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.

On february 28, 2018 TRACON Pharmaceuticals (NASDAQ:TCON), a clinical stage biopharmaceutical company focused on the development and commercialization of novel targeted therapeutics for cancer and wet age‐related macular degeneration, reported financial results for the fourth quarter and year ended December 31, 2017 (Press release, Tracon Pharmaceuticals, FEB 28, 2018, View Source [SID1234524975]).

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Fourth Quarter 2017 and Recent Corporate Highlights

In February, TRACON’s partner, Santen Pharmaceutical Co. Ltd. (Santen), reported Phase I/II data for DE‑122 (carotuximab) in patients with refractory wet age-related macular degeneration (AMD) at the 15th Annual Angiogenesis, Exudation, and Degeneration meeting organized by Bascom Palmer Eye Institute. The study assessed the safety, tolerability and bioactivity of a single intravitreal injection of DE-122 at four dose levels in 12 subjects with wet AMD refractory to vascular endothelial growth factor (VEGF) inhibitors. Improvement was observed in mean change in central retinal subfield thickness (CST) based on the spectral domain optical coherence tomography (SD-OCT) suggesting bioactivity of DE-122 in these refractory wet AMD patients. No serious adverse events were reported. Based on these results, Santen previously advanced DE-122 into a Phase 2a randomized controlled trial assessing the efficacy and safety of intravitreal injections in combination with Lucentis (ranibizumab) compared to Lucentis monotherapy in patients with wet AMD that continues to enroll subjects, and which resulted in TRACON receiving a $7.0 million milestone payment.

In January, initial data from the ongoing open-label, non-randomized Phase 1b/2 study of TRC105 and Nexavar (sorafenib) in patients with advanced hepatocellular carcinoma (HCC) were presented by Dr. Kanwal Raghav from the University of Texas MD Anderson Cancer Center at the 2018 ASCO (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium. Partial responses by RECIST 1.1 occurred in 25% (2 of 8) of evaluable patients and a reduction of 50% or greater in alpha fetoprotein (AFP) concentration occurred in 38% (3 of 8) of evaluable patients, including both partial responses. Reduction in AFP, a tumor marker expressed in patients with HCC, in early treatment may help identify a favorable response to treatment. Adverse events typical of each drug were observed but did not increase in frequency or severity when the drugs were administered concurrently. The 25% response rate seen in this trial is consistent with the 25% (5 of 20) response rate reported in a Phase 1/2 trial of TRC105 and Nexavar conducted by the National Cancer Institute and published in Clinical Cancer Research in 2017. The response rates observed in both trials compare favorably with the 2% and 3% response rates in HCC patients reported for single agent Nexavar in its pivotal studies. The trial is currently ongoing, and we expect to complete the enrollment of approximately 33 patients by the end of 2018.

In December, TRACON licensed development and commercialization rights outside of ophthalmology indications to TRC105 in China, Hong Kong, Macau, and Taiwan to Ambrx. Deal terms included a $3.0 million upfront payment to TRACON, up to $140.5 million in development and commercial milestones and potential high single digit to low teen royalties from net sales of TRC105 in the Ambrx territories. Ambrx is expected to file an IND later this year and to subsequently begin clinical development of TRC105 in China.

In December, Brian Daniels, M.D., was appointed to TRACON’s Scientific Advisory Board. Dr. Daniels was Senior Vice President at Bristol-Myers Squibb until 2014, where he chaired the development investment committee and oversaw the development of Sprycel (dasatinib), Ixempra (ixabepilone), Yervoy (ipilumumab), Empliciti (elotuzumab), and Opdivo (nivolumab). He is currently a venture partner at 5AM Ventures, and previously held senior level development-related positions at Merck and Genentech. In addition, Dr. Daniels previously served in multiple academic roles in the Department of Medicine at the University of California, San Francisco.

In November, the first patient was dosed in a Phase 1b clinical trial of TRC105 in combination with Opdivo in patients with non-small cell lung cancer. The Phase 1b clinical trial is an open-label, dose-escalation and expansion cohort study of TRC105 and Opdivo in patients with non-small cell lung cancer that have received prior chemotherapy. The primary objectives of the Phase 1b study are to assess the safety of TRC105 when dosed with Opdivo, determine its recommended Phase 2 dose and evaluate the response rate. The trial incorporates tumor biopsy testing to determine if there is a correlation of tumor myeloid cell infiltration with response, in order to allow for potential biomarker-directed therapy in lung cancer patients.

In November, TRACON presented updated data from the Phase 1b/2 study of TRC105 and Votrient (pazopanib) in patients with soft tissue sarcoma, including angiosarcoma, at the Connective Tissue Oncology Society (CTOS) annual meeting. Median progression-free survival (PFS) was 7.8 months in 13 VEGF inhibitor naïve angiosarcoma patients treated with the combination of TRC105 and Votrient using either 10 mg/kg weekly dosing or the hybrid dosing schedule of TRC105. The reported median PFS compares favorably to the median PFS of 3 months reported in a retrospective study of single agent Votrient in patients with angiosarcoma. In the 17 patients who received prior treatment for metastatic disease, treatment duration on TRC105 and Votrient exceeded the duration of the most recent prior therapy in 7 of 12 VEGF naïve angiosarcoma patients and 2 of 5 patients who received a VEGF inhibitor as part of their most recent prior therapy. Treatment with the combination of TRC105 and Votrient continued to be well-tolerated and allowed for dosing of the combination for more than two years in the two patients who experienced complete responses to treatment.

Enrollment continues in the Phase 1/2 trial of TRC253, TRACON’s product candidate for treating prostate cancer that was in-licensed from Janssen. The Phase 1/2 trial is designed to assess the safety, determine a recommended Phase 2 dose and assess response by prostate-specific antigen (PSA) levels. If Janssen opts to reacquire TRC253 prior to or following completion of the Phase 1/2 trial, TRACON is entitled to receive a $45.0 million opt-in payment, up to $137.5 million in potential milestone payments and a low single digit royalty.
"We achieved important progress across our pipeline and continued to position the Company well for long-term success during the fourth quarter and throughout 2017," said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. "Most importantly, our pivotal Phase 3 TAPPAS trial of TRC105 in angiosarcoma continues to enroll well across sites in the U.S. and now Europe, with the key interim analysis projected for later this year. Moreover, our recent partnership with Ambrx expands the development and potential commercial opportunity of TRC105 into China."

Expected Additional 2018 Milestones

Completion of the dose escalation portion of the Phase 1/2 trial of TRC253 in patients with prostate cancer is expected in mid-2018.

Announcement of top-line data from the randomized Phase 2 TRAXAR trial of TRC105 in combination with Inlyta for patients with advanced or metastatic renal cell carcinoma is expected in mid-2018.

Announcement of the results of the interim analysis from the Phase 3 pivotal TAPPAS trial of TRC105 in angiosarcoma is expected in the second half of 2018.

Presentation of data from the Phase 1b trial of TRC105 in combination with Opdivo in patients with non-small cell lung cancer is expected in the second half of 2018.
Fourth Quarter 2017 Financial Results

Cash, cash equivalents and short-term investments were $34.5 million at December 31, 2017, compared to $44.4 million at December 31, 2016.

There was no collaboration revenue for the fourth quarter of 2017 compared to $0.6 million for the fourth quarter of 2016.

Research and development expenses for the fourth quarter of 2017 were $4.6 million compared to $4.8 million for the fourth quarter of 2016.

General and administrative expenses for the fourth quarter of 2017 were $1.7 million compared to $1.9 million for the fourth quarter of 2016.

Net loss for the fourth quarter of 2017 was $6.6 million compared to a net loss of $6.3 million for the fourth quarter of 2016.
Investor Conference Call

The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its fourth quarter of 2017. The dial-in numbers are (855) 779‑9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 1894785. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.

After the live webcast, a replay will remain available on TRACON’s website for 60 days.

About Carotuximab (TRC105)

TRC105 is a novel, clinical stage antibody to endoglin, a protein overexpressed on proliferating endothelial cells that is essential for angiogenesis, the process of new blood vessel formation. TRC105 is currently being studied in a pivotal Phase 3 trial in angiosarcoma and multiple Phase 2 clinical trials, in combination with VEGF inhibitors. TRC105 has received orphan designation for the treatment of soft tissue sarcoma in both the U.S. and EU. The ophthalmic formulation of TRC105, DE-122, is currently in a randomized Phase 2 trial for patients with wet AMD. For more information about the clinical trials, please visit TRACON’s website at www.traconpharma.com/clinical_trials.php.

About TRC253

TRC253 is a novel, orally bioavailable small molecule that is a potent, high affinity competitive inhibitor of the androgen receptor (AR) and AR mutations, including the F876L (also known as F877L) mutation. The AR F876L mutation results in an alteration in the AR ligand binding domain that confers resistance to therapies for prostate cancer. Activation of the AR is crucial for the growth of prostate cancer at all stages of the disease. Therapies targeting the AR have demonstrated clinical efficacy by extending time to disease progression, and in some cases, the survival of patients with metastatic castration-resistant prostate cancer. However, resistance to these agents is often observed and several molecular mechanisms of resistance have been identified, including gene amplification, overexpression, alternative splicing, and point mutation of the AR.

On February 27, 2018 Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported its financial results for the fourth quarter and full year 2017 and highlighted recent progress in advancing its business and drug pipeline (Press release, Ionis Pharmaceuticals, FEB 27, 2018, View Source [SID1234524209]).

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"In 2017, our accomplishments were remarkable. The historic launch of SPINRAZA and the acceptance of our regulatory filings for inotersen and volanesorsen, which set up potential launches of these two drugs in 2018, combined with our strong financial performance, demonstrate our ability to execute across all areas of our business. We also advanced our pipeline of novel drugs with positive data readouts from 11 clinical studies, including five with our LICA drugs, and introduced eight new drugs to the pipeline, further highlighting the broad capabilities and potential of our antisense technology platform," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer.

"Looking ahead, we believe 2018 could be an important turning point for Ionis. Most important will be the launch of inotersen and volanesorsen, if approved. We also expect to report data from at least six Phase 2 studies, initiate at least five Phase 2 programs and report data from multiple proof-of-concept clinical studies. These important events build on our recent momentum, solidifying Ionis as a multi-product, commercial company delivering innovative antisense medicines to patients in need," continued Crooke. "As we advance our pipeline of antisense drugs and achieve important milestones in our collaborations, we expect to continue to provide substantial value to our shareholders and the patients we serve."

Financial Results and Highlights

·
Revenues for 2017 increased by more than 45%
o
For the fourth quarter and full year 2017, revenue was $172 million and $508 million, compared to $160 million and $347 million for the same periods in 2016.
o
Commercial revenue for 2017 was $113 million from SPINRAZA royalties and $9 million from other licensing and royalty payments. R&D revenue for 2017 was $386 million and increased by nearly 20% from 2016.

1
·
Operating income for 2017 increased by more than 150%, driven by strong revenues and reflecting prudent expense management
o
GAAP operating income was $25 million for 2017, compared to a GAAP operating loss of $46 million for the same period in 2016. Pro forma operating income was $111 million for 2017, compared to $26 million for the same period in 2016.
o
Operating expenses increased at a much slower rate than revenue with the increase primarily due to higher SG&A expenses as Ionis prepares to commercialize volanesorsen and inotersen this year.

·
Substantial cash position enabled pipeline progress
o
As of December 31, 2017, Ionis had cash, cash equivalents and short-term investments of more than $1 billion compared to $665 million at December 31, 2016.
o
During 2017 Ionis received over $580 million in partner payments. Additionally, Ionis’ cash balance at December 31, 2017 included proceeds from Akcea’s 2017 IPO and Novartis’ strategic investment in Akcea.

"2017 was our first year of commercial revenue in which we earned $113 million in SPINRAZA royalties. It was also our sixth consecutive year of revenue growth, driven by our focus on high-value, innovative drugs like SPINRAZA and multiple other exciting programs in our pipeline," said Elizabeth L. Hougen, chief financial officer of Ionis. Our goal is to be a multi-product, sustainably profitable company. Consistent with this goal, we project 2018 will be our third consecutive year of pro forma operating profitability even as we prepare to launch two new drugs. For 2018, we are projecting R&D expenses of $360 million to $390 million and SG&A expenses of $180 million to $210 million both on a pro forma basis. We project that we will end 2018 with more than $800 million in cash."

All pro forma amounts referred to in this press release exclude non-cash compensation expense related to equity awards. Please refer to the reconciliation of pro forma and GAAP measures, which is provided later in this release.

Recent Pipeline and Technology Highlights

·
SPINRAZA for SMA – one of the most successful orphan drug launches in history
o
SPINRAZA, commercialized by Biogen, generated 2017 global sales of $884 million
o
Results from the ENDEAR study and CHERISH study, in which people with infantile-onset and later-onset SMA, respectively, were treated with SPINRAZA, were published in The New England Journal of Medicine
o
Prestigious 2017 Prix Galien USA Award for Best Biotechnology Product awarded to Ionis and Biogen for SPINRAZA
o
New collaboration with Biogen initiated to discover new antisense drugs with enhanced properties to treat SMA

·
Inotersen for hATTR – potential to transform the lives of people with hATTR
o
Marketing applications accepted, no FDA Advisory Committee recommended, Priority Review in the U.S. and Accelerated Assessment in the EU
o
Preparations for global launch, planned for mid-2018, progressing

2
o
Phase 3 NEURO-TTR study met both primary endpoints demonstrating benefit compared to placebo in multiple measures of quality of life and disease severity; 50% of inotersen-treated patients experienced improvement from baseline in quality of life

·
Volanesorsen for FCS and FPL – potential first treatment for people with FCS
o
Marketing applications accepted in the U.S., EU and Canada with Promising Innovative Medicine designation in the UK and Priority Review in Canada
o
Preparations for global launch for FCS, planned for mid-2018, progressing
o
Phase 3 APPROACH study met primary endpoint of reducing triglyceride levels in people with FCS

·
Pipeline Programs (early and mid-stage) – advancing wholly owned and partnered programs
o
Positive results from seven Phase 2 studies reported, including:
§
Positive data from Phase 1/2 study of IONIS-STAT3-2.5Rx in combination with AstraZeneca’s Imfinzi reported for people with head and neck cancer
§
Robust, dose-dependent reductions of mHTT observed in people with Huntington’s disease treated with IONIS-HTTRx
o
Positive clinical data on five LICA drugs reported, demonstrating consistent, positive performance and sustained target reduction with potential for monthly or less frequent dosing
o
Positive results from six Phase 1 studies reported
o
Nine Phase 2 studies and four Phase 1 studies initiated across multiple therapeutic areas to treat people with both broad and rare diseases

"In 2017, we and our partners advanced key programs in all of our therapeutic areas, including cardiometabolic, oncology, neurology and, severe and rare disease. Of the eight new drugs we added to the pipeline, six were drugs that use our more potent LICA technology or Generation 2.5 chemistry, and two were drugs to treat neurodegenerative disorders. We also added to our pipeline our second orally delivered, locally acting drug for a GI autoimmune disease. These achievements demonstrate the success of our wholly owned and partnered programs," said Brett P. Monia, Ph.D., chief operating officer and senior vice president of antisense drug discovery and translational medicine at Ionis Pharmaceuticals. "Looking ahead, we plan to pursue only those partnerships with infrastructure and resources that complement our own. Simultaneously, we plan to continue to build our wholly owned pipeline of drugs to treat patients with rare and serious diseases."

Expected Events Through Mid-2018

·
Launch of inotersen for people with hATTR, if approved
·
Launch of volanesorsen for people with FCS, if approved
·
Report results from five Phase 2 programs, including data from studies with IONIS-HTTRx in people with Huntington’s disease and data from a 6-12 month study with AKCEA-APO(a)-LRx in people with high Lp(a) and cardiovascular disease
·
Initiate up to six Phase 2 or Phase 3 studies and three Phase 1 studies, including the initiation of a clinical study with follow-on LICA drug, IONIS-TTR-LRx

3
Revenue

Ionis’ revenue for the fourth quarter and full year 2017 was $172.3 million and $507.7 million, compared to $160.3 million and $346.6 million for the same periods in 2016. Ionis’ revenue in 2017 consisted of the following:

Commercial Revenue:

·
$113 million from SPINRAZA royalties; and
·
$9 million from other licensing and royalty payments.

R&D Revenue:

·
$118 million in milestone payments from Biogen, including $90 million in milestone payments for SPINRAZA, $15 million for validating two undisclosed neurological disease targets and $10 million for initiating a Phase 1/2 study of IONIS-MAPTRx;
·
$65 million from Bayer for the license of IONIS-FXI-LRx;
·
$48 million from Roche primarily for the license of IONIS-HTTRx;
·
$10 million from Janssen for license of IONIS-JBI2-2.5Rx and initiation of Phase 1 study of IONIS-JBI1-2.5Rx;
·
$115 million from the amortization of upfront fees; and
·
$30 million from services Ionis performed for its partners, of which more than half related to manufacturing services.

Ionis’ R&D revenue may fluctuate quarterly based on the nature and timing of payments under agreements with its partners and consists primarily of revenue from the amortization of upfront fees, milestone payments and license fees.

At the beginning of 2018, Ionis adopted the new revenue recognition accounting standard on a retrospective basis, which means that starting with the first quarter, Ionis will begin showing all periods presented using the new standard. The Company does not anticipate that there will be a significant impact to its previously reported revenue. Under the new standard, Ionis’ revenue for 2017 will increase by approximately 3%.

Operating Expenses

Operating expenses for the fourth quarter and full year 2017 on a GAAP basis were $174.0 million and $483.1 million, respectively, and on a pro forma basis were $151.7 million and $397.2 million, respectively. This is compared to GAAP operating expenses of $119.2 million and $392.9 million and pro forma operating expenses of $104.0 million and $320.8 million for the same periods in 2016. Operating expenses increased in 2017, compared to 2016, principally due to higher SG&A expenses as Ionis prepares to commercialize volanesorsen and inotersen in 2018. The Company’s SG&A expenses also increased in 2017 compared to 2016 because of fees it owes under its in-licensing agreements related to SPINRAZA.

4
Net Income (Loss)

Ionis reported a net loss of $4.7 million and $17.3 million for the fourth quarter and full year 2017, respectively, compared to net income of $25.9 million and a net loss of $86.6 million for the same periods in 2016, all according to GAAP. On a pro forma basis, Ionis reported net income of $17.7 million and $68.7 million for the fourth quarter and full year 2017, respectively, compared to net income of $41.0 million and a net loss of $14.4 million for the same periods in 2016. Ionis’ GAAP and pro forma net loss improved for 2017 compared to 2016 primarily due to the addition of commercial revenue from SPINRAZA royalties and increased R&D revenue.

Additionally, in 2017, Ionis recorded two non-cash, non-recurring items in other expenses, which contributed to the Company’s net loss. These two items were the previously capitalized fair value of the potential premium Ionis would have received from Novartis if Akcea had not completed its IPO and the loss the Company recognized on the purchase of its primary R&D facility. In 2016, Ionis recorded a $4.0 million non-cash loss related to the early repurchase of its 2¾ percent convertible senior notes, which contributed to the Company’s net loss for 2016.

Net Loss Attributable to Noncontrolling Interest in Akcea Therapeutics, Inc.

Akcea sold shares of its common stock to third parties in its IPO in July 2017. From the closing of the IPO through the end of 2017, Ionis owned 68 percent of Akcea. The shares held by third parties represent an interest in Akcea’s equity that Ionis does not control. However, because Ionis continues to maintain overall control of Akcea through its voting interest, Ionis reflects the assets, liabilities and results of operations of Akcea in Ionis’ consolidated financial statements. Ionis reflects the noncontrolling interest attributable to other holders of Akcea’s common stock in a separate line called "Net loss attributable to noncontrolling interest in Akcea" on Ionis’ statement of operations. Ionis’ net loss attributable to noncontrolling interest in Akcea for the fourth quarter and full year 2017 was $7.4 million and $11.3 million, respectively. Ionis also added a corresponding account in its stockholders’ equity section on its balance sheet called "Noncontrolling interest in Akcea Therapeutics, Inc."

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported net income attributable to Ionis’ common stockholders of $2.7 million for the fourth quarter of 2017, compared to $25.9 million for the same period in 2016. For the full years 2017 and 2016, Ionis reported a net loss attributable to Ionis’ common stockholders of $6.0 million and $86.6 million, respectively. For the fourth quarter of 2017, basic and diluted net income per share were $0.02 compared to basic and diluted net income per share of $0.21 for the same period in 2016. For the full year 2017, basic and diluted net income per share were $0.08 compared to basic and diluted net loss per share of $0.72 for the same period in 2016.

Webcast and Conference Call

Today, at 11:30 a.m. Eastern Time, Ionis will conduct a live webcast conference call to discuss this earnings release and related activities. Interested parties may listen to the call by dialing 877-443-5662 or access the webcast at www.ionispharma.com. A webcast replay will be available for a limited time.

On February 27, 2018 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that it will host a conference call and webcast to provide an update on development plans for GMI-1271 in acute myeloid leukemia (AML) as well as to report its fourth quarter and fiscal year 2017 financial results on Tuesday, March 6, 2018, at 8:30 a.m. ET (Press release, GlycoMimetics, FEB 27, 2018, View Source [SID1234524233]).

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The dial-in number for the conference call is (844) 413-7154 for domestic participants and (216) 562-0466 for international participants, with participant code 1453008. A webcast replay will be available via the "Investors" tab on the GlycoMimetics website for 30 days following the call. A dial-in phone replay will be available for 24 hours after the close of the call by dialing (855) 859-2056 for domestic participants and (404) 537-3406 for international participants, participant code 1453008.

About GMI-1271

GlycoMimetics plans to initiate in mid-2018 a Phase 3 clinical trial evaluating GMI-1271 in relapsed/refractory AML patients. In the recently completed Phase 1/2 clinical trial, GMI-1271 was evaluated in both newly diagnosed elderly and relapsed/refractory patients with acute myeloid leukemia (AML). In both populations, patients treated with GMI-1271 together with standard chemotherapy achieved better than expected remission rates and overall survival compared to historical controls, as well as lower than expected induction-related mortality rates. Importantly, treatment in this patient population was well tolerated with minimal adverse effects. The candidate drug is designed to block E-selectin (an adhesion molecule on cells in the bone marrow) from binding with blood cancer cells as a targeted approach to disrupting well-established mechanisms of leukemic cell resistance within the bone marrow microenvironment.

On February 27, 2018 Ophthotech Corporation (Nasdaq:OPHT) reported financial and operating results for the fourth quarter and full year ended December 31, 2017 and provided a business update (Press release, Ophthotech, FEB 27, 2018, View Source [SID1234524253]).

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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

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The Company also announced today that it has initiated an innovative gene therapy program focused on applying novel gene therapy technology to discover and develop new therapies for ocular diseases. The Company intends to investigate promising gene therapy product candidates and other technologies through collaborations with leading companies and academic institutions in the United States and internationally. For its first gene therapy collaboration, the Company has entered into a series of sponsored research agreements with the University of Massachusetts Medical School (UMMS) and its Horae Gene Therapy Center to utilize their "minigene" therapy approach and other novel gene delivery technologies to target retinal diseases. As a condition of each research agreement, UMMS has granted the Company an option to obtain an exclusive license to any patent or patent applications that result from this research. This announcement will be discussed during today’s conference call/webcast (see press release issued earlier today and call in details below).

"In 2017, our team began to execute on a strategic plan to restructure the Company, broaden our Zimura portfolio to include development programs in both orphan diseases and larger indications in the back of the eye, including dry and wet forms of age-related macular degeneration, and to initiate an aggressive business development outreach," stated Glenn P. Sblendorio, Chief Executive Officer and President of Ophthotech. "We start 2018 with a strong balance sheet, multiple clinical trials in our Zimura program, and have now entered into the gene therapy arena with a collaboration with the Horae Gene Therapy Center at University of Massachusetts Medical School. Looking ahead we will continue to seek collaborations, in-licensing and partnering opportunities that offer novel and differentiating approaches, including gene therapy approaches, for treating diseases of the eye."

Zimura Complement Factor C5 Inhibitor Program

During the second half of 2017, the Company modified its on-going Zimura (avacincaptad pegol) clinical trial for the treatment of geographic atrophy (GA) secondary to dry age related macular degeneration (AMD). The modification of the trial design accelerates the anticipated timeline for obtaining top-line data. Initial top-line data is expected to be available during the second half of 2019. The scientific details of the GA secondary to dry AMD clinical trial were presented at the 41st Annual Macula Society Meeting in Beverly Hills, California, February 21-24, 2018.
During the third quarter of 2017, the Company initiated a dose-ranging, open-label Phase 2a clinical trial of Zimura in combination with the anti-vascular endothelial growth factor (anti-VEGF) agent Lucentis (ranibizumab) in patients with wet AMD who have not been previously treated with any anti-VEGF agents. Based on the current enrollment rate, the Company expects initial top-line data from this trial to be available by the end of 2018.
In January 2018, the first patient was enrolled in the Company’s Phase 2b randomized, double-masked, sham-controlled clinical trial assessing the efficacy and safety of Zimura in patients with autosomal recessive Stargardt disease (STGD1). Initial top-line data is expected to be available in 2020. The scientific details of the Stargardt clinical trial will be presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology in Honolulu, Hawaii, April 29 – May 3, 2018 and at The International Symposium on Ocular Pharmacology and Therapeutics in Tel-Aviv, Israel, March 1-3, 2018.
During the fourth quarter of 2017, the Company initiated an open-label Phase 2a clinical trial evaluating Zimura in combination with the anti-VEGF agent Eylea (aflibercept) for the treatment of idiopathic polypoidal choroidal vasculopathy in treatment experienced patients. Initial top-line data is expected to be available during the second half of 2019.

In January 2018, the Company announced the election of Jane PritchettHenderson, Chief Financial Officer and Senior Vice President of Corporate Development at Voyager Therapeutics, to its Board of Directors. Ms. Henderson has also been elected the Chair of the Ophthotech Audit Committee.

2018 Operational Update

As of December 31, 2017, the Company had $167 million in cash and cash equivalents. Based on the Company’s current 2018 business plan, including continuation of its development programs for Zimura and initiation of its collaborative gene therapy research programs, the Company expects the cash required to fund its operations and capital expenditures for 2018 will range between $50 million and $55 million. This estimate does not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or associated development that the Company may pursue.

Fourth Quarter 2017 Financial Highlights

Revenues: Collaboration revenue was $0 for the quarter ended December 31, 2017, compared to $5.3 million for the same period in 2016. For the year ended December 31, 2017, collaboration revenue was $210.0 million, compared to $50.9 million for 2016. Collaboration revenue variances for both the quarter and the year are the result of the completion of deliverables under the Fovista licensing and commercialization agreement with Novartis Pharma AG and the recognition of all associated deferred revenue during the third quarter of 2017. The recognition of this revenue did not impact the Company’s cash balance.
R&D Expenses: Research and development expenses were $7.9 million for the quarter ended December 31, 2017, compared to $59.4 million for the same period in 2016. For the quarter ended December 31, 2017, research and development expenses included approximately $0.7 million in costs related to the Company’s previously announced reduction in personnel. For the year ended December 31, 2017, research and development expenses were $66.3 million, compared to $196.3 million for 2016. For the year ended December 31, 2017, research and development expenses included approximately $7.5 million in costs related to the Company’s previously announced reduction in personnel. Research and development expenses decreased in both the quarter and year ended December 31, 2017 primarily due to its termination of the Fovista development programs in wet AMD.

G&A Expenses: General and administrative expenses were $6.9 million for the quarter ended December 31, 2017, compared to $13.0 million for the same period in 2016. For the quarter ended December 31, 2017, general and administrative expenses included approximately $0.5 million in costs related to the Company’s previously announced reduction in personnel. For the year ended December 31, 2017, general and administrative expenses were $35.7 million, compared to $50.2 million in 2016. For the year ended December 31, 2017, general and administrative expenses included approximately $5.6 million in costs related to the Company’s previously announced reduction in personnel and its termination of facilities leases. General and administrative expenses decreased in both the quarter and year ended December 31, 2017 primarily due to a decrease in personnel and infrastructure costs to support the Company’s operations.
Net Income: The Company reported a net loss for the quarter ended December 31, 2017 of $9.5 million, or ($0.26) per diluted share, compared to a net loss of $66.3 million, or ($1.86) per diluted share, for the same period in 2016. For the year ended December 31, 2017, the Company reported net income of $114.2 million, or $3.17 per diluted share, compared to a net loss of $193.4 million, or ($5.45) per diluted share, for 2016.

Conference Call/Web Cast Information

Ophthotech will host a conference call/webcast to discuss the Company’s financial and operating results and provide a business update. The call is scheduled for February 27, 2018 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 800-239-9838 (USA) or 323-794-2551 (International), passcode 9171013. A live, listen-only audio webcast of the conference call can be accessed on the Investor Relations section of the Ophthotech website at: www.ophthotech.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA Toll Free), passcode 9171013.