On May 4, 2018 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported financial results for the first quarter of 2018 and highlighted its recent business and pipeline successes (Press release, Ionis Pharmaceuticals, MAY 4, 2018, View Source [SID1234526135]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

"We have successfully executed our commercialization strategy through the expansion of our strategic relationship with Biogen in neurological diseases and our commercialization of inotersen through our affiliate, Akcea. Our strategy is to create tailored commercial solutions for each of our drugs with the aim to maximize the commercial value of the drug and optimize our financial participation in this value. As our most recent collaboration with Biogen demonstrates, we have substantially increased the value of our antisense technology platform in neurological diseases over the last several years. The economics we achieved with our Bayer and Novartis partnerships show that we have done the same in multiple other therapeutic areas," said Stanley T. Crooke, M.D., Ph.D., chairman of the board and chief executive officer. "Looking ahead, we are focused on launching TEGSEDI this year, assuming approval. As Akcea announced yesterday, the FDA decided they needed additional time to review some of our responses to their standard information requests and, therefore, has extended the review period for TEGSEDI. The new PDUFA date is October 6, 2018. We are working closely with the FDA to advance the review of our filing as quickly as possible. This year we also plan to launch WAYLIVRA, assuming approval. The commercialization of these two Ionis drugs will solidify Ionis as a multi-product, profitable company delivering innovative antisense medicines to patients in need."

First Quarter 2018 Financial Highlights

·
Revenues increased by 25%, driven by SPINRAZA royalties
o
Total revenues were $144 million, compared to $116 million in Q1 2017
o
Commercial revenue from SPINRAZA royalties was $41 million, compared to $5 million in Q1 2017
o
R&D revenue included $60 million in licensing fees for two drugs discovered by Ionis under its collaboration with AstraZeneca
o
Beginning in Q2 2018, Ionis’ R&D revenue will include revenue from the amortization of the $500 million technology access fee and equity premium related to Ionis’ expanded strategic research collaboration with Biogen

1
·
GAAP operating and net loss near breakeven; on track for third consecutive year of pro forma operating profitability
o
GAAP operating loss was $3 million in Q1 2018, compared to GAAP operating income of $19 million for the same period in 2017. Pro forma operating income was $25 million in Q1 2018, compared to $40 million for the same period in 2017
o
Operating expenses increased primarily due to higher SG&A expenses as Ionis prepares to commercialize TEGSEDI and WAYLIVRA this year

·
Cash will increase to more than $2 billion, combining Ionis’ first quarter cash balance of more than $1 billion with $1 billion expected upon closing of Ionis’ expanded collaboration with Biogen
o
During Q1 2018, Ionis received more than $155 million in payments from partners

"In the first quarter, we made further progress toward our goal of being a multiproduct, profitable company. We ended the quarter with operating income of $25 million and net income of $27 million, both on a pro forma basis. Our strong first quarter results were driven by a 25% increase in revenue, primarily from substantial SPINRAZA royalties. Completing our seventh consecutive quarter of pro forma operating income keeps us on track for our third consecutive year of proforma operating income even while we prepare to launch two drugs. This strong performance is a direct result of our technology platform delivering high-value drugs combined with our business strategy, which aims to maximize the commercial potential of each of our drugs and optimize our participation in this value," said Elizabeth L. Hougen, chief financial officer of Ionis. "With the $2 billion in cash we expect to have on the closing of the Biogen transaction, we have the financial strength to invest in opportunities that we believe will increase shareholder value, such as advancing and expanding our portfolio of drugs, retaining our drugs longer, and building a growing pipeline of Ionis-owned drugs that we commercialize ourselves through commercial affiliates."

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.

Business Highlights

·
Expanded strategic research collaboration with Biogen for neurological diseases – one of the largest research-stage collaborations ever
o
$1 billion upfront to Ionis, including $625 million to purchase Ionis’ stock at a 25% cash premium of $125 million and a $375 million upfront payment
§
Together, the cash premium and upfront payment represent a $500 million technology access fee
o
Ionis is eligible to receive milestone payments and license fees up to $270 million per drug and royalties up to 20% on net sales
o
Disease areas include dementia, neuromuscular diseases, movement disorders, ophthalmology, diseases of the inner ear, and neuropsychiatry

2
·
SPINRAZA for SMA – one of the most successful orphan disease drug launches in history
o
SPINRAZA, commercialized by Biogen, continued to generate growth with global revenues of $364 million in Q1 2018
o
Increase of over 25% from last quarter in number of patients on SPINRAZA, including a 16% increase in number of patients treated in the U.S. and a more than 50% increase outside the US
o
Access expanding outside the U.S. with reimbursement in 24 countries; Biogen expects reimbursement in at least seven more countries by the end of 2018
o
Presented data from the SHINE open-label study at the American Academy of Neurology (AAN) annual meeting demonstrating continued benefit, improved motor function and mobility, and longer event-free survival for the most severely affected patients treated with SPINRAZA
o
Presented data from the NURTURE study at the Muscular Dystrophy Association (MDA) Clinical Conference demonstrating continued benefit in motor function for infants, teens and young adults treated with SPINRAZA

·
TEGSEDI (inotersen) for hereditary transthyretin amyloidosis (hATTR) – potential to transform the lives of people with hATTR; on-track to launch in 2018
o
Invested in global commercialization of TEGSEDI by licensing TEGSEDI to Ionis’ majority-owned affiliate, Akcea
§
Optimized Ionis’ commercial participation with up to $1.5 billion in milestone payments and a 60% profit share
o
Early access program enrolling in the U.S. and Europe
o
Global commercial organization staffed and focused on disease education; robust patient support program in place; supply chain in place and launch supplies ready to be labeled
o
Presented data from the Phase 3 NEURO-TTR study, the open label extension study and an investigator sponsored Phase 2 study at the International Symposium on Amyloidosis annual meeting and the AAN annual meeting

·
WAYLIVRA (volanesorsen) for FCS and FPL – potential first treatment for people with FCS; global on-track to launch in 2018
o
Early access program enrolling in the U.S. and Europe
o
Global commercial organization staffed and focused on disease education; robust patient support program in place; supply chain in place and launch supplies ready to be labeled
o
Positive scientific opinion to initiate Early Access to Medicines Scheme (EAMS) by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA), for the treatment of people with FCS.

·
Collaboration with AstraZeneca for Cardiovascular, Renal and Metabolic Diseases
o
Earned $60 million for the license of second and third antisense drugs, IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx, to treat a genetically associated form of kidney disease and nonalcoholic steatohepatitis (NASH), respectively, to AstraZeneca
o
As IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx advance, Ionis may receive up to $300 million for each drug in additional development and regulatory milestone payments, as well as tiered royalties on sales of each drug

3
Pipeline and Technology Progress

·
Presented positive IONIS-HTTRx (RG6042) Phase 1/2 data in people with Huntington’s disease (HD) at the annual CHDI HD conference. IONIS-HTTRx is the first drug in development to lower the disease-causing protein in people with HD
·
Presented data at the AAN annual meeting that demonstrated broad potential of antisense drugs for neurological diseases with 14 presentations on Ionis’ drugs to treat neurological diseases, including SMA, hATTR amyloidosis, Huntington’s disease, Alzheimer’s disease, and ALS
o
Presented additional data from the Phase 1/2 study of IONIS-HTTRx that demonstrated correlations between reductions in mutant huntingtin (mHTT) and improvements in clinical measures of HD
·
Published review paper titled, "RNA-targeted Therapeutics" in Cell Metabolism, authored by Stanley Crooke, M.D., Ph.D.; highlights antisense and other RNA-targeting therapeutics as important platforms for drug discovery across multiple diseases

"This year, we plan to launch two new promising drugs for rare diseases, TEGSEDI and WAYLIVRA. We look forward to adding commercial revenue from these drugs, assuming approval, to our growing revenue from SPINRAZA," said Brett P. Monia, Ph.D., chief operating officer and senior vice president of antisense drug discovery and translational medicine at Ionis Pharmaceuticals. "Our next set of commercial opportunities are on the horizon. We have multiple drugs that we or our partners plan to advance into pivotal studies in the next year or so, including IONIS-HTTRx for patients with Huntington’s disease, IONIS-STAT3-2.5Rx for patients with head and neck cancer and AKCEA-APO(a)-LRx in patients with high Lp(a) and risk of cardiovascular disease. Following closely behind these are drugs for rare diseases that have the potential to move quickly toward the market, including IONIS-GHR-LRx for patients with acromegaly and IONIS-TMPRSS6-LRx for patients with beta thalassemia."

Expected Events Through 2018

·
Launch of TEGSEDI for people with hATTR, assuming approval
·
Launch of WAYLIVRA for people with FCS, assuming approval
·
Report results from six Phase 2 programs, including data from a study with AKCEA-APO(a)-LRx in people with high Lp(a) and AKCEA-ANGPTL3-LRx for people with rare hyperlipidemias
·
Initiate up to nine new clinical studies, including a clinical study of AKCEA-TTR-LRx for hereditary and wild-type forms of ATTR

The recent Biogen transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart Scott Rodino Antitrust Improvements Act of 1976 in the United States. Biogen and Ionis expect the deal to close in the second quarter of 2018.

Revenue

At the beginning of 2018, Ionis adopted the new revenue recognition accounting standard on a retrospective basis. Starting with Ionis’ first quarter, all periods presented are shown using the new standard. Ionis has labeled its prior period financial statements "as revised" to indicate the change required under the accounting rules. Whenever Ionis refers to prior period results, they reflect the new accounting rules. This change did not have a significant impact on Ionis’ previously reported revenue.

Ionis’ revenue in the first quarter of 2018 was $144.4 million, compared to $115.8 million for the same period in 2017 and was comprised of the following (amounts in millions):

License fees in the first quarter of 2018 were $62 million primarily from AstraZeneca for the license of IONIS-AZ5-2.5Rx and IONIS-AZ6-2.5-LRx. The first quarter of 2017 included $65 million in a license fee from Bayer for the license of IONIS-FXI-LRx.

Operating Expenses

Operating expenses for the first quarter on a GAAP basis were $147.7 million and on a pro forma basis were $119.3 million compared to GAAP operating expenses of $96.3 million and pro forma operating expenses of $75.4 million for the same period in 2017. Operating expenses increased in Q1 2018, compared to 2017, principally due to higher SG&A expenses as Ionis and its affiliate Akcea prepare to commercialize WAYLIVRA and TEGSEDI. The Company’s SG&A expenses also increased in Q1 2018 compared to Q1 2017 because of fees owed under its in-licensing agreements related to SPINRAZA, which increase as the Company’s SPINRAZA revenue increases. R&D expenses accounted for a smaller portion of the increase in operating expenses. R&D expenses increased primarily from medical affairs expenses and manufacturing costs related to TEGSEDI for the planned launch.

Net Income (Loss)

Ionis reported a net loss of $10.8 million for the first quarter of 2018, compared to net income of $9.0 million for the same period in 2017, all according to GAAP. On a pro forma basis, Ionis reported net income of $17.6 million for the first quarter of 2018, compared to net income of $29.9 million for the same period in 2017. Ionis’ GAAP net loss increased and its pro forma net income decreased in the first quarter of 2018 primarily due to increased operating expenses as Ionis prepares to commercialize TEGSEDI and WAYLIVRA.

5
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 the first quarter in 2018, 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 first quarter of 2018 was $9.4 million. Ionis also added a corresponding account in its stockholders’ equity section on its balance sheet called "Noncontrolling interest in Akcea Therapeutics, Inc."

In April 2018, Ionis received 8 million shares of Akcea stock for the license of TEGSEDI and AKCEA-TTR-LRx to Akcea and purchased an additional 10.7 million shares of Akcea stock for $200 million, increasing Ionis’ ownership percentage to approximately 75 percent. Ionis will reflect this increase in its ownership percentage in the second quarter of 2018.

Net Income (Loss) Attributable to Ionis Common Stockholders

Ionis reported a GAAP net loss attributable to Ionis’ common stockholders of $1.4 million for the first quarter of 2018, compared to GAAP net income of $9.0 million for the same period in 2017. For the first quarter of 2018, basic and diluted net loss per share were $0.01. For the first quarter of 2017, basic and diluted net income per share were $0.07.

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 May 4, 2018 DXC Technology (NYSE: DXC), the world’s leading independent, end-to-end IT services company, reported that it will release financial results for the fourth quarter and full year fiscal 2018 on Thursday, May 24, 2018, at approximately 4:15 p.m. Eastern Daylight Time (EDT) (Press release, DynPort Vaccine Company, MAY 4, 2018, View Source [SID1234526134]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

DXC Technology senior management will host a conference call and webcast on the same day at 5 p.m. EDT. The dial-in number for domestic callers is (800) 289-0438. Callers who reside outside of the United States should dial +1 (323) 794-2423. The passcode for all participants is 6166236. The webcast audio and any presentation slides will be available on DXC Technology’s Investor Relations website.

A replay of the conference call will be available from approximately two hours after the conclusion of the call until May 31, 2018. Replay numbers can be found at the following link. The replay passcode is also 6166236.

On May 4, 2018 Delcath Systems, Inc. (OTCQB:DCTHD), an interventional oncology Company focused on the treatment of primary and metastatic liver cancers, reported that Jennifer K. Simpson, Ph.D., MSN, CRNP, President and Chief Executive Officer of Delcath, will present at the RHK Capital 2018 Disruptive Growth and Healthcare Conference being held May 8-9, 2018 at Reed Smith, LLP in New York. Dr. Simpson’s presentation details are as follows (Press release, Delcath Systems, MAY 4, 2018, View Source;p=RssLanding&cat=news&id=2347110 [SID1234526133]):

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Panel Presentation:

Session: Disruptive Injectables & Oncology
Date and Time: Tuesday, May 8 at 1:45-2:45 p.m. EST
Location: Track 2 – Room C/D
Corporate Presentation:

Date and Time: Tuesday, May 8 at 3:25-3:45 p.m. EST
Location: Room A/B

On May 4, 2018 Varian (NYSE: VAR) commented on the press release of Sirtex Medical Limited (Sirtex) dated May 4, 2018 reported that Sirtex had received an unsolicited non-binding, indicative and conditional proposal from CDH Investments, a China-based alternative asset manager, for the acquisition of all of the issued shares in Sirtex (CDH Proposal) (Press release, Varian Medical Systems, MAY 4, 2018, https://www.varian.com/news/varian-notes-receipt-competing-bid-sirtex [SID1234526132]). The CDH Proposal is subject to a number of conditions following completion of satisfactory confirmatory due diligence, notably the approval of CDH’s Investment Committee and the Australian Foreign Investment Review Board.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

In line with their fiduciary duties, the Sirtex board of directors has determined to engage with CDH Investments to further understand the conditions associated with the CDH Proposal. In light of these developments, Sirtex intends to seek an adjournment of the meeting of Sirtex stockholders to approve the Scheme Implementation Deed between Sirtex and Varian (Varian Scheme), which was scheduled to occur on Monday, May 7, 2018 (Sydney time) to a time and date to be determined.

In connection with this, Varian reiterates its belief that the Varian Scheme remains in the best interests of Sirtex and its stockholders.

"The Varian Scheme has been unanimously approved by both boards of directors, has fully committed financing and has received all necessary regulatory approvals. We believe the Varian Scheme offers more value and carries far less risk for Sirtex stockholders," said Dow Wilson, President and Chief Executive Officer of Varian. "We stand ready to complete the acquisition following the receipt of Sirtex stockholders’ approval and the satisfaction of other closing conditions. We look forward to welcoming our new Sirtex colleagues to Varian."

Varian also notes that the directors of Sirtex continue to believe that the Varian Scheme is in the best interests of Sirtex shareholders and continue to unanimously support and recommend the Varian Scheme.

On May 4, 2018 Celgene Corporation (NASDAQ:CELG) reported net product sales of $3,531 million for the first quarter of 2018, a 20 percent increase from the same period in 2017 (Press release, Celgene, MAY 4, 2018, View Source [SID1234526130]). Celgene reported first quarter 2018 total revenue of $3,538 million, a 19 percent increase compared to $2,962 million in the first quarter of 2017.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Based on U.S. GAAP (Generally Accepted Accounting Principles), Celgene reported net income of $846 million and diluted earnings per share (EPS) of $1.10 for the first quarter of 2018. For the first quarter of 2017, GAAP net income was $932 million and diluted EPS was $1.15.

Adjusted net income for the first quarter of 2018 increased 16 percent to $1,572 million compared to $1,355 million in the first quarter of 2017. For the same period, adjusted diluted EPS increased 23 percent to $2.05 (inclusive of approximately $0.05 dilution from the Juno acquisition) from $1.67.

"Strong global demand and excellent commercial execution drove our exceptional first quarter results, leading to improvement in our 2018 financial guidance," said Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation. "With multiple catalysts for growth expected over the next 12 to 18 months, we are reaffirming our 2020 outlook."

First Quarter 2018 Financial Highlights

Unless otherwise stated, all comparisons are for the first quarter of 2018 compared to the first quarter of 2017. The adjusted operating expense categories presented below exclude share-based employee compensation expense, research and development asset acquisition expense, collaboration-related upfront expense and a benefit associated with the adjustment to clinical trial and development activity wind-down costs. Please see the attached Use of Non-GAAP Financial Measures and Reconciliation of GAAP to Adjusted Net Income for further information relevant to the interpretation of adjusted financial measures and reconciliations of these adjusted financial measures to the most comparable GAAP measures, respectively.

Net Product Sales Performance

REVLIMID sales for the first quarter increased 19 percent to $2,234 million. Sales growth was primarily volume-driven due to increases in treatment duration and market share. U.S. sales of $1,487 million and international sales of $747 million increased 21 percent and 15 percent year-over-year, respectively.
POMALYST/IMNOVID sales for the first quarter were $453 million, an increase of 24 percent year-over-year. U.S. sales were $300 million and international sales were $153 million, an increase of 39 percent and 3 percent year-over-year, respectively. POMALYST/IMNOVID sales growth was primarily volume-driven due to increases in treatment duration and market share.
OTEZLA sales for the first quarter were $353 million, a 46 percent increase year-over-year. First quarter U.S. sales of $276 million and international sales of $77 million increased 39 percent and 79 percent, year-over-year, respectively. OTEZLA sales in the U.S. were primarily volume-driven due to increasing demand and improved access pull-through in contracted health plans. OTEZLA international sales growth was driven primarily by increasing adoption in key ex-U.S. markets.
ABRAXANE sales for the first quarter were $262 million, an 11 percent increase year-over-year. U.S. sales were $159 million and international sales were $103 million, an increase of 12 percent and 10 percent, year-over-year, respectively. ABRAXANE sales were positively impacted by buying patterns. Growth in Europe was driven by market share gains for ABRAXANE in pancreatic cancer.
In the first quarter, all other product sales, which include IDHIFA, THALOMID, ISTODAX, VIDAZA and an authorized generic version of VIDAZA drug product primarily sold in the U.S., were $229 million compared to $226 million in the first quarter of 2017.
Research and Development (R&D)

On a GAAP basis, R&D expenses were $2,203 million for the first quarter of 2018 versus $995 million for the same period in 2017. The first quarter increase was primarily due to an increase in research and development asset acquisition expense relating to our acquisition of Impact Biomedicines, Inc. (Impact), an increase in share-based compensation expense related to our acquisition of Juno Therapeutics, Inc. (Juno), and increased spending related to clinical trial and other R&D activity, partially offset by a reduction of one-time charges related to wind-down costs associated with the GED-0301 clinical trials in Crohn’s disease and certain development activities.

Adjusted R&D expenses were $694 million for the first quarter of 2018 compared to $595 million for the first quarter of 2017. The first quarter increase was primarily due to increased spending related to clinical trial and other R&D activities.

Selling, General, and Administrative (SG&A)

On a GAAP basis, SG&A expenses were $864 million for the first quarter of 2018 compared to $620 million for the same period in 2017. The first quarter increase was primarily due to an increase in share-based compensation expense related to our acquisition of Juno and an increase in promotional activities and legal expenses.

Adjusted SG&A expenses were $671 million for the first quarter of 2018 compared to $539 million for the first quarter of 2017. The first quarter increase was primarily due to an increase in promotional activities and legal expenses.

Cash, Cash Equivalents, Marketable Debt Securities and Publicly-Traded Equity Securities

Operating cash flow was $(325) million in the first quarter of 2018, compared to $853 million for the first quarter of 2017, which was primarily impacted by the $1.1 billion upfront cash payment to acquire Impact Biomedicines (fedratinib).

In the first quarter, Celgene completed two strategic acquisitions for over $10 billion. We repurchased approximately 29.0 million shares at a total cost of approximately $2.7 billion. Celgene raised $4.5 billion in a debt offering to finance a portion of the acquisition of Juno. Celgene ended the quarter with approximately $4.7 billion in cash, cash equivalents, marketable debt securities and publicly-traded equity securities.

Product and Pipeline Updates

Hematology & Oncology

At the 2018 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June, data presentations are expected to include:
Updated durability and safety data from the pivotal TRANSCEND NHL-001 trial evaluating liso-cel (JCAR017) in patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL). In addition, the pivotal TRANSCEND NHL-001 trial completed enrollment in April.
In collaboration with partner bluebird bio, updated data from the CRB-401 phase I trial evaluating bb2121 in patients with relapsed and/or refractory multiple myeloma (RRMM).
Results from the phase III OPTIMISMM trial evaluating POMALYST in combination with bortezomib and dexamethasone (PVd) in patients with second-line multiple myeloma.
Results from the phase III RELEVANCE trial with REVLIMID in combination with rituximab (R²) in patients with previously untreated follicular lymphoma (FL).
Primary progression-free survival (PFS) and safety analysis from the Genentech-sponsored phase III IMpower131 trial evaluating atezolizumab plus chemotherapy (carboplatin and ABRAXANE) as first-line treatment in patients with advanced squamous non-small cell lung cancer (NSCLC).
In April, Celgene initiated the pivotal TRANSCEND WORLD trial evaluating liso-cel in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in the EU and Japan. These data are expected to support international registration submissions for liso-cel in DLBCL.
The phase I TRANSCEND CLL-004 trial evaluating liso-cel in patients with relapsed or refractory chronic lymphocytic leukemia (CLL) initiated in the first quarter.
The phase I EVOLVE trial evaluating JCARH125, a chimeric antigen receptor (CAR) T cell therapy targeting B-cell maturation antigen (BCMA), in patients with RRMM initiated in the first quarter.
Celgene’s partner BeiGene initiated the phase III trial evaluating BGB-A317 (tislelizumab) versus sorafenib in patients with previously untreated advanced hepatocellular carcinoma (HCC) in the first quarter. In addition, a phase II trial evaluating tislelizumab in patients with previously treated advanced HCC was initiated.
Inflammation & Immunology

In February, Celgene announced it received a Refusal To File (RTF) letter from the U.S. Food and Drug Administration (FDA) regarding the New Drug Application (NDA) for ozanimod in relapsing multiple sclerosis (RMS). Following a Type A meeting with the FDA in early April, Celgene expects to resubmit the NDA in the first quarter of 2019.
Following a meeting with European regulatory authorities, Celgene expects to submit a Marketing Authorization Application (MAA) for ozanimod in RMS in the first quarter of 2019.
At the 2018 American Academy of Neurology (AAN) Annual meeting in April, data from new analyses of both pivotal phase III SUNBEAM and RADIANCE Part B trials evaluating ozanimod in RMS were presented. These new analyses show dose-dependent effects of ozanimod on annualized relapse rate (ARR) versus interferon beta-1a (Avonex) across subgroups, including baseline disability and prior exposure to disease-modifying therapies, that were consistent with the overall ARR primary endpoint. In addition, data presentations of exploratory endpoints showed reductions in cortical grey matter and thalamic volume loss consistent with the reductions in whole brain volume loss seen in SUNBEAM at one year and RADIANCE Part B at two years for ozanimod compared with Avonex. The overall safety and tolerability profile for ozanimod has been consistent across the RADIANCE Part A, SUNBEAM and RADIANCE Part B studies.
In February, data from the phase II randomized, double-blind, placebo-controlled proof of concept study evaluating OTEZLA in patients with ulcerative colitis (UC) were presented at the 13th Congress of the European Crohn’s and Colitis Organization (ECCO). In addition, these phase II data have also been accepted for an encore presentation at the Digestive Disease Week (DDW) meeting in June. Celgene plans to initiate the pivotal program with OTEZLA in UC in 2018.
Business Updates

In March, Celgene announced that it completed the acquisition of Juno, a biopharmaceutical company focused on developing innovative cellular immunotherapies for the treatment of cancer. The Juno acquisition positions Celgene as a global cellular immunotherapy company by adding a novel scientific platform and scalable manufacturing capabilities, in addition to liso-cel, an investigational CD19-directed CAR T currently in a clinical development program for relapsed and/or refractory DLBCL.

Celgene acquired all the outstanding shares of common stock of Juno through a tender offer for $87 per share in cash, or an aggregate of approximately $9.1 billion. The transaction was funded through a combination of existing cash, cash equivalents, debt securities available-for-sale and new debt.
In February, Celgene completed the acquisition of Impact, a privately-held biotechnology company developing fedratinib, a highly selective Janus kinase 2 (JAK2) inhibitor, for myelofibrosis. This acquisition strengthens Celgene’s commitment to myelofibrosis, a disease with high unmet medical need, and expands strategic development options within Celgene’s myeloid portfolio of assets.

Under the terms of the agreement, Celgene paid approximately $1.1 billion upfront, contingent consideration based upon regulatory approvals of up to $1.4 billion (including $1.25 billion for myelofibrosis), and contingent consideration of up to $4.5 billion based upon the achievement of sales in any four consecutive calendar quarters between $1.0 billion and $5.0 billion.
In March, Celgene and Prothena Corporation (Prothena) announced a global collaboration to develop new therapies for a broad range of neurodegenerative diseases. The multi-year research and development collaboration is focused on three proteins implicated in the pathogenesis of several neurodegenerative diseases, including tau, TDP-43 and an undisclosed target. For each of the programs, Celgene has an exclusive right to license clinical candidates in the U.S. at Investigational New Drug (IND) filing, and if exercised, would also have a right to expand the license to global rights at the completion of phase I.

Under the terms of the collaboration, Prothena received a $150 million upfront payment (which includes an equity investment) plus future potential exercise payments and regulatory and commercial milestones for each licensed program. Prothena will also receive additional royalties on net sales of any resulting marketed products.
In March, Celgene and Vividion Therapeutics Inc., (Vividion) announced a multi-year strategic research collaboration focused on the identification and development of unique small molecules against targets for a range of oncology, inflammatory and neurodegenerative indications. The collaboration utilizes Vividion’s platform to identify ligands and discover drug candidates against a selected list of high-value, difficult-to-drug targets.

Under the terms of the collaboration, Vividion received an upfront payment of $101 million (which includes an equity investment). Celgene will have the right to opt in to programs at IND acceptance and Celgene will receive exclusive worldwide rights for certain programs, including the first program. In addition, other programs will allow for Celgene and Vividion to share equally either U.S. or worldwide development costs and commercialization profits and losses.
In early March, Celgene’s partner bluebird bio opted in on their right to co-develop and co-promote bb2121, an investigational anti-BCMA CAR T cell therapy for the treatment of patients with RRMM in the United States. The companies originally entered into a broad, global strategic research collaboration in 2013 to discover, develop and commercialize novel therapies in oncology, which included bb2121. Celgene and bluebird bio amended and restated the collaboration agreement in 2015 to focus on developing product candidates targeting BCMA.
Organizational Updates

Celgene recently announced the election of Hans Bishop, Patricia "Pat" Hemingway Hall and John Weiland to the Board of Directors.

Mr. Bishop is a 30-year industry veteran and pioneer in the field of cellular immunotherapy whose expertise will help Celgene lead in this extremely promising area of science. He was most recently President and CEO of Juno, a cellular immunotherapy company that he co-founded in 2013 and led until Juno was acquired by Celgene in March 2018.

Ms. Hemingway Hall has more than 30 years of experience with a focus on the U.S. health insurance market and deep understanding of the U.S. market access landscape which will help to shape Celgene’s strategy in an increasingly complex environment. She most recently was CEO of Health Care Service Corporation (HCSC), the nation’s largest mutual health insurance company, which operates as Blue Cross and Blue Shield in Illinois, Montana, New Mexico, Oklahoma and Texas, from 2008 until her retirement in 2015.

Mr. Weiland has over 30 years in the healthcare industry with significant expertise and experience across therapeutic areas and geographies. His leadership and insight will help to inform and direct Celgene’s long-term growth strategy. He was most recently the President and Chief Operating Officer of C. R. Bard, Inc. (Bard), with worldwide responsibility for all of Bard’s business operations prior to it being acquired by Becton, Dickinson and Company (BD) in December 2017.
First Quarter 2018 Conference Call and Webcast Information

Celgene will host a conference call to discuss the first quarter of 2018 operational and financial performance on Friday, May 4, 2018, at 9 a.m. ET. The conference call will be available by webcast at www.celgene.com. An audio replay of the call will be available from noon May 4, 2018, until midnight ET May 11, 2018. To access the replay in the U.S., dial (855) 859-2056; outside the U.S. dial (404) 537-3406. The participant passcode is 6196716.