On September 27, 2016 Biothera Pharmaceuticals, Inc. reported the presentation of clinical data demonstrating the mechanism of action of Imprime PGG, the Company’s Phase 2 cancer immunotherapy drug, in healthy human volunteers (Press release, Biothera, SEP 27, 2016, View Source [SID:SID1234515441]). These are the first data to show that when administered intravenously to healthy human subjects, Imprime PGG drives the immunopharmacodynamic (IPD) responses observed in ex vivo human and in vivo mouse studies. Specifically, the study demonstrated that formation of an immune complex between Imprime PGG and endogenous anti-beta glucan antibodies (ABA) was critical to elicit Imprime-induced immune activating events in humans. These findings provide additional evidence that ABA levels may be a particularly useful biomarker for selecting patients most likely to respond to Imprime-based therapy. These data, as well as additional preclinical research demonstrating Imprime’s ability to reshape the suppressive immune microenvironment of the tumor and elicit robust anti-tumor immunity, were presented in poster presentations during the CRI-CIMT-EATI-AACR International Cancer Immunotherapy Conference (CIMT) (Free CIMT Whitepaper), taking place in New York City, September 25-28, 2016.

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"For the first time we are showing that Imprime PGG elicits multiple innate immune-activating events after systemic dosing in humans. These immune-activating events are the same events necessary for efficacy in preclinical models," said Jeremy Graff, Ph.D., Biothera’s Chief Scientific Officer and Senior Vice President, Research. "These data also further bolster our extensive research to show that pre-treatment ABA levels represent a viable, non-invasive, mechanism-based patient selection biomarker that will be deployed in our upcoming clinical trials."

Imprime PGG is a Pathogen Associated Molecular Patterning (PAMP) molecule that acts therapeutically as an immunological "ignition switch" to enlist the functionality of the innate immune system and to enhance the efficacy of tumor targeting, anti-angiogenic, and immune checkpoint inhibitor antibodies. Biothera and Merck are advancing a phase 2 clinical research collaboration to evaluate Imprime PGG and Merck’s anti-PD-1 inhibitor KEYTRUDA (pembrolizumab) in patients with either advanced melanoma or metastatic triple negative breast cancer. Biothera and Merck are also collaborating with the Big Ten Cancer Research Consortium to commence an Imprime PGG plus KEYTRUDA Phase 1b/2 study in patients with non-small cell lung cancer.

In a second poster presentation at the conference, Biothera presented preclinical data further demonstrating the ability of Imprime PGG to re-orient the immunosuppressive tumor microenvironment, disabling the immunosuppression that shields cancer cells from immune attack. Imprime PGG promotes the differentiation of human myeloid-derived suppressor cells (MDSCs), significantly upregulating the expression of co-stimulatory molecules (iNOS, CD80, CD86) that drive anti-tumor activity and relieving MDSC-mediated inhibition of T Cell proliferation. Previous preclinical studies have reported that Imprime PGG also promotes repolarization of M2 macrophages to an anti-tumor, M1-like orientation while triggering maturation of critical antigen presenting cells (i.e. dendritic cells) to enable antigen-specific T cell expansion and the production of the potent anti-tumor cytokine interferon gamma. Collectively, these data show that Imprime PGG treatment can reshape the tumor microenvironment favoring robust anti-tumor immune responses.

On September 27, 2016 Galera Therapeutics, Inc., a clinical-stage biotechnology company developing new treatments for cancer patients, reported the presentation of final data, including one-year tumor control, from a Phase 1b/2a clinical trial of GC4419, an investigational drug candidate for the reduction of severe chemoradiation-induced oral mucositis (OM), in an oral presentation at the American Society of Radiation Oncology (ASTRO) Annual Meeting (Press release, Galera Therapeutics, SEP 27, 2016, View Source [SID:SID1234515442]). The OM efficacy data from this study in head and neck cancer patients were previously presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in June and demonstrated marked reductions in the incidence, severity and duration of severe OM when compared to historical experience.

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The new one-year tumor control follow-up data presented today in Boston, Massachusetts further shows that local and distant tumor control, progression-free survival and overall survival compared well with historical experience for patients receiving chemoradiation for head and neck cancers.

This Phase 1b/2a trial assessed the safety and pharmacokinetics of GC4419, administered intravenously prior to each dose of standard intensity modulated radiotherapy (IMRT) and cisplatin therapy, in 46 head and neck cancer patients. The study demonstrated that, compared to historic controls, GC4419 delayed the onset, reduced the incidence, shortened the duration and reduced the intensity of severe OM (defined as WHO Grades 3 and 4 OM). Furthermore, only 4.3% of patients required breaks in IMRT of 5 consecutive fractions or more, as opposed to published rates of 15% in historical studies. In combination, GC4419 had a safety profile consistent with the underlying IMRT and cisplatin regimen.

Now with a full one year of follow-up in all consenting patients (44/46), investigators further report that the overall 1-year local-regional control, the 1-year distant metastasis-free rate and the 1-year overall survival were each 93%. The 1-year progression-free survival was 84%. These rates compare favorably with historic controls.

"We are encouraged to see that after one year of follow-up, tumor control in these patients appears to be maintained," said J. Mel Sorensen, MD, President and CEO of Galera. "These findings support the continuing development of GC4419, now in a randomized double-blinded Phase 2 trial for this patient population. As radiation oncologists know well, severe OM is a debilitating side effect that can result in treatment interruptions of potentially life-saving chemoradiation therapy. We look forward to advancing our clinical development program for GC4419, as well as our pipeline of orally active dismutase mimetics."

About Oral Mucositis (OM)
Oral mucositis is a common debilitating side effect of radiation treatment in head and neck cancer (HNC) patients. Severe OM, defined as Grade 3 or 4 OM on the World Health Organization Oral Mucositis Scale, occurs in 60 to 80 percent of HNC patients who receive radiation therapy. Importantly, severe OM may result in interruptions in radiation treatment, which can compromise the otherwise good prognosis for tumor control in many of these patients. In addition, patients suffer significant pain, may develop serious infections, and may be unable to eat solid food or even drink liquids. Further, the costs of managing these side effects are substantial, particularly when hospitalization and/or surgical placement of PEG tubes to maintain nutrition and hydration is required. There is currently no drug approved to prevent or treat severe OM in head and neck cancer patients.

About GC4419
GC4419 is a superoxide dismutase mimetic, a small molecule drug that is designed to convert superoxide to hydrogen peroxide and oxygen. This mechanism is thought to block the large burst of superoxide induced by radiotherapy, the initiating step in the development of OM, and has been shown to be protective of normal tissue but not tumor in preclinical models. In preliminary clinical studies, GC4419 markedly delayed the onset, shortened the duration and decreased the incidence of severe OM when administered intravenously prior to each dose of intensity modulated radiotherapy (IMRT) and cisplatin. GC4419 has now entered randomized Phase 2 development to reduce the incidence, severity and duration of severe OM in patients receiving radiation and chemotherapy for the treatment of head and neck cancer.

On September 27, 2016 Ipsen (Euronext: IPN; ADR: IPSEY), a global specialty-driven pharmaceutical group, reported that Cabometyx (cabozantinib), Somatuline Autogel (lanreotide) and telotristat ethyl (*previously known as telotristat etiprate) will be the subject of 16 presentations at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 congress:

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Cabozantinib to be featured in eight presentations

CABOSUN results accepted as late-breaker presentation in oral session at the Presidential Symposium
[LBA30] "CABOzantinib versus SUNitinib (CABOSUN) as initial targeted therapy for patients with metastatic renal cell carcinoma (mRCC) of poor and intermediate risk groups: Results from ALLIANCE A031203 Trial (Press release, Ipsen, SEP 27, 2016, View Source [SID:SID1234515444])."
Dr. Toni Choueiri, Director, Lank Center for Genitourinary Oncology, Dana-Farber Cancer Institute, Boston, Massachusetts, USA
Session: Presidential session 3
Oral presentation Monday, October 10, 4:30 – 6:10 p.m. CEST, Copenhagen
Note: This is a National Cancer Institute Cancer Therapy Evaluation Program (NCI-CTEP) study.

Poster Discussion
[774PD] "A phase I study of cabozantinib plus nivolumab (CaboNivo) in patients (pts) with refractory metastatic urothelial carcinoma (mUC) and other genitourinary (GU) tumors."
Dr. Andrea Borghese Apolo, Genitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 4:30 – 5:30 p.m. CEST, Athens
Note: This is an NCI-CTEP study.

Poster Presentations
[787P] "A phase II study of cabozantinib in patients (pts) with relapsed/refractory metastatic urothelial carcinoma (mUC)."
Dr. Rosa Nadal, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Maryland, USA
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 1 – 2 p.m. CEST, Hall E
Note: This is an NCI-CTEP study.

[814P] "Efficacy of cabozantinib (cabo) vs everolimus (eve) by metastatic site and tumor burden in patients (pts) with advanced renal cell carcinoma (RCC) in the phase 3 METEOR trial."
Dr. Thomas Powles, Barts Cancer Institute, Cancer Research UK Experimental Cancer Medicine Centre, Queen Mary University of London, Royal Free NHS Trust, London, GB
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 1 – 2 p.m. CEST, Hall E

[815P] "Evaluation of the novel "trial within a trial" design of METEOR, a randomized phase 3 trial of cabozantinib versus everolimus in patients (pts) with advanced renal cell carcinoma (RCC)."
Colin Hessel, Exelixis, Inc., South San Francisco, California, USA
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 1 – 2 p.m. CEST, Hall E

[816P] "Quality of life (QoL) in the phase 3 METEOR trial of cabozantinib vs everolimus for advanced renal cell carcinoma (RCC)."
Dr. David Cella, Department of Medical Social Sciences, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 1 – 2 p.m. CEST, Hall E

[818P] "Analysis of regional differences in the phase 3 METEOR study of cabozantinib (cabo) versus everolimus (eve) in advanced renal cell carcinoma (RCC)."
Dr. Nizar Tannir, University of Texas, MD Anderson Cancer Center, Houston, Texas, USA
Session: Genitourinary Tumours, Non-Prostate
Poster presented Sunday, October 9, 1 – 2 p.m. CEST, Hall E

[1421TiP] "A randomized double-blind phase II study evaluating the role of maintenance therapy with cabozantinib in high grade undifferentiated uterine sarcoma (HGUS) after stabilization or response to doxorubicin +/- ifosfamide following surgery or in metastatic first line treatment."
Dr. Isabelle Ray-Coquard, Cancer Research Center of Lyon, Lyon, France
Session: Basic science
Poster presented Monday, October 10, 1 – 2 p.m. CEST, Hall E
Note: This is an investigator-sponsored trial.

Lanreotide (Somatuline Autogel) will be featured in 6 presentations:

[438P] "Efficacy of lanreotide autogel/depot (LAN) vs placebo (PBO) for symptomatic control of carcinoid syndrome (CS) in neuroendocrine tumor (NET) patients from the ELECT study"
Dr Edward Wolin, Montefiore Einstein Center for Cancer Care, Bronx, NY, USA
Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1 – 2 p.m. CEST, Hall E

[440P] "Longer term efficacy of lanreotide autogel/depot (LAN) for symptomatic treatment of carcinoid syndrome (CS) in neuroendocrine tumor (NET) patients from the ELECT open label study"

Dr George Fisher. Stanford University School of Medicine, Stanford, CA, USA
Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1-2 p.m. CEST, Hall E

[439P] "Long-term safety/tolerability of lanreotide autogel/depot (LAN) treatment for metastatic intestinal and pancreatic neuroendocrine tumours (NETs): final results of the CLARINET open-label extension (OLE)"

Dr Martyn Caplin, Royal Free Hospital, London, UK
Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1 – 2 p.m. CEST, Hall E

[449TiP] "Safety and Efficacy of Lanreotide Autogel/Depot Every 14 Days for Patients with Pancreatic or Midgut Neuroendocrine Tumours Progressing on Lanreotide Every 28 Days: The Prospective, International CLARINET FORTE Study"

Dr Marianne Pavel. Charité University Medicine, Berlin, Germany
Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1 – 2 p.m. CEST, Hall E

[450TiP] "Safety of lanreotide 120 mg ATG in combination with metformin in patients with advanced well-differentiated gastro-intestinal (GI) or lung carcinoids. A pilot, one-arm, open-label, prospective study: The MetNET-2 trial"

F. De Braud et al.

Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1-2 p.m. CEST, Hall E

Note: This is a National Cancer Institute Milan sponsored study

[451TiP] "Combined Lanreotide Autogel and Temozolomide Therapy in Progressive Neuroendocrine Tumours: The SONNET Study"

Dr Dieter Hörsch, Zentralklinik Bad Berka, Bad Berka, Germany
Session: Endocrine and neuroendocrine tumours
Poster presented Saturday, October 8, 1 – 2 p.m. CEST, Hall E

Telotristat ethyl will be featured in one presentation:

[422PD] "Integrated placebo-controlled safety analysis from clinical studies of telotristat ethyl for the treatment of carcinoid syndrome"

Dr Matthew Kulke, Dana-Farber Cancer Institute, Boston, MA, USA
Session: Endocrine and neuroendocrine tumours
Poster discussion, Monday, October 10, 11-12 a.m. CEST, Room Berlin

In addition, Ipsen supported a collaborative study to understand the epidemiology of NET in European countries:

[424PD] "Prevalence of gastroenteropancreatic and lung neuroendocrine tumours in the European Union"

A. Bergamasco et al.
Session: Endocrine and neuroendocrine tumours
Poster discussion, Monday, October 10, 11-12 a.m. CEST, Room Berlin

Ipsen and Exelixis will host a joint investor / media event on October 10, 2016 at 6:30 p.m. (room 21, 1st floor, press area, Bella Center). Further information will follow with webcast and conference call details.

On September 26, 2016 MabVax Therapeutics Holdings, Inc. (NASDAQ: MBVX), a clinical-stage oncology drug development company, reported that its lead antibody development program, HuMab 5B1, was featured in three separate presentations at the recently held World Molecular Imaging Congress (WMIC) in September (Press release, MabVax, SEP 26, 2016, View Source [SID:SID1234515400]). The presentations were made by investigators from the Department of Radiology at Memorial Sloan Kettering Cancer Center (MSK) describing the novel use of MabVax’s lead antibody as a PET imaging agent and as a radioimmunotherapy agent targeting pancreatic and bladder cancer.

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Summaries of the investigator presentations and key points made are as follows:

"Utilizing antibody fragments for same-day pre-targeted immunoPET imaging in preclinical pancreatic cancer" – Investigators demonstrated that the MabVax HuMab-5B1 antibody can be successfully produced as a smaller fragment called a F(ab’)2 and coupled to multiple imaging agents without impacting immunoreactivity. This smaller fragment was coupled with Zirconium and Copper-based PET imaging agents, and a commonly used fluorescence imaging agent. All three constructs produced promising results for rapid immunoPET or immunofluorescent imaging. The potential advantage of using a smaller fragment is that imaging results could be obtained more rapidly and perhaps within the same day, giving physicians more real-time information and providing increased convenience for patients. This work was led by Jacob Houghton, Ph.D. of the Department of Radiology at MSK.
"Bioorthogonal click chemistry for the development of 225 Ac-radioimmunoconjugates and its application to pretargeting" – Investigators demonstrated that the MabVax HuMab-5B1 antibody was successfully conjugated to the radioimmunotherapy agent 225Actinium without losing immunoreactivity. Actinium has received increased interest among investigators as a potential therapeutic modality because of its alpha particle radiation has a limited range in tissue of a few cell diameters and a greater energy release for selectively killing cancer cells. This work was led by Sophie Poty, Ph.D. from the Department of Radiology and the Molecular Pharmacology Program at MSK.

"Tumor-specific PET Imaging in a Bladder Cancer Model" – The investigators demonstrated that the 89Zirconium-labeled HuMab-5B1 labeled antibody can specifically bind to xenograft tumors of human bladder cancer in animal models, potentially leading to use as an immunoPET radiotracer. There is a large unmet medical need for new treatments for bladder cancer, with an estimated 76,900 new cases each year. MabVax’s 89Zirconium-labeled HuMab-5B1 antibody (MVT-2163) is now in a phase I clinical trial for evaluation as a PET imaging agent for pancreatic cancer targeting the CA19.9 epitope, which is expressed on one-third to one-half of bladder cancers. This work was led by Jeffrey Steckler and Jacob Houghton, Ph.D. of the Department of Radiology at MSK.
David Hansen, CEO of MabVax Therapeutics, said, "We are grateful to Jason S. Lewis, Ph.D. and his team for their continued pioneering work using the HuMab-5B1 platform. They are taking important steps in expanding the clinical utility of our HuMab-5B1 antibody, including (1) demonstrating that smaller fragments of our full-length antibody could provide significant advantages in speeding tumor imaging, (2) demonstrating the utility of our full length antibody with a new radioimmunotherapy approach, (3) helping MabVax to evaluate additional CA19-9 expressing cancers for which our antibody development program may have utility beyond our current focus on pancreatic cancer, and (4) completing investigations supporting our radioimmunotherapy product for which we plan to submit an Investigational New Drug Application later this year."

On September 26, 2016 Myriad Genetics, Inc. (NASDAQ:MYGN), a leader in molecular diagnostics and personalized medicine, reported it will present three new studies at the National Society of Genetic Counselors Annual Education Conference being held Sept. 28-Oct. 1, 2016 in Seattle, Washington (Press release, Myriad Genetics, SEP 26, 2016, View Source [SID:SID1234515405]).

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"We are excited to be co-sponsoring the 35th National Society of Genetic Counselors Annual Education Conference," said Susan Manley, L.C.G.C., M.B.A., vice president of Medical Services, Myriad Genetic Laboratories. "We’re looking forward to presenting important new scientific data, which will improve the practice of genetic counseling. We also are excited to host a patient advocacy event in conjunction with the Conference, which will help raise funds for important patient advocacy groups including Be the Difference Foundation, Hereditary Colon Cancer Foundation and Li-Fraumeni Syndrome Association."

Below is a list of the featured presentations at NSGC (#NSGC16):

Poster Presentations

Title: Average Age of Diagnosis of Ovarian Cancer for Women with Pathogenic Variants in BRIP1, RAD51C and RAD51D​.
Presenter: Susana San Roman.
Date: Friday, September 30, 2016: 11:30 am — 12:45 pm PT.
Poster: C-117.

Title: Ancestry-Based Cancer Risks Associated with APC I1307K​.
Presenter: Lavania Sharma.
Date: Friday, September 30, 2016: 11:30 am — 12:45 pm PT.
Poster: C-123.

Title: Psychosocial Outcomes of Identifying High or Moderate Risk Mutation Carriers by Hereditary Cancer Panel Testing.
Presenter: Julie Culver.
Date: Thursday, September 29, 2016: 5:45 pm — 7:00 pm PT.
Poster: B-71.
For more information about these presentations, including a complete list of abstracts and presentations, please visit the NSGC website at View Source