On February 27, 2018 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported operating results for fourth-quarter and full-year 2017, and provided an update on the Company’s commercial products and development programs (Press release, TESARO, FEB 27, 2018, View Source [SID1234524265]).

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"Following its April 2017 introduction in the U.S., ZEJULA quickly became the market-leading PARP inhibitor for women with ovarian cancer, and in the second half of 2017, six out of ten ovarian cancer patients who were treated with a PARP inhibitor received ZEJULA," said Lonnie Moulder, CEO of TESARO. "Additionally, we are expanding the ZEJULA franchise with our ongoing launches in Europe and a focused clinical development program that utilizes both monotherapy and combination approaches to potentially further lengthen the time women with ovarian cancer are free from disease progression. 2018 will be an exciting year for our immuno-oncology portfolio as we anticipate multiple data readouts, including response data for TSR-042, our anti-PD-1 antibody, in patients with lung cancer and MSI-high tumors, initial results from the combination of TSR-022, our anti-TIM-3 antibody and TSR-042, and initial data from TSR-033, our anti-LAG-3 antibody."

Recent Business Highlights

ZEJULA is the most utilized PARP inhibitor among patients with ovarian cancer in the U.S., with more than 4,000 patients treated in 2017.
Following European Commission (E.C.) approval in November 2017, ZEJULA now has marketing authorization in 32 countries and is the first and only PARP inhibitor authorized for marketing in Europe for the maintenance treatment of patients with recurrent ovarian cancer, regardless of BRCA mutation status. ZEJULA has been launched in Germany and is available in the UK for private pay patients.
TESARO has applied to include ZEJULA in the UK’s Cancer Drug Fund (CDF) and will continue to work closely with the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England on the ZEJULA CDF submission to make this important medicine available as quickly as possible for a broad population of women in the UK.
The unit demand for VARUBI oral tablets increased 43% for Q4 2017 vs. Q4 2016, as the brand continues to penetrate the U.S. oral NK-1 market.
In January 2018, the package insert for VARUBI IV was updated to include mention of new adverse events, including anaphylaxis, anaphylactic shock and other serious hypersensitivity reactions, which were reported in the post-marketing setting following its introduction in late November 2017. Given these dynamics, TESARO believes the market opportunity is more limited than previously anticipated, and will suspend distribution of VARUBI IV while continuing to support VARUBI oral tablets. The Company is considering strategic alternatives for the product, including out-licensing, and will re-direct Company resources in support of ZEJULA.
Clinical trials of niraparib are ongoing to evaluate safety and efficacy in monotherapy and combination therapy for patients with ovarian, breast, and lung cancer:
PRIMA: Phase 3 trial for patients with first-line ovarian cancer will complete enrollment in Q2 2018
QUADRA: Registrational trial for patients with ovarian cancer who have received three or more prior lines of chemotherapy; top-line data will be available in Q1 and an abstract has been submitted to ASCO (Free ASCO Whitepaper)
TOPACIO: Phase 2 trial in combination with anti-PD-1 for patients with platinum-resistant ovarian cancer (data to be presented at SGO) or triple negative breast cancer (abstracts submitted to ASCO (Free ASCO Whitepaper))
AVANOVA: Phase 2 trial in combination with bevacizumab for patients with recurrent ovarian cancer; data are anticipated to be available in 2H 2018 to support an abstract submission
Niraparib tablet: A study is ongoing to advance development of a tablet formulation of niraparib
OVARIO: Phase 2 assessing niraparib in combination with bevacizumab for patients with newly diagnosed ovarian cancer
Janssen continues to advance development of niraparib in prostate cancer in monotherapy and combination therapy.
Zai Lab is advancing the development of niraparib in patients with ovarian, breast and lung cancer in China, and Takeda has initiated development of niraparib in Japan.
TSR-042 is in a registration trial (GARNET) for MSI-high tumors.
An abstract has been submitted to AACR (Free AACR Whitepaper) that includes data from patients with lung and metastatic microsatellite instability-high (MSI-H) cancers
Data are being generated to support the use of TSR-042 in registration studies in multiple tumor types, including lung, breast and ovarian cancer
Clinical trials are ongoing to evaluate TSR-022 (anti-TIM-3 antibody) and TSR-033 (anti-LAG-3 antibody) in combination with TSR-042.
AMBER: Phase 1 trial of TSR-022 in combination with TSR-042 is enrolling three tumor specific cohorts
CITRINO: Phase 1 dose-escalation trial of TSR-033
A retrospective, exploratory analysis of the NOVA trial, presented as part of a Satellite Symposium at the International Meeting of the European Society of Gynaecological Oncology (ESGO) in November 2017 in Austria, identified body weight and baseline platelet counts as the two most significant predictors for grade 3/4 thrombocytopenia.
In November, preliminary Phase 1 data presented at the 2017 Annual Meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) demonstrated TSR-022 is well tolerated across multiple dose levels, with a safety and efficacy profile expected for checkpoint inhibitors.
TESARO entered into a definitive term loan agreement for up to $500 million with Pharmakon Advisors, LP in November 2017, and drew $300 million in December 2017.

2017 Financial Results

TESARO reported total revenue for the fourth quarter of 2017 of $48.0 million, compared to $4.9 million for the same period in 2016. Revenue growth was primarily driven by the launch of ZEJULA in the U.S. in April 2017. Net loss for the fourth quarter of 2017 totaled $182.1 million, or ($3.35) per share, compared to $136.2 million, or ($2.59) per share for the same period in 2016.

Full year 2017 total revenues were $223.3 million, compared to $58.0 million for 2016. Revenue growth was primarily driven by the launch of ZEJULA in the U.S. and the upfront payment received as part of the license agreement with Takeda in the third quarter. Net loss for 2017 totaled $496.1 million, or ($9.17) per share, compared to a net loss of $374.2 million, or ($7.85) per share, for 2016.
(in thousands, except per share amounts) Three Months Ended
December 31, Twelve Months Ended
December 31,
2016
2017
2016
2017

Product revenue, net
ZEJULA - $ 43,436 - $ 108,756
VARUBI/VARUBY $ 2,330 $ 4,541 $ 5,174 $ 11,944
Total product revenue, net $ 2,330 $ 47,977 $ 5,174 $ 120,700
License, collaboration, and other revenue $ 2,591 $ 46 $ 52,844 $ 102,626
Total revenues $ 4,921 $ 48,023 $ 58,018 $ 223,326

Net loss $ (136,240 ) $ (182,065 ) $ (374,224 ) $ (496,126 )

Net loss per share, basic and diluted $ (2.59 ) $ (3.35 ) $ (7.85 ) $ (9.17 )

Product Revenue

Net product sales totaled $48.0 million for the fourth quarter of 2017, and included ZEJULA sales of $43.4 million and VARUBI/VARUBY sales of $4.5 million. This compares to net product sales of $2.3 million for the fourth quarter of 2016. The increase was primarily driven by the launch of ZEJULA in the U.S. in April 2017.

Net product sales for 2017 totaled $120.7 million and included ZEJULA sales of $108.8 million and VARUBI/Y sales of $11.9 million. For 2016, net product sales were $5.2 million.

Other Revenue

License, collaboration and other revenues for 2017 totaled $102.6 million and included the $100.0 million up-front payment received as part of the license agreement with Takeda in the third quarter. For 2016, license, collaboration, and other revenues were $52.8 million and included up-front payments received as part of the license agreements with Zai Lab and Janssen.

Operating Expenses
(in thousands) Three Months Ended
December 31, Twelve Months Ended
December 31,
2016 2017 2016 2017
Cost of sales – product $ 512 $ 30,857 $ 1,203 $ 41,137
Cost of sales – intangible asset amortization $ 464 $ 1,435 $ 1,855 $ 6,158
Research and development (R&D) $ 71,514 $ 97,832 $ 235,144 $ 308,742
Selling, general and administrative (SG&A) $ 54,526 $ 90,569 $ 158,578 $ 336,808
Acquired in-process R&D $ 9,000 $ 3,000 $ 18,940 $ 10,000

For the fourth quarter of 2017, compared to the same period in 2016:

Cost of sales associated with product sales increased to $30.9 million compared to $0.5 million, primarily due to the commercial launch of ZEJULA in the U.S., and inventory write-downs and other charges of $20.3 million related to revised expectations for future VARUBI IV revenue.

Cost of sales associated with intangible asset amortization increased to $1.4 million compared to $0.5 million primarily due to the amortization of milestones recorded upon FDA and European Commission approval of ZEJULA and first commercial sale of VARUBY in Europe.

R&D expenses increased to $97.8 million compared to $71.5 million primarily due to higher manufacturing costs associated with TSR-042 and TSR-022, the expansion of the niraparib, TSR-042 and TSR-022 clinical development programs, and increased headcount.

SG&A expenses increased to $90.6 million compared to $54.5 million primarily due to increased sales and marketing headcount, activities in support of the launches of ZEJULA and VARUBI/Y in the U.S. and Europe, and higher professional service fees.

Acquired in-process R&D expenses totaled $3.0 million compared to $9.0 million and included a milestone payment related to our immuno-oncology portfolio.

Operating expenses include total non-cash, stock-based compensation expense of $23.5 million, compared to $14.4 million.

For full-year 2017, compared to 2016:

Cost of sales associated with product sales increased to $41.1 million compared to $1.2 million primarily due to the commercial launch of ZEJULA in the U.S., and inventory write-downs and other charges related to revised expectations for future VARUBI IV revenue.

Cost of sales associated with intangible asset amortization increased to $6.2 million compared to $1.9 million primarily due to the amortization of milestones recorded upon FDA and E.C. approvals of ZEJULA and first commercial sale of VARUBY in Europe.

R&D expenses increased to $308.7 million compared to $235.1 million due to increased headcount, higher manufacturing costs associated with TSR-042 and TSR-022, the expansion of the niraparib, TSR-042 and TSR-022 clinical development programs, and the advancement of our earlier-stage immuno-oncology portfolio.

SG&A expenses increased to $336.8 million compared to $158.6 million due to increased sales and marketing headcount, activities in support of the launches of ZEJULA and VARUBI/Y in the U.S. and Europe, and higher professional service fees.

Acquired in-process R&D expenses totaled $10.0 million and included milestone payments related to our immuno-oncology portfolio, compared to $18.9 million, which included milestone payments related to ZEJULA and our immuno-oncology portfolio.

Operating expenses include total non-cash, stock-based compensation expense of $90.4 million compared to $48.5 million.

Cash and Cash Equivalents

As of December 31, 2017, TESARO had approximately $643.1 million in cash and cash equivalents and approximately 54.5 million outstanding shares of common stock.

2018 Financial Guidance

In 2018, TESARO expects:
Total Revenue, net, worldwide (FY) $310 to $345 million
ZEJULA (FY) $255 to $275 million
ZEJULA (Q1) $45 to $50 million
Other revenue, including licensing and VARUBI/Y oral (FY) $55 to $70 million
Interest expense (FY) $50 to $60 million, including non-cash interest expense of $14 million

In addition, TESARO anticipates its cash and cash equivalents balance to decline by $150 million during the first quarter. Quarterly declines in cash and cash equivalents are expected to moderate over the course of 2018, and in the fourth quarter of 2018, the decline in cash and cash equivalents balance is expected to be approximately $75 million. The Company plans to draw $200 million in 2018 from its available term loan facility. TESARO anticipates year-end 2018 cash and cash equivalents to be approximately $400 million.

Key Development Milestones

TESARO intends to achieve the following development milestones during 2018:

Ovarian Cancer Franchise:

Complete PRIMA enrollment in Q2 2018
Report TOPACIO platinum-resistant ovarian cancer data in 1H 2018
Initiate FIRST, a Phase 3 clinical trial of niraparib in combination with TSR-042 in first-line ovarian cancer, in 1H 2018
Report QUADRA data in 1H 2018 and submit sNDA in 2H 2018

Breast Cancer:

Report TOPACIO triple negative breast cancer data in 1H 2018
Publish BRAVO data in 2H 2018
Define registration path for niraparib in breast cancer in mid-2018

Lung Cancer:

Report initial data from lung cancer cohort of the GARNET trial of TSR-042 in NSCLC in 1H 2018
Initial data from Phase 2 JASPER study of niraparib in combination with an anti-PD-1 inhibitor to be available in 2H 2018

Prostate Cancer:

Janssen anticipates advancing trials of niraparib in prostate cancer to support U.S. and EU regulatory filings in 2019

Immuno-oncology Portfolio:

Complete enrollment in the MSI-high cohort of the GARNET trial to support a BLA submission to FDA in 2019
Report initial data for the AMBER trial of TSR-022 in combination with TSR-042 in 2H 2018 and define development strategy
Initiate assessment of the combination of TSR-033 plus TSR-042 in the CITRINO trial in Q2 2018 and report Phase 1 monotherapy dose-escalation data for TSR-033 in 2H 2018
Advance IND-enabling studies of PD-1/LAG-3 bi-specific (TSR-075)

Today’s Conference Call and Webcast
TESARO will host a conference call to discuss fourth quarter and full-year operating results and provide an update on its commercial products and development programs today at 4:15 P.M. Eastern time. The accompanying slide presentation and live webcast of the conference call can be accessed by visiting the TESARO website at www.tesarobio.com. The call can be accessed by dialing (877) 853-5334 (U.S. and Canada) or (970) 315-0307 (international). A replay of the webcast will be archived on the Company’s website for 30 days following the call.

About ZEJULA (Niraparib)
ZEJULA (niraparib) is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in a complete or partial response to platinum-based chemotherapy. In preclinical studies, ZEJULA concentrates in the tumor relative to plasma, delivering greater than 90% durable inhibition of PARP 1/2 and a persistent antitumor effect. Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), including some fatal cases, was reported in patients treated with ZEJULA. Discontinue ZEJULA if MDS/AML is confirmed. Hematologic adverse reactions (thrombocytopenia, anemia and neutropenia), as well as cardiovascular effects (hypertension and hypertensive crisis) have been reported in patients treated with ZEJULA. Monitor complete blood counts to detect hematologic adverse reactions, as well as to detect cardiovascular disorders, during treatment. ZEJULA can cause fetal harm and females of reproductive potential should use effective contraception. Please see full prescribing information, including additional important safety information, available at www.zejula.com.

On February 27, 2018 AMAG Pharmaceuticals, Inc. (NASDAQ: AMAG) today reported unaudited consolidated financial results for the fourth quarter and full year ended December 31, 2017 (Press release, AMAG Pharmaceuticals, FEB 27, 2018, View Source [SID1234524190]).

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Total GAAP revenue for the full year of 2017 increased approximately 15% to $609.9 million, driven by increased sales growth across AMAG’s portfolio, which includes Makena (hydroxyprogesterone caproate injection), Feraheme (ferumoxytol injection) and Cord Blood Registry (CBR), as well as the addition of Intrarosa (prasterone) to the company’s portfolio and subsequent launch in July 2017. The company reported a GAAP operating loss of $293.3 million in 2017, due primarily to a third quarter non-cash accounting charge, and non-GAAP adjusted EBITDA of $230.1 million in 2017.1

"2017 was an exciting year for AMAG and we have had a strong start to 2018," said William Heiden, president and chief executive officer. "In early 2017, we expanded our women’s health product portfolio with the in-licensing of Intrarosa and bremelanotide. Our commercial team performed across the board by driving revenue to a record level, with growth coming from each of the key products in our portfolio. Today we also announced strong earnings in a year when we made significant investments in our new products, as well as in next generation opportunities for Makena and Feraheme — which resulted in two sNDA approvals by the FDA this month."

"We have been evolving AMAG into a more fully integrated pharmaceutical company with strong development capabilities. These recent wins from our clinical and regulatory teams, and another NDA filing with the FDA anticipated this quarter, are a testament to the progress that we have made. This new corporate competency, combined with our track record for commercial excellence, positions AMAG well for an exciting future as we continue to work toward bringing new therapies to patients in need," continued Mr. Heiden.

2017 Highlights and Recent Events:
Financial
•
Achieved record revenue of $609.9 million, with every key AMAG product growing over 2016
•
Reduced total debt by nearly 20% and extended maturities, ending 2017 with cash and cash investments of $328.7 million
Business Development
•
Expanded product portfolio with Intrarosa and bremelanotide and established new 170-person women’s health commercial team
Intrarosa
•
Launched Intrarosa; drove broad awareness and access; over 5,000 HCPs have prescribed Intrarosa
•
Phase 3 hypoactive sexual desire disorder (HSDD) study in post-menopausal women initiated by AMAG’s partner, Endoceutics

1 See summaries of GAAP to non-GAAP adjustments at conclusion of this press release.

1

Makena
•
Received FDA approval for subcutaneous auto-injector (February 14, 2018)
•
Achieved record sales of $387.2 million, an increase of 16% over 2016
Feraheme
•
Received FDA approval for broad IDA label (February 2, 2018)
•
Grew sales to $105.9 million, an increase of 9% over 2016
CBR
•
Increased storage revenue by approximately $7.4 million, or 9%, over 2016
•
Grew new first-time enrollments by 4% over 2016
Bremelanotide
•
Completed clinical work with partner, Palatin, for planned new drug application (NDA) submission in the first quarter of 2018

Fourth Quarter Financial Results Ended December 31, 2017 (unaudited)
GAAP Fourth Quarter Financial Results
Total GAAP revenues for the fourth quarter of 2017 were $158.3 million, compared with $151.6 million for the same period in 2016. In the fourth quarter of 2017, sales of Makena increased to $100.4 million, compared with $97.2 million in the same period last year; sales of Feraheme and MuGard were $26.6 million; and service revenue from CBR increased to $29.8 million, compared with $27.7 million in the same period last year.

Total costs and expenses, including costs of product sales and services, were $165.5 million in the fourth quarter of 2017, compared with $137.0 million in the same period in 2016. This increase was primarily related to a $33.9 million increase in amortization expense for the Makena intramuscular intangible asset, which contributed to an operating loss of $7.1 million, compared with operating income of $14.6 million in the prior year period. The company reported net income of $3.5 million, or $0.10 per basic and diluted share, in the fourth quarter of 2017, compared with a net loss of $10.6 million, or $(0.31) per basic and diluted share, in the same period of 2016. Net income in the fourth quarter of 2017 was driven primarily by a decrease in the deferred tax liability due to federal tax reform.

Non-GAAP Fourth Quarter Financial Results1
Non-GAAP revenue totaled $159.7 million in the fourth quarter of 2017, up from $153.0 million in same period last year. Non-GAAP CBR service revenue totaled $31.2 million in the fourth quarter of 2017, compared with $29.1 million in the fourth quarter of 2016. The difference between GAAP and non-GAAP revenue represents CBR purchase accounting adjustments related to deferred revenue.

Total costs and expenses on a non-GAAP basis totaled $94.0 million, resulting in an adjusted EBITDA margin of 41% in the fourth quarter of 2017. Non-GAAP adjusted EBITDA totaled $65.7 million in the fourth quarter of 2017, compared to $77.4 million recorded in the fourth quarter of 2016. The decline in adjusted EBITDA for the fourth quarter of 2017 was in line with the company’s expectations and previously stated plans to invest in the commercial launch of Intrarosa while also advancing bremelanotide as the company prepares to file the NDA in the first quarter of 2018.

Full Year Financial Results Ended December 31, 2017 (unaudited)
GAAP Full Year Financial Results
Total GAAP revenues in 2017 increased 15% to $609.9 million, compared with $532.1 million in 2016. This increase was driven by record sales of Makena in 2017 and increased sales of Feraheme and CBR, as well as the commercial launch of Intrarosa in July 2017. In 2017, net sales of Makena increased 16% to $387.2 million, compared with $334.1 million in 2016; sales of Feraheme and MuGard increased 9% to $106.7 million, compared with $98.1 million in 2016; and service revenues from CBR increased 15% to $114.2 million, compared with $99.6 million in 2016.

2

Total costs and expenses on a GAAP basis, including costs of product sales and services, totaled $903.2 million in 2017, compared with $453.2 million in 2016. The increase in total costs and expenses in 2017 was primarily due to (i) the third quarter non-cash impairment charge related to the Makena intramuscular intangible asset of $319.2 million, (ii) acquired in-process research and development expense in 2017 of $65.8 million primarily due to our license agreement with Palatin Technologies for the rights to bremelanotide, and (iii) higher amortization expense of the Makena intramuscular intangible asset, partially offset by a reduction in our fair value estimate of contingent consideration.

Higher costs and expenses in 2017 (including the third quarter non-cash impairment charge) resulted in an operating loss of $293.3 million, compared to operating income of $78.9 million in 2016. The company reported a net loss of $199.2 million, or $(5.71) per basic and diluted share in 2017, compared with a net loss of $2.5 million, or $(0.07) per basic and diluted share in 2016.

Non-GAAP Full Year Financial Results2
Non-GAAP revenues increased 12% to $615.4 million in 2017, compared with $549.1 million in 2016. Non-GAAP CBR service revenue totaled $119.7 million in 2017, compared with non-GAAP CBR service revenue of $116.6 million in 2016.

Total costs and expenses on a non-GAAP basis totaled $385.3 million, resulting in adjusted EBITDA of $230.1 million in 2017. This compares to costs and expenses of $283.4 million and adjusted EBITDA of $265.7 million in 2016. The increase of approximately $101.9 million in total costs and expenses in 2017, compared with 2016 was consistent with the company’s stated plan to invest in its expanding portfolio of products. The majority of the increase, approximately $81.8 million, was substantially related to the hiring of the Intrarosa commercial team and costs associated with the product launch during the second half of 2017.

Balance Sheet Highlights
The company ended 2017 with $328.7 million in cash and investments and total debt (principal amount outstanding) of $816.4 million. In 2017, the company reduced its overall indebtedness by nearly 20% and extended maturities through a series of financing transactions that were completed in the second quarter.

2018 Financial Guidance2
The company affirms the following financial guidance for 2018.
$ in millions

Total revenue
$500 – $560
GAAP operating loss
($147) – ($117)
Non GAAP adjusted EBITDA
$100 – $130
2 See reconciliation of 2018 GAAP to non-GAAP financial guidance at conclusion of this press release.

"In 2017, we delivered strong top- and bottom-line results while investing aggressively in the products that we expect will drive future growth and shareholder value," said Ted Myles, executive vice president and chief financial officer. "We also improved our liability profile so that our balance sheet is better aligned with our evolving business strategy. We have a solid plan for 2018 focused on executional excellence across our portfolio, and with two FDA approvals this month, we are off to a great start."

Conference Call and Webcast Access
AMAG Pharmaceuticals, Inc. will host a conference call and webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter and full year 2017 financial results and recent developments.

Dial-in Number
U.S./Canada Dial-in Number: (877) 412-6083

3

International Dial-in Number: (702) 495-1202
Conference ID: 5496159

Replay Dial-in Number: (855) 859-2056
Replay International Dial-in Number: (404) 537-3406
Conference ID: 5496159

A telephone replay will be available from approximately 11:00 a.m. ET on February 27, 2018 through midnight on March 5, 2018.

The webcast with slides will be accessible through the Investors section of AMAG’s website at www.amagpharma.com. A replay of the webcast will be archived on the website for 30 days.

Use of Non-GAAP Financial Measures
AMAG has presented certain non-GAAP financial measures, including non-GAAP revenue and non-GAAP adjusted EBITDA (earnings before income taxes, depreciation and amortization). These non-GAAP financial measures exclude certain amounts, revenue, expenses or income, from the corresponding financial measures determined in accordance with accounting principles generally accepted in the U.S. (GAAP). Management believes this non-GAAP information is useful for investors, taken in conjunction with AMAG’s GAAP financial statements, because it provides greater transparency regarding AMAG’s operating performance. Management uses these measures, among other factors, to assess and analyze operational results and trends and to make financial and operational decisions. Non-GAAP information is not prepared under a comprehensive set of accounting rules and should only be used to supplement an understanding of AMAG’s operating results as reported under GAAP, not as a substitute for GAAP. In addition, these non-GAAP financial measures are unlikely to be comparable with non-GAAP information provided by other companies. The determination of the amounts that are excluded from non-GAAP financial measures is a matter of management judgment and depends upon, among other factors, the nature of the underlying expense or income amounts. Reconciliations between these non-GAAP financial measures and the most comparable GAAP financial measures are included in the tables at the conclusion of this press release.

On February 27, 2018 VistaGen Therapeutics, Inc. (NASDAQ: VTGN), a clinical-stage biopharmaceutical company developing new generation medicines for depression and other central nervous system (CNS) disorders, reported that Shawn Singh, Chief Executive Officer, will present at the Cowen and Company 38th Annual Health Care Conference in Boston at 10:00 a.m. Eastern Time on Wednesday, March 14, 2018 (Press release, VistaGen Therapeutics, FEB 27, 2018, View Source [SID1234524267]).

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A live audio webcast of the presentation will be accessible at the time of the presentation on the investor page of VistaGen’s website at ir.vistagen.com. A replay of the webcast will be archived on VistaGen’s website following the conference.

For more information about the conference, or to schedule a one-on-one meeting with VistaGen’s management, please contact your Cowen representative directly, or send an email to [email protected].

MacroGenics 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, MacroGenics, 2018, FEB 27, 2018, View Source [SID1234524215]).

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On February 27, 2018 Atara Biotherapeutics, Inc. (Nasdaq:ATRA), a leading off-the-shelf T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune and viral diseases, today reported financial results for the fourth quarter and full year ended December 31, 2017 and recent operational highlights (Press release, Atara Biotherapeutics, FEB 27, 2018, View Source [SID1234524191]).

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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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"Atara continues to advance its leadership in T-cell immunotherapy innovation, highlighted by our recent initiation of the first Phase 3 clinical studies of an off-the-shelf, allogeneic T-cell technology, tab‑cel, in the U.S.," said Isaac Ciechanover M.D., Chief Executive Officer and President of Atara Biotherapeutics. "We also recently received FDA clearance to expand our pioneering multinational clinical study of off-the-shelf ATA188 for patients with multiple sclerosis in the U.S. We are proud of these accomplishments and believe the next 18 months will be a transformational period for Atara. Our focus is to expand and advance our robust T‑cell immunotherapy pipeline, manufacturing expertise and global commercial capabilities in anticipation of the announcement of the first tab-cel Phase 3 results and submission of an EU conditional marketing authorization application for tab-cel, both expected in the first half of 2019. We also have a well-defined strategy to leverage the potential of our platform in other cancer, autoimmune and viral diseases, as well as initiate development of genetically modified off-the-shelf T-cell immunotherapies to transform the lives of patients with serious medical conditions."

Recent Highlights and Anticipated Upcoming Milestones

Initiated two Phase 3 clinical studies (MATCH and ALLELE) to evaluate tab-cel in patients with EBV+ PTLD who have failed rituximab following hematopoietic cell transplant (HCT) or solid organ transplant (SOT).
° Six clinical sites for the MATCH and eight for the ALLELE pivotal studies are now open for enrollment in the U.S. and the studies continue to expand to additional U.S. sites as well as sites in other countries including EU, Canada and Australia.
° Results from the first tab-cel Phase 3 study to reach the primary endpoint are expected to be announced in the first half of 2019. Atara also plans to submit a Conditional Marketing Authorization (CMA) application for tab-cel in the EU for patients with EBV+ PTLD who have failed rituximab following HCT during the first half of 2019.

Received clearance of an Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to proceed with patient enrollment at U.S. sites for the Company’s ongoing multinational Phase 1 clinical study to evaluate ATA188 in patients with progressive or relapsing-remitting multiple sclerosis (MS).
° The primary objective of the Phase 1 study is to assess the safety of ATA188 in patients followed for at least one year after the first dose. Key secondary endpoints in the study include measures of clinical improvement such as expanded disability status scale (EDSS) and annualized relapse rate (ARR), as well as MRI imaging.
° We believe that ATA188 may allow for a more consistent reactivity against target EBV antigens, which correlated with clinical improvements in a previous autologous ATA190 Phase 1 study in patients with progressive MS.
° The first results from the ATA188 Phase 1 study in patients with progressive MS are expected in the first half of 2019.
Presented positive interim tab-cel results from a multicenter expanded access protocol (EAP) study for patients with EBV associated cancers at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
° In 6 patients with EBV+ PTLD who have failed rituximab following SOT, the Objective Response Rate (ORR) was 83%, with 5 of 6 patients responding to treatment.
° Additionally, in 5 patients with EBV+ PTLD who have failed rituximab following allogeneic HCT, an ORR of 80% was observed, with 4 of 5 patients responding to treatment.
° Safety findings were reported for a total of 23 patients and demonstrated that tab-cel was generally well-tolerated in this study population, which comprised ill, mostly immunosuppressed patients with multiple comorbidities. Five patients experienced treatment-related serious adverse events (SAEs).
Continue to build core commercial and clinical development capabilities in preparation for the expected submission of the tab-cel CMA in the EU and potential launch.
° Appointed Dr. Derrell Porter as Senior Vice President, Global Commercial Head, who brings extensive oncology and specialty commercialization experience to the management team.
° Appointed Dr. Kanya Rajangam as Senior Vice President and Chief Medical Officer, who has leadership experience advancing multiple global, early- and late-stage oncology programs.
° Identified Atara’s EU headquarters in Zug, Switzerland and began recruiting key global functional leadership and staff.
° Plan to announce partner for Atara MatchMeTM, our off-the-shelf T-cell immunotherapy delivery solution, in the first half of 2018.

Plan to initiate a Phase 1/2 clinical study of tab-cel in combination with Merck’s anti-PD-1 (programmed death receptor-1) therapy, KEYTRUDA (pembrolizumab), in patients with platinum-resistant or recurrent EBV-associated nasopharyngeal carcinoma (NPC) in the second half of 2018.

Expect to present updated tab-cel results in patients with EBV+ cancers in the second half of 2018.

Plan to communicate development strategy for CMV and other viral disease programs in the second half of 2018.

Expect operations to commence at Atara T Cell Operations & Manufacturing (ATOM) facility in 2018, with clinical production anticipated in 2019.
Fourth Quarter and Full Year 2017 Financial Results

Cash, cash equivalents and short-term investments as of December 31, 2017 totaled $166.1 million, which the Company believes, along with the $131.4 million net proceeds from the sale of 7,675,072 shares of common stock in an underwritten public offering completed in January 2018, will be sufficient to fund planned operations into the first half of 2020.
The Company reported net losses of $35.3 million, or $1.15 per share and $119.5 million, or $4.00 per share, for the fourth quarter and fiscal year 2017, as compared to $18.2 million, or $0.63 per share and $79.0 million, or $2.75 per share, for the same periods in 2016.
Research and development expenses were $24.8 million and $81.2 million for the fourth quarter and fiscal year 2017, as compared to $13.5 million and $56.5 million for the same periods in 2016. The increases in the fourth quarter and fiscal year 2017 were due to costs associated with the Company’s continuing expansion of research and development activities, including:
° manufacturing and outside service costs related to the preparation for the two tab-cel Phase 3 clinical studies in patients with EBV+ PTLD who have failed rituximab;
° ongoing costs for the Company’s EAP clinical study for tab-cel, which was initiated in mid-2016;
° clinical manufacturing and preparations for the Phase 1 clinical study of allogeneic ATA188, which was initiated in October 2017;
° higher payroll and related costs from increased headcount, and
° an increase in allocated facilities and information technology expenses.
Research and development expenses include $2.5 million and $8.8 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2017, as compared to $0.4 million and $7.6 million for the same periods in 2016.
General and administrative expenses were $11.0 million and $40.3 million for fourth quarter and fiscal year 2017, as compared to $5.3 million and $24.7 million for the same periods in 2016. The increases in the fourth quarter and fiscal year 2017 were primarily due to increases in payroll and related costs driven by increased headcount to support the Company’s expanding operations and higher professional services costs. General and administrative expenses include $3.6 million and $14.3 million of non-cash stock-based compensation expenses for the fourth quarter and fiscal year 2017, as compared to $1.3 million and $9.2 million for the same periods in 2016.
About EBV+ PTLD
Since its discovery as the first human oncovirus, Epstein-Barr virus (EBV) has been implicated in the development of a wide range of lymphoproliferative disorders, including lymphomas and other cancers. EBV is widespread in all human populations and persists as a lifelong, asymptomatic infection. In immunocompromised patients, such as those undergoing allogeneic hematopoietic cell transplants (HCT) or solid organ transplants (SOT), EBV associated post-transplant lymphoproliferative disorder (EBV+ PTLD), represents a life-threatening condition. Median overall survival in patients with EBV+ PTLD following HCT who have failed rituximab-based first line therapy is 16-56 days. In EBV+ PTLD following SOT, patients failing rituximab experience increased chemotherapy-induced treatment-related mortality compared to other lymphoma patients. One- and two-year survival in patients with high-risk EBV+ PTLD following SOT is 36% and 0%, respectively.

About tab-cel (tabelecleucel; formerly known as ATA129)
Atara’s most advanced T-cell immunotherapy in development, tab-cel, is a potential treatment for patients with Epstein-Barr virus (EBV) associated post-transplant lymphoproliferative disorder (EBV+ PTLD) who have failed rituximab, as well as other EBV associated hematologic and solid tumors, including nasopharyngeal carcinoma (NPC). In February 2015, FDA granted tab-cel Breakthrough Therapy Designation for EBV+ PTLD following allogeneic hematopoietic cell transplant (HCT) and in October 2016, tab-cel was accepted into the EMA Priority Medicines (PRIME) regulatory pathway for the same indication, providing enhanced regulatory support. Atara also received positive regulatory feedback from Health Canada in September 2017 supporting the submission of tab-cel for an expedited approval pathway. In addition, tab-cel has orphan status in the U.S. and EU. Tab-cel is in Phase 3 clinical development for the treatment of EBV+ PTLD following an allogeneic hematopoietic cell transplant (MATCH study) or solid organ transplant (ALLELE study), and a Phase 1/2 study in NPC is planned for 2018. Tab-cel is also available to eligible patients with EBV associated hematologic and solid tumors through an ongoing multicenter expanded access protocol clinical study, positive interim results of which were presented in December 2017 at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

About Multiple Sclerosis (MS)
MS is a chronic neurological autoimmune disease that affects an estimated 2.3 million people around the world. Relapsing-remitting MS (RRMS) is the most common form of MS and is characterized by episodes of new or worsening signs or symptoms (relapses) followed by periods of recovery. Despite available disease-modifying treatments, most individuals with RRMS continue to experience disease activity and disability progression.

Progressive MS (PMS) is a severe form of the disease with few therapeutic options. PMS comprises two conditions, both characterized by persistent progression and worsening of MS symptoms and physical disability over time. Primary Progressive MS (PPMS) occurs when continuous progressive disease is present at diagnosis and occurs in approximately 15% of newly diagnosed cases. Secondary Progressive MS (SPMS) initially begins as RRMS and develops into a progressive form. Up to 80% of people with RRMS will eventually develop SPMS. There is substantial unmet medical need for new and effective therapies for patients with PPMS and SPMS. Most treatment options that work well in reducing flares in RRMS have not been shown to be effective in slowing or reversing disability in PMS.

About allogeneic ATA188 and autologous ATA190
Epstein-Barr Virus (EBV) is associated with a wide range of hematologic malignancies and solid tumors, as well as certain autoimmune conditions such as multiple sclerosis (MS). T-cells are a critical component of the body’s immune system and can selectively target specific EBV antigens believed to be important for the potential treatment of MS. Off-the-shelf ATA188 and autologous ATA190, using the Company’s complementary T-cell immunotherapy technology developed by Professor Rajiv Khanna at QIMR Berghofer, have the potential to precisely recognize and eliminate EBV-infected B-cells and plasma cells in the central nervous system that may catalyze autoimmune responses and MS pathophysiology. Professor Michael Pender from The University of Queensland presented updated results from the first autologous ATA190 study, which was partially funded by MS Research Australia, MS Queensland and Perpetual Foundation, at MSParis 2017 Congress, the 7th Joint ECTRIMS and ACTRIMS Meeting in October 2017. This study tested adoptive immunotherapy in patients with MS and showed that autologous ATA190 led to encouraging clinical improvements in MS symptoms that correlated with autologous ATA190’s reactivity against target EBV antigens (EBV reactivity). In addition to the ongoing Phase 1 autologous ATA190 clinical study in patients with progressive MS, Atara also initiated a multinational Phase 1 ATA188 clinical study in patients with progressive or relapsing-remitting MS in Australia in the fourth quarter of 2017 with patient enrollment at U.S. sites beginning in early 2018.

About CMV
In patients with weakened immune systems, including bone marrow and solid organ transplant recipients, newborns with immature immune systems and those with human immunodeficiency virus (HIV), cytomegalovirus (CMV) can cause potentially life-threatening disease or may result in blindness, brain damage, and deafness. While small molecule antiviral drugs are approved to treat and prevent CMV infection, there remains a high unmet need due to viral resistance, modest neurodevelopmental activity and adverse effects, such as toxicity and reduction in white blood cell count impairing the ability to fight other infections, with these agents.

About ATA230
ATA230, an allogeneic T-cell immunotherapy targeting antigens expressed by cytomegalovirus (CMV), has been investigated in one Phase 1 and two Phase 2 clinical studies in immunocompromised patients with CMV viremia or disease who are refractory or resistant to antiviral drug treatment in the post-transplant setting. In October 2017, Atara announced that ATA230 was granted Rare Pediatric Disease Designation by the FDA for the treatment of congenital CMV infection, and in September 2017, ATA230 received orphan drug designation in the U.S. for the treatment of CMV viremia and disease in immunocompromised patients. The European Medicines Agency (EMA) in October 2016 also issued a positive orphan drug designation opinion for ATA230 for the treatment of CMV infection in patients with impaired cell-mediated immunity. Given the opportunity to pursue a CMA in the EU for tab-cel, we have decided to prioritize our EBV related programs ahead of ATA230 at this time, and plan to further evaluate our development strategy for ATA230 in 2018.