On February 28, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for fourth-quarter 2016 and provided an update on the Company’s development programs (Press release, TESARO, FEB 28, 2017, View Source [SID1234517988]).

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"2017 is poised to be an eventful year for TESARO as we prepare for four product launches across the United States and Europe," said Lonnie Moulder, CEO of TESARO. "2016 was an important year for the Company, highlighted by the landmark NOVA trial results for niraparib, which were published in the New England Journal of Medicine and presented in a presidential session at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) annual meeting. We intend to build upon these successes in 2017 with the planned launch of niraparib in the U.S. during the first half of this year and in Europe by year end. Looking beyond ovarian and breast cancer, we aim to finalize our registration program for niraparib in lung cancer in the first half of this year. Lastly, we are excited about the progress in our immuno-oncology pipeline, and expect to initiate a registrational program for TSR-042 in the coming months."

Recent Business Highlights

The U.S. launch of VARUBI continues, with sequential unit volume growth for the fourth quarter of 2016. For the month of December, VARUBI achieved greater than 40% market share in the oral NK-1 market in the U.S., which represents the market-leading position in the category.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) rendered a positive opinion for the marketing authorization application (MAA) for VARUBY for the prevention of delayed nausea and vomiting associated with highly and moderately emetogenic cancer chemotherapy in adults.
In January, TESARO received a Complete Response Letter regarding the rolapitant IV New Drug Application (NDA) for the prevention of delayed nausea and vomiting associated with initial and repeat courses of emetogenic cancer chemotherapy. TESARO expects to be able to address the questions from the U.S. Food and Drug Administration (FDA) and resubmit the NDA to enable approval in the first half of 2017.
The NDA for niraparib was accepted for review by the FDA for patients with recurrent ovarian cancer following response to platinum-based chemotherapy. The FDA granted priority review for the niraparib NDA and established a Prescription Drug User Fee Act (PDUFA) goal date of June 30, 2017.
An expanded access program (EAP) for niraparib opened in the U.S. in January, through which niraparib is being made available for eligible women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer following a complete or partial response to platinum-based chemotherapy. An EAP for niraparib is planned to open in Europe in the first half of 2017, and will be initiated on a country-by-country basis.
Enrollment continues in the Phase 3 PRIMA trial of niraparib for patients with first-line ovarian cancer, the Phase 3 BRAVO trial for patients with germline BRCA-mutated, metastatic breast cancer, and in the QUADRA trial of niraparib for the treatment of patients with ovarian cancer who have received three or more prior lines of chemotherapy.
Based upon the anti-tumor activity observed in Phase 1 of the TOPACIO trial of niraparib plus KEYTRUDA (pembrolizumab) in patients with ovarian cancer or with triple negative breast cancer, the trial has expanded to Phase 2 and is enrolling patients. Initial data are expected at an upcoming medical meeting.
Following the presentation of data at ASCO (Free ASCO Whitepaper) in 2016, the Phase 2 portion of the AVANOVA trial of niraparib plus bevacizumab in patients with ovarian cancer continues to enroll, and updated data are expected in 2H 2017.
Dose escalation and identification of a fixed dose and schedule was completed for the Phase 1 trial of TSR-042, an anti-PD-1 antibody candidate, and a registration trial is planned to begin in 1H 2017.
Enrollment continues in the Phase 1 dose escalation study of TSR-022, an anti-TIM-3 antibody candidate.
A lead PD-1/LAG-3 bi-specific antibody candidate was identified.
Fourth Quarter 2016 Financial Results

TESARO reported revenue of $4.2 million for the fourth quarter of 2016, compared to revenue of $0.2 million for the fourth quarter of 2015. Net product revenue for the fourth quarter of 2016 totaled $2.5 million and included sales of VARUBI from specialty pharmacy customers to patients and from specialty distributors to providers that were made during the fourth quarter. License, collaboration and other revenue for the fourth quarter of 2016 totaled $1.8 million and included amortization of up-front payments and shipments of clinical materials under our license agreements with Hengrui and Janssen.

Research and development expenses increased to $71.5 million for the fourth quarter of 2016, compared to $42.9 million for the fourth quarter of 2015, driven primarily by higher costs related to the ongoing registration trials of niraparib, manufacturing and other research and development costs related to niraparib, advancement of our immuno-oncology portfolio, and increased headcount.

Selling, general and administrative expenses increased to $54.5 million for the fourth quarter of 2016, compared to $27.9 million for the fourth quarter of 2015, primarily due to commercial activities in support of the launch of VARUBI, pre-launch activities related to niraparib, expansion of the European commercial organization, increased headcount and higher professional service fees.

Acquired in-process research and development expenses totaled $9.0 million for the fourth quarter of 2016 and included milestone payments related to niraparib, compared to $1.0 million for the fourth quarter of 2015, which included a milestone payment related to our immuno-oncology portfolio.

Operating expenses, as described above, include total non-cash, stock-based compensation expense of $14.4 million for the fourth quarter of 2016, compared to $8.4 million for the fourth quarter of 2015.

Net loss totaled $136.9 million, or ($2.60) per share, for the fourth quarter of 2016, compared to a net loss of $75.8 million, or ($1.89) per share, for the fourth quarter of 2015.

As of December 31, 2016, TESARO had approximately $785.9 million in cash and cash equivalents and approximately 53.6 million outstanding shares of common stock. Excluding the proceeds from the November 2016 financing, our cash and cash equivalents balance declined $95.7 million in the fourth quarter.

Full-Year 2016 Financial Results

TESARO reported revenue of $44.8 million for 2016, compared to revenue of $0.3 million for 2015. Net product revenue for 2016 totaled $6.9 million and included sales of VARUBI from specialty pharmacy customers to patients and from specialty distributors to providers through December 31, 2016. License, collaboration and other revenue for 2016 totaled $37.9 million and included amortization of up-front payments and shipments of clinical materials under our license agreements with Hengrui and Janssen.

Research and development expenses increased to $235.1 million for 2016, compared to $155.4 million for 2015, driven primarily by three ongoing registration trials of niraparib, increased headcount, and advancement of our immuno-oncology portfolio.

Selling, general and administrative expenses increased to $158.6 million for 2016, compared to $78.7 million for 2015, primarily due to increased headcount, commercial and pre-launch activities in support of VARUBI and niraparib, expansion of the European commercial organization, and higher professional service fees.

Acquired in-process research and development expenses totaled $18.9 million for 2016 and included milestone payments related to niraparib and our immuno-oncology portfolio, compared to $2.0 million for 2015, which included milestone payments related to our immuno-oncology portfolio.

Operating expenses, as described above, include total non-cash, stock-based compensation expense of $48.5 million for 2016, compared to $25.9 million for 2015.

Net loss totaled $387.5 million, or ($8.13) per share, for 2016, compared to a net loss of $251.4 million, or ($6.38) per share, for 2015.

In anticipation of four product launches in 2017, TESARO will continue to invest in pre-launch inventory manufacturing and development of supply chain capabilities and capacity, in addition to making milestone payments for regulatory submissions. TESARO expects its cash and cash equivalents balance to decline by approximately $110 to $120 million on average, per quarter, during the first half of 2017, excluding one-time regulatory milestones of $35 million expected to be paid at the time of the first commercial sale of VARUBY oral in Europe and approval of niraparib in the U.S.

2017 Corporate Objectives

TESARO anticipates achieving the following key objectives:

VARUBI (rolapitant):

Launch VARUBI IV into the U.S. market in mid-2017, pending FDA approval; and
Launch VARUBY oral in Europe in 2Q 2017, pending EMA approval.
Niraparib:

Continue commercial preparations in support of the launches of niraparib in the U.S. in 1H 2017 and Europe by year-end 2017, pending regulatory approvals;
Report initial data from TOPACIO trial at upcoming medical meeting;
Report QUADRA data in 2H 2017;
Report Phase 3 BRAVO data in 2H 2017;
Continue to enroll the Phase 3 PRIMA trial throughout 2017;
Finalize a potential lung cancer registration strategy in 1H 2017 and initiate the development program;
Update AVANOVA data in 2H 2017; and
Describe the potential registration strategy for niraparib plus TSR-042 in ovarian and other cancers in 2H 2017.
Immuno-Oncology Portfolio:

Identify a dose and schedule for TSR-022 (anti-TIM-3 antibody) by mid-2017;
Submit an IND for TSR-033 (anti-LAG-3 antibody) in 2Q 2017;
Finalize the TSR-042 registration strategy and initiate a registration program in 1H 2017;
Identify the first clinical candidate within the MD Anderson collaboration in 1H 2017; and
Initiate a Phase 1 clinical trial of TSR-022 in combination with an anti-PD-1 antibody in mid-2017.

On February 28, 2017 Portola Pharmaceuticals, Inc. (NASDAQ: PTLA) reported a corporate update and reported its financial results for the fourth quarter and year ended December 31, 2016today provided (Filing, Q4/Annual, Portola Pharmaceuticals, 2016, FEB 28, 2017, View Source [SID1234517963]).
"We are working towards gaining approval of both betrixaban, our investigational oral Factor Xa inhibitor, and andexanet alfa, our investigational antidote for oral Factor Xa inhibitors, in the United States and Europe over the next year," said Bill Lis, chief executive officer of Portola. "Each has the potential to become the first product approved for their respective indications and both are highly anticipated by the medical community. We are confident in the robust clinical data for both products, and are focused on addressing the regulatory risks that remain."
Recent Achievements, Upcoming Events and Milestones
Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE; designated Fast Track status by the U.S. Food and Drug Administration (FDA)

• The FDA accepted the New Drug Application (NDA), granted priority review, and established a Prescription Drug User Fee Act (PDUFA) action date of June 24, 2017

• FDA informed Portola at the mid-cycle review that it does not plan to schedule an Advisory Committee meeting to discuss the NDA

• The European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) under a standard 210-day review period; the Committee for Medicinal Products for Human Use (CHMP) opinion is expected by the end of the year
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding; designated a Breakthrough Therapy and an Orphan Drug by the FDA

• Expanded the existing agreement with Daiichi Sankyo; Portola received a $15 million upfront payment and is eligible to receive up to an additional $10 million upon meeting certain milestones; upon AndexXa’s approval, Daiichi will be eligible to receive a capped royalty

• Resubmission of the Biologics License Application (BLA) is anticipated in the second quarter of 2017

• Continued to enroll patients in the ongoing Phase 3b/4 ANNEXA-4 Study in patients with acute major bleeding; the Data and Safety Monitoring Board (DSMB) successfully completed its third review
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients

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

• Entered into an exclusive worldwide licensing agreement with Dermavant Sciences for the development and commercialization of cerdulatinib in topical applications beyond oncology; Portola retains full rights to all non-topical formulations of cerdulatinib, including oral formulations
Corporate

• Entered into a $50 million loan agreement with Bristol-Myers Squibb Company and Pfizer Inc. for continued development and clinical studies of andexanet alfa

• Entered into a $150 million royalty agreement in the first quarter of 2017 with HealthCare Royalty Partners in exchange for a tiered, mid-single-digit royalty based on worldwide andexanet alfa sales
Fourth Quarter and Year-End Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Dermavant Sciences was $13.7 million for the fourth quarter of 2016 compared with $4.4 million for the fourth quarter of 2015. Collaboration and license revenue for the year ended December 31, 2016, was $35.5 million compared with $12.1 million for the year ended December 31, 2015.
Total operating expenses for the fourth quarter of 2016 were $68.9 million compared with $70.7 million for the same period in 2015. Total operating expenses for the fourth quarter of 2016 included $7.9 million in stock-based compensation expense compared with $6.8 million for the same period in 2015. Total operating expenses for the year ended December 31, 2016, were $305.1 million compared with $239.2 million for 2015. Total operating expenses for the full year ended December 31, 2016, included $30.4 million in stock-based compensation expense compared with $22.9 million for 2015.
Research and development expenses for the fourth quarter of 2016 were $56.0 million compared with $59.8 million for the same period in 2015. Research and development expenses were $246.9 million for the year ended December 31, 2016, compared with $200.4 million for 2015. The increase in R&D expenses was primarily due to increased program costs to advance andexanet alfa of $64.7 million, including a $27.3 million one-time charge to write off certain prepaid manufacturing costs and increased program costs to support cerdulatinib and early research programs of $3.8 million, partially offset by decreased program costs related to betrixaban of $22.0 million following the completion of the APEX clinical trial enrollment.
Selling, general and administrative expenses for the fourth quarter of 2016 were $12.9 million compared with $10.9 million for the same period in 2015. Selling, general and administrative expenses for the year ended December 31, 2016, were $58.2 million compared with $38.9 million for 2015. The increase in SG&A expenses was primarily due to increased headcount-related costs, commercial launch preparation activities and business development-related costs, and costs associated with professional and accounting fees of $2.0 million.
For the fourth quarter of 2016, Portola reported a net loss of $53.8 million, or $0.95 net loss per share, compared with a net loss of $66.1 million, or $1.23 net loss per share, for the same period in 2015. Shares used to compute net loss per share attributable to common stockholders
were 56.5 million for the fourth quarter of 2016 compared with 53.6 million for the same period in 2015. Net loss for the year ended December 31, 2016, was $269.0 million, or $4.76 net loss per share, compared with a net loss of $226.5 million, or $4.36 net loss per share, for the same period in 2015. Shares used to compute net loss per share attributable to common stockholders were 56.5 million for 2016 compared with 52.0 million for 2015.
Cash, cash equivalents and investments at December 31, 2016, totaled $318.8 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.
2017 Annual Financial Guidance
For the fiscal year 2017, Portola expects total GAAP operating expenses to be between $323 million and $344 million. For the fiscal year 2017, Portola expects total pro-forma operating expenses to be between $290 million and $310 million, excluding stock-based compensation. These expenses will be primarily for manufacturing of both andexanet and betrixaban, support of ongoing clinical trials and preparation for the potential commercial launches of betrixaban and AndexXa.

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On February 28, 2017 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported a corporate progress update and reported financial results for the year ended December 31, 2016 (Filing, 8-K, MacroGenics, FEB 28, 2017, View Source [SID1234517914]).

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"MacroGenics continues to advance its broad pipeline of clinical compounds, including those in our HER2, B7-H3 and PD-1 franchises as well as the many bispecific product candidates based on our DART platform," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Across our portfolio of proprietary antibody and DART-based oncology programs, we now have initial evidence of on-target engagement, manageable safety and anti-tumor activity. In addition, I am very encouraged by the biological activity observed in subjects treated with MacroGenics’ autoimmune DART molecule, MGD010. We will continue to advance our robust pipeline in 2017, including defining future development strategies for multiple programs based on data expected later this year."
Pipeline Updates
Margetuximab. Recent highlights related to the Company’s Fc-optimized monoclonal antibody that targets the human epidermal growth factor receptor 2, or HER2, include:
•
Phase 3 Metastatic Breast Cancer Study. The pivotal SOPHIA study is evaluating the efficacy of margetuximab plus chemotherapy compared to trastuzumab plus chemotherapy in approximately 530 relapsed/refractory HER2-positive metastatic breast cancer patients. MacroGenics continues to expect to complete enrollment of this study by late 2018. In addition, in consultation with the U.S. Food and Drug Administration (FDA) and the Committee for Medicinal Products for Human Use of the European Medicines Agency, the patient population eligible for participation in SOPHIA has been further expanded to include ado-trastuzumab emtansine-naïve patients.
•
Phase 2 Gastric Cancer Study. The Company continues to enroll advanced HER2-positive gastric and gastroesophageal junction cancer patients in its combination study of margetuximab with an anti-PD-1 antibody. Preliminary data from the dose escalation portion of the study, including patients with objective response following progression on previous lines of treatment with trastuzumab and chemotherapy, was presented at the Company’s R&D Day in December 2016. MacroGenics expects to complete enrollment of this study in 2017.
B7-H3 Franchise. MacroGenics is developing a portfolio of therapeutics that target B7-H3, a member of the B7 family of molecules involved in immune regulation. The Company is advancing multiple programs that target B7-H3 through complementary mechanisms of action that take advantage of this antigen’s broad expression across multiple solid tumor types. These molecules include:
•
Enoblituzumab: The Company continues to recruit patients in multiple ongoing studies of enoblituzumab, an Fc-optimized monoclonal antibody that targets B7-H3. These studies include one monotherapy study expanded to include additional bladder and prostate cancer cohorts and two combination studies with either an anti-CTLA-4 mAb (ipilimumab) or anti-PD-1 mAb (pembrolizumab). In addition, two monotherapy Phase 1 studies were recently initiated: a study for children with various solid tumors, including neuroblastoma, and an investigator-sponsored study in men with localized intermediate and high-risk prostate cancer in the neoadjuvant setting.

•
MGD009: This DART molecule targeting B7-H3 and CD3 is being evaluated in a Phase 1 study across multiple solid tumor types. Adverse events have been manageable to date and initial signs of antitumor activity have been observed, as previously disclosed at the Company’s R&D Day in December 2016. The Company expects to establish the dose and schedule for MGD009 administration as well as initiate dose expansion cohorts in 2017.
•
MGC018: The Company is conducting Investigational New Drug (IND)-enabling activities to support an IND application for this anti-B7-H3 antibody drug conjugate (ADC) in 2018. The Company will feature a poster presentation on MGC018 at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in April.
PD-1-Directed Immuno-Oncology Franchise. MacroGenics is advancing several PD-1-directed programs, which will enable both a broad set of combination opportunities across the Company’s portfolio and provide further differentiation from existing PD-1-based treatment options. The first of these are:
•
MGA012. The Company’s Phase 1 clinical study of its proprietary anti-PD-1 monoclonal antibody was initiated in November 2016. With anti-PD-1 therapy becoming a mainstay of cancer treatment across multiple tumor types, MacroGenics believes MGA012 will be the basis for combination therapy with several of the molecules in its pipeline.
•
MGD013. MacroGenics is developing the DART molecule, MGD013, to provide co-blockade of two immune checkpoint molecules expressed on T cells, PD-1 and LAG-3, for the potential treatment of a range of malignancies. The Company has completed IND-enabling studies and plans to submit an IND for MGD013 in the first half of 2017.
Additional DART Clinical Programs. Additional DART molecules in Phase 1 clinical development include flotetuzumab (CD123 x CD3, also known as MGD006 and S80880); MGD007 (gpA33 x CD3); MGD010 (CD32B x CD79B); duvortuxizumab (CD19 x CD3, also known as MGD011), which is being developed by Janssen; and PF-06671008 (P-cadherin x CD3), which is being developed by Pfizer. At its R&D Day in December 2016, MacroGenics provided an in-depth update on its portfolio of proprietary, clinical DART programs. The Company highlighted the promising features of these DART molecules, including on-target engagement, manageable safety as well as preliminary evidence of biological activity. Updates on three of these programs for which MacroGenics leads development include:
•
Flotetuzumab. MacroGenics continues to recruit patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) in the U.S. and Europe in the Phase 1 study of flotetuzumab. The Company expects to establish the dose and schedule as well as initiate dose expansion cohorts for this study in 2017. In late 2016, the FDA granted orphan drug designation to flotetuzumab for the treatment of AML.
•
MGD007. MacroGenics continues to recruit patients with colorectal cancer in the Phase 1 study of MGD007. The Company expects to establish the dose and schedule for this study in 2017.
•
MGD010. During the fourth quarter of 2016, MacroGenics completed the Phase 1 study of MGD010 in healthy subjects. This DART molecule is being developed for the potential treatment of autoimmune disorders. The Company plans to report updated clinical pharmacodynamic activity results from this study in 2017.
Corporate Update
•
New Board Members: In January 2017, MacroGenics announced the expansion of its Board of Directors from six to eight members and the appointments of Karen J. Ferrante, M.D., and Scott Jackson to fill the newly

created vacancies. Dr. Ferrante, a hematologist-oncologist, most recently served as Chief Medical Officer and Head of Research and Development at Tokai Pharmaceuticals. Mr. Jackson served as Chief Executive Officer and as a member of the Board of Directors of Celator Pharmaceuticals, Inc. until its acquisition in July 2016.
•
Commenced Build-out of GMP Manufacturing Suite: In January 2017, the Company began the expansion of its manufacturing capacity by commencing the construction of a GMP suite in its headquarters building in Rockville, MD to support larger-scale clinical and commercial manufacturing.
2016 Financial Results and Cash Runway Guidance

•
Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2016, were $285.0 million, compared to $339.0 million as of December 31, 2015.
•
Revenue: Total revenue, consisting primarily of revenue from collaborative agreements, was $91.9 million for the year ended December 31, 2016, compared to $100.9 million for the year ended December 31, 2015. Revenue from collaborative agreements includes the recognition of deferred revenue from payments received in previous periods as well as payments received during the year.
•
R&D Expenses: Research and development expenses were $122.1 million for the year ended December 31, 2016, compared to $98.3 million for the year ended December 31, 2015. This increase was due primarily to increased activity in the Company’s preclinical immune checkpoint programs, including MGD013 and MGA012, MGD014 (funded by NIAID/NIH) and the initiation of two Phase 1 clinical trials combining enoblituzumab with other compounds. These increases were partially offset by decreased manufacturing costs for margetuximab.
•
G&A Expenses: General and administrative expenses were $29.8 million for the year ended December 31, 2016, compared to $22.8 million for the year ended December 31, 2015. This increase was primarily due to increased staff, recruiting costs and stock-based (non-cash) compensation expense and patent expense.
•
Net Loss: Net loss was $58.5 million for the year ended December 31, 2016, compared to net loss of $20.1 million for the year ended December 31, 2015.
•
Shares Outstanding: Shares outstanding as of December 31, 2016 were 34,870,607.
•
Cash Runway Guidance: MacroGenics expects that its current cash, cash equivalents and marketable securities, combined with anticipated funding under its current strategic collaborations, should fund the Company’s operations through late 2018.

Conference Call Information

MacroGenics will host a conference call today at 4:30 pm (EDT) to discuss the 2016 financial results and provide a corporate update. To participate in the conference call, please dial (877) 303-6253 (domestic) or (973) 409-9610 (international) five minutes prior to the start of the call and provide the Conference ID: 58247768.
The recorded, listen-only webcast of the conference call can be accessed under "Events & Presentations" in the Investor Relations section of the Company’s website at View Source A replay of the webcast will be available shortly after the conclusion of the call and archived on the Company’s website for 30 days following the call.

MACROGENICS, INC.
SELECTED CONSOLIDATED BALANCE SHEET DATA
(Amounts in thousands)

As of December 31,

2016

2015
Cash, cash equivalents and marketable securities
$
284,982

$
339,049

Total assets
311,263

359,269

Deferred revenue
14,306

18,497

Total stockholders’ equity
268,751

313,337

MACROGENICS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS
(Amounts in thousands, except share and per share data)

Year Ended December 31,

2016

2015

2014
Revenues:

Revenue from collaborative agreements
$
86,582

$
99,368

$
47,264

Revenue from government agreements
5,298

1,486

533

Total revenues
91,880

100,854

47,797

Costs and expenses:

Research and development
122,091

98,271

70,186

General and administrative
29,831

22,765

15,926

Total costs and expenses
151,922

121,036

86,112

Loss from operations
(60,042
)

(20,182
)

(38,315
)

Other income
1,514

42

2

Net loss
(58,528
)

(20,140
)

(38,313
)

Other comprehensive loss:

Unrealized loss on investments
(77
)

(5
)

—

Comprehensive loss
$
(58,605
)

$
(20,145
)

$
(38,313
)

Basic and diluted net loss per common share
$
(1.69
)

$
(0.63
)

$
(1.40
)
Basic and diluted weighted average number of common shares
34,685,274

31,801,645

27,384,990

On February 28, 2017 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported a corporate update and reported its financial results for the fourth quarter and year ended December 31, 2016 (Press release, Portola Pharmaceuticals, FEB 28, 2017, View Source;p=RssLanding&cat=news&id=2250316 [SID1234517893]).

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"We are working towards gaining approval of both betrixaban, our investigational oral Factor Xa inhibitor, and andexanet alfa, our investigational antidote for oral Factor Xa inhibitors, in the United States and Europe over the next year," said Bill Lis, chief executive officer of Portola. "Each has the potential to become the first product approved for their respective indications and both are highly anticipated by the medical community. We are confident in the robust clinical data for both products, and are focused on addressing the regulatory risks that remain."

Recent Achievements, Upcoming Events and Milestones

Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE; designated Fast Track status by the U.S. Food and Drug Administration (FDA)

The FDA accepted the New Drug Application (NDA), granted priority review, and established a Prescription Drug User Fee Act (PDUFA) action date of June 24, 2017
FDA informed Portola at the mid-cycle review that it does not plan to schedule an Advisory Committee meeting to discuss the NDA
The European Medicines Agency (EMA) validated the Marketing Authorization Application (MAA) under a standard 210-day review period; the Committee for Medicinal Products for Human Use (CHMP) opinion is expected by the end of the year
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding; designated a Breakthrough Therapy and an Orphan Drug by the FDA

Expanded the existing agreement with Daiichi Sankyo; Portola received a $15 million upfront payment and is eligible to receive up to an additional $10 million upon meeting certain milestones; upon AndexXa’s approval, Daiichi will be eligible to receive a capped royalty
Resubmission of the Biologics License Application (BLA) is anticipated in the second quarter of 2017
Continued to enroll patients in the ongoing Phase 3b/4 ANNEXA-4 Study in patients with acute major bleeding; the Data and Safety Monitoring Board (DSMB) successfully completed its third review
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients

Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies
Entered into an exclusive worldwide licensing agreement with Dermavant Sciences for the development and commercialization of cerdulatinib in topical applications beyond oncology; Portola retains full rights to all non-topical formulations of cerdulatinib, including oral formulations
Corporate

Entered into a $50 million loan agreement with Bristol-Myers Squibb Company and Pfizer Inc. for continued development and clinical studies of andexanet alfa
Entered into a $150 million royalty agreement in the first quarter of 2017 with HealthCare Royalty Partners in exchange for a tiered, mid-single-digit royalty based on worldwide andexanet alfa sales
Fourth Quarter and Year-End Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, Daiichi Sankyo and Dermavant Sciences was $13.7 million for the fourth quarter of 2016 compared with $4.4 million for the fourth quarter of 2015. Collaboration and license revenue for the year ended December 31, 2016, was $35.5 million compared with $12.1 million for the year ended December 31, 2015.

Total operating expenses for the fourth quarter of 2016 were $68.9 million compared with $70.7 million for the same period in 2015. Total operating expenses for the fourth quarter of 2016 included $7.9 million in stock-based compensation expense compared with $6.8 million for the same period in 2015. Total operating expenses for the year ended December 31, 2016, were $305.1 million compared with $239.2 million for 2015. Total operating expenses for the full year ended December 31, 2016, included $30.4 million in stock-based compensation expense compared with $22.9 million for 2015.

Research and development expenses for the fourth quarter of 2016 were $56.0 million compared with $59.8 million for the same period in 2015. Research and development expenses were $246.9 million for the year ended December 31, 2016, compared with $200.4 million for 2015. The increase in R&D expenses was primarily due to increased program costs to advance andexanet alfa of $64.7 million, including a $27.3 million one-time charge to write off certain prepaid manufacturing costs and increased program costs to support cerdulatinib and early research programs of $3.8 million, partially offset by decreased program costs related to betrixaban of $22.0 million following the completion of the APEX clinical trial enrollment.

Selling, general and administrative expenses for the fourth quarter of 2016 were $12.9 million compared with $10.9 million for the same period in 2015. Selling, general and administrative expenses for the year ended December 31, 2016, were $58.2 million compared with $38.9 million for 2015. The increase in SG&A expenses was primarily due to increased headcount-related costs, commercial launch preparation activities and business development-related costs, and costs associated with professional and accounting fees of $2.0 million.

For the fourth quarter of 2016, Portola reported a net loss of $53.8 million, or $0.95 net loss per share, compared with a net loss of $66.1 million, or $1.23 net loss per share, for the same period in 2015. Shares used to compute net loss per share attributable to common stockholders were 56.5 million for the fourth quarter of 2016 compared with 53.6 million for the same period in 2015. Net loss for the year ended December 31, 2016, was $269.0 million, or $4.76 net loss per share, compared with a net loss of $226.5 million, or $4.36 net loss per share, for the same period in 2015. Shares used to compute net loss per share attributable to common stockholders were 56.5 million for 2016 compared with 52.0 million for 2015.

Cash, cash equivalents and investments at December 31, 2016, totaled $318.8 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.

2017 Annual Financial Guidance
For the fiscal year 2017, Portola expects total GAAP operating expenses to be between $323 million and $344 million. For the fiscal year 2017, Portola expects total pro-forma operating expenses to be between $290 million and $310 million, excluding stock-based compensation. These expenses will be primarily for manufacturing of both andexanet and betrixaban, support of ongoing clinical trials and preparation for the potential commercial launches of betrixaban and AndexXa.

Non-GAAP Financial Projection
This press release and the reconciliation table included herein include a non-GAAP projection of 2017 operating expenses, excluding stock-based compensation. A reconciliation to projected GAAP 2017 operating expenses is provided in the accompanying table entitled "Reconciliation of GAAP to Non-GAAP Projected Operating Expenses." Portola management believes this non-GAAP information is useful for investors because it provides information about the Company’s ability to independently fund and advance its operations with existing capital resources. Share-based compensation is not an expense that requires cash settlement, and therefore, the Company excludes these charges for purposes of evaluating our ability to fund future operations.

On February 28, 2017 Cancer Research UK, Cancer Research Technology (CRT), the charity’s commercial arm, and HitGen Ltd, a privately held biotech company focused on early drug discovery, reported that they have entered into a licence agreement to develop a novel class of drugs against lung cancer (Press release, Cancer Research Technology, 28 28, 2017, View Source [SID1234523167]).

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The work will be carried out by scientists at Cancer Research UK’s Drug Discovery Unit at The University of Manchester, after the new family of compounds was identified using HitGen’s leading technology platform. This involved screening vast DNA encoded libraries, containing many hundreds of millions of small molecules with drug-like properties synthesized on chemically diverse scaffolds. A number of new small molecule leads for this important therapeutic target in lung cancer nominated by Cancer Research UK were revealed.

This is the first licence taken through the ongoing collaboration between CRT, Cancer Research UK’s Manchester Institute Drug Discovery Group and HitGen, who are eligible for milestone payments as the project progresses.

Dr Jin Li, CEO of HitGen, said: "We’re delighted to announce this significant project milestone. We look forward to seeing the progress made by the Cancer Research UK Manchester Institute."

Dr Donald Ogilvie, head of drug discovery at the Cancer Research UK Manchester Institute at The University of Manchester, said: "We’re very pleased to work with HitGen to find promising leads against these more difficult targets that may otherwise not be developed."

"As part of the Cancer Research UK Lung Cancer Centre of Excellence, we’re determined to get new lung cancer treatments to patients quicker. Identifying this promising candidate drug offers the potential to help boost survival from this devastating disease."