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

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Revenues for 2017 increased by more than 45%
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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.
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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.

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Operating income for 2017 increased by more than 150%, driven by strong revenues and reflecting prudent expense management
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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.
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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.

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Substantial cash position enabled pipeline progress
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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.
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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

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SPINRAZA for SMA – one of the most successful orphan drug launches in history
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SPINRAZA, commercialized by Biogen, generated 2017 global sales of $884 million
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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
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Prestigious 2017 Prix Galien USA Award for Best Biotechnology Product awarded to Ionis and Biogen for SPINRAZA
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New collaboration with Biogen initiated to discover new antisense drugs with enhanced properties to treat SMA

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

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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

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

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Pipeline Programs (early and mid-stage) – advancing wholly owned and partnered programs
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Positive results from seven Phase 2 studies reported, including:
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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
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Robust, dose-dependent reductions of mHTT observed in people with Huntington’s disease treated with IONIS-HTTRx
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Positive clinical data on five LICA drugs reported, demonstrating consistent, positive performance and sustained target reduction with potential for monthly or less frequent dosing
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Positive results from six Phase 1 studies reported
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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

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Launch of inotersen for people with hATTR, if approved
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Launch of volanesorsen for people with FCS, if approved
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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
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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:

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

R&D Revenue:

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$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;
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$65 million from Bayer for the license of IONIS-FXI-LRx;
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$48 million from Roche primarily for the license of IONIS-HTTRx;
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$10 million from Janssen for license of IONIS-JBI2-2.5Rx and initiation of Phase 1 study of IONIS-JBI1-2.5Rx;
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$115 million from the amortization of upfront fees; and
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$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.

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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 Celgene Corporation (NASDAQ:CELG) reported that it will present at two upcoming investor conferences where Celgene management will provide an overview of the Company (Press release, Celgene, FEB 27, 2018, View Source [SID1234524195]). The conferences will be webcast live and will be available in the Investor Relations section of the Company’s website at www.celgene.com.

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Tuesday, March 13, 2018, Celgene will present at the Barclays Global Healthcare Conference in Miami at 3:20 pm ET

Wednesday, March 14, 2018, Celgene will present at the Cowen and Company 38th Annual Health Care Conference in Boston at 8:40 am ET

On February 27, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported that it will release its audited annual financial results for the year ended December 31, 2017 on Tuesday, March 6, 2018, before the US markets open (Press release, BioLineRx, FEB 27, 2018, View Source;p=RssLanding&cat=news&id=2334829 [SID1234524194]).

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The Company will host a conference call on Tuesday, March 6, 2018 at 10:00 a.m. EST featuring remarks by Philip Serlin, Chief Executive Officer. The conference call will be available via webcast and can be accessed through the Investor Relations page of BioLineRx’s website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast.

To dial into the conference call, please dial +1-888-668-9141 from the U.S. or +972-3-918-0609 internationally. A replay of the conference call will be available approximately two hours after completion of the live conference call on the Investor Relations page of BioLineRx’s website. A dial-in replay of the call will be available until March 9, 2018; please dial +1-888-326-9310 from the U.S. or +972-3-925-5901 internationally.

On February 27, 2018 BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) reported financial results for the fourth quarter and year ended December 31, 2017 (Press release, BioCryst Pharmaceuticalsa, FEB 27, 2018, View Source [SID1234524193]).

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"Our team made significant progress in 2017 and we are off to a strong start in 2018," said Jon P. Stonehouse, President & Chief Executive Officer. "We are keenly focused on continuing that momentum by advancing our pipeline, adding additional programs and driving our BCX7353 oral prophylactic program toward approval and launch. We are on track to report top-line results from the APeX-2 pivotal trial of BCX7353 and to initiate a Phase 1 clinical trial for our recently unveiled ALK2 inhibitor program for treating FOP in the first half of 2019."

Mr. Stonehouse continued, "In January, we announced our proposed merger with Idera Pharmaceuticals, Inc. that we believe will build greater and more sustainable value for the benefit of stockholders as well as patients with rare diseases beyond what we could achieve alone. The BioCryst Board determined this combination was compelling from both a strategic and financial perspective following a careful evaluation of a range of strategies to enhance long-term stockholder value. The transaction will create a leading rare disease company with a robust pipeline including two promising Phase 3 programs and combines synergistic discovery engines that will not only expand the number of rare diseases we can target but create meaningful opportunities for differentiation in the market through joint small molecule and oligo treatments. Importantly, joining with Idera will also enable us to achieve cost synergies and increase our financial strength and flexibility."

Fourth Quarter Financial Results

For the three months ended December 31, 2017, total revenues were $3.9 million, compared to $9.0 million in the fourth quarter of 2016. The decrease in revenue was primarily due to the recognition of $2.3 million of RAPIVAB product sales to commercial partners in 2016 that did not recur in 2017 and approximately a $2.5 million decline in collaborative revenue in 2017, associated with a decrease in development activity under U.S. Government development contracts.

Research and Development (R&D) expenses for the fourth quarter of 2017 increased to $16.9 million from $12.2 million in the fourth quarter of 2016, primarily due to additions in R&D personnel, as well as increased spending to advance the Company’s hereditary angioedema (HAE) portfolio. These increases were partially offset by a decrease in the Company’s galidesivir development expenses in 2017.

General and administrative (G&A) expenses for the fourth quarter of 2017 increased to $4.7 million, compared to $2.6 million in the fourth quarter of 2016. The increase was primarily due to approximately $1.5 million of merger-related costs associated with the Company’s previously announced definitive merger agreement with Idera Pharmaceuticals, Inc. (Idera).

Interest expense was $2.2 million in the fourth quarter of 2017, compared to $2.1 million in the fourth quarter of 2016. Also, a $71,000 mark-to-market gain on the Company’s foreign currency hedge was recognized in the fourth quarter of 2017, as compared to a $5.7 million mark-to-market gain in the fourth quarter of 2016. These changes result from periodic changes in the U.S. dollar/Japanese yen exchange rate.

Net loss for the fourth quarter of 2017 was $19.5 million, or $0.20 per share, compared to a net loss of $4.5 million, or $0.06 per share, for the fourth quarter 2016.

Full Year 2017 Financial Results

For the year ended December 31, 2017, total revenues decreased to $25.2 million from $26.4 million in 2016. The decrease in 2017 revenue was primarily due to lower collaborative revenue under U.S. Government development contracts as well as lower revenue from product sales to corporate partners. These decreases were largely offset by $7.0 million in milestone payments associated with U.S. pediatric and Canadian regulatory approvals of RAPIVAB.

R&D expenses for 2017 increased to $67.0 million from $61.0 million in 2016, primarily due to increased spending on the Company’s HAE program, partially associated with the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial, as well as an increase in R&D personnel. These increases were partially offset by a decrease in galidesivir development expenses under U.S. Government development contracts.

G&A expenses for 2017 increased to $13.9 million, compared to $11.3 million in 2016. The increase was due primarily to the achievement of a performance-based stock option grant related to the successful completion of the APeX-1 clinical trial as well as merger-related costs associated with the Company’s definitive merger agreement with Idera.

Interest expense was $8.6 million in 2017, compared to $6.5 million in 2016. The increase in interest expense was due primarily to the closing of the Company’s $23 million senior credit facility in September 2016. A $1.8 million mark-to-market loss on the Company’s foreign currency hedge was recognized in 2017, as compared to a $1.7 million mark-to-market loss in 2016. These losses result from periodic changes in the U.S. dollar/Japanese yen exchange rate. During 2017 and 2016, the Company also realized currency gains of $966,000 and $811,000, respectively, from the exercise of a U.S. Dollar/Japanese yen currency option within its foreign currency hedge.

Net loss for 2017 was $65.8 million, or $0.78 per share, compared to a net loss of $55.1 million, or $0.75 per share for the same period last year.

Cash, cash equivalents and investments totaled $159.0 million at December 31, 2017, and reflect an increase from $65.1 million at December 31, 2016. Net operating cash use for 2017 was $41.8 million, which excludes $134.0 million of net proceeds from the March and September 2017 public offerings.

Clinical Development Update & Outlook

Enrollment in the 750 mg cohort of the Zenith-1 proof-of-concept Phase 2 clinical trial of a liquid formulation of BCX7353 for treatment of acute angioedema attacks in HAE has been completed and the 500 mg cohort is currently enrolling. We expect to report top-line results from the first cohort in the second half of 2018.

On January 5, 2018, BioCryst announced that it had advanced a discovery program exploring activin receptor-like kinase-2 (ALK2) inhibitors for treatment of Fibrodysplasia Ossificans Progressiva (FOP) into Investigational New Drug Application (IND) enabling nonclinical development. The Company’s optimized lead candidates, BCX9250 and BCX9499, are projected to enter Phase 1 clinical trials during the first half of 2019.

On January 22, 2018, BioCryst and Idera jointly announced the signing of a definitive merger agreement to create a company focused on the development and commercialization of medicines to serve patients suffering from rare diseases. The combined company will be renamed upon closing, and will be led by Vincent Milano, CEO of Idera. Jon Stonehouse will serve as a member of the Board of Directors. The transaction is subject to approval by the stockholders of both companies, as well as the satisfaction of customary closing conditions. The transaction is expected to be completed by the end of the second quarter of 2018.
Financial Outlook for 2018

Based upon development plans and the Company’s awarded government contracts, on a stand-alone basis, BioCryst expects its 2018 net operating cash use to be in the range of $67 to $90 million, and its 2018 operating expenses to be in the range of $85 to $110 million. The Company’s operating expense range excludes equity-based compensation expense due to the difficulty in reliably projecting this expense, as it is impacted by the volatility and price of the Company’s stock, as well as by the vesting of the Company’s outstanding performance-based stock options.

Company and Idera File Joint Preliminary Proxy Statement / Prospectus and Updated Merger Presentation

The Company also today provided an updated investor presentation regarding the proposed merger with Idera Pharmaceuticals, which was announced on January 22, 2018. The presentation and a joint preliminary proxy statement / prospectus were filed today with the U.S. Securities and Exchange Commission (the "SEC"), and both can be accessed by visiting the "Investors" section of the Company’s website at www.BioCryst.com.

Conference Call and Webcast

BioCryst’s leadership team will host a conference call and webcast Tuesday, February 27, 2018 at 11:00 a.m. Eastern Time to discuss these financial results and recent corporate developments. To participate in the conference call, please dial 1-877-303-8027 (United States) or 1-760-536-5165 (International). No passcode is needed for the call. The webcast can be accessed live or in archived form in the "Investors" section of the Company’s website at www.BioCryst.com. An accompanying slide presentation may also be accessed via the BioCryst website. Please connect to the website at least 15 minutes prior to the start of the conference call to ensure adequate time for any software download that may be necessary.

About BCX7353

Discovered by BioCryst, BCX7353 is a novel, oral, once-daily, selective inhibitor of plasma kallikrein currently in development for the prevention and treatment of angioedema attacks in patients diagnosed with HAE. BCX7353 has been generally safe and well tolerated in the Phase 2 APeX-1 clinical trial. BioCryst is also conducting the ongoing ZENITH-1 clinical trial. ZENITH-1 is a proof-of-concept Phase 2 clinical trial testing an oral liquid formulation of BCX7353 for the treatment of acute angioedema attacks.

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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"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.