On August 2, 2017 Kite Pharma, Inc., (Nasdaq:KITE), a leading cell therapy company, reported that patients with relapsed/refractory indolent B-cell non-Hodgkin lymphoma (iNHL) are now being treated in its Phase 2 ZUMA-5 trial with its lead investigational candidate, axicabtagene ciloleucel (axi-cel) (Press release, Kite Pharma, AUG 2, 2017, View Source [SID1234519984]). This study builds upon early clinical results using the same axi-cel anti-CD19 construct previously published in the March 2012 issue of Blood, "B-cell Depletion and Remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells,"1 in which patients with relapsed/refractory iNHL experienced a high response rate and durable disease remissions in a clinical trial at the National Cancer Institute (NCI).

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"Despite recent advances in the treatment of NHL, iNHL generally remains an incurable disease. As a result, there is a great unmet need in patients with high-risk disease which is why the focus of this study has the opportunity to make a difference," said David Chang, M.D., Ph.D., Executive Vice President of Research and Development and Chief Medical Officer of Kite. "The dosing of the first patient in ZUMA-5 marks an important milestone in our commitment to serving patients. We will continue to explore the potential for axi-cel to treat hematologic malignancies beyond aggressive NHL, the foundation of our CAR-T program."

B-cell iNHL is expected to account for approximately 25 percent of 2017 newly diagnosed NHL cases in the United States (U.S.). Follicular lymphoma is the most common type of iNHL and one of the most common subtypes of NHL. The company estimates that there will be approximately 4,700 patients in 2017 with follicular lymphoma in the U.S. who could potentially benefit from CAR-T therapy.

ZUMA-5 is a single-arm, open-label, multi-center study in patients with iNHL whose disease has relapsed within two years of first line treatment, is refractory to second line or greater therapy or has relapsed at any point after transplant. The study will enroll approximately 50 patients. Additional details about this study can be found on ClinicalTrials.gov, using identifier NCT: 03105336.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA) for aggressive non-Hodgkin’s lymphoma and was granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL).

On August 2, 2017 Celsion Corporation (NASDAQ:CLSN) reported findings from the translational research data from its Phase Ib dose escalating clinical trial (the OVATION Study) combining GEN-1, the Company’s IL-12 gene-mediated immunotherapy, with the standard of care for the treatment of newly-diagnosed patients with Stage III and IV ovarian cancer who will undergo neoadjuvant chemotherapy (NACT) followed by interval debulking surgery (Press release, Celsion, AUG 2, 2017, View Source [SID1234519983]).

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Translational research data was reviewed with leading immuno-oncology experts from the Roswell Park Cancer Institute. The analysis of peritoneal fluid and blood samples collected immediately before and 24 hours after IP administration of multiple doses of GEN-1 (36, 47, 61, 72 mg/m2) and standard NACT (carboplatin every 21 days and Taxol weekly) shows clear evidence of IL-12 gene transfer by dose dependent increases in IL-12 levels and immune system activity and significant increases in interferon-gamma (IFN- γ) and decreases in VEGF levels. The treatment-related changes in immune activating cytokines and pro-tumor VEGF levels followed a dose-dependent trend and were predominantly in the peritoneal fluid compartment with little to no changes observed in the patients’ systemic blood stream.

Key translational research findings from the first 12 of 15 patients&supl; enrolled in four patient cohorts are summarized below:
The treatment-related changes in immune activating cytokines and pro-tumor VEGF and IFN- γ levels followed a dose-dependent trend and were predominantly in the peritoneal fluid compartment with little to no changes observed in the patients’ systemic circulation. The observed immunological changes are consistent with an IL-12 based mechanism.

Effects observed in the IHC analysis were pronounced decreases in the density of immunosuppressive T-cell signals (FoxP3, PD-1, PDL-1, IDO-1) and increases in CD8+ cells in the tumor microenvironment.

The ratio of CD8+ cells to immunosuppressive cells was increased in approximately 75% of patients suggesting an overall shift in the tumor microenvironment from immunosuppressive to pro-immune stimulatory following treatment with GEN-1. An increase in CD8+ to immunosuppressive T-cell populations is a leading indicator and believed to be a good predictor of improved overall survival.

"These translational research findings demonstrate that GEN-1 in ovarian cancer patients is biologically active and creates a shift in the tumor microenvironment in the peritoneal cavity in a dose-dependent manner and promotes a pro-immune T-cell population dynamic in the tumor microenvironment," said Dr. Khursheed Anwer, Celsion’s executive vice president and chief science officer. "These distinct immunological changes in the local disease environment appear to translate into clinical benefit and warrant the continued development of our GEN-1 IL-12 immunotherapy as a potential adjuvant, in both first and second-line ovarian cancer. Furthermore, pro-immune changes in the tumor microenvironment appear to support research combining GEN-1 with other exciting immuno-oncology therapies including adaptive T-cell and check point inhibitors."

The Company previously announced the latest clinical findings from the OVATION Study in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Annual Meeting in June 2017. The presentation summarized clinical findings for all fourteen patients treated in the trial to-date.

Of the fourteen patients treated to date, two (2) patients demonstrated a complete response, ten (10) patients demonstrated a partial response and two (2) patients demonstrated stable disease, as measured by RECIST criteria.
This translates to a 100% disease control rate (DCR) and an 86% objective response rate (ORR).

Of the five patients treated in the highest dose cohort, there was a 100% objective response rate with one (1) complete response and four (4) partial responses.

Fourteen patients had successful resections of their tumors, with nine (9) patients (64%) having an R0 resection, which indicates a margin-negative resection in which no gross or microscopic tumor remains in the tumor bed.

Of the five patients treated at the highest dose cohort, all five patients (100%) experienced a R0 surgical resection.

Seven out of eight (87%) patients in the highest two dose cohorts experienced a R0 surgical resection.

Of the seven patients who have received GEN-1 treatment over one year ago and are being followed, only one patient’s cancer has progressed after 11.7 months. This compares favorably to the historical median progression free survival (PFS) of 12 months for newly-diagnosed patients with Stage III and IV ovarian cancer that undergo neoadjuvant chemotherapy followed by interval debulking surgery². Of the remaining six patients who have been on the study for over one year, their average PFS is 16.4 months with the longest progression-free patient at over 22 months.

"The impressive early trends in tumor response, surgical resections and progression-free survival are consistent with the dose dependent increases in IFN- γ levels, decreases in VEGF levels and immune system activity observed in the translational data," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "Ovarian cancer patients have a very poor prognosis. These data along with other published, pre-clinical data, underscore the potential of GEN-1 to serve as an effective, safe IL-12 immunotherapy in this underserved population."

On August 2, 2017 CTI BioPharma Corp. (NASDAQ and MTA: CTIC) reported the completion of enrollment in the Phase 3 PIX306 trial of PIXUVRI (pixantrone) (Press release, CTI BioPharma, AUG 2, 2017, View Source [SID1234519970]). The PIX306 trial is evaluating PIXVURI combined with rituximab in comparison to that of rituximab combined with gemcitabine in patients with aggressive B-cell non-Hodgkin lymphoma (NHL). Patients eligible to be enrolled in the trial had failed front line CHOP-R and were not eligible for autologous stem cell transplant (ASCT) (2nd line) or failed ASCT (3rd or 4th line). PIXUVRI has previously been granted conditional marketing authorization from the European Commission for the treatment of adult patients with multiply relapsed or refractory aggressive B-cell NHL.i The trial is being conducted as a post-authorization requirement of conditional marketing authorization. If positive, the results from this trial could support broader indications. Top-line results are event-driven and are expected in the first half of 2018.

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About PIXUVRI (pixantrone)

PIXUVRI is a cytotoxic medicine that works by interfering with the DNA within cells and preventing them from making more copies of DNA. This means that the cancer cells in B-cell NHL cannot divide and eventually die.ii

PIXUVRI is conditionally approved in the EU as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive B-cell NHL. The benefit has not been established in patients when used as fifth line or greater chemotherapy in patients who are refractory to last therapy.

The Summary of Product Characteristics (SmPC) has the full prescribing information, including the safety and efficacy profile of PIXUVRI in the approved indication. The SmPC is available at www.servier.com.

About NHL

NHL is an uncommon type of cancer that affects the lymphatic system, which is defined as a network of vessels and glands that run throughout the body.iii The lymphatic system is a key component of the immune system, as it plays a role in destroying old or abnormal cells and fighting bacteria and other infections.iv

Around 93,500 new cases of NHL were diagnosed in Europe in 2012, making it the eleventh most common cancer on the continent.v

NHL comprises more than 60 subtypes, with each requiring a different diagnostic evaluation and treatment approaches. Lymphoma patient groups around the world, led by the umbrella group Lymphoma Coalition, have been recently calling for accurate subtype reporting to allow patients to clearly understand their subtype and have better communication with their doctors. Given the complexities of the condition, access to information is essential to empower patients.

On August 2, 2017 AstraZeneca and its haematology research and development centre of excellence, Acerta Pharma, reported that the US Food and Drug Administration (FDA) has accepted and granted Priority Review for the New Drug Application (NDA) for acalabrutinib, a highly-selective, potent, Bruton tyrosine kinase (BTK) inhibitor (Press release, AstraZeneca, AUG 2, 2017, View Source [SID1234519969]).

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The NDA is based on results from the Phase II ACE-LY-004 clinical trial, which evaluated the safety and efficacy of acalabrutinib in patients with relapsed/refractory mantle cell lymphoma (MCL) who have received at least one prior therapy. This follows the FDA’s recent Breakthrough Therapy Designation for acalabrutinib.

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer, said: "FDA’s acceptance of the acalabrutinib application and Priority Review illustrates the impact it could have on patients with relapsed or refractory mantle cell lymphoma as we work to bring this potential medicine to those in need as quickly as possible."

Priority Review is granted to applications for medicines that, if approved, would offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions.1 The Prescription Drug User Fee Act (PDUFA) date is during the first quarter of 2018.

Flavia Borellini, PhD, Acerta Pharma Chief Executive Officer, said: "We believe acalabrutinib has the potential to be a very important treatment option for patients with this life-threatening blood cancer. The FDA’s NDA acceptance exemplifies our progress in the acalabrutinib development programme and continues our momentum as we seek to transform care for people with haematologic malignancies."

Results from the ACE-LY-004 clinical trial will be submitted for presentation at a forthcoming medical meeting. The acalabrutinib development programme includes both monotherapy and combination therapy strategies in a broad range of blood cancers and solid tumours. The programme includes the Phase III ACE-LY-308 clinical trial evaluating acalabrutinib as a 1st-line treatment for patients with MCL.2


About mantle cell lymphoma (MCL)

Mantle cell lymphoma (MCL) is an aggressive B-cell non-Hodgkin lymphoma (NHL) with poor prognosis.3,4,5,6 MCL accounts for approximately 3% to 6% of new NHL cases in Western countries each year, with an annual incidence of 0.5 per 100,000 persons and an estimated prevalence of 3.5/100,000.5,7 The median age at diagnosis is 68 years, with a 3:1 male predominance.5

About acalabrutinib

Acalabrutinib is a highly-selective, potent, covalent inhibitor of Bruton tyrosine kinase (BTK) with minimal off-target activity observed in pre-clinical trials.8,9,10 This potential new medicine is in development for the treatment of multiple B-cell and other cancers. The acalabrutinib development programme includes both monotherapy and combination therapy strategies in chronic lymphocytic leukaemia (CLL), MCL, Waldenström macroglobulinemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma, as well as monotherapy and combination trials in solid tumours. In total, more than 25 acalabrutinib clinical trials with more than 2,000 patients are underway or have completed. Acalabrutinib was granted Orphan Drug Designation by the FDA for the treatment of patients with MCL in September 2015 and by the European Commission in March 2016 for the treatment of patients with CLL, MCL and WM. Acalabrutinib was granted Breakthrough Therapy Designation by the FDA in August 2017 for the treatment of patients with MCL who have received at least one prior therapy. Acalabrutinib is a potential new medicine not approved for any current use.

On August 2, 2017 Ono Pharmaceutical Co., Ltd. (“The Company”) reported its consolidated financial results for three months ended June 30, 2017 (Press release, Ono, AUG 2, 2017, View Source [SID1234519982]).

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The consolidated financial statements have been prepared in accordance with International Financial Reporting Standards (“IFRSs”).

This First Quarter Flash Report 2018 (unaudited) is summary information extracted from the financial statements announced, and the financial statements and the figures contained herein are prepared for reference only for the convenience of readers outside Japan with certain modifications and reclassifications made from the original financial statements presented in Japanese language.

The translations of Japanese yen amounts into U.S. dollar amounts are included solely for the convenience of readers outside Japan using the rate of 112 to $1, the approximate rate of exchange at June 30, 2017.

Amounts of less than one million yen and one thousand U.S. dollars have been rounded to the nearest million yen and one thousand U.S. dollars in the presentation of the accompanying consolidated financial statements.

Financial Highlights
Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries

Revenue ¥ 58,757 ¥ 244,797 ¥ 60,913 $ 543,865 Profit (Owners of the parent company) Total equity 477,791 524,211 510,218 4,555,517 Total assets 540,405 617,461 577,330 5,154,731 Basic earnings per share ¥ 25.81 ¥ 105.27 ¥ 22.31 $ 0.20 Diluted earnings per share ¥ 25.81 ¥ 105.26 ¥ 22.31 $ 0.20 105,129 ONO PHARMACEUTICAL CO., LTD. Thousands of US$ Annual 12 months 2017 3 months ended Jun. 30, US$ 13,680 1st Quarter 3 months ended Jun. 30, 2016 55,793 11,774 1st Quarter 2017 Millions of yen ended Mar. 31, 2017 1st Quarter 3 months ended Jun. 30, 1
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017

Future Outlook
Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries
Revenue ¥ 112,500 $ 1,004,464 ¥ 236,000 $ 2,107,143 Operating profit 13,200 117,857 36,500 325,893 Profit before tax 14,500 129,464 39,000 348,214 Profit 10,700 95,536 29,000 258,929 (Owners of the parent company) Basic earnings per share ¥ 20.19 $ 0.18 ¥ 54.72 $ 0.49

(*)The foregoing are forward-looking statements based on a number of assumptions and beliefs in light of the information currently available to management and are subject to risks and uncertainties. Actual financial results may differ materially depending on a number of economic factors, including conditions and currency exchange rate fluctuations.

Six months ending Year ending Yen US$ Yen US$ September 30, 2017 March 31, 2018 Millions of yen Thousands of US$ Millions of yen Thousands of US$ 2
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017

Consolidated Statement of Financial Position
Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries

Current assets Cash and cash equivalents ¥ 146,323 ¥ 92,491 $ 825,810 Trade and other receivables 73,255 74,435 664,597 Marketable securities 17,560 15,620 139,467 Other financial assets 819 810 7,228 Inventories 25,334 26,733 238,684 Other current assets 7,742 9,990 89,192 Total current assets 271,033 220,078 1,964,979 Non-current assets Property, plant, and equipment 83,659 85,169 760,441 Intangible assets 45,237 49,005 437,549 Investment securities 176,573 184,785 1,649,864 Investments in associates 114 119 1,063 Other financial assets 26,836 26,708 238,462 Deferred tax assets 10,739 7,685 68,619 Other non-current assets 3,271 3,781 33,755 Total non-current assets 346,428 357,252 3,189,752 Total assets ¥ 617,461 ¥ 577,330 $ 5,154,731 ASSETS As of March 31, 2017 As of June 30, 2017 As of June 30, 2017 Millions of yen Thousands of US$ 3
Current liabilities Trade and other payables ¥ 30,905 ¥ 27,736 $ 247,645 Borrowings 423 436 3,891 Other financial liabilities 5,814 5,320 47,497 Income taxes payable 24,777 3,746 33,446 Provisions 6,086 7,134 63,700 Other current liabilities 14,928 12,743 113,781 Total current liabilities 82,933 57,115 509,959 Non-current liabilities Borrowings 542 468 4,180 Other financial liabilities 11 12 106 Retirement benefit liabilities 2,805 2,658 23,734 Provisions 30 30 268 Deferred tax liabilities 881 893 7,972 Long-term advances received 5,276 5,170 46,158 Other non-current liabilities 772 766 6,837 Total non-current liabilities 10,316 9,997 89,255 Total liabilities 93,250 67,112 599,214 Equity Share capital 17,358 17,358 154,985 Capital reserves 17,144 17,155 153,173 Treasury shares (59,382) (81,881) (731,078) Other components of equity 51,752 58,844 525,393 Retained earnings 492,237 493,596 4,407,110 Non-controlling interests 5,101 5,145 45,935 Total equity 524,211 510,218 4,555,517 Total liabilities and equity ¥ 617,461 ¥ 577,330 $ 5,154,731 Millions of yen Thousands of US$ Equity attributable to owners of the parent company 519,110 505,073 4,509,582 LIABILITIES AND EQUITY As of June 30, 2017 As of March 31, 2017 As of June 30, 2017 4
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017

Consolidated Statement of Income
Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries Revenue
¥ 58,757 ¥ 60,913 $ 543,865 Cost of sales (16,202) (15,140) (135,176) Gross profit 42,555 45,773 408,689 Selling, general, and administrative expenses (14,054) (16,240) (144,999) Research and development costs (11,119) (14,938) (133,376) Other income 21 62 555 Other expenses (159) (382) (3,410) Operating profit 17,244 14,275 127,459 Finance income 1,531 1,523 13,594 Finance costs (540) (8) (69) 10 6 50 Profit before tax 18,245 15,796 141,035 Income tax expense (4,541) (3,992) (35,644) Profit for the period 13,704 11,804 105,391 Profit for the period attributable to: Owners of the parent company 13,680 11,774 105,129 Non-controlling interests 24 29 261 Profit for the period 13,704 11,804 105,391 Earnings per share: Basic earnings per share 25.81 22.31 0.20 Diluted earnings per share 25.81 22.31 0.20 Yen US$ Millions of yen Thousands of US$ Share of profit (loss) from investments in associates 1st Quarter 3 months 1st Quarter 3 months ended June 30, 2017 ended June 30, 2016 1st Quarter 3 months ended June 30, 2017 5
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017

Consolidated Statement of Comprehensive Income
Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries Profit for the period
¥ 13,704 ¥ 11,804 $ 105,391 Other comprehensive income: (1,910) 7,084 63,250 (206) 185 1,651 (0) (0) (1) (2,117) 7,269 64,900 Items that may be reclassified subsequently to profit or loss: (470) 19 171 (25) 6 57 (495) 26 228 Total other comprehensive income (loss) (2,612) 7,294 65,129 Total comprehensive income for the period 11,092 19,098 170,519 Comprehensive income for the period attributable to: Owners of the parent company 11,073 19,052 170,103 Non-controlling interests 19 47 416 Total comprehensive income for the period 11,092 19,098 170,519 Net fair value gain (loss) on derivatives under hedge accounting Net gain (loss) on financial assets measured at fair value through other comprehensive income Remeasurement of defined benefit plans Share of net gain (loss) on financial assets measured at fair value through other comprehensive income of investments in associates Millions of yen Thousands of US$ 1st Quarter 3 months ended June 30, 1st Quarter 3 months ended June 30, 2016 Total of items that will not be reclassified to profit or loss Items that will not be reclassified to profit or loss: Total of items that may be reclassified subsequently to profit or loss Exchange differences on translation of foreign operations 1st Quarter 3 months ended June 30, 2017 2017 6
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017 Consolidated Statement of Changes in Equity Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries Share capital Capital reserves Treasury shares Other components of equity Retained earnings Equity attributable to owners of the parent company Noncontrolling interests Total equity Balance at April 1, 2016 ¥17,358 ¥17,103 (¥59,358) ¥43,307 ¥452,983 ¥471,393 ¥4,862 ¥476,255 Profit for the period 13,680 13,680 24 13,704 Other comprehensive income (2,607) (2,607) (5) (2,612) Total comprehensive income for the period – – – (2,607) 13,680 11,073 19 11,092 Purchase of treasury shares (21) (21) (21) Cash dividends (9,540) (9,540) (3) (9,544) Share-based payments 8 8 8 Transfer from other components of equity to retained earnings 206 (206) – – Total transactions with the owners – 8 (21) 206 (9,747) (9,553) (3) (9,556) Balance at June 30, 2016 ¥17,358 ¥17,111 (¥59,379) ¥40,906 ¥456,916 ¥472,912 ¥4,879 ¥477,791 Share capital Capital reserves Treasury shares Other components of equity Retained earnings Equity attributable to owners of the parent company Noncontrolling interests Total equity Balance at April 1, 2017 ¥17,358 ¥17,144 (¥59,382) ¥51,752 ¥492,237 ¥519,110 ¥5,101 ¥524,211 Profit for the period 11,774 11,774 29 11,804 Other comprehensive income 7,277 7,277 17 7,294 Total comprehensive income for the period – – – 7,277 11,774 19,052 47 19,098 Purchase of treasury shares (22,499) (22,499) (22,499) Cash dividends (10,600) (10,600) (3) (10,604) Share-based payments 11 11 11 Transfer from other components of equity to retained earnings (185) 185 – – Total transactions with the owners – 11 (22,499) (185) (10,415) (33,088) (3) (33,091) Balance at June 30, 2017 ¥17,358 ¥17,155 (¥81,881) ¥58,844 ¥493,596 ¥505,073 ¥5,145 ¥510,218 Share capital Capital reserves Treasury shares Other components of equity Retained earnings Equity attributable to owners of the parent company Noncontrolling interests Total equity Balance at April 1, 2017 $154,985 $153,074 ($530,195) $462,070 $4,394,976 $4,634,909 $45,546 $4,680,456 Profit for the period 105,129 105,129 261 105,391 Other comprehensive income 64,974 64,974 155 65,129 Total comprehensive income for the period ––– 64,974 105,129 170,103 416 170,519 Purchase of treasury shares (200,883) (200,883) (200,883) Cash dividends (94,646) (94,646) (28) (94,674) Share-based payments 99 99 99 Transfer from other components of equity to retained earnings (1,651) 1,651 – – Total transactions with the owners – 99 (200,883) (1,651) (92,995) (295,430) (28) (295,458) Balance at June 30, 2017 $154,985 $153,173 ($731,078) $525,393 $4,407,110 $4,509,582 $45,935 $4,555,517 Millions of yen Equity attributable to owners of the parent company Millions of yen Equity attributable to owners of the parent company Thousands of US $ Equity attributable to owners of the parent company 7
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017 Consolidated Statement of Cash Flows Ono Pharmaceutical Co., Ltd. and Consolidated Subsidiaries Cash flows from operating activities Profit before tax ¥ 18,245 ¥ 15,796 $ 141,035 Depreciation and amortization 1,680 2,217 19,794 Impairment losses 9 – – Interest and dividend income (1,526) (1,488) (13,282) Interest expense 3 4 33 (Increase) Decrease in inventories (1,143) (1,420) (12,683) (Increase) Decrease in trade and other receivables (16,415) (1,186) (10,585) Increase (Decrease) in trade and other payables (268) (3,243) (28,958) Increase (Decrease) in provisions (103) 1,048 9,358 Increase (Decrease) in retirement benefit liabilities 100 120 1,074 Increase (Decrease) in long-term advances received (198) (106) (949) Other 6,752 (7,259) (64,812) Subtotal 7,137 4,483 40,026 Interest received 39 22 194 Dividends received 1,487 1,464 13,071 Interest paid (3) (4) (33) Income taxes paid (6,588) (24,693) (220,475) Net cash provided by (used in) operating activities 2,072 (18,728) (167,218) Cash flows from investing activities Purchases of property, plant, and equipment (8,751) (2,844) (25,392) Purchases of intangible assets (606) (4,478) (39,985) Purchases of investments – (40) (357) Proceeds from sales and redemption of investments 6,000 4,000 35,714 Other (74) 133 1,187 Net cash provided by (used in) investing activities (3,432) (3,229) (28,833) Cash flows from financing activities Dividends paid to owners of the parent company (8,700) (9,310) (83,125) Dividends paid to non-controlling interests (3) (3) (30) Repayments of long-term borrowings (94) (104) (926) Net increase (decrease) in short-term borrowings (12) 18 165 Purchases of treasury shares (20) (22,499) (200,883) Net cash provided by (used in) financing activities (8,830) (31,898) (284,800) Net increase (decrease) in cash and cash equivalents (10,190) (53,855) (480,851) Cash and cash equivalents at the beginning of the period 110,485 146,323 1,306,460 Effects of exchange rate changes on cash and cash equivalents (204) 23 201 Cash and cash equivalents at the end of the period ¥ 100,091 ¥ 92,491 $ 825,810 Millions of yen Thousands of US$ 1st Quarter 3 months ended June 30, 1st Quarter 3 months ended June 30, 2017 1st Quarter 3 months ended June 30, 2016 2017 8
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017 Sales of Major Products Supplemental Data For information purpose only Opdivo Agent for treatment of unresectable melanoma, unresectable, advanced or recurrent non-small cell lung cancer, unresectable or metastatic renal cell carcinoma, relapsed or refractory classical hodgkin lymphoma, and recurrent or metastatic head and neck cancer ¥ 198 ¥ ᇞ 54 ᇞ 21.4 % ¥ 740 ¥ ᇞ 299 ᇞ 28.8 % Glactiv Agent for type II diabetes 70 ᇞ 7 ᇞ 8.6 % 295 +1 +0.4 % Orencia SC Agent for rheumatoid arthritis 33 +6 +23.1 % 145 +29 +25.2 % Opalmon Circulatory system agent 38 ᇞ 9 ᇞ 18.3 % 140 ᇞ 30 ᇞ 17.8 % Recalbon Agent for osteoporosis 27 ᇞ 2 ᇞ 6.0 % 110 ᇞ 3 ᇞ 2.6 % Forxiga Agent for type II diabetes 26 +8 +45.7 % 100 +22 +28.1 % Rivastach Agent for Alzheimer’s disease 22 ᇞ 0 ᇞ 0.4 % 100 +11 +12.9 % Emend/Proemend Agent for Chemotherapy-induced nausea and vomiting 25 ᇞ 1 ᇞ 3.5 % 100 +1 +1.2 % Kyprolis Agent for relapsed or refractory multiple myeloma 12 +12 60 +40 +206.1 % Onoact Agent for tachyarrhythmia during and post operation 15 +0 +0.5 % 60 +3 +4.8 % Onon Agent for bronchial asthma and allergic rhinitis 13 ᇞ 4 ᇞ 22.9 % 55 ᇞ 13 ᇞ 19.0 % Staybla Agent for overactive bladder (pollakiuria and urinary incontinence) 11 ᇞ 2 ᇞ 17.0 % 45 ᇞ 3 ᇞ 5.7 % Parsabiv Agent for secondary hyperparathyroidism 6 +6 30 +28 +1439.8 % Onon dry syrup Agent for pediatric bronchial asthma and allergic rhinitis 8 ᇞ 3 ᇞ 26.0 % 30 ᇞ 11 ᇞ 26.9 % Foipan Agent for chronic pancreatitis and postoperative reflux esophagitis 8 ᇞ 3 ᇞ 24.9 % 30 ᇞ 8 ᇞ 21.7 % Kinedak Agent for diabetic peripheral neuropathy 6 ᇞ 2 ᇞ 27.7 % 25 ᇞ 4 ᇞ 13.2 % Note: Sales of products are shown in a gross sales basis. Launched in August 2016 Forecast Increase/Decrease Launched in February 2017 Hundreds of Millions of yen 1st Quarter 3 months ended June 30, 2017 Year ending March 31, 2018 Results Increase/Decrease 9
First Quarter (April 1 – June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017 Breakdown of Revenue Supplemental Data For information purpose only (Hundreds of Millions of yen) 1st Quarter 3 months ended June 30, 2016 1st Quarter 3 months ended June 30, 2017 485 536 124 51 609 588 Note: In “Royalty and Other Revenue”, royalty revenue of “Opdivo Intravenous Infusion” is included, which is 43 hundreds of millions of yen for the 1st quarter 3 months ended June 30, 2016 and 89 hundreds of millions of yen for the 1st quarter 3 months ended June 30, 2017. Information about Revenue by Geographic Area Supplemental Data For information purpose only (Hundreds of Millions of yen) 1st Quarter 3 months ended June 30, 2016 1st Quarter 3 months ended June 30, 2017 　　Japan 484 537 　　Americas 115 43 　　Asia 11 6 　　Europe 0 1 588 609 Note: Revenue by geographic area is attributable to countries or regions based on the customer location. Revenue of Goods and Products Royalty and Other Revenue Total Total 10
First Quarter (April 1– June 30, 2017) Flash Report (unaudited) Three months ended June 30, 2017 Supplemental Information Status of Development Pipeline as of July 28, 2017 I. Main Status of Development Pipelines (Oncology) 1. Development Status in Japan < Approved > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form In-house＊） / In-license Kyprolis for Intravenous Infusion *1 Additional dosage and administration Multiple myeloma / Proteasome inhibitor Injection In-license (Amgen Inc.) Changes from Flash Report for the Fiscal Year ended March 2017 *1: Approval for the partial change in approved items of the manufacturing and marketing approval for Kyprolis for Intravenous Infusion was obtained in Japan for the treatment of patients with relapsed or refractory multiple myeloma. Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. < Filed > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Gastric cancer Injection In-house (Co-development with Bristol-Myers Squibb) Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. < Clinical Trial Stage > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Esophageal cancer Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Gastro-esophageal junction cancer and esophageal cancer Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Small cell lung cancer Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Hepatocellular carcinoma Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Glioblastoma Injection III In-house (Co-development with Bristol-Myers Squibb) 11
Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Urothelial cancer Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Malignant pleural mesothelioma Injection III In-house (Co-development with Bristol-Myers Squibb) Additional indication Ovarian cancer Injection III In-house (Co-development with Bristol-Myers Squibb) Kyprolis for Intravenous Infusion Change of dosage and administration Multiple myeloma / Proteasome inhibitor Injection III In-license (Amgen Inc.) ONO-7643 / Anamorelin New chemical entities Cancer anorexia / cachexia / Ghrelin mimetic Tablet III In-license (Helsinn Healthcare, S.A.) ONO-7702 / Encorafenib New chemical entities Melanoma *2 / BRAF inhibitor Capsule III In-license (Array Biopharma Inc.) ONO-7703 / Binimetinib New chemical Entities Melanoma *3 / MEK inhibitor Tablet III In-license (Array Biopharma Inc.) Opdivo Intravenous Infusion Additional indication Solid tumor (Cervix carcinoma, Uterine body cancer, Soft tissue sarcoma) Injection II In-house (Co-development with Bristol-Myers Squibb) Additional indication Central nervous system lymphoma, Primary testicular lymphoma Injection II In-house (Co-development with Bristol-Myers Squibb) Additional indication Multiple myeloma *4 Injection II In-house (Co-development with Bristol-Myers Squibb) Additional indication Virus positive / negative solid carcinoma Injection I / II In-house (Co-development with Bristol-Myers Squibb) ONO-5371 / Metyrosine New chemical entities Pheochromocytoma / Tyrosine hydroxylase inhibitor Capsule I / II In-license (Valeant Pharmaceuticals North America LLC.) ONO-4686 (BMS-986207) New chemical entities Solid tumor / Anti-TIGIT antibody Injection I / II In-license (Co-development with Bristol-Myers Squibb) ONO-4059 / Tirabrutinib New chemical entities Central nervous system lymphoma *5 / Bruton’s tyrosine kinase (Btk) inhibitor Tablet I / II In-house Opdivo Intravenous Infusion Additional indication Biliary tract cancer Injection I In-house (Co-development with Bristol-Myers Squibb) 12
Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase In-house＊） / In-license ONO-4481 (BMS-663513) / Urelumab New chemical entities Solid tumor / Anti-CD137 antibody Injection I In-license (Co-development with Bristol-Myers Squibb) ONO-4482 (BMS-986016) New chemical entities Solid tumor / Anti-LAG-3 antibody Injection I In-license (Co-development with Bristol-Myers Squibb) ONO-4687 (BMS-986227) / Cabiralizumab New chemical entities Solid tumor and hematologic cancer / Anti-CSF-1R antibody Injection I In-license (Co-development with Bristol-Myers Squibb) ONO-7701 (BMS-986205) New chemical entities Solid tumor and hematologic cancer / IDO1 Inhibitor Capsule I In-license (Co-development with Bristol-Myers Squibb) ONO-4483 (BMS-986015) / Lirilumab New chemical entities Solid tumor / Anti-KIR antibody Injection I In-license (Co-development with Bristol-Myers Squibb) ONO-4578 New chemical entities Solid tumor / PG receptor (EP4) antagonist Tablet I In-house Changes from Flash Report for the Fiscal Year ended March 2017 *2: BRAF inhibitor (ONO-7702) is under Phase III for the treatment of melanoma. *3: MEK inhibitor (ONO-7703) is under Phase III for the treatment of melanoma. *4: Phase II of Opdivo was initiated for the treatment of multiple myeloma. *5: Phase I/II of Btk inhibitor (ONO-4059) was initiated for the treatment of central nervous system lymphoma. * Phase I of ONO-7268MX1 and ONO-7268MX2 for the treatment of hepatocellular carcinoma were discontinued due to the strategic reason. Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. 13
2. Development Status in S. Korea and Taiwan < Filed > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Non-small cell lung cancer (Non-Squamous) Injection Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Head and neck cancer Injection Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Hodgkin lymphoma *6 Injection Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Urothelial cancer *7 Injection Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Gastric cancer *8 Injection Taiwan In-house (Co-development with Bristol-Myers Squibb) Changes from Flash Report for the Fiscal Year ended March 2017 *6: A supplemental application for a partial change in the approved items of the importing and marketing approval for Opdivo was submitted in Taiwan for the treatment of hodgkin lymphoma. *7: A supplemental application for a partial change in the approved items of the importing and marketing approval for Opdivo was submitted in Taiwan for the treatment of urothelial cancer. *8: A supplemental application for a partial change in the approved items of the importing and marketing approval for Opdivo was submitted in Taiwan for the treatment of gastric cancer. Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. < Clinical Trial Stage > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Head and neck cancer Injection III South Korea In-house (Co-development with Bristol-Myers Squibb) Additional indication Gastric cancer Injection III South Korea In-house (Co-development with Bristol-Myers Squibb) Additional indication Esophageal cancer Injection III South Korea, Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Gastro-esophageal junction cancer and esophageal cancer Injection III South Korea, Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Small cell lung cancer Injection III South Korea, Taiwan In-house (Co-development with Bristol-Myers Squibb) 14
Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Hepatocellular carcinoma Injection III South Korea, Taiwan In-house (Co-development with Bristol-Myers Squibb) Additional indication Urothelial cancer Injection III South Korea In-house (Co-development with Bristol-Myers Squibb) ONO-7702 / Encorafenib New chemical entities Colon cancer *9 / BRAF inhibitor Capsule III South Korea In-license (Array Biopharma Inc.) New chemical entities Melanoma *10 / BRAF inhibitor Capsule III South Korea In-license (Array Biopharma Inc.) ONO-7703 / Binimetinib New chemical entities Colon cancer *11 / MEK inhibitor Tablet III South Korea In-license (Array Biopharma Inc.) New chemical entities Melanoma *12 / MEK inhibitor Tablet III South Korea In-license (Array Biopharma Inc.) Opdivo Intravenous Infusion Additional indication Virus positive / negative solid carcinoma Injection I / II South Korea, Taiwan In-house (Co-development with Bristol-Myers Squibb) Changes from Flash Report for the Fiscal Year ended March 2017 *9: BRAF inhibitor (ONO-7702) is under Phase III for the treatment of colon cancer in South Korea. *10: BRAF inhibitor (ONO-7702) is under Phase III for the treatment of melanoma in South Korea. *11: MEK inhibitor (ONO-7703) is under Phase III for the treatment of colon cancer in South Korea. *12: MEK inhibitor (ONO-7703) is under Phase III for the treatment of melanoma in South Korea. Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. 15
3. Development Status in Europe and the United States < Approved > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Urothelial cancer *13 Injection Europe In-house (Co-development with Bristol-Myers Squibb) Changes from Flash Report for the Fiscal Year ended March 2017 *13: Approval for the partial change in approved items of the manufacturing and marketing approval for Opdivo was obtained in Europe for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC). Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. < Filed > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Colon cancer Injection USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Hepatocellular carcinoma *14 Injection USA In-house (Co-development with Bristol-Myers Squibb) Changes from Flash Report for the Fiscal Year ended March 2017 *14: A supplemental application for the partial change in approved items of the manufacturing and marketing approval for Opdivo was submitted in USA for the treatment of previously treated hepatocellular carcinoma. Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. < Clinical Trial Stage > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Glioblastoma Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Small cell lung cancer Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Hepatocellular carcinoma Injection III Europe In-house (Co-development with Bristol-Myers Squibb) Additional indication Esophageal cancer Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Multiple myeloma Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) 16
Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase Area In-house＊） / In-license Opdivo Intravenous Infusion Additional indication Gastro-esophageal junction cancer and esophageal cancer Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Gastric cancer Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Malignant pleural mesothelioma Injection III Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Diffuse large B cell lymphoma Injection II Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Follicular lymphoma Injection II Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Central Nervous System Lymphoma, Primary Testicular Lymphoma Injection II Europe USA In-house (Co-development with Bristol-Myers Squibb) ONO-4059 / Tirabrutinib New chemical entities B cell lymphoma / Bruton’s tyrosine kinase (Btk) inhibitor Tablet II Europe In-house (Out-license to Gilead Sciences, Inc.) ONO-7579 New chemical entities Solid tumor / Tropomyosin receptor kinase (Trk) inhibitor Tablet I / II Europe USA In-house Opdivo Intravenous Infusion Additional indication Colon cancer Injection I / II Europe In-house (Co-development with Bristol-Myers Squibb) Additional indication Solid tumors (Triple negative breast cancer, Gastric cancer, Pancreatic cancer, Small cell lung cancer, Urothelial cancer, Ovarian cancer) Injection I / II Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Virus positive/negative solid carcinoma Injection I / II Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Hematologic cancer (T-cell lymphoma, Multiple myeloma, Chronic leukemia, etc.) Injection I Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Chronic myeloid leukemia Injection I Europe USA In-house (Co-development with Bristol-Myers Squibb) ONO-4059 / Tirabrutinib New chemical entities B cell lymphoma / Bruton’s tyrosine kinase (Btk) inhibitor Tablet I USA In-house (Out-license to Gilead Sciences, Inc.) ONO-7475 New chemical entities Acute leukemia / Axl / Mer inhibitor Tablet I USA In-house Note: “In-house” compounds include a compound generated from collaborative research. In the case of clinical development of the anticancer compound in the same indication, the most advanced clinical phase is described. 17
II. Main Status of Development Pipelines (Non-Oncology) 1. Development Status in Japan < Filed > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form In-house＊） / In-license Orencia IV Additional indication Juvenile Idiopathic Arthritis / T-cell activation inhibitor Injection In-license (Bristol-Myers Squibb) Note: “In-house” compounds include a compound generated from collaborative research. < Clinical Trial Stage > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase In-house＊） / In-license Orencia IV Additional indication Lupus nephritis / T-cell activation inhibitor Injection III In-license (Bristol-Myers Squibb) Orencia SC Additional indication Untreated rheumatoid arthritis / T-cell activation inhibitor Injection III In-license (Bristol-Myers Squibb) Additional indication Primary sjögren syndrome / T-cell activation inhibitor Injection III In-license (Bristol-Myers Squibb) Additional indication Polymyositis / Dermatomyositis *15 / T-cell activation inhibitor Injection III In-license (Bristol-Myers Squibb) ONO-1162 / Ivabradine New chemical entities Chronic heart failure / If channel inhibitor Tablet III In-license (Les Laboratoires Servier) Onoact for Intravenous Infusion 50 mg / 150 mg (ONO-1101) Additional indication for pediatric use Tachyarrhythmia in low cardiac function / Short acting beta 1 blocker Injection II / III In-house Additional indication Ventricular arrhythmia / Short acting beta 1 blocker Injection II / III In-house ONO-2370 / Opicapone New chemical entities Parkinson’s disease / Long acting COMT inhibitor Tablet II In-license (Bial) ONO-8577 New chemical entities Overactive bladder / Bladder smooth muscle relaxant Tablet II In-house Opdivo Intravenous Infusion Additional indication Sepsis *16 Injection I / II In-house (Co-development with Bristol-Myers Squibb) Changes from Flash Report for the Fiscal Year ended March 2017 *15: Phase III of Orencia SC was initiated for the treatment of polymyositis/dermatomyositis. *16: Phase I/II of Opdivo was initiated for the treatment of sepsis. * Phase I of ONO-2160/CD (levodopa pro-drug) for the treatment of parkinson’s disease was discontinued due to no expected treatment effect. Note: “In-house” compounds include a compound generated from collaborative research. 18
2. Development Status in Overseas < Clinical Trial Stage > Product Name / Development Code / Generic Name Classification Target indication / Pharmacological Action Dosage form Phase Area In-house＊） / In-license ONO-4474 New chemical entities Osteoarthritis / Tropomyosin receptor kinase (Trk) inhibitor Capsule II Europe In-house ONO-4059 / Tirabrutinib New chemical entities Sjögren syndrome / Bruton’s tyrosine kinase (Btk) inhibitor Tablet II USA In-house (Out-license to Gilead Sciences, Inc.) Opdivo Intravenous Infusion Additional indication Hepatitis C Injection I Europe USA In-house (Co-development with Bristol-Myers Squibb) Additional indication Sepsis Injection I USA In-house (Co-development with Bristol-Myers Squibb) ONO-8055 New chemical entities Underactive bladder / PG receptor (EP2 / EP3) agonist Tablet I Europe In-house Note: “In-house” compounds include a compound generated from collaborative research.