On February 28, 2018 Heat Biologics, Inc. (NASDAQ: HTBX), a biopharmaceutical company developing drugs designed to activate a patient’s immune system against cancer, reported interim results from its Phase 2 study investigating HS-110 in combination with Bristol-Myers Squibb’s (Press release, Heat Biologics, FEB 28, 2018, View Source [SID1234524236]).

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anti-PD-1 checkpoint inhibitor, nivolumab (Opdivo), in patients with advanced non-small cell lung cancer (NSCLC) whose cancers have progressed after treatment with one or more lines of therapy.

Among the 35 patients in the Intent-to-Treat ("ITT") population, 6 patients (17%) achieved a partial response and 14 patients (40%) achieved disease control. Evaluable ITT patients (those who underwent at least one follow-up scan regardless of treatment duration) demonstrated overall response and disease control rates of 26% and 67%, respectively. Overall responses appeared durable and long lasting. The survival data are still maturing, and median overall survival has not yet been reached. The combination of HS-110 and nivolumab was well tolerated, with no additional toxicities compared to what has been observed with single agent checkpoint inhibitors.

As predefined in the clinical protocol, patient subgroups were evaluated for levels of tumor infiltrating lymphocytes ("TIL") and for PD-L1 checkpoint protein expression on tumor cells. Four of 9 "cold tumor" patients with low TIL levels (<10%) at baseline had partial responses. HS-110 also showed a durable effect in patients with low levels of PD-L1, with 3 of 9 patients responding. Both of these patient populations respond poorly to checkpoint inhibitors.

George Peoples, M.D., Chief Medical Officer, stated, "The results from this combination trial with HS-110 and nivolumab are very promising, demonstrating durable responses in those patients with low levels of TIL and PD-L1. These patients represent the most difficult-to-treat patient groups and comprise the majority of the NSCLC population."

"We look forward to continuing patient enrollment to better define the optimal NSCLC population and inform the design of a pivotal trial," commented Jeff Wolf, Chairman and CEO. "These data are consistent with the mechanism of action of our T-cell Activation Platform that promotes a robust T-cell immune response, an important component of an effective immunotherapy combination against cancer."

DURGA Trial Design

The ongoing DURGA trial is a single arm multicenter trial that was designed to evaluate the combination of HS-110 and nivolumab in patients with NSCLC. Patients with advanced and previously treated NSCLC were treated with weekly HS-110 for 18 weeks and nivolumab 3 mg/kg every 2 weeks until disease progression or death. The primary endpoints are 1) safety and tolerability, and 2) objective response rate as defined by RECIST 1.1 criteria. Secondary endpoints include disease control rate, duration of response, peripheral blood immune response, progression-free survival and overall survival.

Live Webcast with Slides

The interim data from the DURGA trial will be presented at an analyst event, taking place at 8am Eastern Time, today, Wednesday February 28th. To register and watch a live webcast of the event, visit View Source

On February 28, 2018 Galera Therapeutics, Inc., a clinical-stage biotechnology company developing drugs targeting oxygen metabolic pathways with the potential to transform cancer radiotherapy, reported the first patient with locally advanced pancreatic cancer (LAPC) has been dosed in a Phase 1/2 clinical trial of lead candidate GC4419, a highly selective and potent small molecule dismutase mimetic, at The University of Texas MD Anderson Cancer Center in Houston, Texas (Press release, Galera Therapeutics, FEB 28, 2018, View Source [SID1234524232]).

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Pancreatic cancer is the fourth-leading cause of cancer-related death in the United States, with more than 55,000 patients diagnosed annually. Although its five-year overall survival rate is approximately less than 5 percent, it has the potential to improve to more than 25 percent following successful surgical resection, according to the American Cancer Society. Unfortunately, fewer than 10 percent of patients have resectable tumors at diagnosis. A goal of treatment for patients with LAPC is to use a combination of aggressive chemotherapy and radiation to shrink the unresectable tumor and improve the possibility of resectability.

Preclinical data demonstrate GC4419 has the potential to improve the effectiveness of radiation on cancer cells while preventing toxicity in normal tissue. GC4419 leverages Galera’s dismutase mimetic platform to rapidly convert the superoxide generated by radiation therapy into hydrogen peroxide, which is lethal to cancer cells in high concentrations. The most profound effects are seen when combining GC4419 with targeted, high doses of radiation like that used in stereotactic body radiation therapy (SBRT). This is because the amount of hydrogen peroxide created by the conversion of superoxide increases as the dose of radiation increases when combined with GC4419, producing a magnified anti-tumor effect.

"Our recently completed 223-patient Phase 2b trial of GC4419 in head and neck cancer demonstrated GC4419’s ability to limit radiation-induced healthy tissue damage by reducing the duration, incidence and severity of radiation and chemotherapy-induced oral mucositis," said Mel Sorensen, M.D., President and CEO of Galera. "We seek to build upon these results with this anti-tumor trial in LAPC and generate robust data to demonstrate GC4419’s potential to change the management of radiation therapy by both protecting normal tissue and improving the effectiveness of radiation, making more surgical resections possible."

The adaptive, Phase 1/2 dose escalation trial will evaluate the safety and anti-tumor effect (i.e. tumor response and improvement in resectability) of GC4419 in combination with SBRT, compared with SBRT alone, in LAPC patients. The trial, which will enroll 48 patients, will also assess safety and tolerability to determine the maximum tolerated dose of SBRT when combined with GC4419 or placebo.

"GC4419 offers the potential to create two opportunities to improve radiation therapy – by synergistic anti-tumor efficacy and by protecting healthy cells at higher doses of radiation," said Jon T. Holmlund, M.D., Chief Medical Officer of Galera. "This trial will indicate whether SBRT and GC4419 can offer a powerful combination therapy that may improve the survival for LAPC patients by making inoperable tumors operable, which may have important implications for the treatment of pancreatic and other cancers."

About GC4419

GC4419 is a highly selective and potent small molecule dismutase mimetic that closely mimics the activity of human superoxide dismutase enzymes. GC4419 works to reduce elevated levels of superoxide caused by radiation therapy by rapidly converting superoxide to hydrogen peroxide and oxygen. Left untreated, elevated superoxide can damage noncancerous tissues and lead to debilitating side effects, including oral mucositis (OM), which can limit the anti-tumor efficacy of radiation therapy. Conversion of elevated superoxide to hydrogen peroxide, which is selectively more toxic to cancer cells, can also enhance the effect of radiation on tumors, particularly with stereotactic body radiation therapy (SBRT), which produces high levels of superoxide.

GC4419 has been studied in patients with head and neck cancer, GC4419’s lead indication, for its ability to reduce the duration, incidence and severity of radiation-induced severe oral mucositis (SOM). Results from Galera’s 223-patient, double blind, randomized, placebo-controlled Phase 2b clinical trial demonstrated GC4419’s ability to dramatically reduce the duration of SOM from 19 days to 1.5 days (92 percent), the incidence of SOM through completion of radiation by 34 percent and the severity of patients’ OM by 47 percent, while demonstrating acceptable safety when added to a standard radiotherapy regimen. In addition, in multiple preclinical studies, GC4419 demonstrated an increased tumor response to radiation therapy while preventing toxicity in normal tissue.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to GC4419 for the reduction of the duration, incidence and severity of SOM induced by radiation therapy with or without systemic therapy. The FDA also granted Fast Track designation to GC4419 for the reduction of the severity and incidence of radiation and chemotherapy-induced OM.

On February 28, 2018 Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE:CFBI), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address cancer, liver and inflammatory diseases, reported an update on its Phase II clinical trial with drug candidate Namodenoson (CF102) in the treatment of NAFLD/NASH (Press release, Can-Fite BioPharma, FEB 28, 2018, View Source [SID1234524224]).

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The current Phase II study is being conducted in three Israeli sites including Hadassah Medical Center, Jerusalem and the Rabin Medical Center, Petach Tikva. Patients who suffer from NAFLD/NASH with evidence of active inflammation are treated twice daily with 12.5 or 25 mg of oral Namodenoson vs. placebo. The primary end point of the Phase II study is the anti-inflammatory effect of the drug, as determined by ALT blood levels, and the secondary end points include percentage of liver fat, as measured by MRI-PDFF (proton density fat fraction). The company anticipates the completion of patient enrollment toward the end of 2018 and data release in the first half of 2019.

Recent safety data showed that Namodenoson has a favorable profile and lack of hepatotoxicity in patients. Preclinical data demonstrate robust anti-inflammatory, anti-fibrogenic and anti-steatotic effects, supporting its development for the NAFLD/NASH indication.

Can-Fite CEO Dr. Pnina Fishman commented, "We are pleased with the progress so far in our clinical trial with Namodenoson for the treatment of NAFLD/NASH, and we look forward to data release later in H1/2019."

There is currently no U.S. FDA approved drug for the treatment of NASH, which is an addressable pharmaceutical market estimated to reach $35-40 billion by 2025.

About Namodenoson

Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). Namodenoson is being evaluated in Phase II trials for two indications, as a second line treatment for hepatocellular carcinoma, and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.

On February 28, 2018 Bayer reported group sales increased by 1.5 percent (Fx & portfolio adj.) to 35.015 billion euros (Press release, Bayer, FEB 28, 2018, View Source;parent=news-overview-category-search-en&ccm=001&presskit=1 [SID1234524316]).

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In 2017, Bayer’s operational business was at the prior-year level. The company made good progress strategically. "We took major steps toward the proposed acquisition of Monsanto," Management Board Chairman Werner Baumann said on Wednesday at the Financial News Conference in Leverkusen. Pharmaceuticals achieved another record year in business operations. Sales and earnings declined at both Consumer Health and Crop Science – in the latter case, however, this development was attributable to the difficult situation in Brazil. Animal Health posted an increase in sales and earnings. "We remain focused on our objectives and are convinced of our long-term perspective. We therefore have every reason to look to the future with optimism," said Baumann.

Bayer received further approvals last year for the proposed acquisition of Monsanto. "Only recently, the Brazilian antitrust authorities gave the green light. That is an important milestone on the road to closing this transaction. After all, Brazil is one of the world’s most important agricultural markets," Baumann said. Overall, more than half of the around 30 authorities worldwide have now approved the acquisition. Although Bayer is continuing to cooperate closely with the institutions involved, it is becoming evident that the examination procedures will require more time. "Our goal now is to be able to close the transaction in the second quarter of 2018," Baumann explained. "This does not affect our expectation of a successful conclusion to the regulatory review, nor our conviction that this is the right step."

Of importance last year in connection with the proposed acquisition of Monsanto and the associated merger control proceedings was the contractual agreement to sell certain Crop Science businesses to BASF. "We have now also committed to divest our entire vegetable seed business. Certain additional business activities of Bayer and Monsanto may also be sold or out-licensed," Baumann said. Thus Bayer is actively addressing observations expressed by antitrust authorities. Any sales and licenses would be subject to a successful closing of the proposed acquisition of Monsanto, which remains subject to customary closing conditions, including receipt of required regulatory approvals.

According to Baumann, the company last year took a big step toward its goal of achieving full separation from Covestro in the medium term by selling approximately 36 percent of the interest in that company for 4.7 billion euros. Bayer ceded de facto control of Covestro and deconsolidated the company at the end of September. Bayer’s direct interest in Covestro currently amounts to 14.2 percent. Another 8.9 percent is held by Bayer Pension Trust.

"Operationally, 2017 was a year of ups and downs," said the Bayer CEO. Sales of the Bayer Group rose by 1.5 percent on a currency- and portfolio-adjusted basis (Fx & portfolio adj.) (reported: 0.2 percent) to 35.015 billion euros. At 9.288 billion euros, EBITDA before special items was level with the previous year despite negative currency effects. EBIT rose by 2.9 percent to 5.903 billion euros after special charges of 1.227 billion euros (2016: 1.088 billion euros). The special charges comprised mainly impairment losses on intangible assets, expenses in connection with the proposed acquisition of Monsanto and efficiency improvement programs, and provisions for litigations and legal risks. EBIT before special items increased by 4.5 percent to 7.130 billion euros. Net income increased by 61.9 percent to 7.336 billion euros and core earnings per share from continuing operations by 1.0 percent to 6.74 euros.

Operating cash flow from continuing operations climbed by 2.7 percent to 6.611 billion euros. "We are pleased that we were able to substantially reduce our net financial debt in 2017," said Chief Financial Officer Johannes Dietsch. Net financial debt declined by 69.5 percent to 3.595 billion euros. There were cash inflows from operating activities and from the sale of Covestro shares. "We are therefore in a good position for the pending financing activities connected with the proposed acquisition of Monsanto," said Dietsch.

Pharmaceuticals: Earnings growth stronger than sales increase

Sales of prescription medicines (Pharmaceuticals) increased by 4.3 percent (Fx & portfolio adj.) to 16.847 billion euros – a new record. "The success of the division was once again driven by our key growth products," said Baumann. Total sales of the anticoagulant Xarelto, the eye medicine Eylea, the cancer drugs Stivarga and Xofigo and the pulmonary hypertension treatment Adempas advanced by 16.3 percent (Fx adj.) to 6.196 billion euros. The development in sales of Xofigo was particularly strong at 25.6 percent (Fx adj.), due mainly to its market launch in Japan in 2016 and to higher demand in the United States. Business with Xarelto expanded by 13.9 percent (Fx adj.), primarily on account of higher volumes in Europe, Japan and China. Bayer also posted gains for its license revenues – recognized as sales – in the United States, where Xarelto is marketed by a subsidiary of Johnson & Johnson. Sales of Eylea climbed by 18.5 percent (Fx adj.), due especially to expanded volumes in Europe, Canada and Japan.

Among the other leading pharmaceutical products, the hormone-releasing intrauterine devices of the Mirena product family posted sales growth of 9.2 percent (Fx adj.), benefiting from higher volumes of the low-dose intrauterine device Kyleena, particularly in the United States and Europe. Marked sales gains were also achieved with Aspirin Cardio for the secondary prevention of heart attacks (Fx adj. 10.5 percent) and the diabetes treatment Glucobay (Fx adj. 13.0 percent), mainly as a result of a continued positive business performance in China. Sales of the Kogenate/Kovaltry blood-clotting medicines were down by 15.9 percent (Fx adj.) due to lower order volumes being placed for the active ingredient by a distribution partner ahead of the planned contract termination at the end of the year. Adjusted for this development, sales of this product were at the prior-year level.

EBITDA before special items of Pharmaceuticals increased by 8.8 percent to 5.711 billion euros. Growth was mainly driven by higher volumes. By contrast, negative currency effects diminished earnings by 98 million euros.

Sales and earnings of Consumer Health down

Sales of self-care products (Consumer Health) declined by 1.7 percent (Fx and portfolio adj.) to 5.862 billion euros. "This was due to persistently weak business development in the United States," said Baumann. Furthermore, the Chinese authorities unexpectedly changed the legal status of two of Bayer’s medicated skincare brands from OTC to prescription, which led to sales declines in the fourth quarter of 2017. By contrast, business expanded slightly in Europe/Middle East/Africa, while sales in Latin America came in at the prior-year level (Fx adj.).

Business with the Bepanthen / Bepanthol wound and skin care products expanded by 6.6 percent (Fx adj.), with gratifying sales gains particularly in Europe/Middle East/Africa, and especially in Germany. Sales of the Canesten skin and intimate health products grew by 3.5 percent (Fx adj.), in a development that was mainly attributable to a positive business performance in China and the United Kingdom. There was a substantial 7.9 percent (Fx adj.) decline in sales of the analgesic Aleve, which had benefited in 2016 from a product line extension and faced intense competition in the United States in 2017.

EBITDA before special items of Consumer Health declined by 12.8 percent to 1.231 billion euros. This was largely due to lower volumes, in part as a consequence of the reverse switch in China and the associated EBITDA effect of around 50 million euros. Earnings were also held back by a higher cost of goods sold, primarily as a result of inventory impairments, as well as by currency effects of 25 million euros and higher selling expenses. Positive contributions came from one-time gains, including particularly 80 million euros from the divestment of noncore brands.

Crop Science held back by situation in Brazil

Sales in the agriculture business (Crop Science) moved back by 2.2 percent (Fx & portfolio adj.) to 9.577 billion euros. This was mainly due to the situation in the Brazilian crop protection business, where volumes were held back by unexpectedly high inventories in the market. "We have initiated a number of measures to normalize this situation. For example, we took back crop protection products from our distribution partners and concluded new agreements at amended terms," said Baumann. "We are now seeing that these measures are taking effect." When the Brazilian business is excluded, sales of Crop Science rose by 3.0 percent (Fx & portfolio adj.). Sales declined by 18.0 percent (Fx adj.) in Latin America but grew strongest, by 5.8 percent (Fx adj.), in North America, followed by Asia/Pacific and Europe/Middle East/Africa.

The Seeds business (seed and plant traits) posted positive development, with sales gains of 9.1 percent (Fx & portfolio adj.). Environmental Science, the business with products for nonagricultural applications, saw sales increase by an even more substantial 14.0 percent (Fx & portfolio adj.). By contrast, there was a decline of 5.3 percent (Fx & portfolio adj.) at Crop Protection. Fungicides (Fx & portfolio adj.: minus 9.9 percent) and Insecticides (Fx & portfolio adj.: minus 6.1 percent) saw disproportionate declines in sales – unlike Herbicides and SeedGrowth (seed treatment products), where the declines were much less marked at 1.6 and 0.3 percent (Fx & portfolio adj.), respectively.

EBITDA before special items of Crop Science declined by 15.6 percent to 2.043 billion euros. This decline is largely attributable to the situation in Brazil, which resulted in lower selling prices and volumes. Negative currency effects of 63 million euros were an additional factor.

Animal Health posts gains in Asia/Pacific and North America

Sales of Animal Health increased by 2.0 percent (Fx and portfolio adj.) to 1.571 billion euros. Business in the Asia/Pacific region developed especially positively due to higher demand and price increases. Sales also grew in North America. The Seresto flea and tick collar posted continued strong growth of 25.1 percent (Fx adj.). Sales of the Advantage family of flea, tick and worm control products decreased by 7.8 percent (Fx adj.) year on year. EBITDA before special items rose by 9.2 percent to 381 million euros. Price increases, the Cydectin business acquired by Bayer in January 2017 and lower selling expenses had a positive impact on earnings.

Core earnings per share increased in the fourth quarter of 2017

Sales of the Bayer Group in the fourth quarter of 2017 rose by 2.7 percent (Fx & portfolio adj.), to 8.596 billion euros. EBITDA before special items declined by 1.3 percent to 1.783 billion euros. By contrast, EBIT climbed by 6.7 percent to 625 million euros. Net income fell by 67.3 percent to 148 million euros. This included a negative special effect of 455 million euros that relates to the tax reform in the United States. By contrast, core earnings per share from continuing operations improved by 28.2 percent to 1.41 euros.

Sales and earnings in 2018 expected to be at the prior-year level despite currency losses

Based on the exchange rates as of December 31, 2017, Bayer expects sales of around 35 billion euros for 2018. Sales, EBITDA before special items and core earnings per share are expected to be at the prior-year level. On a currency- and portfolio-adjusted basis, Bayer expects sales to increase by a low- to mid-single-digit percentage, while EBITDA before special items and core earnings per share from continuing operations are each predicted to grow by a mid-single-digit percentage after adjusting for currency effects. The forecast takes into account temporary supply interruptions due to remediation measures in production. Bayer expects the impact on adjusted EBITDA to be about 300 million euros. The largest proportion of this amount is related to the Pharmaceuticals Division and a minor part to the Consumer Health Division.

For Pharmaceuticals, Bayer plans to generate sales of more than 16.5 billion euros. This corresponds to a low-single-digit percentage increase on a currency- and portfolio-adjusted basis. The division aims to raise sales of the key growth products Xarelto, Eylea, Stivarga, Xofigo and Adempas toward 7 billion euros. Bayer expects EBITDA before special items to decline by a low-single-digit percentage (Fx adj.: increase by a low-single-digit percentage) and anticipates a slight decline in the EBITDA margin before special items.

At Consumer Health, Bayer expects sales of more than 5.5 billion euros, which would be at the prior-year level on a currency- and portfolio-adjusted basis. Bayer expects EBITDA before special items to decline by a low-single-digit percentage (Fx adj.: increase by a low-single-digit percentage).

For Crop Science, Bayer sees sales coming in at more than 9.5 billion euros. This corresponds to a mid-single-digit percentage increase on a currency- and portfolio-adjusted basis. Bayer expects to increase EBITDA before special items by a mid- to high-single-digit percentage (Fx adj.: mid-teens percentage increase).

At Animal Health, Bayer expects a currency- and portfolio-adjusted increase in sales by a low-single-digit percentage. The company expects EBITDA before special items to decline by a mid-single-digit percentage (Fx adj.: at the prior-year level). Both sales and EBITDA before special items are negatively impacted by revised financial reporting standards (IFRS 15).

Through the expected acquisition of Monsanto in the second quarter of 2018, Bayer anticipates a significant increase in sales and EBITDA before special items. Based on current assumptions about the equity and financing measures to be undertaken, Bayer expects a moderate decline in core earnings per share. For the first full year following the acquisition, Bayer continues to expect a significant increase in sales and EBITDA before special items, and an increase in core earnings per share.

On February 28, 2018 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 2017 (Press release, argenx, FEB 28, 2018, View Source;p=RssLanding&cat=news&id=2335501 [SID1234524272]).

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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 dial in number from the list included below, and use the confirmation code 6683242. The webcast may be accessed on the homepage of the argenx website at www.argenx.com or by clicking here.

"2017 was a year of tremendous momentum for argenx as we made progress on our pipeline and increased value for our shareholders. As a result of this focused execution, we believe we are well-positioned to advance our pipeline through several key clinical milestones in the year ahead," commented Tim Van Hauwermeiren, CEO of argenx. "In December, we announced positive clinical data from our Phase 2 trial of ARGX-113 for the treatment of generalized myasthenia gravis and plan to advance ARGX-113 to Phase 3 in this indication. This also marked the first dataset from our Phase 2 program aimed at evaluating the role of ARGX-113 in indications where the reduction of pathogenic autoantibodies may mediate disease. We continue to evaluate the asset in two additional Phase 2 indications, immune thrombocytopenia and pemphigus vulgaris, and expect to report data from both in the second half of 2018. We also advanced our lead oncology asset and reported interim data from our Phase 1/2 trial of ARGX-110 in acute myeloid leukemia, which we expect to transition into a Phase 2 trial this year. We feel that our achievements in 2017 have further validated our disciplined and differentiated approach to antibody development and look forward to providing updates on several important catalysts from our wholly-owned pipeline and strategic collaborations in 2018."

FOURTH QUARTER 2017 AND RECENT HIGHLIGHTS

Reported positive topline results from Phase 2 proof-of-concept trial of ARGX-113 (efgartigimod) in generalized myasthenia gravis (MG) showing a strong clinical improvement over placebo throughout the entire duration of the trial as well as a favorable tolerability profile consistent with Phase 1 data.
Launched Phase 1 trial with subcutaneous formulation of ARGX-113 evaluating pharmacokinetics (PK), pharmacodynamics (PD), safety and tolerability.
Presented updates on Phase 1/2 clinical trials of ARGX-110 in acute myeloid leukemia (AML) and cutaneous T-cell lymphoma (CTCL) during American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
Raised approximately $266 million in gross proceeds in an upsized U.S. public offering.

FINANCIAL HIGHLIGHTS (as of December 31, 2017) (compared to financial highlights as of December 31, 2016)

Operating income of €41.3 million (December 31, 2016: €17.2 million).
Net loss of €28.1 million (December 31, 2016: €21.4 million).
Cash position of €359.8 million (cash, cash equivalents and current financial assets) allowing us to pursue development of our pipeline as planned.

DETAILS OF OPERATIONAL RESULTS

Products in Clinical Development:

ARGX-113 (efgartigimod)

Reported positive topline results from Phase 2 proof-of-concept trial of ARGX-113 in generalized MG. ARGX-113 treatment resulted in a strong clinical improvement over placebo as measured by all four predefined clinical efficacy scales throughout the entire duration of the study. Among ARGX-113 treated patients, 75% had a clinically meaningful and statistically significant improvement for a period of at least six consecutive study weeks versus 25% of patients on placebo. Primary endpoint analysis revealed ARGX-113 to be well-tolerated in all patients and consistent with Phase 1 data.
Nearing complete enrollment in the Phase 2 clinical trial of ARGX-113 in immune thrombocytopenia (ITP). Amended ITP trial protocol to extend the follow-up period from eight weeks to 21 weeks. In addition, a one-year open label extension study was added as a second amendment to allow (re)treatment of the ITP patients from the first study (dosed at 10 mg/kg).
Launched Phase 1 trial to evaluate the PK, PD, safety and tolerability of a subcutaneously administered formulation of ARGX-113 in healthy volunteers.

ARGX-110

Provided updates on Phase 1/2 clinical trials of ARGX-110 in AML and CTCL during ASH (Free ASH Whitepaper) Annual Meeting.
Data from the Phase 1/2 clinical trial of ARGX-110 in combination with azacitidine in six newly diagnosed AML patients unfit for intensive chemotherapy showed signs of clinical activity, including complete remission (3/6 patients), complete remission with incomplete blood count recovery (1/6 patients) and partial response (2/6 patients). One of the patients that achieved a complete remission bridged to allogeneic stem cell transplant after five cycles. The preliminary data from the first set of patients suggest ARGX-110 is active both at the circulating and bone marrow blast levels and at the leukemic stem cell level. The data showed a favorable tolerability profile with no exacerbation of azacitidine toxicity.
Amended Phase 1/2 AML trial protocol to add a 20mg/kg dose cohort to the dose escalation to best inform the recommended dose for a Phase 2 study.
Data from the Phase 1/2 clinical trial of ARGX-110 in relapsed/refractory CTCL patients. Of the 22 CTCL patients under analysis, we observed one complete response, two partial responses and 10 patients with stable disease. ARGX-110 was observed to have a favorable tolerability profile in these CTCL patients.

Collaborations

Bird Rock Bio, Inc. (BRB) and argenx have mutually agreed to terminate BRB’s license agreement to develop and commercialize ARGX-109 (gerilimzumab). Genor, a sublicensee of Bird Rock Bio, will continue to develop ARGX-109 for the Chinese market.

Corporate

Intellectual property portfolio now includes 80 granted and 109 pending patents as of December 31, 2017.
Company expanded to 95 employees and consultants in support of the growth of the business.
Established argenx US, Inc., a subsidiary located in Boston, MA.
Nominated James Michael Daly as a new member of the Board of Directors, subject to approval by the shareholders at the annual general meeting.

OUTLOOK 2018
argenx continues to implement its business plan through advancing its deep pipeline of differentiated antibody-based therapies, including ARGX-113, ARGX-110, ARGX-115 and ARGX-112, the forging of collaborations with pharmaceutical companies and leading academic labs under its Innovative Access Program and the strengthening of its shareholder base.

In 2018, argenx aims to execute its ambitious business plan as follows:

Report full data from the Phase 2 proof-of-concept trial for ARGX-113 in generalized MG at the American Academy of Neurology conference (April 21-27, 2018,Los Angeles, CA)
Report topline data of the Phase 2 proof-of-concept trial for ARGX-113 in ITP and interim data of the Phase 2 proof-of-concept trial in pemphigus vulgaris in the second half of 2018. Report full data of the Phase 2 proof-of-concept trial for ARGX-113 in ITP before the end of the year.
Progress ARGX-113 into Phase 3 clinical development in generalized MG before the end of the year.
Report the full data of the Phase 1 healthy volunteer trial with the subcutaneous formulation of ARGX-113 during the second quarter of the year.
Provide an update on the Phase 1/2 clinical trial in AML in the second half of the year.
Report the full data of the AML Phase 1/2 and CTCL Phase 2 clinical trials of ARGX-110 by the end of the year.
Launch the Phase 2 proof-of-concept trial for ARGX-110 in AML before the end of the year.

KEY FIGURES (CONSOLIDATED)
Year Ended
December 31,
in thousands of € 2017 2016 Variance
Revenue € 36,415 € 14,713 € 21,702
Other operating income 4,841 2,439 2,402
Total operating income 41,256 17,152 24,104
Research and development expenses (51,740) (31,557) (20,183)
Selling, general and administrative expenses (12,448) (7,011) (5,437)
Operating loss € (22,932) € (21,416) € (1,516)
Financial income 1,250 73 1,177
Financial expenses – – –
Exchange losses (5,797) (31) (5,766)
Loss before taxes € (27,479) € (21,374) € (6,105)
Income tax income expense € (597) € – € (597)
TOTAL COMPREHENSIVE LOSS OF THE PERIOD € (28,076) € (21,374) € (6,702)

Net increase in cash, cash equivalents and current financial assets compared to year-end 2016 and 2015 263,047 54,402
Cash, cash equivalents and current financial assets at the end of the period 359,775 96,728

DETAILS OF THE FINANCIAL RESULTS

Cash, cash equivalents and current financial assets totaled €359.8 million for the year ended on December 31, 2017, compared to €96.7 million on December 31, 2016. The increase in the year-end cash balance on December 31, 2017 resulted primarily from €304.7 million of net proceeds received from the initial and follow-on U.S. public offerings of American Depositary Shares on the Nasdaq Global Select Market completed respectively in May and December 2017.

Operating income increased by €24.1 million for the year ended December 31, 2017 to reach €41.3 million, compared to €17.2 million for the year ended December 31, 2016. The increase primarily related to a €11.0 million increase in the recognition of upfront payments and a €9.2 million increase in milestone payments from our collaboration partners.

Research and development expenses totaled €51.7 million and €31.6 million for the years ended December 31, 2017 and 2016, respectively. The increase is mainly the result of higher external research and development expenses, reflecting higher clinical trial costs and manufacturing expenses related to the development of our product candidate portfolio.

Selling, general and administrative expenses totaled €12.4 million and €7.0 million for the years ended December 31, 2017 and 2016, respectively. The increase of €5.4 million in selling, general and administrative expenses for the year ended December 31, 2017 primarily resulted from higher personnel expenses, office costs and consulting fees incurred to support our growth and prepare the company to become and operate as a Nasdaq-listed company.

For the year ended December 31, 2017, financial income amounted to €1.3 million compared to €0.1 million for the year ended December 31, 2016. The increase of €1.2 million relates to (i) a €0.9 million realized gain on the sale of a participation in FairJourney Biologics LDA in December 2017 and (ii) an increase in the interest received on our cash, cash equivalents and current financial assets.

Exchange losses totaled €5.8 million for the year ended December 31, 2017 compared to €0.03 million for the year ended December 31, 2016. The increase is mainly attributable to unrealized exchange rate losses on our cash and current financial assets position in U.S. dollars due to the unfavourable fluctuation of the EUR/USD exchange rate.

The total comprehensive loss for the year ended December 31, 2017 was €28.1 million compared to €21.4 million for the year ended December 31, 2016.

U.S. SEC and statutory financial reporting
argenx’s primary accounting standard for quarterly earnings releases and annual reports is International Financial Reporting Standars (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, 2017 presented in this press release are unaudited.

Annual Report 2017
argenx expects to publish its 2017 Annual Report based on IFRS as issued by the IASB and its 2017 Annual Report for statutory purposes based on IFRS as adopted by the EU on March 26, 2018. These Annual Reports will be available on www.argenx.com.

FINANCIAL CALENDAR:

May 8, 2018: Annual General Meeting
May 9, 2018: Q1 2017 Business Update and financial results
August 2, 2018: Half-year 2017 Business Update and financial results
October 25, 2018: Q3 2017 Business Update and financial results

Dial-in numbers:
Please dial in 5-10 minutes prior to 3 pm CET/ 9 am EST using the number and conference ID below.

Confirmation Code: 6683242
Belgium 0800 58228
Belgium +32 (0)2 404 0659
France 0805 101 219
France +33 (0)1 76 77 22 74
Netherlands 0800 023 1436
Netherlands +31 (0) 20 721 9251
United Kingdom 0800 358 6377
United Kingdom +44 (0)330 336 9105
United States 888-394-8218
United States +1 323-794-2149

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.