On February 28, 2019 Aduro Biotech, Inc. (NASDAQ: ADRO) reported that three abstracts will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019, which will be held from March 29 through April 3, 2019 in Atlanta, GA (Press release, Aduro Biotech, FEB 28, 2019, View Source;p=RssLanding&cat=news&id=2389463 [SID1234533780]). Presentations will highlight the company’s lead programs, STING agonist ADU-S100, and anti-APRIL antibody BION-1301.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Details of the presentations are as follows:

Abstract 1202: Tumor cell intrinsic STING signaling demonstrates minimal contribution to the anti-tumor response elicited by the STING agonist ADU-S100 (MIW815)

Session: Tumor-immune Interactions
Date/Time: Monday Apr 1, 2019 8:00 AM – 12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 9

Abstract CT107: Phase 1/2 Safety and Pharmacokinetics of BION-1301 Targeting APRIL, a Proliferation-Inducing Ligand, in Adults with Relapsed or Refractory Multiple Myeloma

Session: Phase I-III Trials in Progress: Part 1
Date/Time: Monday Apr 1, 2019 1:00 PM – 5:00 PM
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 17

Abstract 1203: Preclinical development of ADU-1805, a highly selective pan-allele anti-SIRPα antibody that blocks the SIRPα-CD47 innate immune checkpoint

Session: Tumor-immune Interactions
Date/Time: Monday, Apr 1, 2019 8:00 AM – 12:00 PM
Location: Georgia World Congress Center, Exhibit Hall B, Poster Section 9

To view these abstracts, please visit the AACR (Free AACR Whitepaper) website located at View Source

On February 28, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases, reported that the Marginal Zone Lymphoma (MZL) cohort of the UNITY-NHL Phase 2b pivotal trial evaluating umbralisib (TGR-1202), our novel, once daily, PI3K delta inhibitor, met the primary endpoint of Overall Response Rate (ORR) as determined by Independent Review Committee (IRC) for all treated patients (n=69) (Press release, Manhattan Pharmaceuticals, FEB 28, 2019, View Source [SID1234533795]). The results met the Company’s target guidance of 40-50% ORR.

Interim safety and efficacy data from this study will be presented in an oral presentation at the upcoming American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting on April 1, 2019 and full data from this study are expected to be presented at a medical meeting later this year. The company plans to discuss the results with the U.S. Food and Drug Administration (FDA) regarding a potential new drug application (NDA) filing for accelerated approval. Umbralisib was recently granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of adult patients with MZL who have received at least one prior anti-CD20 regimen.

Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "We are extremely pleased to announce that the UNITY-NHL marginal zone lymphoma cohort evaluating umbralisib monotherapy met the primary endpoint of ORR. While this was an early look at the response data, we were excited to have already met the target ORR, which we previously stated was approximately 40%-50%. Importantly, with many patients still on study, we anticipate the ORR will continue to improve with additional follow-up, which will also provide us with critical information on duration of response, progression free survival and long-term safety and tolerability necessary to support an NDA filing."

Mr. Weiss continued, "There are no fully approved drugs for MZL, and thus remains an unmet medical need and we are excited by the potential to offer a novel treatment for this underserved population. We look forward to discussing the results with the FDA as soon as possible and if all goes well, we believe we could be in a position to file for accelerated approval for umbralisib by year-end."

About the UNITY-NHL Phase 2b Study—Marginal Zone Lymphoma Cohort

The multicenter, open-label, UNITY-NHL Phase 2b study – Marginal Zone Lymphoma cohort was designed to evaluate the safety and efficacy of single agent umbralisib, the Company’s novel, once daily, PI3K delta inhibitor, in patients with MZL who have received at least one prior anti-CD20 regimen. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. The primary analysis of ORR will be conducted once all treated patients have had at least 9 cycles (Cycle = 28 days) of follow-up. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

The MZL cohort completed enrollment in August 2018 with a total of 69 patients enrolled and receiving at least one dose of umbralisib. The positive ORR outcome announced today was based on all 69 enrolled and treated patients, however at this time all patients have not yet been followed for a minimum of 9 cycles as required for the primary analysis of ORR. Accordingly, the study is on-going and patients with benefit on therapy (stable disease or in response) remain on study. Safety data are currently being analyzed.

2019 AACR (Free AACR Whitepaper) Oral Presentation Details

Abstract titles are now available online and can be accessed on the AACR (Free AACR Whitepaper) meeting website at www.aacr.org. The complete text of the abstract will be available on the meeting website on Friday, March 29, 2019. The presentation is expected to include a subset of patients from the UNITY-NHL-Marginal Zone Cohort with long-term follow-up as of the data cut-off. Additional details regarding the presentation are included below.

● Title: Umbralisib monotherapy demonstrates efficacy and safety in patients with relapsed/refractory marginal zone lymphoma: A multicenter, open-label, registration directed Phase II study
o Session Date and Time: Monday April 1, 2019 3:00 PM – 5:00 PM ET
o Session Title: The Next Generation of Clinical Trials in Molecularly-driven Therapy
o Session Location: Marcus Auditorium- Bldg A-GWCC
o Presenter: Nathan Fowler, MD, Associate Professor, Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX
o Abstract Number: 7821

Following the presentation, the data presented will be available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

CONFERENCE CALL INFORMATION

The Company will host a conference call today, Thursday, February 28, 2019 at 8:30 AM ET to discuss the UNITY-NHL MZL results.

In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Update Call.

A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

ABOUT MARGINAL ZONE LYMPHOMA

Marginal zone lymphoma (MZL) comprises a group of indolent (slow growing) B-cell non-Hodgkin lymphomas (NHLs) that begin forming in the marginal zone of lymphoid tissue. With an annual incidence of approximately 7,500 newly diagnosed patients, MZL is the third most common B-cell NHL accounting for approximately eight percent of all NHL cases.i MZL consists of three different subtypes: extranodal MZL of the mucosal-associated lymphoid tissue (MALT), nodal marginal zone lymphoma (NMZL), and splenic marginal zone lymphoma (SMZL).

ABOUT BREAKTHROUGH THERAPY DESIGNATION

The FDA’s Breakthrough Therapy designation is intended to expedite the development and review of a drug candidate that is planned to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over available therapies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On February 28, 2019 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported financial results for the fourth quarter and year ended December 31, 2018 (Press release, Puma Biotechnology, FEB 28, 2019, View Source [SID1234533837]). Unless otherwise stated, all comparisons are for the fourth quarter and full year 2018, compared to the fourth quarter and full year 2017 .

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Product revenue, net consists entirely of sales revenue from NERLYNX, Puma’s first commercial product. Net NERLYNX revenue in the fourth quarter of 2018 was $61.1 million, compared to net NERLYNX revenue of $20.1 million in the fourth quarter of 2017. Net NERLYNX revenue for the full year 2018 was $200.5 million, compared to net NERLYNX revenue of $26.2 million in 2017. Puma received approval from the U.S. Food and Drug Administration (FDA) for NERLYNX (neratinib) for the treatment of early stage HER2-positive breast cancer following adjuvant trastuzumab-based therapy on July 17, 2017, and the Company began shipment to wholesalers at the end of July 2017.

Based on accounting principles generally accepted in the United States (GAAP), Puma reported a net loss of $30.7 million, or $0.80 per share, for the fourth quarter of 2018, compared to a net loss of $64.1 million, or $1.71 per share, for the fourth quarter of 2017. Net loss for the full year 2018 was $113.6 million, or $2.99 per share, compared to $292.0 million, or $7.85 per share, for the full year 2017.

Non-GAAP adjusted net loss was $12.2 million, or $0.32 per share, for the fourth quarter of 2018, compared to non-GAAP adjusted net loss of $38.6 million, or $1.03 per share, for the fourth quarter of 2017. Non-GAAP adjusted net loss for the full year 2018 was $26.7 million, or $0.70 per share, compared to non-GAAP adjusted net loss of $183.3 million, or $4.93 per share, for the full year 2017. Non-GAAP adjusted net loss excludes stock-based compensation expense. For a reconciliation of GAAP net loss to non-GAAP adjusted net loss and GAAP net loss per share to non-GAAP adjusted net loss per share, please see the financial tables at the end of this news release.

Net cash provided by operating activities for the fourth quarter of 2018 was $7.1 million, compared to net cash used in operating activities of $35.6 million for the fourth quarter of 2017. Net cash used in operating activities for the full year 2018 was $24.1 million, compared to $172.5 million for the full year 2017. At December 31, 2018, Puma had cash and cash equivalents of $108.4 million and marketable securities of $57.0 million, compared to cash and cash equivalents of $81.7 million at December 31, 2017.

"2018 was a strong year for Puma as we continued to grow NERLYNX sales, expanded our global presence and made progress in expanding the potential indications for the drug," said Alan H. Auerbach, Chairman, Chief Executive Officer and President of Puma. "This was highlighted by the European Commission granting marketing authorization in Europe, additional licensing agreements designed to provide access to NERLYNX in China, Latin America, Israel and Canada, and announcing data from the Phase III NALA trial, Phase II CONTROL trial, and Phase II SUMMIT trials."

Mr. Auerbach added, "During 2019, we anticipate the following key milestones for Puma: (i) meeting with the FDA and the European Medicines Agency to discuss data from the Phase III trial in third-line metastatic breast cancer patients in the first half of 2019; (ii) meeting with the FDA to discuss the clinical development and regulatory strategy for the SUMMIT trial in the first half of 2019; (iii) announcing regulatory decisions on neratinib for the extended adjuvant HER2-positive early stage breast cancer indication in countries outside of the United States and Europe in the first half of 2019; (iv) presenting data from the Phase III trial in third-line HER2-positive metastatic breast cancer treatment (NALA) in the first half of 2019; (v) reporting additional data from the Phase II CONTROL trial in the first half of 2019; and (vi) reporting Phase II data from the SUMMIT basket trial of neratinib in patients with HER2 mutations in the first half of 2019."

Revenue

Total revenue consists of product revenue, net from sales of NERLYNX, Puma’s first commercial product, and license revenue. For the fourth quarter ended December 31, 2018, total revenue was $71.1 million, of which $61.1 million was net NERLYNX revenue and $10.0 million was license revenue. For the year ended December 31, 2018, total revenue was $251.0 million, of which $200.5 million was net NERLYNX revenue and $50.5 million was license revenue received from Puma’s sub-licensees. This compares to total revenue of $27.7 million in 2017, of which $26.2 million was net NERLYNX revenue and $1.5 million was license revenue.

Operating Costs and Expenses

Total operating costs and expenses were $89.7 million for the fourth quarter of 2018, compared to $85.2 million for the fourth quarter of 2017. Total operating costs and expenses were $345.7 million for the full year 2018 compared to $320.1 million for the full year 2017.

Cost of Sales:

Cost of sales was $10.3 million for the fourth quarter and $34.6 million for the full year 2018, compared to $4.1 million for the fourth quarter and $5.6 million for the full year 2017.

Selling, General and Administrative Expenses:

Selling, general and administrative expenses were $41.0 million for the fourth quarter of 2018, compared to selling, general and administrative expenses of $30.9 million for the fourth quarter of 2017. Selling, general and administrative expenses for full year 2018 were $146.2 million, compared to $106.7 million for full year 2017, an increase of approximately $39.5 million. Increases in SG&A expenses for the full year 2018 primarily related to the hiring of a sales force and the commercial launch of NERLYNX, including approximately $18.9 million in payroll and related expenses, $8.2 million in travel and meetings, $6.0 million in professional fees, such as marketing, market access and analytics, $3.7 million in stock-based compensation and $2.6 million in other expenses, such as software and facilities, to support overall corporate growth.

Research and Development Expenses:

Research and development expenses were $38.4 million for the fourth quarter of 2018, compared to $50.2 million for the fourth quarter of 2017. Research and development expenses for the full year 2018 were $164.9 million, compared to $207.8 million for the full year 2017. The decrease of $42.9 million during the full year 2018 compared to the full year 2017 resulted primarily from decreases of approximately $25.5 million in stock-based compensation, $16.8 million in clinical trial expense, $2.6 million in consultant and contractor expense, partially offset by increases of approximately $2.0 million in R&D expenses primarily related to headcount additions in the medical affairs, quality assurance, regulatory affairs and pharmacovigilance.

Total Other Income (Expenses)

Total other expenses were $12.1 million for the fourth quarter of 2018, compared to total other expenses of $0.5 million for the fourth quarter of 2017. Total other expenses were $18.9 million for the year ended December 31, 2018, compared to total other income of $0.4 million for the year ended December 31, 2017. Other expense recorded in the fourth quarter of 2018 includes $9.0 million that represents an initial estimate of potential amounts that may be owed to class action participants as a result of the February 2019 jury verdict in a class action lawsuit, Hsu vs. Puma Biotechnology, Inc., et al.. The total amount of aggregate class-wide damages is uncertain and will be ascertained only after an extensive claims process and the exhaustion of any appeals. It is possible that the total damages will be higher than this estimate.

Conference Call

Puma will host a conference call to report its fourth quarter and full year 2018 financial results and provide an update on the Company’s business and outlook at 1:30 p.m. PST/4:30 p.m. EST on Thursday, February 28, 2019. The call may be accessed by dialing 1-877-709-8150 (domestic) or 1-201-689-8354 (international) at least 10 minutes prior to the start of the call and referencing the "Puma Biotechnology Conference Call." A live webcast of the conference call and presentation slides may be accessed on the Investors section of the Puma Biotechnology website at View Source A replay of the call will be available approximately one hour after completion of the call and will be archived on the Company’s website for 90 days.

On February 28, 2019 Halozyme Therapeutics, Inc. (NASDAQ: HALO), a biotechnology company developing novel oncology and drug-delivery therapies, reported that Genentech, a member of the Roche Group, has received approval from the U.S. Food and Drug Administration (FDA) for Herceptin Hylecta, a subcutaneous fixed-dose combination of trastuzumab and hyaluronidase-oysk (Press release, Halozyme, FEB 28, 2019, View Source [SID1234533861]). Herceptin Hylecta is approved for the treatment of certain people with HER2-positive early breast cancer (node-positive, or node-negative and ER/PR-negative or with one high-risk feature) in combination with chemotherapy and HER2-positive metastatic breast cancer in combination with paclitaxel or alone in people who have received one or more chemotherapy regimens for metastatic disease. Herceptin Hylecta is a co-formulation of trastuzumab with Halozyme’s proprietary recombinant human hyaluronidase enzyme (ENHANZE technology). Herceptin Hylecta is a ready-to-use formulation that can be administered in two to five minutes, compared to 30 to 90 minutes for intravenous trastuzumab.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are delighted that Herceptin Hylecta has been approved in the U.S. and that the potential for two- to five-minute administration time will now be available to patients in most of the major developed markets," said Dr. Helen Torley, president and chief executive officer. "Herceptin Hylecta is the third product co-formulated with ENHANZE to receive FDA approval, which represents an important achievement."

About ENHANZE Technology
Halozyme’s proprietary ENHANZE drug-delivery technology is based on its patented recombinant human hyaluronidase enzyme (rHuPH20). rHuPH20 has been shown to remove traditional limitations on the volume of biologics that can be delivered subcutaneously (just under the skin). By using rHuPH20, some biologics and compounds that are administered intravenously may instead be delivered subcutaneously. ENHANZE may also benefit subcutaneous biologics by reducing the need for multiple injections. This delivery has been shown in studies to reduce health care practitioner time required for administration and shorten time for drug administration.

On February 28, 2019 argenx (Euronext & Nasdaq: ARGX), a clinical-stage biotechnology company developing a deep pipeline of differentiated antibody-based therapies for the treatment of severe autoimmune diseases and cancer, reported its fourth quarter business update and full year results for 2018 (Press release, argenx, FEB 28, 2019, View Source [SID1234533796]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The full year results will be discussed during a conference call and webcast presentation today at 3 pm CET/9 am EST. To participate in the conference call, please select your phone number below and use the confirmation code 9795988. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"By translating fundamental immunology breakthroughs into novel product candidates, we have built a robust pipeline of differentiated antibodies with the potential to truly impact patients’ lives," commented Tim Van Hauwermeiren, CEO of argenx. "We made great progress in 2018 with our wholly owned lead asset efgartigimod, advancing development across four severe autoimmune indications, in both intravenous and subcutaneous formulations. We signed a transformational global collaboration and license agreement with Janssen to develop cusatuzumab in AML while retaining key commercial rights to the product in the U.S. We also announced that AbbVie exercised its option to in-license ARGX-115 targeting GARP.

"Based on our deep and diversified product candidate portfolio, a solid cash position and an ambitious business plan we are facing an abundance of opportunity ahead in 2019. We will continue to build out our novel pipeline through our Innovative Access Program and our commercial capabilities as we drive enrollment of the Phase 3 ADAPT trial of efgartigimod in gMG and plan for the start of a second Phase 3 trial in ITP."

FOURTH QUARTER 2018 AND RECENT HIGHLIGHTS

argenx continues to build a deep pipeline of differentiated antibody-based product candidates, including wholly-owned efgartigimod for the treatment of severe autoimmune diseases and cusatuzumab for the treatment of acute myeloid leukemia (AML) and high-risk myelodysplastic syndromes (MDS) in collaboration with Janssen. Through its Innovative Access Program, argenx grows its proprietary and partnered pipeline by leveraging its antibody expertise to match fundamental biology breakthroughs at leading research institutions.

Efgartigimod (ARGX-113) Program

· Full Phase 2 immune thrombocytopenia (ITP) trial data presented during American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting

o Correlation between IgG antibody reduction, platelet count increase and reduction of bleeding events following efgartigimod treatment.

o Increasing separation between treatment arms and placebo at increasing response thresholds observed, with response rates of 46% and 67% in primary trial and first dosing cycle of open-label extension trial, respectively.

o Primary endpoint analysis revealed efgartigimod to be well-tolerated in all patients consistent with tolerability analysis from Phase 1 healthy volunteer study.

· Orphan drug designation granted in February 2019 by U.S. Food and Drug Administration (FDA) for use for primary ITP.

o argenx additionally received orphan drug designation for efgartigimod in generalized myasthenia gravis (gMG) from FDA and the European Medicines Agency, which was announced, respectively, in September 2017 and March 2018.

· By mid-2019, argenx expects to close its ongoing open-label extension study as part of the completed Phase 2 proof-of-concept trial in ITP in anticipation of the start of a more chronic dosing regimen in its Phase 3 trial.

Cusatuzumab (ARGX-110) Program

· Updated Phase 1/2 AML trial data in combination with azacitidine presented during ASH (Free ASH Whitepaper) Annual Meeting.

o Overall response rate (ORR) of 92% in 12 newly diagnosed AML patients unfit for intensive chemotherapy, including 10 patients (91%) with a complete remission with or without hematologic recovery (CR/CRi) and 1 patient (9%) with partial remission (PR).

o Five patients (42%) achieved minimal residual disease (MRD) negativity. Translational data demonstrated that cusatuzumab monotherapy and in combination with azacitidine significantly reduced leukemic stem cells in the bone marrow of AML patients.

o Well-tolerated with no exacerbation of azacitidine toxicity.

· Orphan drug designation granted by FDA in January 2019 for use of cusatuzumab for AML.

Collaborations

· Entered global collaboration and license agreement with Halozyme for ENHANZE technology (February 2019).

· Gained access to ENHANZE subcutaneous delivery technology for up to three targets, including exclusive rights to develop therapeutic products targeting human neonatal Fc receptor FcRn.

· Upfront payment of $30 million paid to Halozyme with potential future payments up to $160 million per selected target subject to achievement of specified development, regulatory and sales-based milestones.

· Entered exclusive global collaboration and license agreement with Janssen for cusatuzumab

· Deal totaling up to $1.6 billion, plus royalties ranging from the low double digits to the high teens, to develop cusatuzumab in AML, MDS and other hematological malignancies.

Received $300 million upfront cash payment, and Johnson & Johnson Innovation made equity investment of €176.7 million ($200.0 million based on exchange rate on date of signing) in argenx, representing 4.68% of then-outstanding share capital.

· argenx retains right to co-promote cusatuzumab in the U.S. and share such royalties with Janssen on a 50-50 basis.

Corporate Updates

· Awarded €2.6 million grant from Flanders Innovation and Entrepreneurship (VLAIO) agency to explore new applications and modes of action of proprietary ABDEG technology, the Fc engineering technology used in the design of efgartigimod to augment clearance of disease-causing autoantibodies.

· Selected for BEL 20 Index on Euronext Brussels and awarded ‘Best BEL 20 Company’ for 2018.

· Intellectual property portfolio (which includes both owned and in-licensed patent rights) now includes 105 granted and 106 pending patents, covering lead assets and core technologies.

· Company expanded to 132 employees and consultants in support of growing business needs.

OUTLOOK 2019

In 2019, argenx expects to execute on its business plan towards the below milestones:

· Continue enrollment of efgartigimod in ongoing Phase 3 ADAPT and ADAPT+ trials in gMG

· Launch Phase 3 clinical trial with efgartigimod in ITP in second half of 2019

· Launch Phase 2 clinical trial with subcutaneous formulation of efgartigimod in ITP in first half of 2019

· Launch cohort 3 of Phase 2 proof-of-concept trial of efgartigimod in pemphigus vulgaris in first half of 2019

· Launch Phase 2 clinical trial with efgartigimod in chronic inflammatory demyelinating polyneuropathy before end of 2019

· Launch Phase 1 clinical trial with subcutaneous ENHANZE formulation of efgartigimod in healthy volunteers

· Host R&D day (May 22, 2019) to present new pipeline programs ARGX-117 and ARGX-118

Details of the Financial Results

Cash, cash equivalents and current financial assets totaled €564.6 million as of December 31, 2018, compared to €359.8 million on December 31, 2017. The increase in the year-end cash balance on December 31, 2018 resulted primarily from €240.9 million of net proceeds received from the follow-on public offering of American Depositary Shares on the Nasdaq Global Select Market in September 2018.

Operating income decreased by €12.1 million for the year ended December 31, 2018 to €29.2 million, compared to €41.3 million for the year ended December 31, 2017. The decrease in 2018 was primarily related to a €11.5 million decrease in the recognition of upfront payments.

Research and development expenses totaled €83.6 million and €51.7 million for the years ended December 31, 2018 and 2017, respectively. The increase in 2018 resulted primarily from higher external research and development expenses and personnel expenses, reflecting higher clinical trials costs and manufacturing expenses related to the development of the argenx product candidate portfolio and the recruitment of additional employees to support its research and development activities. argenx employed 75 employees in its research and development functions on December 31, 2018, compared to 58 employees on December 31, 2017.

Selling, general and administrative expenses totaled €27.5 million and €12.4 million for the years ended December 31, 2018 and 2017, respectively. The increase of €15.1 million in selling, general and administrative expenses for the year ended December 31, 2018 primarily resulted from higher personnel expenses and consulting fees related to the preparation for potential future commercialization of lead product candidate efgartigimod. On December 31, 2018, argenx employed 30 employees in its selling, general and administrative functions, compared to 15 employees on December 31, 2017.

For the year ended December 31, 2018, financial income amounted to €3.7 million compared to €1.3 million for the year ended December 31, 2017. The increase of €2.4 million in 2018 related primarily to an increase in the interest received on cash, cash equivalents and current financial assets.

Exchange gains totaled €12.3 million for the year ended December 31, 2018 compared to the €5.8 million exchange losses incurred for the year ended December 31, 2017. The increase was mainly attributable to unrealized exchange rate gains on the cash and current financial assets position in U.S. dollars due to the favorable fluctuation of the EUR/USD exchange rate in 2018.

The total comprehensive loss for the year ended December 31, 2018 was €66.6 million, compared to €28.1 million for the year ended December 31, 2017.

U.S. SEC and statutory financial reporting

argenx’s primary accounting standard for quarterly earnings releases and annual reports is International Financial Reporting Standards (IFRS) as issued by the International Accounting Standards Board (IASB). Quarterly summarized statements of profit and loss based on IFRS as issued by the IASB are available on www.argenx.com.

In addition to reporting financial figures in accordance with IFRS as issued by the IASB, argenx also reports financial figures in accordance with IFRS as adopted by the European Union (EU) for statutory purposes. The consolidated statement of financial position, the consolidated statements of profit and loss, the consolidated statements of cashflow, and the consolidated statement of changes in equity are not affected by any differences between IFRS as issued by the IASB and IFRS as adopted by the EU.

The consolidated statement of profit and loss data of argenx SE as of December 31, 2018 presented in this press release is unaudited.

Annual Report 2018

argenx expects to publish its 2018 Annual Report based on IFRS as issued by the IASB and its 2018 Annual Report for statutory purposes based on IFRS as adopted by the EU on March 26, 2019. These Annual Reports will be available on www.argenx.com.

EXPECTED 2019 FINANCIAL CALENDAR

· May 7, 2019: Annual General Meeting

· May 9, 2019: Q1 2019 business update and financial results

· August 1, 2019: HY 2019 business update and financial results

· October 24, 2019: Q3 2019 business update and financial results

Dial-in numbers:

Please dial in 5–10 minutes prior to 3 p.m. CET/ 9 a.m. ET using the number and conference ID below.

Confirmation Code: 9795988

Belgium

+32 (0)2 400 9874

Belgium

0800 48740

France

+33 (0)1 767 00794

France

0805 103028

Netherlands

+31 (0)20 714 3545

Netherlands

0800 0249557

United Kingdom

+44 (0)844 571 8892

United Kingdom

0800 376 7922

United States

+1 (631) 510 7495

United States

+1 (866) 966 1396

A question and answer session will follow the presentation of the results. Go to www.argenx.com to access the live audio webcast. The archived webcast will also be available (90 days) for replay shortly after the close of the call from the "Downloads" section of the argenx website.