On May 25, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the presentation of three ZEJULA (niraparib) abstracts at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, June 2 to June 6, 2017, in Chicago (Press release, TESARO, MAY 25, 2017, View Source [SID1234519300]). In addition, TESARO will host an investor and analyst briefing in Chicago on Saturday, June 3 at 6:00 PM local time in conjunction with the ASCO (Free ASCO Whitepaper) Annual Meeting.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"We’re excited to see presentations of additional data from the landmark ENGOT-OV16/NOVA trial presented at this year’s ASCO (Free ASCO Whitepaper) Annual Meeting," said Mary Lynne Hedley, Ph.D., President and COO, TESARO. "We also look forward to sharing initial data from the TOPACIO trial of niraparib plus pembrolizumab, as well as data from the Phase 1 trial of TSR-042, our anti-PD-1 antibody, at our ASCO (Free ASCO Whitepaper) investor briefing."

Please plan to visit TESARO at Booth #18097 to learn more about ZEJULA (niraparib), VARUBI (rolapitant) and our immuno-oncology pipeline.

Presentation Details:

Saturday, June 3, 2017, 1:15 PM to 4:45PM
Efficacy of Niraparib on Progression-free Survival (PFS) in Patients (Pts) with Recurrent Ovarian Cancer (OC) with Partial Response to the Last Platinum-based Chemotherapy
Abstract #5517, Poster Board #339, Location: Hall A
Poster Discussion: Saturday, June 3, 2017, 4:45 PM to 6:00 PM, Location: Aerie Crown Theater

Saturday, June 3, 2017, 1:15 PM to 4:45PM
Long-Term Benefit of Niraparib Treatment of Recurrent Ovarian Cancer (OC)
Abstract #5534, Poster Board #356, Location: Hall A

Saturday, June 3, 2017, 1:15 PM to 4:45PM
The Successful Phase 3 Niraparib ENGOT-OV16/NOVA Trial Included a Substantial Number of Patients with Platinum Resistant Ovarian Cancer
Abstract #5560, Poster Board #382, Location: Hall A

On May 25, 2017 GlycoMimetics, Inc. (NASDAQ: GLYC) reported that the European Commission, based on a favorable recommendation from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products, has granted orphan designation for the company’s drug candidate GMI-1271 for the treatment of acute myeloid leukemia (AML) (Press release, GlycoMimetics, MAY 25, 2017, View Source [SID1234519296]). The U.S. Food and Drug Administration (FDA) previously granted orphan drug designation for GMI-1271 for the treatment of AML in May of 2015.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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GMI-1271, a specific E-selectin inhibitor is being evaluated in the company’s ongoing Phase 1/2 clinical trial, in which clinicians are evaluating the use of GMI-1271 along with chemotherapy in patients with relapsed or refractory AML as well as those with newly diagnosed AML. Earlier this month, the company announced that GMI-1271 had been granted Breakthrough Therapy designation by the FDA. The company also announced that abstracts had been published by both the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and the European Hematology Association (EHA) (Free EHA Whitepaper) highlighting new data from the Phase 2 portion of the company’s ongoing Phase 1/2 trial that will be presented at their upcoming annual meetings in June.

"The European orphan designation will provide incentives for the commercialization and development of GMI-1271 in AML, where there are limited therapies available to patients," said Helen Thackray, M.D., FAAP, Senior Vice President, Clinical Development and Chief Medical Officer of GlycoMimetics. "We believe that GMI-1271, when combined with standard chemotherapy, has the potential to address an unmet therapeutic need for individuals living with AML, and we are encouraged by both our clinical results to date and achieving this designation from the European Commission."

The European Commission grants orphan designation to drugs intended to treat, prevent or diagnose life-threatening or chronically debilitating rare disorders, defined as diseases with prevalence of no more than 5 in 10,000 in the EU, for which no satisfactory method of diagnosis, prevention or treatment yet exists. Orphan designation provides benefits including commercialization incentives, protection of intellectual property, including 10 years of market exclusivity and protocol assistance through the EMA’s Scientific Advice program.

About AML

AML is a cancer of the blood and bone marrow. AML is the most common type of acute leukemia in adults. The National Cancer Institute estimates that there will be over 21,000 new cases of AML diagnosed in 2017 in the United States, and over 10,000 people will die from all forms of the disease in 2017. AML is more commonly present in elderly patients. Unlike other cancers that start in an organ and spread to the bone marrow, AML is known for rapid growth of abnormal white blood cells that gather in the bone marrow, getting in the way of normal blood cell production. The lack of normal blood cells can cause some of the symptoms of AML, including anemia (shortage of red blood cells resulting in tiredness and weakness), neutropenia (shortage of white blood cells that may lead to increased infections), and thrombocytopenia (shortage of platelets in the blood that may lead to excessive bleeding). Current treatment options for AML consist of reducing and eliminating cancer cells mainly through chemotherapy, radiation therapy, and stem cell transplantation.

On May 25, 2017 X4 Pharmaceuticals, a clinical stage biotechnology company developing novel CXCR4 inhibitor drugs to improve immune cell trafficking to treat cancer and rare diseases, reported dosing of the first patient in the Phase 2 portion of the ongoing Phase 1/2 study evaluating X4P-001, the company’s lead CXCR4 inhibitor, in combination with Inlyta (axitinib) in patients with advanced clear cell renal cell carcinoma (ccRCC) (Press release, X4 Pharmaceuticals, MAY 25, 2017, View Source [SID1234519294]). Full results from the Phase 1 portion of the study including the selection of the once daily oral dose used in the expansion cohort will be presented at an upcoming medical meeting.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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In addition to safety and tolerability, the Phase 2 portion of the study will evaluate clinical efficacy as measured by objective response rate (ORR), duration of response (DOR), and progression free survival (PFS) and will explore the correlation of biomarkers with efficacy. Multiple cancer centers with leading renal cell carcinoma researchers in the U.S. and South Korea are participating in the study.

"Efficiently progressing this study into Phase 2 is an important milestone for the development of X4P-001 and the CXCR4-targeted therapeutic approach," said Sudha Parasuraman, MD, Chief Medical Officer of X4. "Having established the Phase 2 combination dose of X4P-001, we are now focused on augmenting proof of concept data for this critically important biological axis known to play a key role in immune cell trafficking."

In addition to this Phase 1/2 study of X4P-001 in combination with Inlyta, a VEGF kinase inhibitor, X4 has additional oncology clinical studies ongoing, including a Phase 1/2 study in patients with advanced ccRCC to evaluate X4P-001 in combination with Opdivo (nivolumab), and a Phase 1b biomarker study in patients with advanced melanoma to evaluate X4P-001 in combination with Keytruda (pembrolizumab).

About X4P-001 in Cancer

X4P-001, the company’s lead drug candidate, is currently in Phase 1/2 testing in refractory clear cell renal cell carcinoma (ccRCC) and other solid tumor indications. Based on promising preclinical studies, X4P-001 is being evaluated in clinical studies in combination with approved cancer therapies, including tyrosine kinase inhibitors and checkpoint inhibitors. X4P-001 is an oral, small molecule inhibitor of CXCR4, or C-X-C receptor type 4, the receptor for the chemokine CXCL12. Recent studies demonstrate that CXCR4/CXCL12 is a primary receptor-ligand pair that cancer cells and surrounding stromal cells use to block normal immune function and promote angiogenesis through the trafficking of T-effector and T-regulatory cells, as well as myeloid derived suppressor cells (MDSCs), in the tumor microenvironment.1,2

About Renal Cell Carcinoma

Kidney cancer is among the ten most common cancers in both men and women with more than 60,000 new diagnoses each year in the United States.3 Clear cell renal cell carcinoma (ccRCC) is the most common form of kidney cancer, and advanced ccRCC accounts for approximately 20% of the patient population. Therapies for advanced ccRCC include immunotherapies, mammalian target of rapamycin (mTOR) kinase inhibitors, and angiogenesis inhibitors, such as vascular endothelial growth factor (VEGF) inhibitors.4 There continue to be unmet medical needs with advanced ccRCC because durable responses remain a serious clinical challenge for patients with advanced disease.

On May 25, 2017 MabVax Therapeutics Holdings, Inc. (Nasdaq: MBVX), a clinical-stage biotechnology company with a fully human antibody discovery platform focused on the rapid translation into clinical development of products to address unmet medical needs in the treatment of cancer, reported that results from the Phase I clinical trial of MabVax’s therapeutic antibody MVT-5873, being evaluated in advanced pancreatic cancer and other CA19-9 positive cancers, will be presented in a poster presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting to be held in Chicago, IL, June 2 – 6, 2017 (Press release, MabVax, MAY 25, 2017, View Source [SID1234519293]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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MabVax Therapeutics Logo (PRNewsfoto/MabVax Therapeutics Holdings, I)

As part of the presentation, MabVax will discuss the results of the Company’s dose-escalation and safety trial, conducted over the last year in 32 patients with advanced pancreatic and colon cancer. Additionally, single agent MVT-5873 safety profile, efficacy, and reductions in serum CA19-9 levels over time will be presented.

Title of Presentation: Single agent HuMab-5B1 (MVT-5873), a monoclonal antibody targeting sLea, in patients with pancreatic cancer and other CA19-9 positive malignancies.
Abstract Number: 4110
Location: Gastrointestinal (Non-colorectal) Cancer Session
Session Date: Saturday, June 3rd
Time: 8:00 AM to 11:30 AM (CDT)

The Phase I therapeutic trial is an open-label, dose-escalation study evaluating the safety, tolerability and pharmacokinetics of MVT-5873. The first group of patients enrolled assess safety and determined the recommended Phase II dose of the antibody. The second patient group will establish the safety and dose of the antibody when administered with a standard-of-care chemotherapy. Dr. Eileen O’Reilly, associate Director of the David M. Rubenstein Center for Pancreatic Cancer Research, attending physician, member at Memorial Sloan Kettering Cancer Center and Professor of Medicine at Weill Cornell Medical College is the lead investigator in the MVT-5873 Phase I clinical trial.

On May 25, 2017 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported the highlights of numerous advances in precision oncology and cancer immunotherapy using the nCounter platform that will be presented at the 53rd Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (Press release, NanoString Technologies, MAY 25, 2017, View Source [SID1234519292]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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"The breadth of nCounter-based research at this year’s ASCO (Free ASCO Whitepaper) conference demonstrates the scientific momentum and significant commercial advances that we’ve made in our key markets, most notably immuno-oncology," said Brad Gray, president and chief executive officer of NanoString. "In addition, our new Digital Spatial Profiling technology continues to demonstrate its unique value in characterizing the tumor microenvironment to inform decisions in drug development programs."

More than 35 abstracts will be presented at the ASCO (Free ASCO Whitepaper) Annual Meeting, which is being held June 2nd through June 6th, 2017 in Chicago, Illinois. The research being presented spans a wide breadth of applications including Targeted Discovery, Biomarker Development and Clinical Practice across multiple oncology indications, including the following.

Targeted Discovery
Multidimensional spatial characterization of the tumor microenvironment (TME) in synchronous melanoma metastases (SMM) to yield insights into mixed responses to therapy in metastatic melanoma (MM) patients (pts). (Abstract # 9575)
NanoString’s new Digital Spatial Profiling research platform was used for spatial characterization of 30 immune markers and signaling proteins in melanoma patient samples to understand correlations and determinants of response.

Molecular characterization of immune-related severe adverse events (irSAE). (Abstract #3076)
NanoString Digital Spatial Profiling was used to profile the immune cell population in tissue affected by immune checkpoint inhibitors-mediated inflammation. Similarities and differences between autoimmune disease and colon-irSAEs were identified at the gene expression and proteomic levels, as regions of inflammation showed higher CD68 and PD-L1 positivity in colon-irSAE specimens versus normal colon or Crohn’s specimens, and reduced beta-catenin levels in both Crohn’s and colon-irSAE specimens relative to normal controls.

Distinct gene expression, mutational profile and clinical outcomes of V600E and V600K/R BRAF-mutant metastatic melanoma (MM). (Abstract #9541)
Gene expression and mutational profiling were used to investigate potential mechanisms explaining the observation that V600K/R metastatic melanoma has inferior response and shorter survival with MAPKi than V600E.

Biomarker Development
Correlation of constitutive PD-1 resistance in HNC with GM-CSF expression and presence of myeloid derived suppressor cells (MDSCs). (Abstract #6049)
A 638-gene immune gene expression panel was used to explore why the majority of INF-G inflamed head and neck squamous cell carcinomas (HNC) tumors do not respond to PD-1 checkpoint blockade. Constitutive resistance to PD-1 checkpoint blockade in inflamed HNC associates with expression of GM-CSF and Myeloid Derived Suppressor Cell (MDSC) markers. Strategies to deplete MDSCs, such as chemotherapy, should be considered in combination or sequentially with anti-PD-1.

Cellular immune biomarkers to prognosticate for survival to adoptive T-cell therapy in advanced nasopharyngeal cancer. (Abstract #6047)
nCounter platform and reagents were used for longitudinal modular transcriptome analysis of PBMC from patients with stage 4c nasopharyngeal carcinoma who received first line chemo-immunotherapy with the aim of identifying signatures associated with positive clinical outcomes.
Results from this study showed that 2-year survivors displayed significant decreased amounts of monocytic myeloid-derived suppressor cells (mMDSCs), compared to non-survivors.

Phase II study of durvalumab (anti-PD-L1 antibody) in combination with R-CHOP or lenalidomide plus R-CHOP in previously untreated, high-risk diffuse large B-cell lymphoma. (Abstract # TPS7573)
NanoString’s Lymphoma Subtyping Test is being used to determine Cell of Origin in an ongoing clinical trial. The primary study objective is to explore the clinical activity of durvalumab with R-CHOP in non-activated B-cell-like (non-ABC) and durvalumab with lenalidomide + R-CHOP (R2-CHOP) in ABC previously untreated DLBCL; secondary objectives are to evaluate safety and identify biomarkers predictive of clinical response.

Association of molecular subtype, proliferation, and immune genes with efficacy of carboplatin versus gemcitabine addition to taxane-based, anthracycline-free neoadjuvant chemotherapy in early triple-negative breast cancer (TNBC): Results of the randomized WSG ADAPT-TN trial. (Abstract #573)
NanoString assays combining the PAM50 breast cancer assay and immune profiling were used to characterize patients in a clinical trial. In early TNBC, basal-like subtype, higher Ki67 (by IHC), and lower HER-2 score were associated with chemo-sensitivity for both neoadjuvant arms. Chemo-resistance pathways differed between the two taxane-based combinations. The positive predictive impact of immunological genes in the nab-pac – carbo arm could influence optimal patient selection for immune-modulative therapy.

Impact of consensus molecular subtyping (CMS) on overall survival (OS) and progression free survival (PFS) in patients (pts) with metastatic colorectal cancer (mCRC): Analysis of CALGB/SWOG 80405 (Alliance). (Abstract #3511)

CALGB 80405 was a randomized Ph3 trial showing no OS or PFS difference in mCRC pts treated with Bevacizumab (Bev) or Cetuximab (Cet) in the first line. A Nanostring platform was used to determine the CMS classification of 392 KRAS wt (codon 12 and 13) primary tumors and correlated it with OS and PFS in patients enrolled in 80405. Data suggest that CMS is associated with OS and PFS in first line therapy in mCRC patients. Preliminary data suggest that certain CMS may be associated with efficacy of Bev and Cet based chemotherapy.

Clinical Practice
Impact of the Prosigna (PAM50) assay on adjuvant clinical decision making in patients with early stage breast cancer: Results of a prospective multicenter public program. (Abstract # e12062)

In this prospective decision impact study, Prosigna results led to a 39% change in adjuvant therapy indication. Patients with initial indication of CHT were changed to HT alone in > 50% of cases. Thus, Prosigna results influenced the treatment decisions and reinforced its clinical utility in real-world settings. The intrinsic subtype classification based on IHC didn’t show to be an adequate surrogate for the genomic subtypes as determined by Prosigna.

At the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting, NanoString will showcase its nCounter platform, Digital Spatial Profiling and 3D Biology capabilities at booth #20097.



Abstract # Summary Hyperlink

e23198 Molecular classification with NanoString nCounter system in triple-negative breast cancer. View Source

e23103 Molecular sequencing and gene fusion detection in non-small cell lung cancer (NSCLC) patients:
Impact of co-existing alterations. View Source


3076 Molecular characterization of immune-related severe adverse events (irSAE). View Source

8015 Pembrolizumab (Pembro) plus lenalidomide (Len) and low-dose dexamethasone (Dex)
for relapsed/refractory multiple myeloma (RRMM): Efficacy and biomarker analyses. View Source


e21030 Immune cell profiling of melanoma metastases from patients treated with TriMixDC-MEL
dendritic cell therapy in combination with ipilimumab. View Source


3509 Clinical utility of colon cancer molecular subtypes: Validation of two main colorectal molecular
classifications on the PETACC-8 phase III trial cohort. View Source


530 Effects of age, immune landscape, and response to trastuzumab (H) in HER-2 positive (HER2+)
breast cancer in NCCTG (Alliance)-N9831. View Source


TPS7573 Phase II study of durvalumab (anti-PD-L1 antibody) in combination with R-CHOP or lenalidomide
plus R-CHOP in previously untreated, high-risk diffuse large B-cell lymphoma. View Source


E12062 Impact of the Prosigna (PAM50) assay on adjuvant clinical decision making in patients with early
stage breast cancer: Results of a prospective multicenter public program. View Source

7512 Clinical and biologic covariates of outcomes in ZUMA-1: A pivotal trial of axicabtagene ciloleucel
(axi-cel; KTE-C19) in patients with refractory aggressive non-Hodgkin lymphoma (r-NHL). View Source

9575 Multidimensional spatial characterization of the tumor microenvironment (TME) in synchronous
melanoma metastases (SMM) to yield insights into mixed responses to therapy in metastatic melanoma
(MM) patients (pts). View Source

e14614 Intra-tumour heterogeneity in the regulation of immune-tolerogenic pathways in primary and metastatic
hepatocellular carcinoma (HCC). View Source

e13052 Molecular profiling of cancer outliers. View Source

e12134 Immune biomarkers and treatment (tx) outcome in hormone receptor-positive (HR+) breast cancer
(BC) patients (pts) treated with preoperative chemotherapy (preop chemo) plus bevacizumab (bev). View Source

3511 Impact of consensus molecular subtyping (CMS) on overall survival (OS) and progression free survival
(PFS) in patients (pts) with metastatic colorectal cancer (mCRC): Analysis of CALGB/SWOG 80405
(Alliance). View Source

e12134 Different patterns of non immediate allergic reaction to BRAF inhibitor in two patients with metastatic
melanoma. View Source


e20028 Novel prognostic markers for epithelioid malignant pleural mesothelioma. View Source

e21052 Different patterns of non immediate allergic reaction to BRAF inhibitor in two patients with metastatic
melanoma. View Source

e23091 Effect of bavituximab in combination with nivolumab on tumor immune response in a 3D ex vivo
system of lung cancer patients. View Source

7547 Rapid, real-time central pathology review for E1412: A novel and successful paradigm for future
National Clinical Trials Network diffuse large B cell lymphoma studies. View Source

e20050 Prognostic gene signatures for lung adenocarcinoma using digital multiplexed gene expression in
formalin-fixed paraffin embedded tissue. View Source

9541 Distinct gene expression, mutational profile and clinical outcomes of V600E and V600K/R BRAF-mutant
metastatic melanoma (MM). View Source

5591 High-intermediate risk endometrial cancer: Can gene expression predict recurrence? View Source

6047 Cellular immune biomarkers to prognosticate for survival to adoptive T-cell therapy in advanced
nasopharyngeal cancer. View Source

e12541 Identification of differentially expressed genes associated with clinical response after treatment of
breast cancer skin metastases with imiquimod. View Source

10503 Molecular alterations to predict survival and response to chemotherapy of pediatric low-grade glioma. View Source

e23090 Anti-PD1 treatment to induce M1 polarization of tumor infiltrating macrophages in a 3D ex vivo system
of lung cancer patients. View Source

8557 Biomarkers of pembrolizumab (P) activity in mesothelioma (MM): Results from a phase II trial. View Source

6049 Correlation of constitutive PD-1 resistance in HNC with GM-CSF expression and presence of myeloid
derived suppressor cells (MDSCs). View Source

511 Seven-year (yr) follow-up of adjuvant paclitaxel (T) and trastuzumab (H) (APT trial) for node-negative,
HER2-positive breast cancer (BC). View Source

529 Impact of DNA repair deficiency signature on outcomes in triple negative breast cancer (TNBC) patients
treated with AC chemotherapy (SWOG S9313). View Source

e20610 Coexistence of rearranged during transfection (RET) variants and activating EGFR mutations with their
molecular implications in lung adenocarcinomas. View Source

e23205 In silico validation of a prostate cancer recurrence prognostic signature based on pathways related to
stem cells. View Source

e13090 Characterization of germline and tumor genomic profile in unselected young black breast cancer patients. View Source

8573 Pembrolizumab in patients with recurrent thymic carcinoma: Results of a phase II study. View Source

TPS594 CORALLEEN: A phase 2 clinical trial of chemotherapy or letrozole plus ribociclib as neoadjuvant treatment
for postmenopausal patients with luminal B/HER2-negative breast cancer. View Source


11553 CCL5 expression and tumor infiltrating immune cells in triple negative breast cancer. View Source

573 Association of molecular subtype, proliferation, and immune genes with efficacy of carboplatin versus
gemcitabine addition to taxane-based, anthracycline-free neoadjuvant chemotherapy in early triple-negative
breast cancer (TNBC): Results of the randomized WSG ADAPT-TN trial. View Source