On February 24, 2016 bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, reported business highlights and financial results for the fourth quarter and full year ended December 31, 2015 (Press release, bluebird bio, FEB 24, 2016, View Source;p=RssLanding&cat=news&id=2142878 [SID:1234509175]).

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"In 2015 bluebird bio defined an accelerated regulatory path for LentiGlobin in ß-thalassemia and established a powerful reason to believe in LentiGlobin in sickle cell disease, though there is still more to learn as we treat additional patients. We also fully enrolled our Starbeam study of Lenti-D in cerebral adrenoleukodystrophy and made significant advances in building a competitive T cell oncology franchise," said Nick Leschly, chief bluebird. "In 2016 we are excited to learn even more across all of our programs and continue to innovate and improve. We are particularly looking forward to sharing data from the Starbeam study for the first time and presenting more data with longer follow-up from all three of our LentiGlobin clinical studies."

Recent Highlights

ADVANCED FIRST ONCOLOGY PROGRAM INTO THE CLINIC – Earlier in February, the first patient was infused in the CRB-401 study of bb2121 in relapsed/refractory multiple myeloma. Additionally, Celgene has exercised its option to exclusively license bb2121, under the terms of the collaboration agreement between the two companies. bluebird bio will receive a $10.0 million option exercise payment from Celgene and is also eligible to receive specified development and regulatory milestone payments and royalty payments on net sales.

PRESENTED UPDATED CLINICAL DATA IN ß-THALASSEMIA FROM HGB-204 AND HGB-205 CLINICAL STUDIES OF LENTIGLOBIN AT ASH (Free ASH Whitepaper) – In December 2015, investigators presented data from bluebird bio’s ongoing clinical studies in transfusion-dependent ß-thalassemia (TDT) and severe sickle cell disease (SCD). Data in patients with TDT from the HGB-204 and HGB-205 studies showed that 100% of patients with non-ß0/ß0 genotypes achieved sustained transfusion independence as of the data cut-off, ranging from 7.1 months to 23.4 months. Patients with the ß0/ß0 genotype all saw reductions in their transfusion needs, ranging from a 33% to 100% reduction.

PRESENTED CLINICAL DATA IN SICKLE CELL DISEASE FROM HGB-205 AND HGB-206 CLINICAL STUDIES OF LENTIGLOBIN AT ASH (Free ASH Whitepaper) –Marina Cavazzana, M.D., Ph.D., of Hospital Necker, University Paris Descartes, presented updated data on one patient with SCD from the HGB-205 study, who remained free of transfusions, hospitalizations and acute SCD-related events for more than nine months as of the data cut-off. At the 12-month post-drug infusion follow-up, the proportion of anti-sickling hemoglobin in this patient accounted for 49% (47% HbAT87Q + 2% HbF) of all hemoglobin production – well above the 30% threshold anticipated to achieve a disease-modifying effect. John Tisdale, M.D., of the National Institutes of Health presented early data from the HGB-206 study, in which two patients had at least three months of post-infusion follow-up. At the three-month post-infusion follow-up for Subject 1301, anti-sickling hemoglobin accounted for 17% of all hemoglobin production (4% HbAT87Q + 13% HbF). At the six-month post-infusion follow-up for subject 1303, anti-sickling hemoglobin accounted for 16 percent of all hemoglobin production (12% HbAT87Q + 4% HbF).

PRESENTED PRE-CLINICAL AND MANUFACTURING DATA FROM CAR T ONCOLOGY PROGRAMS AT ASH (Free ASH Whitepaper) – bluebird bio scientists presented three posters at ASH (Free ASH Whitepaper), covering critical basic research, translational and manufacturing aspects of the Company’s T cell oncology programs. One poster discussed an important observation made by bluebird bio scientists: culturing anti-BCMA CAR T cells with a PI3K inhibitor generated a product with many of the properties of younger, less differentiated T cells. Consistent with a younger T cell phenotype, this product showed improved in vivo efficacy and persistence in multiple model systems.
SHARED DATA ON PLATFORM IMPROVEMENTS – In an investor event at ASH (Free ASH Whitepaper), bluebird bio chief scientific officer Philip Gregory, D.Phil., presented data from ongoing research to improve the cell transduction process for LentiGlobin. The presentation showed that in preclinical experiments, adding selected compounds to the transduction process resulted in substantially increased vector copy number and transduction efficiency (i.e. percentage of corrected cells). Importantly, the new process was shown to be robust with similar improvements seen across multiple donors and vector lots.

ENTERED INTO CAR T LICENSE WITH VIROMED – Signed exclusive license agreement with Viromed Co., Ltd., to research, develop and commercialize CAR T therapies using Viromed’s proprietary humanized antibody to an undisclosed cancer target in solid tumors.

Upcoming Anticipated Milestones

Presentation of interim data from the Starbeam study of Lenti-D in patients with cerebral adrenoleukodystrophy (CALD) at the American Academy of Neurology annual meeting in April 2016

Update on LentiGlobin process improvements in the second half of 2016

Initiation of the HGB-207 study in patients with TDT with the non-ß0/ß0 genotype in the second half of 2016

Presentation of updated data from the HGB-204, HGB-205 and HGB-206 studies at the ASH (Free ASH Whitepaper) annual meeting in December 2016
Fourth Quarter and Full Year 2015 Financial Results and Financial Guidance

Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2015 were $865.8 million, compared to $492.0 million as of December 31, 2014, an increase of $373.8 million, which was primarily driven by the June 2015 equity financing partially offset by cash used to fund operations.

Revenues: Collaboration revenue was $1.5 million for the fourth quarter of 2015 and $14.1 million for the year ended December 31, 2015, compared to $6.3 million and $25.0 million in the comparable periods in 2014. The decrease is a result of an amendment to our collaboration agreement with Celgene in the second quarter of 2015.

R&D Expenses: Research and development expenses were $35.7 million for the fourth quarter of 2015 and $134.0 million for the year ended December 31, 2015, compared to $20.5 million and $62.6 million for the comparable periods in 2014. The increase in research and development expenses was primarily attributable to increased employee compensation expense due to increased headcount, in-licensing milestones and fees, and manufacturing and clinical trial-related costs to support our advancing pipeline.

G&A Expenses: General and administrative expenses were $14.4 million for the fourth quarter of 2015 and $46.2 million for the year ended December 31, 2015, compared to $5.3 million and $23.2 million for the comparable periods in 2014. The increase in general and administrative expenses was primarily attributable to increased employee compensation expense due to increased headcount, and consulting and facilities-related costs to support our overall growth.

Net Loss: Net loss was $47.3 million for the fourth quarter of 2015 and $166.8 million for the year ended December 31, 2015, compared to net loss of $19.5 million and $48.7 million for the comparable periods in 2014.

Financial guidance: bluebird bio expects that its cash, cash equivalents and marketable securities of $865.8 million as of December 31, 2015 will be sufficient to fund its current operations through 2018.

On February 24, 2016 The Clinical Center at the National Institutes of Health reported that they are investigating the potential use of a new generation of a computerized tomography (CT) scanner, called a photon-counting detector CT scanner, in a clinical setting (Press release, NIH, FEB 24, 2016, View Source [SID:1234509173]). The prototype technology is expected to replicate the image quality of conventional CT scanning, but may also provide health care specialists with an enhanced look inside the body through multi-energy imaging. Patients could receive a minimum amount of radiation, while the maximal amount of information needed would be delivered to health care providers.

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Over the next five years, David Bluemke, M.D., Ph.D., chief of the Department of Radiology and Imaging Sciences, and his team will continue to develop scan protocols and image processing algorithms, which could improve screening, imaging, and treatment planning for health conditions like cancer and cardiovascular disease.

"The NIH Clinical Center has helped shape and share research advances and health care for decades. Now is an exciting time for us and for our study participants here in the Clinical Center as we help test and develop this CT technology so that it may one day help patients around the world and impact the health care they receive," said Dr. Bluemke.

As the world’s largest hospital solely dedicated to research, the NIH Clinical Center sees thousands of patients every year, many of whom have rare and complicated illnesses. In the treatment and study of disease, surgery is often viewed as the last option. CT scanning is one way that doctors can examine the body’s internal features in a non-surgical way. In collaboration and through a partnership known as a cooperative research and development agreement with the manufacturer, Siemens Healthcare, and researchers in the CT technology field, the Clinical Center is testing this technology to help the health care field optimize the scanner for clinical use across the U.S. and around the globe.

Image of photon-counting CT scan
Photon CT scan image of a research subject at the NIH. Greater amounts of iodine contrast are shown in brighter, yellow colors.
The Clinical Center is one of three sites in the world to use this technology and is the first hospital-based research setting of the device. More than 45 volunteers enrolled in a research protocol have benefited from this cutting edge equipment. Initial findings have been reported in Radiology (link is external).

By advancing this technology, researchers aim to improve the diagnosis that doctors can offer by increasing the resolution and contrasts available for analysis. Areas of research investigation with the new technology include:

Doctors can identify materials in the body with anatomic precision. A dye, or contrast, is often given to a patient so that researchers can see a selected area in more detail. Different materials in the body can be displayed in different colors for faster diagnosis and precision.
The new technology may be used to help identify and characterize tumors, plaques or vessels that are smaller than half a millimeter. For many patients, finding a tumor that size may make a difference in identifying if it is benign or could be cancerous.
The technology may help to more accurately identify soft tissues such as proteins, tendons or collagen which are hard to differentiate with current equipment.

On February 24, 2016, At its meeting today, the Supervisory Board of Bayer AG announced that they have approved the Board of Management’s recommendation that a dividend payment of EUR 2.50 (2014: EUR 2.25) per share be proposed to the Annual Stockholders’ Meeting on April 29, 2016 (Press release, Bayer, FEB 24, 2016, View Source [SID:1234509168]). "2015 was a very good year for Bayer. We would like our stockholders to share appropriately in this success," explained Bayer CEO Dr. Marijn Dekkers. With 826,947,808 shares entitled to the dividend, the total dividend payment would amount to EUR 2,067 million (2014: EUR 1,861 million), an increase of 11.1 percent.

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The Bayer Group’s consolidated financial statements for 2015 will be presented and discussed at the Financial News Conference on February 25, 2016.

On February 24, 2016 Provectus Biopharmaceuticals, Inc. (NYSE MKT: PVCT, www.pvct.com), a clinical-stage oncology and dermatology biopharmaceutical company ("Provectus" or "The Company"), reported that data on intralesional PV-10 and co-inhibitory blockade in a melanoma model will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper)’s ("AACR") Annual Meeting 2016 on Wednesday, April 20, 2016, from 8 am to 12 Noon Central Standard Time (Filing, 8-K, Provectus Pharmaceuticals, FEB 24, 2016, View Source [SID:1234509165]).

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The poster presentation is titled "T Cell Mediated Immunity after Combination Therapy with Intralesional PV-10 and Co-Inhibitory Blockade in a Melanoma Model." Scheduled for presentation at Section 26 of the exhibition area, the data are from a team of researchers at the H. Lee Moffitt Cancer Center in Tampa, led by Dr. Shari Pilon-Thomas.

The AACR (Free AACR Whitepaper) Annual Meeting 2016 is being held at the Ernest N. Morial Convention Center in New Orleans, Louisiana, from April 16-20, 2016.

On February 24, 2016 Forty Seven Inc., a clinical-stage immuno-oncology company, reported that it has completed the first half of a committed $75 million Series A financing round and has licensed the rights to multiple immuno-oncology programs from Stanford University (Press release, Forty Seven, FEB 24, 2016, View Source [SID:1234509244]).

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The Series A financing was led by Lightspeed Venture Partners and Sutter Hill Ventures with participation from Clarus Ventures and GV (formerly Google Ventures). The license includes rights to over 100 issued or pending U.S. or foreign patents that cover the antibody Hu5F9-G4 and several other novel immune checkpoint inhibitors and cancer-specific antibodies.
Forty Seven is committed to the advancement of immuno-oncology through the engagement of new and complementary phagocytic pathways that enhance anti-tumor efficacy and selectivity. The financing will allow Forty Seven to continue the clinical development of its lead molecule, Hu5F9-G4, a humanized monoclonal antibody against human CD47 that potentially has broad applications spanning multiple tumor types and treatment modalities.

CD47 is a molecule that is overexpressed on the surface of the majority of tumors and transmits a "don’t eat me" signal, enabling cancer cells to evade phagocytosis by macrophages. The molecule was originally identified as a cancer target by researchers at Stanford. In preclinical models, Hu5F9-G4 facilitated phagocytosis and elimination of cancer cells from multiple human tumor types as a monotherapy. Additionally, when used in combination therapy, it engaged macrophages as effector cells to enhance the efficacy of cancer-specific antibodies via Antibody-Dependent Cellular Phagocytosis (ADCP). Importantly, Hu5F9-G4 also could prime an effective antitumor T-cell response through cross-presentation of cancer cell antigens by macrophages, preventing engraftment of tumors expressing a cross-presented antigen into animals.

Company founder Irv Weissman said, "Targeting CD47 integrates the adaptive and innate immune systems creating synergy with existing cancer-specific antibodies like rituximab, cetuximab and trastuzumab through ADCP, and potentially with T-cell checkpoint inhibitors through cross-presentation. We are grateful to the California Institute for Regenerative Medicine (CIRM) for funding the preclinical studies and the current solid tumor clinical trial at Stanford, and to Ludwig Cancer Research for funding much of the research."

"The founders, Irv Weissman, Ravi Majeti, Mark Chao and Jens Volkmer, have studied the CD47 pathway extensively since they initially identified it as a cancer target in two papers published in 2009," commented Chris Schaepe, Partner at Lightspeed Venture Partners. "The company’s scientific founders have done an outstanding job of advancing Hu5F9-G4 into two Phase 1 clinical trials in patients with relapsed or refractory solid tumors or acute myeloid leukemia (AML)."

"Forty Seven’s accomplishments pre-Series A funding are unusual as is the breadth of its patent portfolio which also has broad potential applications outside of cancer," remarked Jeff Bird, Partner at Sutter Hill Ventures.

Forty Seven will use the proceeds of the Series A financing to complete the two ongoing clinical studies, fund additional clinical trials in 2016 to assess Hu5F9-G4 in combination therapy and advance some preclinical programs towards IND.

"Forty Seven has hired a strong management team with deep industry experience. I feel confident that this combined team along with the support we have from our Series A investors and our scientific founders will enable us to fully explore the clinical utility of our lead molecule and the licensed technology," said Jonathan MacQuitty, CEO of Forty Seven Inc.

Forty Seven Senior Management
Chief Executive Officer, Jonathan MacQuitty, Ph.D., M.B.A. former Partner at Abingworth and former CEO of GenPharm.
Chief Business Officer, Craig Gibbs, Ph.D., M.B.A. former VP and Head of Commercial Strategy, Corporate Development and Biology Research at Gilead.
Chief Medical Officer, Chris Takimoto M.D. Ph.D. former VP Experimental Medicine Early
Development Oncology at Janssen/J&J.
Chief Patent Officer, Norm Kruse Ph.D., J.D. former Head of Intellectual Property at Verinata Health and Maxygen.
VP Clinical Operations, Hassan Movahhed, M.S. former SVP and Head of Clinical Operations at United Therapeutics.

Forty Seven Board of Directors
Jeff Bird, Sutter Hill Ventures.
Dennis Henner, Clarus Ventures.
Jonathan MacQuitty, Forty Seven Inc.
Ravi Majeti, Stanford University School of Medicine.
Chris Schaepe, Lightspeed Venture Partners.
Irv Weissman, Stanford University School of Medicine.

About Forty Seven Inc.
Forty Seven Inc. is a clinical-stage immuno-oncology company that is developing therapies licensed from Stanford University targeting cancer immune evasion pathways. The lead program Hu5F9-G4 is a monoclonal antibody against the CD47 receptor, a "don’t eat me" signal that cancer cells commandeer to avoid being ingested by the immune system. This antibody is currently being evaluated in two Phase 1 clinical studies in patients with solid tumors and in patients with acute myeloid leukemia. Forty Seven is located at 1661 Page Mill Road, Suite C, Palo Alto, CA 94304, U.S.A. For more information please visit www.fortyseveninc.com or contact Chief Business Officer Craig Gibbs at (650)-352-4136.