On January 2, 2019 Karyopharm Therapeutics Inc. (Nasdaq:KPTI), a clinical-stage pharmaceutical company, reported that Michael Kauffman, MD, PhD, Chief Executive Officer, will present at the 37th Annual J.P. Morgan Healthcare Conference on Monday, January 7, 2019 at 7:30 a.m. PT at the Westin St. Francis in San Francisco (Press release, Karyopharm, JAN 2, 2019, View Source [SID1234532330]).

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A live webcast of the event will be available on the "Events & Presentations" page in the Investors section of the Company’s website at View Source A replay of the webcast will be archived on the Company’s website for 90 days following the presentation.

On January 1, 2019 HJB and MabSpace Biosciences (MabSpace) reported that the two companies have entered into a definitive merger agreement to create Transcenta Holding Ltd. (Transcenta), a world-class biotherapeutics company with fully-integrated capabilities in research, development, regulatory and manufacturing of biologics (Press release, Mabspace, JAN 1, 2019, View Source [SID1234552801]). Dr. Xueming Qian, former Founder, Chairman and CEO of MabSpace, will serve as Chief Executive Officer while Dr. Jonathan Yining Zhao, Co-founder and CEO of HJB, will be the Executive Chairman of the combined company, respectively.

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Founded in 2013, MabSpace is a clinical stage biotech company focused on discovery, clinical research and commercial development of innovative biologic medicines, particularly in the field of antibody-based therapeutics for cancer and other diseases. MabSpace is headquartered in Hong Kong, with a 50,000 square-foot, fully-integrated antibody discovery and development facility in Biobay, Suzhou Industrial Park, China. MabSpace employs a sixty-person team covering discovery and translational research, process development, clinical and commercial development and regulatory affairs. With its proprietary antibody generation platform technology, Immune Tolerance Breaking Technology, MabSpace has established a pipeline of over ten innovative fast follow-on or first-in-class antibody programs in oncology, ophthalmology as well as nephrology.

Founded in 2016, HJB is dedicated to designing and applying innovative bioprocessing technologies to accelerate biologics R&D and manufacturing. HJB has global sites established in Shanghai, Hangzhou and Boston, and a global talent pool of over 100 scientists, clinicians and bioengineers. With its 140,000 square feet state-of-the-art POD-based manufacturing facility and next-generation continuous processing technology, HJB can enable speedy development of high-quality biotherapeutic agents and significantly reduce the cost of production to make these biologic medicines more affordable to a global population. Through in-licensing, HJB has obtained either China or global rights for several first-in-class next-generation immunotherapy antibody programs.

By leveraging and combining the complimentary capabilities of HJB and MabSpace, Transcenta will be equipped with a senior team with extensive global industrial experience in biologics-based therapeutics discovery and development, and fully integrated in-house capabilities in biologic therapeutics discovery, development and manufacturing. Transcenta will have a global footprint: Discovery and Translational Research Center in Suzhou, Process and Product Development Center and Manufacturing Facility in Hangzhou, and Clinical Development Centers in Shanghai, Beijing and Boston, US. The combined entity will have over 10 innovative pipeline molecules. Transcenta aims to shorten the timeline from target to BLA, and will make the high-quality, next-generation GMP facility available to support the development and commercialization of both in-house and strategic partner’s pipeline molecules.

"We are excited to merge with MabSpace. The newly merged entity will combine the strength of MabSpace and HJB in discovery, development and manufacturing," said Transcenta’s executive chairman, Dr. Jonathan Y. Zhao, "MabSpace and HJB have a combined pipeline with more than 10 pre-clinical and clinical pipeline products. Leveraging the HJB’s strength in process and clinical development and integrated biomanufacturing, Transcenta will continue to create greater values by providing high-quality biologics at affordable prices to patients around the world. "

"I would like to thank our investors from both sides for their full-hearted support of this strategic combination. With the merger of HJB and MabSpace, we can achieve a critical mass, significantly accelerate the development and commercialization of our innovative pipeline and establish Transcenta as a truly fully integrated biotherapeutics-focused global biotech company. This not only enables shorter development timelines, but also provides high-quality biotherapeutics at a much lower cost to ensure affordability and sustained profitability. We hope to build Transcenta as an enduring biotech company that delivers high quality, innovative medicines to our patients and value to our shareholders around the world," added Dr. Xueming Qian, Transcenta’s Co-Founder and CEO.

HJB and MabSpace have altogether raised approximately $160 million thus far from prominent investors, including Lilly Asia Ventures, Temasek, Sequoia China, ARCH Venture Partners, Teng Yue Partners and Taikang, etc. Transcenta is open to strategic collaboration with global biotech companies to further expand its portfolio and enhance its technology capabilities.

On January 1, 2019 Antengene Corporation (Antengene), a clinical stage therapeutics company focused on oncology, reported that it has completed a $120 million Series B financing (Press release, Antengene, JAN 1, 2019, View Source [SID1234532325]). The Series B financing was jointly led by Boyu Capital and FountainVest, with participation from Celgene Corporation, WuXi Corporate Venture Fund, and Taikang. Previous investors, Qiming Venture Partners and TF Capital, also participated in this round. This new round follows a $21 million Series A financing in 2017, led by Qiming Venture Partners.

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Founded in 2017, Antengene is a biopharmaceutical company that focuses on the development and commercialization of novel therapeutics. Headquartered in China, Antengene is committed to delivering innovative drugs and combination therapies to diseases with high unmet medical needs in China and the Asia-Pacific region.

Proceeds from the financing will be primarily used to fund the continuing development of the company’s lead programs ATG-008 and ATG-010 (selinexor) and other clinical-stage assets, to expand the company’s pipeline through internal R&D and external partnerships, and to prepare the commercial launch of late-stage drug candidates. A 169,984 square feet manufacturing and research facility in Shaoxing, China, currently undergoing construction for GMP manufacturing, will provide both the clinical and commercial drug supply for the company’s pipeline products.

ATG-010 (selinexor) is a first-in-class Selective Inhibitor of Nuclear Export (SINE) compound in late clinical development for the treatment of multiple hematological malignancies and solid tumors, including multiple myeloma, diffuse large B-cell lymphoma, liposarcoma, etc. ATG-008 is a second generation TORC1/2 inhibitor currently in late stage development for the treatment of hepatitis B virus positive (HBV+) hepatocellular carcinoma. This program has also been selected as a "Key Project for Novel Drug Development in China".

"We are grateful for the support and recognition from these prestigious and well-established investors, and that we share a common vision of delivering cutting-edge therapies to help patients with life-threatening diseases. We look forward to creating value for patients, investors and our partners," said Dr. Jay Mei, founder, chairman, and CEO of Antengene. "This round of financing is critical for Antengene’s growth. We will continue to maintain and advance rigorous, science-driven, and patient-centered R&D, while actively preparing for the commercialization of our lead products in China and the Asia-Pacific Region."

Commented Yanling Cao, Managing Director of Boyu Capital, "As a long-term investor in China’s biopharmaceutical industry, Boyu Capital is much honored to be the lead investor in the Series B financing of Antengene. We view in-licensing of first-in-class/best-in-class drug candidates as an effective solution to the unmet clinical needs in China. In addition, we are very impressed with the progress that Antengene is making and the pipeline they are building. Therefore, we are excited about the opportunity to work with the exceptional team at Antengene and bring novel therapeutics to Chinese and Asian patients."

"In the coming decade, China will rapidly fill the gap in healthcare compared to western countries. This trend will continuously bring opportunities to innovative biopharmaceutical companies such as Antengene. But it also requires patient capital and financial investor with strategic angle. That’s how FountainVest position ourselves," said Zhen Li, Managing Director of FountainVest. "We are delighted and fortunate to lead this round of investment in Antengene, and look forward to best deploying FountainVest’s resources and expertise in the field to contribute to the healthcare industry in China and the rest of Asia together with Antengene."

On January 1, 2019 Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops and commercializes high quality medicines, reported that the first patient has been dosed in a phase III clinical trial (ORIENT-15) that is to evaluate Tyvyt (fully human anti-PD-1 therapeutic monoclonal antibody, generic name: sintilimab injection), in combination with paclitaxel and cisplatin, as first-line treatment in patients with advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) (Press release, Innovent Biologics, JAN 1, 2019, View Source [SID1234532324]).

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The ORIENT-15 study is a randomized, double-blind, multi-center, phase III trial conducted in China to evaluate the efficacy and safety of Tyvyt (sintilimab injection) or placebo in combination with chemotherapy as first-line treatment in subjects with unresectable, locally advanced, recurrent or metastatic ESCC. The phase III study will enroll 640 patients. The study follows a phase Ib study that evaluated Tyvyt (sintilimab injection) in patients with esophageal cancer and a phase II study that evaluated Tyvyt (sintilimab injection) versus paclitaxel/irinotecan as second-line therapy for patients with advanced/metastatic ESCC.

"The incidence of esophageal squamous cell carcinoma in Asian countries is much higher than in western countries. Today, patients have no treatment options other than chemotherapy and radiation therapy. Immune checkpoint inhibitors have brought new hope to patients with this life-threatening disease. Based on the efficacy signals and the safety profile from previous trials, we hope to validate the therapeutic potential of Tyvyt (sintilimab injection) in combination with chemotherapy in ORIENT-15, a phase III trial," said Professor Lin Shen from the Beijing Cancer Hospital.

"Esophageal cancer is the third most common malignant tumor in China. The development of new agents for the treatment of advanced esophageal squamous cell carcinoma has been stagnant, so there is a huge unmet medical need. Based on the preliminary result or the ongoing phase II study, we have decided to conduct ORIENT-15, a phase III study as a first-line treatment for patients with esophageal squamous cell carcinoma. Our goal is to provide more effective cancer treatment options for these patients and for their families," said Michael Yu, Founder, Chief Executive Officer and Chairman of Innovent.

About Tyvyt (sintilimab injection)

Tyvyt (sintilimab injection) is an innovative drug jointly developed by Innovent and Eli Lilly and Company in China. Tyvyt (sintilimab injection) is a type of immunoglobulin G4 monoclonal antibody, which binds to the PD-1 molecule on the surface of T-cells, blocks the PD-L1（Programmed Cell Death-1 Ligand-1, PD-L1 pathway）and reactivates T-cells to kill cancer cells. Tyvyt (sintilimab injection) is the only PD-1 antibody in China branded by both a local biopharmaceutical company and a global pharmaceutical company.

About ORIENT-15 Study

The ORIENT-15 study is a randomized, double-blind, multi-center, phase III trial that evaluates the efficacy and safety of Tyvyt (sintilimab injection) in combination with chemotherapy as first-line treatment in subjects with unresectable, locally advanced recurrent or metastatic ESCC in China. Patients will receive Tyvyt (sintilimab injection) or placebo in combination with paclitaxel and cisplatin until disease progression. Participants will be randomly assigned in a 1:1 ratio into experimental or control groups. The study will enroll 640 patients. The primary endpoint is overall survival in both the entire population and in PD-L1 positive population of patients.

About Advanced or Metastatic Esophageal Squamous Cell Carcinoma (ESCC)

Esophageal cancer is the eighth most common cancer in the world, and the sixth leading cause of cancer death. Nearly four out of five cases occur in developing countries. China has the largest population of patients with esophageal cancer in the world. The incidence and mortality in China are higher than the worldwide average, ranking 3rd and 4th respectively. The histopathological type of esophageal cancer in China is different than that in Europe and the United States. In China the major type of esophageal cancer is squamous cell carcinoma which accounts for more than 90% of cases. In the United States and Europe adenocarcinoma of the esophagus is the predominant histopathology. The prognosis of patients with advanced and metastatic esophageal cancer is poor with an overall survival of about 10 months.

On January 31, 2019 Celgene Corporation (NASDAQ: CELG) reported operating results for the fourth quarter and full year of 2018 (Press release, Celgene, JAN 31, 2018, View Source [SID1234532985]). For the fourth quarter of 2018, net product sales were $4,036 million, an increase of 16 percent year-over-year. Fourth quarter total revenue increased 16 percent year-over-year to $4,037 million.

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Net product sales for the full year of 2018 were $15,265 million, an increase of 18 percent year-over-year. Total revenue for the full year of 2018 was $15,281 million, an increase of 18 percent year-over-year.

Based on U.S. GAAP (Generally Accepted Accounting Principles), Celgene reported net income of $1,073 million and diluted earnings per share (EPS) of $1.50 for the fourth quarter of 2018. For the fourth quarter of 2017, GAAP net loss was $81 million and diluted EPS was ($0.10). Full year GAAP net income for 2018 was $4,046 million and diluted EPS was $5.51. Full year GAAP net income for 2017 was $2,940 million and diluted EPS was $3.64.

Adjusted net income for the fourth quarter of 2018 increased 7 percent to $1,709 million compared to $1,592 million in the fourth quarter of 2017. For the same period, adjusted diluted EPS increased 20 percent to $2.39 from $2.00.

Adjusted net income for the full year of 2018 increased 8 percent to $6,511 million. Adjusted diluted EPS increased 19 percent to $8.87 from $7.44 for the full year of 2017.

"2018 was another year of excellent operating results and significant progress advancing our innovative early-, mid- and late-stage pipeline," said Mark J. Alles, Chairman and Chief Executive Officer of Celgene Corporation. "With five near-term product launches and many promising assets advancing, we are very optimistic about our potential for long-term growth as part of the new Bristol-Myers Squibb."

Fourth Quarter and Full Year 2018 Financial Highlights

Unless otherwise stated, all comparisons are for the fourth quarter and full year of 2018 compared to the fourth quarter and full year of 2017. The adjusted operating expense categories presented below exclude share-based employee compensation expense, collaboration-related upfront expense, research and development asset acquisition expense, IPR&D asset impairment charges, clinical trial and development activity wind-down costs and a litigation-related loss contingency accrual expense. 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 fourth quarter increased 16 percent to $2,549 million. Fourth quarter U.S. sales of $1,729 million and international sales of $820 million increased 17 percent and 15 percent, respectively. REVLIMID sales growth was driven by increases in treatment duration and market share. Full year REVLIMID sales were $9,685 million, an increase of 18 percent year-over-year.
POMALYST/IMNOVID sales for the fourth quarter were $567 million, an increase of 28 percent year-over-year. Fourth quarter U.S. sales of $393 million and international sales of $174 million increased 39 percent and 9 percent, respectively. POMALYST/IMNOVID sales growth was driven primarily by increases in treatment duration and market share. Full year POMALYST/IMNOVID sales were $2,040 million, an increase of 26% year-over-year.
OTEZLA sales in the fourth quarter were $448 million, a 21 percent increase year-over-year. Fourth quarter U.S. sales of $360 million and international sales of $88 million increased 19 percent and 29 percent, respectively. OTEZLA sales growth in the U.S. was driven by increases in demand. OTEZLA international sales were driven by launch uptake in key ex-U.S. markets, including Japan. Full year OTEZLA sales were $1,608 million, an increase of 26 percent year-over-year.
ABRAXANE sales for the fourth quarter were $269 million, an increase of 7 percent year-over-year. Fourth quarter U.S. sales of $178 million and international sales of $91 million increased 15 percent and decreased 5 percent, respectively. ABRAXANE sales growth was driven primarily by demand. Full year ABRAXANE sales were $1,062 million, an increase of 7 percent year-over-year.
In the fourth 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 $203 million compared to $227 million in the fourth quarter of 2017. Full year sales for these products were $870 million compared to $901 million for the full year 2017.
Research and Development (R&D)

On a GAAP basis, R&D expenses were $1,138 million for the fourth quarter of 2018 versus $2,738 million for the same period in 2017. Full year 2018 R&D expenses were $5,673 million compared to $5,915 million for 2017.

Adjusted R&D expenses were $919 million for the fourth quarter of 2018 compared to $766 million for the same period in 2017. For the full year 2018, adjusted R&D expenses were $3,509 million compared to $2,749 million for the full year 2017. Both the fourth quarter and full year 2018 increases in R&D expenses were primarily driven by the inclusion of R&D expenses associated with the acquisition of Juno Therapeutics (Juno) and regulatory submission-related work on multiple programs. Additional R&D expenses (only included on a GAAP basis) decreased in 2018, as outlined in the attached Reconciliation of GAAP to Adjusted Net Income.

Selling, General, and Administrative (SG&A)

On a GAAP basis, SG&A expenses were $850 million for the fourth quarter of 2018 compared to $774 million for the same period in 2017. Full year SG&A expenses were $3,250 million for 2018 compared to $2,941 million for 2017.

Adjusted SG&A expenses were $762 million for the fourth quarter of 2018 compared to $687 million for the same period in 2017. For full year 2018, adjusted SG&A expenses were $2,747 million versus $2,279 million in 2017. Both the fourth quarter and full year 2018 increases in SG&A expenses were primarily driven by the inclusion of SG&A expense associated with the acquisition of Juno and marketing-related expenses. Additional SG&A expenses (only included on a GAAP basis) increased in 2018, as outlined in the attached Reconciliation of GAAP to Adjusted Net Income.

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

Operating cash flow was $5.2 billion for both 2018 and 2017. Celgene ended the fourth quarter of 2018 with approximately $6.0 billion in cash, cash equivalents, marketable debt securities and publicly-traded equity securities.

**Not meaningful as the 2019 measures exclude the impact of any strategic transactions, impairments, loss contingencies, changes in the fair value of equity investments, costs associated with the Bristol-Myers Squibb Company (Bristol-Myers Squibb) and Celgene transaction and non-operating tax adjustments that have not yet occurred.

Portfolio Updates

At the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December, data were presented on Celgene’s marketed and pipeline hematology assets. Select data presentations included:
In collaboration with partner Acceleron Pharma, data from the phase III MEDALIST and BELIEVE trials with luspatercept in patients with low-to-intermediate risk myelodysplastic syndromes (MDS) and transfusion-dependent beta-thalassemia, respectively;
Data from the phase I TRANSCEND CLL-004 trial evaluating liso-cel in patients with relapsed and/or refractory chronic lymphocytic leukemia (CLL); and,
Data from the phase III AUGMENT trial evaluating REVLIMID in combination with rituximab (R²) in patients with relapsed and/or refractory indolent non-Hodgkin lymphoma (NHL)
A New Drug Application (NDA) was submitted to the U.S. Food and Drug Administration (FDA) for fedratinib for the treatment of patients with myelofibrosis. Celgene plans to submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) in the first half of 2019. In addition, the phase III myelofibrosis program (FREEDOM and FREEDOM-2 trials) evaluating fedratinib in patients resistant or intolerant to ruxolitinib is initiating
A supplemental NDA (sNDA) was submitted to the U.S. FDA for REVLIMID in combination with rituximab (AUGMENT trial) for the treatment of patients with relapsed and/or refractory indolent NHL. The anticipated U.S. FDA action date for this application is in the second half of 2019
In the fourth quarter, Celgene and partner bluebird bio announced the completion of enrollment for the KarMMa pivotal trial evaluating bb2121 in patients with relapsed and/or refractory multiple myeloma (RRMM)
The phase II TRANSCEND OUTREACH trial evaluating liso-cel (JCAR017) in patients with relapsed and/or refractory diffuse large B-cell lymphoma (DLBCL) in the outpatient setting initiated in the fourth quarter
The phase II pivotal trial evaluating liso-cel in patients with relapsed and/or refractory CLL is initiating
The phase III ADVANCE trial evaluating OTEZLA in patients with mild to moderate plaque psoriasis is initiating
Business Update Summary

In January, Celgene and Bristol-Myers Squibb (BMS) announced that they have entered into a definitive merger agreement under which BMS will acquire Celgene for approximately $74 billion (based on closing price of BMS on date of the agreement). Under the terms of the agreement, Celgene shareholders will receive for each Celgene share $50 plus one BMS share and one tradeable Contingent Value Right (CVR), which will entitle the holder to receive a cash payment of $9.00 upon the achievement of FDA approval of all three products (ozanimod, liso-cel and bb2121) within specified time periods. The transaction is subject to approval by BMS and Celgene stockholders and the completion of customary closing conditions and regulatory approvals. BMS and Celgene expect to close the transaction in the third quarter of 2019.
Q4 and Full Year 2018 Conference Call and Webcast Information

Celgene will host a conference call to discuss the fourth quarter and full year of 2018 operational and financial performance on Thursday, January 31, 2019, 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 January 31, 2019, until midnight ET February 7, 2019. To access the replay in the U.S., dial 1-855-859-2056; outside the U.S. dial 404-537-3406. The participant passcode is 7075709.