On February 28, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, today explains PharmaCyte’s plans to conduct a Phase 2b clinical trial (Press release, PharmaCyte Biotech, FEB 28, 2018, View Source [SID1234524256]).

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When PharmaCyte met with the FDA in January of 2017, PharmaCyte’s pre-IND meeting submission was predicated on PharmaCyte conducting a Phase 2b trial. During discussions with the U.S. Food and Drug Administration (FDA), PharmaCyte asked whether the data from that planned trial could be considered "pivotal" and thus support registration for marketing purposes. The FDA indicated that this was a possibility, but that the trial would have to be much larger than PharmaCyte was planning and the data would have to be markedly superior to the data seen with the comparator treatment.

PharmaCyte’s decision to conduct a Phase 2b trial rather than a pivotal trial was made relatively recently based on advice from PharmaCyte’s consulting oncologists, Chief Medical Officer and information obtained from PharmaCyte’s Advisory Board. PharmaCyte must rely on data from two European trials from 27 patients that were conducted about 20 years ago. The data from these trials are incomplete when compared to what is now required by the FDA to support a pivotal trial.

The Phase 2b trial is designed to determine how effective and safe multiple courses of ifosfamide will be in patients with locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) and how PharmaCyte’s treatment compares to a commonly used treatment for LAPC after patients’ tumors no longer respond following 4 to 6 months of the combination therapy of gemcitabine and Abraxane. PharmaCyte is designing a Phase 2b clinical trial that, if successful, it believes will give the company a much more solid foundation for dealing with the FDA with the goal of bringing its pancreatic cancer therapy to market.

The planned Phase 2b trial will be significantly larger than the original Phase 2b trial PharmaCyte previously discussed with the FDA and will include multiple courses of low dose ifosfamide (the earlier trials used only two courses). This trial will also provide better statistical analyses of PharmaCyte’s therapy for LAPC and the comparator arm in terms of survival and safety. Also, the possibility exists that if the data from PharmaCyte’s therapy are significantly better than the data from the comparator arm, this may allow PharmaCyte to apply to the FDA for accelerated approval.

Valeant has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Valeant, 2018, FEB 28, 2018, View Source [SID1234524250]).

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On February 28, 2018 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported financial results for the fourth quarter and full year ended December 31, 2017 and provided a corporate update (Press release, Portola Pharmaceuticals, FEB 28, 2018, View Source;p=RssLanding&cat=news&id=2335410 [SID1234524257]).

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"2017 was a year of significant achievement for Portola, highlighted by the FDA approval of our first medicine, Bevyxxa, and the regulatory application for FDA approval of our breakthrough-designated Factor Xa-inhibitor antidote, AndexXa," said Bill Lis, chief executive officer of Portola. "We are pleased with the early indicators of the U.S. commercial launch of Bevyxxa, which began in January, and the potential to have a major public health impact on millions of patients in the U.S. and beyond. We also are rapidly approaching the FDA action date for AndexXa, which, if approved, would position Portola with two first-in-class medicines in the field of thrombosis. In the meantime, we are working with European regulatory authorities on the path forward for both medicines and are continuing to advance our dual, oral Syk/JAK inhibitor, cerdulatinib, for hematologic cancers."

Recent Achievements, Upcoming Events and Milestones

Bevyxxa (betrixaban) – an oral, once-daily Factor Xa inhibitor approved for extended prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE.

Initiated U.S. commercial launch of Bevyxxa in January 2018
Received FDA approval for the manufacturing of betrixaban in Cork, Ireland
Completed an oral explanation to the European Committee for Medicinal Products for Human Use (CHMP); determining next steps in light of negative CHMP trend vote
New APEX trial results published in The American Heart Journal showing betrixaban’s effect on symptomatic VTE and VTE-related deaths; 12th major peer-reviewed publication

AndexXa (andexanet alfa) – an antidote for Factor Xa inhibitor treated patients with life-threatening bleeding; designated a Breakthrough Therapy and an Orphan Drug by the FDA.

The FDA set a new action date of May 4, 2018 for the Biologics License Application (BLA)
Successfully completed the first commercial campaign for Gen 2 product
Enrollment remains on track for the ongoing Phase 3b/4 ANNEXA-4 study in patients with acute major bleeding
Late-breaking clinical trial presentation on March 12, 2018 at the American College of Cardiology’s 67th Annual Scientific Session & Expo (ACC.18) featuring new interim data from ANNEXA-4
Successfully completed an oral explanation to the European CHMP; received a positive CHMP trend vote and are working with regulatory authorities to address their accompanying request for additional data

Cerdulatinib – an oral, dual-spleen tyrosine kinase (Syk) and janus kinase (JAK) inhibitor in development for the treatment of relapsed/refractory B-cell and other T-cell malignancies in patients who have failed multiple therapies.

Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell and T-cell malignancies who have failed multiple therapies

Fourth Quarter and Full Year 2017 Financial Results
Collaboration and license revenue earned under Portola’s collaboration and license agreements with Bristol-Myers Squibb Company, Pfizer, Bayer Pharma, Janssen Pharmaceuticals, Daiichi Sankyo and Dermavant Sciences was $9.8 million for the fourth quarter of 2017, compared with $13.7 million for the fourth quarter of 2016. Collaboration and license revenue for the year ended December 31, 2017 was $22.5 million, compared with $35.5 million for the year ended December 31, 2016.

Total operating expenses for the fourth quarter of 2017 were $95.7 million, compared with $68.9 million for the same period in 2016. Total operating expenses for the fourth quarter of 2017 included $10.9 million in stock-based compensation expense, compared with $7.9 million for the same period in 2016. Total operating expenses for the year ended December 31, 2017 were $295.2 million, compared with $305.1 million for 2016. Total operating expenses for the full year ended December 31, 2017 included $43.3 million in stock-based compensation expense compared with $30.4 million for 2016.

Research and development expenses were $68.5 million for the fourth quarter of 2017, compared with $56.0 million for the fourth quarter of 2016. Research and development expenses were $203.7 million for the year ended December 31, 2017, compared with $246.9 million for 2016.

Selling, general and administrative expenses for the fourth quarter of 2017 were $26.9 million, compared with $12.9 million for the same period in 2016. Selling, general and administrative expenses for the year ended December 31, 2017 were $91.1 million, compared with $58.2 million for 2016.

For the fourth quarter of 2017, Portola reported a net loss of $91.8 million, or $1.41 net loss per share, compared with a net loss of $53.8 million, or $0.95 net loss per share, for the same period in 2016. Shares used to compute net loss per share attributable to common stockholders were $65.3 million for the fourth quarter of 2017 compared with $56.5 million for the same period in 2016. Net loss for the year ended December 31, 2017 was $286.1 million, or $4.81 net loss per share, compared with a net loss of $269.0 million, or $4.76 net loss per share, for the same period in 2016. Shares used to compute net loss per share attributable to common stockholders were $59.5 million for 2017 compared with $56.5 million for 2016.

Cash, cash equivalents and investments at December 31, 2017 totaled $534.2 million, compared with cash, cash equivalents and investments of $318.8 million as of December 31, 2016.

If the FDA approves andexanet alfa in Q2 2018, the Company will be entitled to receive an additional $100 million from its royalty-based financing with Health Care Royalty Partners.

2018 Annual Financial Guidance
For the fiscal year 2018, Portola expects total GAAP operating expenses to be between $390 million and $430 million, including stock based compensation. These expenses are primarily for manufacturing of both andexanet alfa and betrixaban, ongoing clinical trials, support for the commercial launch of Bevyxxa and preparation for the potential commercial launch of AndexXa.

Conference Call Details
Portola will host a conference call today, Wednesday, February 28, 2018, at 4:30 p.m. ET, during which time management will provide fourth quarter and full year 2017 financial results, updates on the U.S. launch of Bevyxxa and other matters. The live call can be accessed by phone by dialing (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 4893459. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call

Ionis Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing, 10-K, Ionis Pharmaceuticals, 2018, FEB 28, 2018, View Source [SID1234524271]).

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On February 28, 2018 Daiichi Sankyo, Inc. ("the Company") reported that it will reorganize its U.S. Commercial organization as it continues to maximize opportunities for its current portfolio and prepare for its upcoming oncology pipeline (Press release, Daiichi Sankyo, FEB 28, 2018, View Source [SID1234524227]). The U.S. restructuring will streamline the Company’s operations, enable it to best serve its current patients and maximize its contribution to the development of medicines that change the standard of care. As part of the reorganization, the Company will reduce headcount by approximately 280 employees from various locations in the U.S.

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"Our priorities are to bring spending in line with revenue, shift resources to maximize Injectafer and our abuse-deterrent pain treatments, and prepare for exciting potential new treatments for patients with cancer being developed by our R&D organization," said Ken Keller, President, Administrative and Commercial, Daiichi Sankyo, Inc. "While the decision to reduce our workforce is a very difficult one, the changes are necessary to position us for long-term success. We are grateful for the contributions of all employees, and we are committed to making this process as easy and as streamlined as possible."

Daiichi Sankyo will offer outplacement services, severance and other support to all employees who are directly affected.