On December 6, 2017 Fate Therapeutics, Inc. (NASDAQ:FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported a partnership with the University of California San Diego to develop off-the-shelf, chimeric antigen receptor (CAR)-targeted natural killer (NK) cell cancer immunotherapies (Press release, Fate Therapeutics, DEC 6, 2017, View Source [SID1234522400]). The two-year collaboration is being led by Dan S. Kaufman, M.D., Ph.D., Professor of Medicine in the Division of Regenerative Medicine and Director of Cell Therapy at UC San Diego School of Medicine.

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"NK cells have the inherent ability to target a diversity of stress-induced ligands on tumor cells and can be safely administered without the need for individualized patient matching. Additionally, NK cells engineered with chimeric antigen receptors can be targeted to tumors with high specificity. This duality provides CAR NK cells with the unique potential to overcome antigen escape and address tumor heterogeneity, which are distinct advantages over patient-specific CAR T-cell immunotherapies," said Dr. Kaufman. "We have now identified several CAR constructs optimized for NK cell signaling, persistence and cytotoxicity, and combined our targeting content with Fate Therapeutics’ induced pluripotent stem cell product platform for development of off-the-shelf CAR-targeted NK cell products using clonal engineered master pluripotent cell lines."

The CAR constructs identified by the collaborators contain transmembrane and co-stimulatory domains that enhance antigen-specific NK cell activation and improve the effector function of NK cells. Fate Therapeutics holds an exclusive license to the intellectual property covering these CAR constructs and maintains an option to exclusively license intellectual property arising from all research and development activities under the collaboration.

At the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, Dr. Kaufman and Fate Therapeutics will present preclinical data on Saturday, December 9, 2017 highlighting CAR-targeted NK cells derived from an induced pluripotent stem cell (iPSC) engineered with a specific CAR construct containing a NKG2D transmembrane domain, a 2B4 co-stimulatory domain and a CD3ζ signaling domain. In preclinical studies using an ovarian cancer xenograft model, the collaborators have shown that a single dose of CAR-targeted NK cells derived from iPSCs engineered with this specific CAR construct markedly inhibited tumor growth and significantly enhanced survival as compared to NK cells containing a CAR construct commonly used for T-cell immunotherapy. Dr. Kaufman was recently awarded $5.15 million by the California Institute for Regenerative Medicine (CIRM) to advance clinical translation of NK cells derived from pluripotent stem cells into a standardized treatment for treating hematologic malignancies.

iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The engineering of iPSCs can be done as a one-time genetic modification event and a single iPSC can be selected for creation of a clonal master pluripotent cell line. Similar to master cell lines used for the manufacture of therapeutic antibodies, a clonal master pluripotent cell line can be used to repeatedly create clonal populations of effector cells. This first-of-kind approach enables large-scale generation of off-the-shelf, targeted, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments.

On December 6, 2017 Geron Corporation (Nasdaq:GERN) reported that the company’s analyst and investor meeting to discuss imetelstat clinical data will take place in New York, N.Y., on Tuesday, December 19, 2017 (Press release, Geron, DEC 6, 2017, View Source;p=RssLanding&cat=news&id=2321341 [SID1234522399]). The presentation will begin at 8:30 a.m. ET.

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Dr. Azra Raza, M.D., Professor of Medicine and Director of the Myelodysplastic Syndromes (MDS) Center at New York-Presbyterian/Columbia University Medical Center in New York City, will present data from Part 1 of IMerge, the ongoing Phase 2/3 clinical trial of the telomerase inhibitor imetelstat in patients with lower risk MDS being conducted by Janssen Research & Development, LLC. These data have been accepted as a poster presentation at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

A live audio webcast of the presentation will be available through the Investors section of Geron’s website (www.geron.com) under Events. Following the live presentation, the webcast will be archived and available for replay for a period of 30 days.

To attend the meeting in person, please send an email to Gitanjali Jain Ogawa at The Trout Group ([email protected]) for further information.

On December 6, 2017 AstraZeneca, along with Acerta Pharma, its haematology research and development centre of excellence, and MedImmune, its global biologics research and development arm, will highlight significant progress in blood cancer research at the 59th 2017 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exhibition in Atlanta, USA (Press release, AstraZeneca, DEC 6, 2017, View Source [SID1234522396]). Presentations will include new data from AstraZeneca’s emerging haematology portfolio in several cancer types including mantle cell lymphoma (MCL), chronic lymphocytic leukaemia (CLL), hairy cell leukaemia (HCL), acute myeloid leukaemia (AML), multiple myeloma and diffuse large B-cell lymphoma (DLBCL).

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Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "Following the recent accelerated approval of AstraZeneca’s first medicine for a blood cancer, Calquence, we will share a broad range of new data at ASH (Free ASH Whitepaper) highlighting our scientific progress in haematology as we seek to develop potential medicines that advance patient care."

Efficacy and safety of Calquence in the management of previously-treated MCL

Following the US Food and Drug Administration (FDA) accelerated approval of Calquence (acalabrutinib), a kinase inhibitor indicated for the treatment of adult patients with MCL who have received at least one prior therapy, data from the pivotal Phase II ACE-LY-004 clinical trial on which the accelerated approval was based, will be presented for the first time (Abstract #155). New details of the trial will be shared, including median time to response, pre-specified patient subgroup efficacy analyses, as well as safety analyses, further characterising the clinical profile of Calquence in this patient population.

Calquence as monotherapy and in combination in multiple CLL patient populations

Results will be presented from the Phase Ib/II ACE-CL-003 trial evaluating Calquence and obinutuzumab in treatment-naïve and previously-treated CLL patients (Abstract #432), which highlight the safety profile and activity of the combination. Long-term follow-up safety and efficacy data from the Phase I/II ACE-CL-001 clinical trial which tested Calquence as a monotherapy in a large cohort of patients with relapsed or refractory CLL (Abstract #498) will expand on findings previously reported; these data will highlight the favourable duration of response in this patient population.

Early-stage haematology portfolio

AstraZeneca will present additional data from its haematology portfolio, including findings from a Phase I trial of moxetumomab pasudotox, an anti-CD22 recombinant immunotoxin and potential new medicine in development for the treatment of people with previously-treated HCL (Abstract: #2765)
Early data on AZD2811, a novel nanoparticle inhibitor of aurora kinase B being tested in AML (Abstract #1368)
Preclinical data from trials on MEDI2228, a BCMA-targeting pyrrolobenzodiazepine-linked antibody drug conjugate being tested in multiple myeloma (Abstract #3153)
Data from a trial of vistusertib (AZD2014), a dual mTORC1/2 inhibitor being tested in DLBCL (Abstract #4113).
NOTES TO EDITORS

A full list of company-sponsored abstracts to be presented at ASH (Free ASH Whitepaper) are as follows:

Abstract Number
Title
Presentation Details
Abstract #155
Efficacy and safety of acalabrutinib monotherapy in patients with relapsed/refractory mantle cell lymphoma in the Phase II ACE-LY-004 study
Oral session, Saturday, December 9, 1 p.m. EST

Location: Georgia World Congress Center, Building A, Level 4, A411-A412
Abstract #1741
Pharmacodynamic evaluation of acalabrutinib in relapsed/refractory and treatment-naive patients with chronic lymphocytic leukemia in the Phase I/II ACE-CL-001 study
Poster sessions, Saturday, December 9, 5:30-7:30 p.m. EST

Location: Georgia World Congress Center, Building A, Level 1, Hall A2
Abstract #1268
Exposure-response of the Bruton tyrosine kinase inhibitor, acalabrutinib in the treatment of hematologic malignancies
Abstract: #1243
Concurrent treatment with Pim kinase inhibitor downregulates alternative non-homologous end-joining repair and decreases genomic instability in FLT3-ITD cells treated with topoisomerase 2 inhibitors
Abstract #1368
Preclinical and early Phase I clinical data of AZD2811 nanoparticle in AML, an aurora B kinase inhibitor
Abstract #432
Acalabrutinib with obinutuzumab in relapsed/refractory and treatment-naive patients with chronic lymphocytic leukemia: The Phase Ib/II ACE-CL-003 study
Oral session, Sunday, December 10, 1:15 p.m. EST

Location: Georgia World Congress Center, Building B, Level 5, Murphy BR 3-4
Abstract #498
Acalabrutinib monotherapy in patients with relapsed/refractory chronic lymphocytic leukemia: Updated results from the Phase I/II ACE-CL-001 study
Oral session, Sunday, December 10, 5:45 p.m. EST

Location: Georgia World Congress Center, Building B, Level 5, Murphy BR 3-4
Abstract: #2765
Negative minimal residual disease associated with extended response to moxetumomab pasudotox in patients with relapsed/refractory hairy cell leukemia: Long-term follow-up of bone marrow immunohistochemistry analyses from a Phase I study
Poster Session, Sunday, December 10, 6-8 p.m. EST

Location: Georgia World Congress Center, Building A, Level 1, Hall A2
Abstract: #3153
Preclinical evaluation of MEDI2228, a BCMA-targeting pyrrolobenzodiazepine-linked antibody drug conjugate for the treatment of multiple myeloma
Abstract #3442
Adverse events, resource use, and economic burden in patients with mantle cell lymphoma in the United States
Abstract #4326
Pooled analysis of safety data from clinical trials evaluating acalabrutinib monotherapy in hematologic malignancies
Poster sessions, Monday, December 11, 6-8 p.m. EST

Location: Georgia World Congress Center, Building A, Level 1, Hall A2

Abstract #4060
Understanding ibrutinib treatment discontinuation patterns for chronic lymphocytic leukaemia
Abstract #4684
MCL treatment patterns and outcomes: A community oncology practice experience
Abstract #4113
Combined inhibition of mTOR and BTK signaling is required for optimal long-term growth inhibition in DLBCL models
About Calquence

Calquence (acalabrutinib; previously known as ACP-196) is a selective inhibitor of BTK. Calquence binds covalently to BTK, irreversibly inhibiting its activity, and has demonstrated this with minimal interactions with other immune cells in pre-clinical studies.5 In B cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion.1

The recommended dose of Calquence is one 100mg capsule taken orally approximately every twelve hours until disease progression or unacceptable toxicity.1 Calquence may be taken with or without food.1

Calquence is also in development for the treatment of multiple B-cell malignancies and other cancers including 1st-line MCL, chronic lymphocytic leukaemia (CLL), Waldenström macroglobulinemia (WM), follicular lymphoma, diffuse large B-cell lymphoma, and multiple myeloma. It is also being developed as a monotherapy and in combination trials for solid tumours. More than 35 clinical trials across 40 countries with more than 2,500 patients are underway or have been completed.

Calquence was granted Orphan Drug Designation by the US FDA for the treatment of adult patients with MCL in September 2015 and by the European Commission in March 2016 for the treatment of adult patients with CLL, MCL and WM.

About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly-growing portfolio of new medicines that have the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advance New Oncology as one of AstraZeneca’s five Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On December 6, 2017 Foundation Medicine, Inc. (NASDAQ:FMI) reported that new data generated with FoundationOneHeme, its comprehensive genomic profiling (CGP) assay for hematologic malignancies and sarcomas, will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (Press release, Foundation Medicine, DEC 6, 2017, View Source [SID1234522397]). Data from a broad range of blood cancers, including acute myeloid leukemia (AML), myeloproliferative neoplasms (MPN), and non-Hodgkin lymphoma (NHL), including primary central nervous system lymphoma (PCL), demonstrate the value of integrating FoundationOneHeme into clinical care. The data presented is expected to demonstrate the potential for CGP to improve disease classification, to offer personalized prognostic information and to support therapeutic treatment decision making by informing treating physicians about the use of novel treatment options, including cancer immunotherapies.

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"Comprehensive genomic profiling (CGP) is proving to be an essential component for personalized cancer care, particularly as we learn more about the diverse genomic alterations in blood cancers," said Vincent Miller, M.D., chief medical officer at Foundation Medicine. "FoundationOneHeme is at the forefront of helping to identify new treatment approaches, including cancer immunotherapy, for individuals with blood cancers. Our new findings to be presented at ASH (Free ASH Whitepaper) support the ability of CGP to address high unmet medical needs across a wide range of hematologic malignancies, including rare conditions for which there are few treatment options."

Cancer immunotherapy is emerging as a therapeutic tool for patients with diverse hematologic malignancies. New results to be presented at ASH (Free ASH Whitepaper) using FoundationOneHeme show that more than one-quarter of patients with PCL had high tumor mutational burden (TMB), a genomic biomarker that has been shown across several cancer types to predict response to immuno-oncology treatment strategies, such as checkpoint inhibitors. Less than 50 percent of PCL patients achieve complete remission with current standard of care treatments, underscoring a need for new treatment options for these patients.

New results also support the role of FoundationOneHeme to guide therapy selection and predict response to treatment. In an oral study to be presented, FoundationOneHeme detected NTRK fusions in a wide variety of hematologic malignancies, and clinical response to a TRK inhibitor was subsequently observed in a patient with refractory AML and an ETV6-NTRK2 fusion. In another study to be presented, CGP offered insights that may facilitate risk-adapted clinical management decisions in patients with intermediate-and favorable-risk AML, potentially informing optimal use of autologous stem cell transplantation (auto-SCT) over conventional consolidation chemotherapy. For example, the study showed the presence of PTPN11 mutations to predict long term clinical outcomes following an auto-SCT.

Other results to be presented at ASH (Free ASH Whitepaper) demonstrate the ability of FoundationOneHeme to detect both known and novel BCL6 rearrangements in NHL, including cases that previously tested negative with standard-of-care single marker testing. BCL6 rearrangements have known diagnostic and prognostic utility in specific subtypes of NHL.

Foundation Medicine and collaborators will present a total of nine studies, including four oral presentations and five poster presentations, at the ASH (Free ASH Whitepaper) Annual Meeting from December 9-12, 2017 in Atlanta. The planned presentations are as follows:

Dec 9:

115 – Unrecognized clonal hematopoiesis of indeterminate potential in solid tumors: Implications for interpretation of molecular testing, Dec 9, 9:30am, C208-210 (Oral Presentation)
1423 – The role of comprehensive mutational profiling in predicting patients who may benefit from autologous hematopoietic cell transplant for acute myeloid leukemia, Dec 9, 5:30pm-7:30pm, Hall A2 (Poster Presentation)
1657 – A distinct mutation profile might contribute to the dismal outcome of triple negative patients with primary myelofibrosis, Dec 9, 5:30pm-7:30pm, Hall A2 (Poster Presentation)
Dec 10:

417 – Comprehensive genomic profiling identifies novel BCL6 rearrangements in diverse subtypes of Non-Hodgkin lymphoma as well as known rearrangements not detected using standard of care assays, Dec 10, 12:30pm, Marcus Auditorium (Oral Presentation)
476 – Comprehensive genomic profiling identifies genomic alterations that define Philadelphia-like B-acute lymphoblastic leukemia, Dec 10, 4:45pm, B213-B214 (Oral Presentation)
Dec 11:

794 – Characterization of NTRK fusions and therapeutic response to NTRK inhibition in hematologic malignancies, Dec 11, 4:45pm, B207-208 (Oral Presentation)
3800 – Recurrent copy number variants are highly prevalent in acute myeloid leukemia, Dec 11, 6:00pm-8:00pm, Hall A2 (Poster Presentation)
3996 – Comprehensive genomic profiling demonstrates differences in primary CNS lymphoma and systemic diffuse large B cell lymphoma and reveals biomarkers indicating potential benefit from immune checkpoint inhibitors, Dec 11, 6:00pm-8:00pm, Hall A2 (Poster Presentation)
4016 – Next generation sequencing of Castleman disease and follicular dendritic cell sarcomas associated with Castleman disease, Dec 11, 6:00pm-8:00pm, Hall A2 (Poster Presentation)

On December 6, 2017 Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) reported that it will have two presentations at the 59th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting being held Dec. 9-12, 2017 in Atlanta (Press release, ProMetic Life Sciences, DEC 6, 2017, View Source [SID1234522398]).

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An oral presentation, entitled, "Pivotal Trial with Intravenous Plasminogen Replacement in Patients with Plasminogen Deficiency Demonstrates Long-Term Efficacy for Treatment and Prevention of Ligneous Lesions" will be presented by Dr. Charles T. Nakar, Indiana Hemophilia and Thrombosis Center. During the oral session, Dr. Nakar will present 48-week data demonstrating the long-term safety and efficacy of intravenous plasminogen replacement (RyplazimTM) in patients with plasminogen deficiency.

A poster presentation, entitled, "Computer Modeling Using Historical Data Demonstrates a Significant Reduction in Expected Extravascular Fibrinous Lesions Due to Congenital Plasminogen Deficiency While Receiving Intravenous Plasminogen Replacement" will be presented by Dr. Joseph M. Parker, Senior Director of clinical development of Prometic. Dr. Parker will present a poster of historical data computer modeling highlighting the significant treatment effect of RyplazimTM in reducing the extravascular ligneous lesions in pediatric and adult subjects with plasminogen deficiency.

"Data from these presentations demonstrates the remarkable safety and efficacy profile of plasminogen treatment we have observed to date," said Pierre Laurin, Chief Executive Officer at Prometic. "No currently-available treatment options have demonstrated a complete resolution of lesions in patients with plasminogen deficiency. We are continuing to work closely with the FDA with the goal of making our plasminogen replacement therapy available to patients as soon as possible."

Details of the oral presentation are as follows:

Presentation Title: Pivotal Trial with Intravenous Plasminogen Replacement in Patients with Plasminogen Deficiency Demonstrates Long-Term Efficacy for Treatment and Prevention of Ligneous Lesions

Presenter: Charles T. Nakar, M.D.

Session Title: Disorders of Coagulation or Fibrinolysis: Novel Therapies and Clinical Trials in Bleeding Disorders
Date/Time: Saturday, December 9, 2017 at 9:30 a.m. EST
Room: Georgia World Congress Center, Bldg B, Lvl 2, B207-B208

Details of the poster presentation are as follows:

Presentation Title: Computer Modeling Using Historical Data Demonstrates a Significant Reduction in Expected Extravascular Fibrinous Lesions Due to Congenital Plasminogen Deficiency While Receiving Intravenous Plasminogen (PLG) Replacement

Presenter: Joseph M. Parker, M.D.
Session Title: Disorders of Coagulation or Fibrinolysis: Poster II
Date/Time: Sunday, December 10, 2017 from 6:00 p.m. to 8:00 p.m. EST
Room: Georgia World Congress Center, Bldg A, Lvl 1, Hall A2