On March 1, 2018 Apricus Biosciences, Inc. (Nasdaq:APRI), a biopharmaceutical company advancing innovative medicines in urology and rheumatology, reported financial results for the fourth quarter of 2017 and provided a corporate update on its near-term priorities (Press release, Apricus Biosciences, MAR 1, 2018, View Source;p=RssLanding&cat=news&id=2335731 [SID1234524293]).

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"Since the end of the third quarter of 2017, we have been focused on supporting the Vitaros NDA resubmission and strengthening our balance sheet. While we were successful achieving the latter, we were extremely disappointed with the FDA’s recent decision regarding the approval of Vitaros. As such, we will submit a request to the FDA for an end-of-review meeting in the coming weeks with a meeting expected to be scheduled in April per FDA guidelines. Our objective for this meeting is to determine the specific requirements needed to address the deficiencies noted in the Complete Response," stated Richard W. Pascoe, Chief Executive Officer. "In parallel, we will focus our efforts on identifying and evaluating opportunities or business combinations to maximize shareholder value, as we look reduce our expenses and extend our cash runway."

Fourth Quarter and Full Year Financial Results

Net loss during the quarter ended December 31, 2017 was $2.4 million, or loss per share of $0.16, compared to a net loss of $0.3 million, or loss per share of $0.04, during the fourth quarter of 2016. Net income during the year ended December 31, 2017 was $0.3 million, or income per share of $0.02, compared to a net loss of $7.4 million, or loss per share of $1.15, during the year ended December 31, 2016.

Net income during the year ended December 31, 2017 was primarily due to the $12.3 million gain recorded upon the sale of our ex-U.S. Vitaros rights and assets to Ferring, offset by the $1.5 million regulatory milestone payment made to Allergan upon the FDA’s acknowledgment of our Vitaros NDA resubmission, Vitaros commercial preparation activities, as well as other general and administrative expenses.

For all periods presented, financial statement activity related to our ex-U.S. Vitaros business has been presented as discontinued operations. As of December 31, 2017, the Company’s cash totaled $6.3 million, compared to $2.1 million as of December 31, 2016. As of February 26, 2018, the Company’s cash totaled $6.3 million, which is expected to fund operations through the end of 2018.

Conference Call Details

Apricus will host a live conference call and webcast today at 4:30 p.m. Eastern Time to discuss the Company’s financial results and provide a corporate update. To participate by telephone, please dial (855) 780-7196 (Domestic) or (631) 485-4867 (International). The conference ID number is 3687726. The live and archived audio webcast can be accessed through the Investors Relations’ section of the Company’s website at www.apricusbio.com. Please log in approximately five to ten minutes before the event to ensure a timely connection. The archived webcast will be available for 30 days following the live call.

On March 1, 2018 OPKO Health, Inc. (NASDAQ: OPK) reports business highlights and financial results for the three months ended December 31, 2017 (Press release, Opko Health, MAR 1, 2018, View Source [SID1234524313]).

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Business Highlights

RAYALDEE total prescriptions reported by IMS increased 47% in Q4 2017 compared with Q3 2017: Total prescriptions were more than 3,900 during the three months ended December 31, 2017. As of January 1, 2018, more than 79% of patients have access to RAYALDEE under their insurance plans.

4Kscore utilization increased 15% in Q4 2017 compared with Q4 2016: OPKO has undertaken a number of initiatives to drive utilization of the 4Kscore test, the Company’s blood test that gives a man with elevated prostate specific androgen (PSA) levels a personalized prediction of his chance of having or developing an aggressive form of prostate cancer. OPKO launched regional television ads in the Northeast for the 4Kscore test on November 21, 2017 and expanded the television ads to Florida in February 2018.

Premarket Approval (PMA) application for Claros point-of-care (POC) PSA test submitted to FDA: On November 6, 2017, OPKO submitted a PMA for a PSA test utilizing the Claros 1 immunoassay analyzer, a novel diagnostic instrument that can provide rapid, quantitative blood test results in 10 minutes in the physician’s office with only a finger stick drop of whole blood. A second product on the same platform for testosterone is advancing toward a 510(k) submission to the FDA later this year.

Global and Japan Phase 3 and registration studies for hGH-CTP in pediatric growth hormone deficient children are making good progress in enrolling patients: The global pediatric study is a pivotal, non-inferiority design comparing a single weekly administration of hGH-CTP with daily injections of a currently marketed growth hormone product. This study is expected to complete enrollment this year. The global and Japanese pediatric studies utilize the pen device and formulation that will be launched commercially upon approval. The pediatric segment represents more than 80% of the commercial market for treatment of hGH deficiency.

Initiated a Phase 2b clinical trial for OPK88004, an orally administered selective androgen receptor modulator (SARM): In November 2017, OPKO initiated a Phase 2b dose-ranging study for the treatment of men with benign prostatic hypertrophy (BPH), or enlarged prostate. OPK88004 is expected to improve the symptoms of BPH by reducing prostate size and, on the basis of data from a previous trial in 350 men, increase muscle mass and bone strength and decrease fat mass. BPH affects approximately 50 million men in the U.S.

Initiation of four Phase 2 clinical trials anticipated in 2018:

• RAYALDEE line extension in dialysis patients with secondary hyperparathyroidism (SHPT): Together with its partners Vifor Fresenius and Japan Tobacco, OPKO is developing RAYALDEE for Stage 5 chronic kidney disease (CKD) patients with SHPT undergoing dialysis and anticipates initiating a global Phase 2 trial in dialysis centers in the second quarter of this year.
• OPK88003, a once-weekly oxyntomodulin dual GLP1-Glucagon agonist for type 2 diabetes and obesity: OPKO expects to initiate a Phase 2b dose-escalation study with OPK88003 in the second quarter of this year. In a 420-patient Phase 2 trial in patients with type 2 diabetes, OPK88003 reduced HbA1c levels similar to exenatide extended-release (Ex ER). The drug also showed statistically significantly greater weight loss and lowering of total cholesterol and triglycerides compared to once-weekly Ex ER, with a good safety profile.
• OPK88002, an NK-1 antagonist to treat pruritus (itching) in Stage 5 CKD patients undergoing dialysis: An Investigational New Drug application was submitted to the FDA and plans are being finalized to begin a single-dose Phase 2a trial of OPK88002 in dialysis patients to treat severe itching. Approximately 50% of renal dialysis patients experience difficult to control pruritus.
• OPK88001 for the treatment of Dravet Syndrome: Three clinical research centers in the United States are expected to participate in this first in human clinical study of the AntagoNAT for treatment of Dravet Syndrome.
Financial Highlights

Net loss of $213.9 during the three months ended December 31, 2017 included $147.7 million of non-recurring or non-cash items consisting of:

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$73.3 million of revenue adjustments
$13.2 million of intangible impairment related to VARUBI
$61.2 million Income tax provision
Consolidated revenues for the three months ended December 31, 2017 were $193.7 million compared to $275.5 million for the comparable period of 2016. During the three months ended December 31, 2017, revenue from services were negatively impacted by non-recurring reimbursement adjustments from commercial and federal payor programs of $73.3 million and by reduced sample volume of $9.3 million.Revenue from products included $9.1 million of revenue from RAYALDEE, including $6.2 million related to revenue previously deferred through September 30, 2017.
During the three months ended December 31, 2017, operating expenses included investment in the commercial activities supporting the launch of RAYALDEE of $7.9 million, as well as continued investment in the Company’s pharmaceutical pipeline, with R&D expense increasing to $34.2 million. In addition, fourth quarter 2017 operating expenses included a $13.2 million impairment related to our VARUBI intangible assets as a result of our licensee’s discontinuation of the IV formulation.
During the three months ended December 31, 2017, a $61.2 million income tax provision was recorded, principally as a result of the Tax Cuts and Jobs Act ($31.8 million) as well as recording a valuation allowance against our U.S. based deferred tax assets. The comparable period of 2016 includes an income tax benefit of $31.5 million.
Cash, cash equivalents and marketable securities were $91.5 million as of December 31, 2017.
OPKO strengthened its balance sheet with a $55 million private placement of convertible notes issued on February 27, 2018.

CONFERENCE CALL & WEBCAST INFORMATION

OPKO’s senior management will provide a business update and discuss results in greater detail in a conference call and live audio webcast at 4:30 p.m. Eastern time today. The conference call dial-in and webcast information is as follows:

DOMESTIC DIAL-IN:
866-634-2258
INTERNATIONAL DIAL-IN:
330-863-3454
PASSCODE:
1973978
WEBCAST:
View Source
For those unable to participate in the live conference call or webcast, a replay will be available beginning March 1, 2018 approximately two hours after the close of the conference call. To access the replay, dial 855-859-2056 or 404-537-3406. The replay passcode is 1973978. The replay can be accessed for a period of time on OPKO’s website at View Source.

Juno 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, Juno, 2018, MAR 1, 2018, View Source [SID1234524277]).

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Supernus 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, Supernus, 2018, MAR 1, 2018, View Source [SID1234524319]).

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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.