activities on track, including:Build-out of supply chain to ensure ample commercial product available at launch to meet early demandHiring of experienced, neurology-focused sales team with aim to have approximately 70 sales representatives in place for launchOngoing education efforts with key stakeholder groups, including patients, physicians and payorsLaunched pre-approval access program (PAA) in the U.S. and Europe to open availability of efgartigimod to people living with gMG who have a high degree of unmet clinical need and are not able to participate in a clinical trialSeven global trials to be ongoing in 2021 of efgartigimod across IV and subcutaneous (SC) formulations; proof-of-concept now demonstrated in four indications, which strategically fit within growing commercial franchises.ADAPT-SC: Enrollment ongoing in registrational trial evaluating non-inferiority based on pharmacodynamic effect of SC efgartigimod compared to IV efgartigimod for treatment of gMG; trial expected to enroll approximately 50 patientsADHERE: Enrollment ongoing in registrational trial evaluating SC efgartigimod for treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) following interim analysis of safety data as well as efficacy assessments that surpassed pre-defined “GO” threshold; trial expected to enroll approximately 130 patientsADVANCE and ADVANCE-SC: Registrational trials ongoing of IV and SC efgartigimod for treatment of primary immune thrombocytopenia (ITP); trials expected to each enroll approximately 156 patientsADDRESS: Registrational trial ongoing of SC efgartigimod for treatment of pemphigus (vulgaris and foliaceus); trial expected to enroll approximately 150 patientsEnrollment in fifth and sixth indications to begin in 2021Agreement with Zai Lab Limited expected to expand and accelerate global development of efgartigimod, including into additional autoimmune indications             
Data expected mid-2021 from Phase 1 healthy volunteer trial of ARGX-117, a potential first-in-class C2 antagonist and second program with broad applicability in severe autoimmunity.
      -Trial to evaluate safety and tolerability of single and multiple ascending doses of IV and SC ARGX-117, and to identify dose to take forward into potential Phase 2 proof-of-concept trials, including for multifocal motor neuropathy (MMN)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.Data update from Phase 2 CULMINATE trial evaluating cusatuzumab in combination with azacitidine for treatment of newly diagnosed AML to be presented in peer-reviewed forumDecision to initiate additional cusatuzumab studies under collaboration will be determined following review of all available data including ongoing Phase 1b ELEVATE trial (NCT04150887), which is evaluating cusatuzumab in combination with venetoclax and azacitidine for treatment of newly diagnosed AMLImmunology Innovation Program (IIP) continues to drive pipeline expansion by identifying potential value-creation opportunities through collaboration with leading disease biologists.Preclinical work ongoing in early-stage pipeline, including ARGX-118, ARGX-119 and ARGX-12017 discovery programs under evaluation that emerged from IIPargenx continues its transition to a global, integrated, immunology organization.Geneva office opened to support commercial infrastructure ahead of expected EU launch of efgartigimodPlanned transition agreement in place for Chief Financial Officer Eric Castaldi as part of evolution to commercial-stage company; recruitment efforts ongoing for U.S.-based successorYvonne Greenstreet, President and Chief Operating Officer of Alnylam, has been nominated to Board of Directors to fill position of Dr. David Lacey, who intends to transition to an advisory role for the CompanyCompleted public offering of 3,593,750 ordinary shares in February 2021 with gross proceeds of $1.15 billion             Q4 AND FY2020 FINANCIAL RESULTS