On March 1, 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, MAR 1, 2018, View Source [SID1234524294]).

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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.

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.

On March 1, 2018 Paratek Pharmaceuticals, Inc. (Nasdaq:PRTK), a biopharmaceutical company focused on the development and commercialization of innovative therapies based upon tetracycline chemistry, reported financial results and provided an update on financial, clinical, and regulatory filing activities for the quarter and full year ended December 31, 2017 (Press release, Paratek Pharmaceuticals, MAR 1, 2018, View Source [SID1234524314]).

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"We had a strong finish to the year as we initiated our Phase 2 program in urinary tract infections and, importantly, we recently announced the completion of the rolling NDA submissions for omadacycline," said Michael Bigham, Chairman and Chief Executive Officer, Paratek. "We continue to expand our commercial readiness activities for omadacycline as we await the FDA’s acceptance of our NDAs".

Recent Highlights

Completed submission of New Drug Applications (NDAs) with the U.S. Food and Drug Administration (FDA) on February 2, 2018 for the once-daily oral and intravenous formulations of omadacycline for the treatment of community-acquired bacterial pneumonia (CABP) and acute skin and skin structure infections (ABSSSI)

Advanced commercial readiness activities, including initiation of manufacturing validation and commercial product supply and expansion of the medical affairs and payer account management teams in readiness for payer formulary discussions

Initiated the first of two Phase 2 studies of omadacycline in urinary tract infections (UTI)

Announced FDA acceptance of Allergan’s NDA for Seysara

Raised $50.0 million through a registered underwritten public offering of common stock

Upcoming Milestones

FDA acceptance of omadacycline NDAs anticipated in April 2018

Formulary discussions with payers to begin post FDA acceptance of NDA filing

Presentation of clinical and microbiological data at the upcoming European Congress of Clinical Microbiology and Infectious Disease, including nine posters and one oral presentation of the OASIS-2 study of oral-only omadacycline in ABSSSI

European pre-submission meetings planned for the second quarter of 2018

Fourth Quarter and Full Year 2017 Financial Results

For the fourth quarter of 2017, Paratek reported a net loss of $21.9 million, or ($0.78) per share, compared to a net loss of $26.5 million, or ($1.16) per share, for the same period in 2016. For the year ended December 31, 2017, Paratek reported a net loss of $89.1 million, or ($3.32) per share, compared to a net loss of $111.6 million, or ($5.51) per share, for the same period in 2016.

Revenue earned during the year ended December 31, 2017 primarily consists of a $7.5 million upfront payment

received as part of a collaboration agreement with Zai Lab and a $5.0 million milestone payment earned from Allergan on FDA acceptance of the Seysara NDA.

Research and development expenses were $14.2 million and $60.1 million for the quarter and year ended December 31, 2017, respectively, compared to $19.7 million and $83.5 million for the same periods in 2016. The decrease was driven primarily by lower clinical study costs associated with the completion of the Phase 3 program for omadacycline. This decrease is partially offset by higher employee compensation costs, NDA preparation and related user fees, and an increase in medical affairs activity.

General and administrative expenses were $11.7 million and $37.0 million for the quarter and year ended December 31, 2017, respectively, compared to $6.5 million and $26.4 million for the same periods in 2016. The increase was driven primarily by higher employee compensation costs as the Company continues to expand its commercial team, costs associated with pre-commercial activities, and business development efforts.

As of December 31, 2017, Paratek had cash, cash equivalents, and marketable securities of $151.7 million. Based on current assumptions, including full commercial buildout and launch of omadacycline, Paratek’s existing capital resources as well as the $50 million in proceeds from its January 2018 public offering of common stock, future contingent regulatory and commercial milestone payments from collaborations with Allergan and Zai Lab, anticipated extension of the interest-only period for the Hercules Term Loan, and estimated omadacycline product sales will enable Paratek to fund operating expenses and capital expenditure requirements into late 2019.

Conference Call and Webcast

Paratek’s earnings conference call for the quarter ended December 31, 2017 will be broadcast at 8:30 a.m. EDT on March 1, 2018. The live webcast can be accessed under "Events and Presentations" in the Investor Relations section of Paratek’s website at www.paratekpharma.com.

Domestic investors wishing to participate in the call should dial: 877-407-0792 and international investors should dial: 201-689-8263. The conference ID is 13675875. Investors can also access the call at View Source

Replays of the call will be available through March 15, 2018. Domestic investors can access the replay by dialing 844-512-2921 and international investors can access the replay by dialing 412-317-6671. The PIN code to access the replay is 13675875.

Website Information

Paratek routinely posts important information for investors on the Investor Relations section of its website at www.paratekpharma.com. Paratek intends to use this website as a means of disclosing material, non-public information and for complying with its disclosure obligations under Regulation FD. Accordingly, investors should monitor the Investor Relations section of Paratek’s website, in addition to following its press releases, SEC filings, public conference calls, presentations and webcasts. The information contained on, or that may be accessed through, Paratek’s website is not incorporated by reference into, and is not a part of, this document.

Valeant has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Valeant, 2018, FEB 28, 2018, View Source [SID1234524250]).

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On February 28, 2018 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported financial results for the fourth quarter and full year ended December 31, 2017 and provided a corporate update (Press release, Portola Pharmaceuticals, FEB 28, 2018, View Source;p=RssLanding&cat=news&id=2335410 [SID1234524257]).

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"2017 was a year of significant achievement for Portola, highlighted by the FDA approval of our first medicine, Bevyxxa, and the regulatory application for FDA approval of our breakthrough-designated Factor Xa-inhibitor antidote, AndexXa," said Bill Lis, chief executive officer of Portola. "We are pleased with the early indicators of the U.S. commercial launch of Bevyxxa, which began in January, and the potential to have a major public health impact on millions of patients in the U.S. and beyond. We also are rapidly approaching the FDA action date for AndexXa, which, if approved, would position Portola with two first-in-class medicines in the field of thrombosis. In the meantime, we are working with European regulatory authorities on the path forward for both medicines and are continuing to advance our dual, oral Syk/JAK inhibitor, cerdulatinib, for hematologic cancers."

Recent Achievements, Upcoming Events and Milestones

Bevyxxa (betrixaban) – an oral, once-daily Factor Xa inhibitor approved for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE.

Initiated U.S. commercial launch of Bevyxxa in January 2018
Received FDA approval for the manufacturing of betrixaban in Cork, Ireland
Completed an oral explanation to the European Committee for Medicinal Products for Human Use (CHMP); determining next steps in light of negative CHMP trend vote
New APEX trial results published in The American Heart Journal showing betrixaban’s effect on symptomatic VTE and VTE-related deaths; 12th major peer-reviewed publication

AndexXa (andexanet alfa) – an antidote for Factor Xa inhibitor treated patients with life-threatening bleeding; designated a Breakthrough Therapy and an Orphan Drug by the FDA.

The FDA set a new action date of May 4, 2018 for the Biologics License Application (BLA)
Successfully completed the first commercial campaign for Gen 2 product
Enrollment remains on track for the ongoing Phase 3b/4 ANNEXA-4 study in patients with acute major bleeding
Late-breaking clinical trial presentation on March 12, 2018 at the American College of Cardiology’s 67th Annual Scientific Session & Expo (ACC.18) featuring new interim data from ANNEXA-4
Successfully completed an oral explanation to the European CHMP; received a positive CHMP trend vote and are working with regulatory authorities to address their accompanying request for additional data

Cerdulatinib – an oral, dual-spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor in development for the treatment of relapsed/refractory B-cell and other T-cell malignancies in patients who have failed multiple therapies.

Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell and T-cell malignancies who have failed multiple therapies

Fourth Quarter and Full Year 2017 Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company, Pfizer, Bayer Pharma, Janssen Pharmaceuticals, Daiichi Sankyo and Dermavant Sciences was $9.8 million for the fourth quarter of 2017, compared with $13.7 million for the fourth quarter of 2016. Collaboration and license revenue for the year ended December 31, 2017 was $22.5 million, compared with $35.5 million for the year ended December 31, 2016.

Total operating expenses for the fourth quarter of 2017 were $95.7 million, compared with $68.9 million for the same period in 2016. Total operating expenses for the fourth quarter of 2017 included $10.9 million in stock-based compensation expense, compared with $7.9 million for the same period in 2016. Total operating expenses for the year ended December 31, 2017 were $295.2 million, compared with $305.1 million for 2016. Total operating expenses for the full year ended December 31, 2017 included $43.3 million in stock-based compensation expense compared with $30.4 million for 2016.

Research and development expenses were $68.5 million for the fourth quarter of 2017, compared with $56.0 million for the fourth quarter of 2016. Research and development expenses were $203.7 million for the year ended December 31, 2017, compared with $246.9 million for 2016.

Selling, general and administrative expenses for the fourth quarter of 2017 were $26.9 million, compared with $12.9 million for the same period in 2016. Selling, general and administrative expenses for the year ended December 31, 2017 were $91.1 million, compared with $58.2 million for 2016.

For the fourth quarter of 2017, Portola reported a net loss of $91.8 million, or $1.41 net loss per share, compared with a net loss of $53.8 million, or $0.95 net loss per share, for the same period in 2016. Shares used to compute net loss per share attributable to common stockholders were $65.3 million for the fourth quarter of 2017 compared with $56.5 million for the same period in 2016. Net loss for the year ended December 31, 2017 was $286.1 million, or $4.81 net loss per share, compared with a net loss of $269.0 million, or $4.76 net loss per share, for the same period in 2016. Shares used to compute net loss per share attributable to common stockholders were $59.5 million for 2017 compared with $56.5 million for 2016.

Cash, cash equivalents and investments at December 31, 2017 totaled $534.2 million, compared with cash, cash equivalents and investments of $318.8 million as of December 31, 2016.

If the FDA approves andexanet alfa in Q2 2018, the Company will be entitled to receive an additional $100 million from its royalty-based financing with Health Care Royalty Partners.

2018 Annual Financial Guidance
For the fiscal year 2018, Portola expects total GAAP operating expenses to be between $390 million and $430 million, including stock based compensation. These expenses are primarily for manufacturing of both andexanet alfa and betrixaban, ongoing clinical trials, support for the commercial launch of Bevyxxa and preparation for the potential commercial launch of AndexXa.

Conference Call Details
Portola will host a conference call today, Wednesday, February 28, 2018, at 4:30 p.m. ET, during which time management will provide fourth quarter and full year 2017 financial results, updates on the U.S. launch of Bevyxxa and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 4893459. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call

Ionis Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Ionis Pharmaceuticals, 2018, FEB 28, 2018, View Source [SID1234524271]).

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