On November 7, 2016 Heat Biologics, Inc. (Nasdaq:HTBX), a leader in the development of gp96-based immunotherapies that activate a patient’s immune system to fight cancer, reported that it will present a poster on its ComPACT platform at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting, in National Harbor, Maryland, on Friday, November 11th .

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The details are:

Title: Gp96-Ig/Costimulator (OX40L, ICOSL, or 4-IBBL) Combination Vaccine Improves T-cell Priming and Enhances Immunity, Memory and Tumor Elimination

Date and Time: November 11, 2016, 6:15-7:30 PM

Poster Number: 211

On November 7, 2016 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that eleven abstracts, including four oral presentations, supporting the company’s hematology and oncology portfolio will be presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in San Diego, California, December 3-6, 2016 (Press release, Jazz Pharmaceuticals, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220278 [SID1234516378]).

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"The data presentations at ASH (Free ASH Whitepaper) reflect our efforts in advancing our diversified pipeline of programs in hematology and oncology, including rare blood disorders such as acute lymphoblastic leukemia (ALL) and AML, and in complications of hematopoietic stem-cell transplantation (HSCT) such as hepatic VOD," said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer at Jazz Pharmaceuticals. "Of note, we look forward to sharing a post-hoc sub-analysis of Phase 3 survival data following allogeneic HSCT in older high-risk AML patients that compares CPX-351, also known as Vyxeos, with the standard of care."

The following oral and poster presentations focusing on Defitelio (defibrotide sodium) injection, Erwinaze (asparaginase Erwinia chrysanthemi) and CPX-351(cytarabine and daunorubicin liposome injection) will be presented at ASH (Free ASH Whitepaper).

Defitelio Related Oral and Poster Presentations

Presentation Title
Author
Presentation Number / Date / Time / Location
Timing of Initiation of Defibrotide Post-Diagnosis of Hepatic Veno-Occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome (SOS) Post-Hematopoietic Stem Cell Transplantation (HSCT): Exploratory Age-Group Analysis From an Expanded Access Study

Grupp S, et al.
Oral Presentation 66:
– December 3; 8:45 AM (PT); Manchester Grand Hyatt San Diego, Grand Hall B
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Post-Transplant Complications
Impact on Outcomes of Baseline Bilirubin in Patients with Hepatic VOD/SOS Receiving Defibrotide Treatment: A Post-Hoc Analysis

Richardson P, et al.
Poster Presentation 2213:
– December 3; 5:30-7:30 PM (PT); San Diego Convention Center, Hall GH
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster I
Treatment of Hepatic VOD/SOS Post-HSCT in Patients With Acute Leukemias: A Subgroup Analysis From the Defibrotide Expanded-Access Program
Richardson P, et al.
Poster Presentation 3412:
– December 4; 6:00-8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Poster II
VOD Related Abstract

Abstract Title
Author
Details
Stratification of Allogeneic HSCT Patients by Risk of Developing VOD: A Model for Assigning a Risk Score

Strouse C, et al.
Oral Presentation 983:
– December 5; 3:45 PM (PT); Manchester Grand Hyatt San Diego, Grand Hall B
Session 721: Clinical Allogeneic Transplantation: Conditioning Regimens, Engraftment, and Acute Transplant Toxicities: Post-Transplant Complications II
Diagnosis of VOD/SOS With or Without Multi-Organ Dysfunction (MOD) After HSCT: Analysis of a Multicenter Chart Review
Doede T, et al.
Online Only Publication;
Abstract 5756
Erwinaze Related Oral and Poster Presentations

Presentation Title
Author
Presentation Date / Time / Location
Population Pharmacokinetic Modeling of Intravenous Asparaginase Erwinia Chrysanthemi: Impact of Varied Infusion Rates on Exposure

Zomorodi K, et al.
Poster Presentation 1631:
– December 3; 5:30-7:30 PM (PT), San Diego Convention Center, Hall GH
– 614: Acute Lymphoblastic Leukemia: Therapy, excluding Transplantation: Poster I
CPX-351 Related Oral and Poster Presentations

Presentation Title
Author
Presentation Date / Time / Location
Analysis of Efficacy by Age for Patients Aged 60–75 With Untreated Secondary Acute Myeloid Leukemia (AML) Treated With CPX-351 Liposome Injection Versus Conventional Cytarabine and Daunorubicin in a Phase III Trial
Medeiros B, et al.
Oral Presentation 902:
– December 5; 3:00 PM (PT); Marriot Marquis San Diego Marina, San Diego Ballroom AB
– Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Clinical Trials of Novel Drugs and Combinations in AML
Survival Following Allogeneic Hematopoietic Cell Transplantation in Older High-Risk Acute Myeloid Leukemia Patients Initially Treated With CPX-351 Liposome Injection Versus Standard Cytarabine and Daunorubicin: Subgroup Analysis of a Large Phase III Trial
Lancet J, et al.
Oral Presentation 906:
– December 5; 4:00 PM (PT); Marriot Marquis San Diego Marina, San Diego Ballroom AB
– Session: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Clinical Trials of Novel Drugs and Combinations in AML
Enhanced Cytarabine and Daunorubicin Population Pharmacokinetics When Administered as CPX-351: A Novel Liposomal Formulation Not Requiring Dose Reduction for Mild Renal or Hepatic Dysfunction

Nikanjam M, et al.
Poster Presentation 3955:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 604: Molecular Pharmacology and Drug Resistance in Myeloid Diseases: Poster III

AML Related Poster

Presentation Title
Author
Presentation Number / Date / Time / Location
Burden of Acute Myeloid Leukemia (AML) Among Older Newly-Diagnosed Patients
Sacks N, et al.
Poster Presentation 4780:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 904: Outcomes Research—Malignant Conditions: Poster III
Additionally, one Jazz-sponsored Investigator Initiated Research poster presentation focusing on CPX-351 as an investigational agent for the treatment of AML will be presented at ASH (Free ASH Whitepaper).

Presentation Title
Author
Presentation Number / Date / Time / Location
CPX-351 for the Treatment of High-Risk Patients (pts) With Acute Myeloid Leukemia (AML)
Assi, R, et al.

Poster Presentation 4047:
– December 5; 6:00 – 8:00 PM (PT); San Diego Convention Center, Hall GH
– Session 616: Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III
Full details of the ASH (Free ASH Whitepaper) 2016 annual meeting can be found here (View Source) and abstracts can be found here (View Source).

About Defitelio1
In the U.S., Defitelio (defibrotide sodium) injection 80mg/mL received FDA marketing approval on March 30, 2016 for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT) and is the first and only FDA-approved therapy for patients with this rare, potentially fatal complication.

Defitelio is contraindicated in patients currently taking anticoagulants or fibrinolytics and in patients who are allergic to Defitelio or any of its ingredients. Defitelio may increase the risk of bleeding and should be withheld or stopped if significant bleeding occurs. Patients should be monitored for allergic reactions, especially if there is a history of previous exposure to Defitelio. The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds.

Please see full Prescribing Information for Defitelio. (View Source)

In Europe, defibrotide is marketed under the name Defitelio▼(defibrotide). In October 2013, the European Commission granted marketing authorization to Defitelio under exceptional circumstances for the treatment of severe VOD in patients undergoing HSCT therapy. It is the first and only approved treatment in Europe for severe VOD. In Europe, Defitelio is indicated in patients over one month of age. It is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic therapy. ▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system found under section 4.8 of the SmPC. (View Source)

About VOD
HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous hematologic disorders such as leukemia, lymphoma and aplastic anemia, and congenital immunodeficiency and autoimmune disorders.2 VOD is a rare complication of HSCT, which occurs in approximately 9-14% of HSCT patients.3,4 Hepatic VOD, also known as SOS, is an early and life-threatening complication affecting the sinusoidal endothelial cells of the liver, which can typically occur within the first 21 days following HSCT.4,5 Hepatic VOD progresses to multi-organ dysfunction in approximately 30-50% of cases.5 VOD with multi-organ dysfunction (MOD) is associated with an overall mortality (death) rate of 84%.3 MOD is characterized by the presence of renal or pulmonary dysfunction.6,7 VOD is often characterized by sudden weight gain, hepatomegaly (abnormally enlarged liver), and elevated bilirubin.6,7

About Erwinaze
Erwinaze (asparaginase Erwinia chrysanthemi) is currently approved in the U.S. for administration via intramuscular injection or via intravenous infusion in conjunction with chemotherapy. It is indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.8 Erwinaze is derived from the bacterium Erwinia chrysanthemi and is therefore immunologically distinct from E. coli-derived asparaginase and suitable for patients with hypersensitivity to E. coli-derived treatments9. Outside of the U.S., Erwinaze is sold under the name Erwinase. Please consult local labeling for product information specific to your country.

Erwinaze is contraindicated in patients who have had serious allergic reactions to Erwinaze, or had serious swelling of the pancreas, serious blood clots, or serious bleeding with past L-asparaginase treatment. Erwinaze should be discontinued if any of the following occur: serious allergic reactions, including a feeling of tightness in the throat, unusual swelling/redness in the throat and/or tongue, or trouble bleeding; or severe inflammation of the pancreas. Glucose intolerance has been reported, which in some cases may be irreversible. If blood clots of bleeding occur, discontinue Erwinaze until symptoms resolve. The most common side effects of Erwinaze are allergic reactions, too much sugar in the blood, fever, swelling of the pancreas, local reactions (swelling, rash, etc. where the needle entered the skin), vomiting, nausea, blood clots, liver problems, stomach pain/discomfort, and diarrhea. Please see full Prescribing Information for Erwinaze. (View Source)

About Vyxeos (CPX-351)
CPX-351 (cytarabine and daunorubicin liposome injection) is an investigational product being evaluated for the treatment of AML and is a combination of cytarabine and daunorubicin encapsulated within a nano-scale liposome at a 5:1 molar ratio. The proposed trade name, Vyxeos, is conditionally approved by the FDA and is subject to confirmation upon approval of the NDA. CPX-351 was granted orphan drug status by the FDA and the European Commission for the treatment of acute myeloid leukemia. CPX-351 was granted Breakthrough Therapy Designation for the treatment of adults with therapy-related AML or AML with myelodysplasia-related changes and was also granted Fast Track designation by the FDA for the treatment of older patients with secondary AML. On October 3, 2016 Jazz announced the initiation of a rolling submission of a New Drug Application (NDA) to the FDA, seeking marketing approval of CPX-351 for the treatment of AML.

About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a rapidly progressing and life-threatening blood cancer that rises in frequency with age.16 The American Cancer Society estimates that there will be 19,950 new cases of AML and 10,430 deaths from AML in the U.S. in 2016.10 In the European Union, the number of new cases is estimated to be 20,100 in 2016.11

The median age at diagnosis is 67 and with rising age there is progressive worsening of prognosis.10,12 Advancing age is associated with increasing risk of specific chromosomal/mutational changes and risk of pre-malignant marrow disorders which give rise to more aggressive and less responsive forms of AML.13,14 As patients age there is also reduced tolerance for intensive chemotherapy.15 As a consequence, advances in supportive care, intensive chemotherapy, and bone marrow transplantation have primarily benefitted younger patients with approximately one third of patients 18-60 years of age achieving cure.13,15 Older patients have not achieved higher rates of cure or improved upon a 5-year survival rate of 10-20% in spite of 40 years of research.15,16

On November 7, 2016 Threshold Pharmaceuticals, Inc. (Nasdaq:THLD), a clinical-stage biopharmaceutical company developing novel therapies for cancer, reported financial results for the third quarter ended September 30, 2016 and provided an update on the Company’s corporate and clinical development activities (Filing, Q3, Threshold Pharmaceuticals, 2016, NOV 7, 2016, View Source [SID1234516782]).

"We plan to initiate a clinical trial evaluating evofosfamide in combination with checkpoint inhibitors with our collaborators at MD Anderson Cancer Center while at the same time continue to pursue registration of evofosfamide in Japan for the treatment of pancreatic cancer. In addition, we continue to pursue potential collaborations to expand our development pipeline while we complete IND-enabling toxicology studies with TH-3424 with the goal of reaching the clinic with a new compound in 2017", said Barry Selick, Ph.D., Chief Executive Officer of Threshold.

Recent Highlights

Evofosfamide – The Company’s lead product candidate is an investigational hypoxia-activated prodrug that is designed to be activated under tumor hypoxic conditions, a hallmark of many cancers.

· First meeting scheduled with the Japanese PMDA (Pharmaceutical and Medical Devices Agency) to present the improvement in overall survival that was observed in the Japanese sub-population of the MAESTRO Phase 3 trial;

· The Company announced a partnership with Caris Life Sciences to develop a customized assay for pancreatic cancer patients to predict likelihood of response to evofosfamide;

· Clinical development collaborations investigating evofosfamide in patients with pancreatic neuroendocrine tumors (pNET), recurrent glioblastoma (GBM) and hepatocellular carcinoma (HCC) remain ongoing;

· A recent presentation by one of the Company’s collaborators from the MD Anderson Cancer Center highlighted the promise of evofosfamide in combination with a class of checkpoint inhibitor antibodies to improve the efficacy of this class of potent anti-cancer therapies; and

· The Company plans to initiate a clinical trial in various solid tumors evaluating evofosfamide in combination with one or more checkpoint inhibitor antibodies in development.

Tarloxotinib – The Company had been prosecuting Phase 2 proof of concept studies with tarloxotinib, a hypoxia-activated epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), which was designed to selectively release an irreversible EGFR-TKI in hypoxic tumors.

· During the quarter, the Company announced interim results from two proof-of-concept, Phase 2 clinical trials in patients with advanced non-small cell lung cancer (NSCLC) and patients with metastatic squamous cell carcinoma of the head and neck and skin. While results in patients with squamous cell carcinoma of the skin were minimally sufficient for continuation, the response rate in the NSCLC trial and the head and neck arms of the squamous cell cancer trial did not meet the minimum response rate hurdles for study continuation. Accordingly, enrollment in both Phase 2 clinical trials and further development of tarloxotinib has been discontinued.

TH-3424 – TH-3424 is the Company’s small-molecule drug candidate being evaluated for the treatment of hepatocellular (liver) cancer (HCC), castrate resistant prostate cancer (CRPC), T-cell acute lymphoblastic leukemias (T-ALL), and other cancers expressing high levels of aldo-keto reductase family 1 member C3 (AKR1C3). Tumors overexpressing AKR1C3 can be resistant to radiation therapy, chemotherapy and anti-androgen therapy. TH-3424 is a prodrug that selectively releases a potent DNA cross-linking agent in the presence of AKR1C3.

· TH-3424 has been selected as a lead candidate and Investigational New Drug (IND)-enabling studies have been initiated in collaboration with Ascenta Pharmaceuticals, Ltd.

Third Quarter 2016 Financial Results

· Cash, cash equivalents and marketable securities totaled $28.1 million at September 30, 2016 compared to $33.6 million at June 30, 2016; the net decrease of $5.5 million was a result of operating cash requirements for the quarter ended September 30, 2016. With the previously announced decision to cease joint development of evofosfamide under the Company’s former collaboration with Merck KGaA, the decision in September to discontinue further development of tarloxotinib, and the workforce reduction, the Company continues to expect its operating cash requirements to be lower for the fourth quarter of fiscal year 2016 and fiscal year 2017.

· No revenue was recognized in the third quarter ended September 30, 2016 compared to $3.7 million for the same period of 2015. Revenue for the quarter ended September 30, 2015 related to the amortization of the aggregate of $110 million in upfront and milestone payments received from the Company’s former collaboration with Merck KGaA, Darmstadt, Germany. The revenue from the upfront payment and milestone payments received under the agreement were previously being amortized over the relevant performance period, rather than being immediately recognized when the upfront payment and milestones were earned or received. As a result of Merck KGaA, Darmstadt, Germany’s and the Company’s decision to cease further joint development of evofosfamide in December 2015, the Company immediately recognized all of the remaining deferred revenue into revenue during the quarter ending December 31, 2015. Also as a result of the termination of the agreement, the Company is no longer eligible to receive any further milestone payments from Merck KGaA, Darmstadt, Germany.

· Research and development expenses were $3.5 million for the third quarter ended September 30, 2016, compared to $8.1 million for the same period in 2015. The decrease in research and development expenses, net of reimbursement for Merck KGaA, Darmstadt, Germany’s 70 percent share of total eligible collaboration expenses for evofosfamide, was due primarily to a $2.8 million decrease in employee related expenses, including a $0.4 million decrease in non-cash stock-based compensation expense and a $1.8 million decrease in clinical development expenses and consulting expenses. The Company expects research and development expenses to continue to decline in 2016 as a result of the decision to cease further joint development of evofosfamide under the Company’s former collaboration with Merck KGaA and the workforce reduction.

· General and administrative expenses were $1.7 million for the third quarter ended September 30, 2016 compared to $2.4 million for the same period in 2015. The decrease in general and administrative expenses was due primarily to a $0.5 million decrease in employee related expenses and a $0.2 million decrease in consulting expenses.

· Non-cash stock-based compensation expense included in total operating expenses was $0.8 million for the third quarter of 2016 compared to $1.5 million for the same period in 2015. The decrease in stock-based compensation expense was due to the amortization of a smaller number of options with lower fair values.

· Net loss for the third quarter of 2016 was $5.7 million compared to $6.4 million for the same period in 2015. Included in the net loss for the third quarter of 2016 was an operating loss of $5.2 million and non-cash expense of $0.5 million compared to an operating loss of $6.8 million and non-cash income of $0.3 million for the third quarter of 2015. The non-cash income or expense is related to changes in the fair value of the Company’s outstanding and exercised warrants that was classified as other income (expense).

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About Evofosfamide
Evofosfamide (previously known as TH-302) is an investigational hypoxia-activated prodrug of a bis-alkylating agent that is preferentially activated under severe hypoxic tumor conditions, a feature of many solid tumors. Areas of low oxygen levels (hypoxia) in solid tumors are due to insufficient blood vessel supply. Similarly, the bone marrow of patients with hematological malignancies has also been shown, in some cases, to be severely hypoxic.

About Tarloxotinib Bromide
Tarloxotinib bromide (previously known as TH-4000), or "tarloxotinib", is a prodrug designed to selectively release a covalent (irreversible) EGFR tyrosine kinase inhibitor under severe hypoxia, a feature of many solid tumors. In September 2016, the Company announced the discontinuation of any further development of tarloxotinib.

About TH-3424
TH-3424 is small-molecule drug candidate being evaluated for the potential treatment of hepatocellular (liver) cancer (HCC), castrate resistant prostate cancer (CRPC), T-cell acute lymphoblastic leukemias (T-ALL), and other cancers expressing high levels of aldo-keto reductase family 1 member C3 (AKR1C3). Tumors overexpressing AKR1C3 can be resistant to radiation therapy and chemotherapy. TH-3424 is a prodrug that selectively releases a potent DNA cross-linking agent in the presence of AKR1C3. Preliminary nonclinical toxicology studies suggested an adequate therapeutic index that the Company believes warrants conducting Investigational New Drug (IND)-enabling toxicology studies, which are being done in collaboration with Ascenta Pharmaceuticals, Ltd.

On November 7, 2016 Novocure (NASDAQ:NVCR) reported that 38 abstracts focusing on Tumor Treating Fields (TTFields) will be presented at the 21st Annual Scientific Meeting of the Society for Neuro-Oncology (SNO) on Nov. 17-20, in Scottsdale, Arizona (Press release, NovoCure, NOV 7, 2016, View Source [SID1234516379]). Of the 38 abstracts, 28 were prepared by external researchers, representing a growing interest in Novocure’s proprietary TTFields therapy.

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"Every year at the SNO annual meeting, we observe an increased interest in TTFields, as is evident from a growing number of abstracts accepted along with an increasing number of institutions that are studying TTFields," said Dr. Eilon Kirson, Novocure’s Chief Science Officer and Head of Research and Development. "Research conducted by external scientists strengthens our understanding of TTFields, often validating TTFields as a potential treatment for a number of solid tumors."

Abstracts span a variety of topics from preclinical and clinical data to treatment delivery and patient education. Notable topics to be presented include, amongst others, TTFields in combination with immunotherapy, radiation, triple chemotherapy and cytostatic agents, a case report on the use of TTFields in brain metastasis from ovarian cancer and a pilot study of Optune for recurrent atypical and anaplastic meningioma.

The long-term survival analysis of the full dataset from its phase 3 pivotal EF-14 trial of Optune in combination with temozolomide for the treatment of newly diagnosed glioblastoma (GBM) will be presented on Friday, Nov. 18, by Roger Stupp, M.D., Professor at the University of Zurich and Director of Department of Oncology at the Zurich University Hospital, Zurich, Switzerland, and EF-14 Principal Investigator. The long-term analysis confirms the interim analysis results published in the Journal of the American Medical Association (JAMA)1 in December 2015, showing significant extension of both progression free and overall survival in newly diagnosed GBM patients receiving Optune with temozolomide compared to temozolomide alone. The long-term analysis shows survival rates were significantly higher four years from randomization in patients receiving Optune with temozolomide compared to patients receiving temozolomide alone. The safety profile in the long-term analysis was consistent with the interim analysis of the EF-14 trial.

The following abstracts will be presented:

Preclinical

Human glioma cell migration and invasion properties are inhibited by exposure to tumor treating fields (TTFields) in vitro; RS Schneiderman et al; Saturday, Nov. 19; abstract: #EXTH-03

Tumor treating fields (TTFields) induce autophagy in glioma cells; Y Porat et al; Saturday, Nov. 19; abstract: #EXTH-30

Effects of tumor treating fields (TTFields) and temozolomide in MGMT expressing and non-expressing patient-derived glioblastoma cells; PA Clark et al; Saturday, Nov. 19; abstract: #EXTH-25

Tumor Treating Field (TTFields) effects on glioblastoma cells are augmented by mitotic checkpoint inhibition; AF Kessler et al; Saturday, Nov. 19; abstract: #EXTH-07

Cytostatic agents combined with tumor treating fields (TTFields) in glioma cell lines; M Groves et al; Saturday, Nov. 19; abstract: #NIMG-39 Tumor cells induce immune activation phenotype in immune cells following TTFields activation; T Holtzman et al; Friday, Nov. 18; abstract: #IMST-26 Evaluating the in-vitro effects of tumor treating fields on T cell responses; G Diamant et al; Friday, Nov. 18; abstract: #IMST-30 Clinical METIS: A phase III study of radiosurgery with TTFields for 1-10 brain metastases; M Mehta et al; Saturday, Nov. 19; abstract: #BMET-03 One year with Optune in routine use: first report on clinical experiences in Germany; M Misch et al; Friday, Nov. 18; abstract: #RARE-44 Analysis of glioblastoma physical characteristics in patients benefiting from tumor treating electric fields therapy; PP San et al; Friday, Nov. 18; abstract: #RTHP-19 Compliance and duration of treatment with tumor treating fields (TTFields) in adjuvant treatment for newly diagnosed glioblastomas (GBMs) improves progression-free survival (PFS) and overall survival (OS); SA Jeyapalan et al; Friday, Nov. 18; abstract: #RTHP-29 Pilot study of Optune (Novo-TTF-100A) for recurrent atypical and anaplastic meningioma; G Wu et al; Friday, Nov. 18; abstract: #ACTR-43 Classification of dermatologic adverse events and management strategies in patients receiving therapy with Optune for high grade gliomas; D Jennings et al; Friday, Nov. 18; abstract: #QLIF-11 The use of a programmable shunt in a patient, treated with Optune: feasibility report; L Schreiber et al; Friday, Nov. 18; abstract: #RARE-24 Survival benefit with triple chemotherapy and TTFields for glioblastoma; G Lu et al; Friday, Nov. 18; abstract: #PDCT-16 Patterns of glioblastoma recurrence in low field intensity regions during TTFields treatment; J Battiste et al; Saturday, Nov. 19; abstract: #NIMG-61 Patterns of compliance in the use of tumor treating fields (Optune) for brain tumors; M Pandey et al; Friday, Nov. 18; abstract: #ACTR-31 Use of tumor treating fields in brain metastases from ovarian cancer, a case report; M Pandey et al; Saturday, Nov. 19; abstract: #BMET-21 Imaging the early metabolic response during tumor treating fields (TTFields) therapy in recurrent glioblastoma; C Juhasz et al; Saturday, Nov. 19; abstract: #NIMG-37 Rates and impact of compliance with tumor treating fields therapy in a glioma cohort; Y Odia et al; Friday, Nov. 18; abstract: #RTHP-37 Rates and outcomes of combination tumor treating fields and immunotherapy in a glioma cohort; Y Odia et al; Friday, Nov. 18; abstract: #ATIM-34 A phase II, single arm study of Optune in bevacizumab-naive subjects with recurrent WHO grade III malignant glioma; D O’Connell et al; Friday, Nov. 18; abstract: #ACTR-41 Use of tumor treating fields in the first line treatment of glioblastoma; M Pandey et al; Friday, Nov. 18; abstract: #ACTR-28 Clinical experiences of tumor treating fields (TTFields) in pediatric GBM patients; J Crawford et al; Saturday, Nov. 19; abstract: #NIMG-66 Beneficial effect of tumor treating field therapy in Gliomatosis Cerebri; N Blondin; Friday, Nov. 18; abstract: #ACTR-08 Enhanced therapeutic benefits of tumor treating fields (TTFields) on superficially located glioblastoma multiforme (GBM); V Kumar et al; Friday, Nov. 18; abstract: #RTHP-30 Stability of shunt programmable valve settings with simultaneous use of the Optune transducer array; A Chan et al; Friday, Nov. 18; abstracts: #SURG-25 Treatment Delivery/Patient Education First report of user experience, compliance outcomes, and alarm states with the second generation Optune system; A Kinzel et al; Saturday, Nov. 18; abstract: #NIMG-50 Does personalizing tumor treating fields (TTFields) with NovoTAL in glioblastoma treatment planning make a difference? Insights from electric field simulation studies; D Garcia Carracedo et al; Saturday, Nov. 19; abstract: #NIMG-06 Compliance of tumor treating electric fields therapy and overall survival in glioblastoma; ET Wong et al; Friday, Nov. 18; abstract: #RTHP-17 Development of practice algorithms to guide treatment planning with TTFields for the management of glioblastoma; J Trusheim et al; Saturday, Nov. 19; abstract: #NIMG-23 Shared Care Model for glioblastoma patient management; J Battiste et al; Saturday, Nov. 19; abstract: #NIMG-52 Periodic transducer array shifting preserves both TTFields intensity in the gross tumor volume (GTV) and promotes scalp health during the course of glioblastoma therapy; A Naveh et al; Saturday, Nov. 19; abstract: #NIMG-26 Influence of 2 contrasting treatment planning approaches on tumor treating fields (TTFields) intensity in the gross tumor volume (GTV) and peritumoral brain zone (PBZ) in glioblastoma; D Garcia Carracedo et al; Saturday, Nov. 19; abstract: #NIMG-07 Placement of scalp electrodes (Novo TTF) does not significantly change radiation dosimetry when delivering cranial intensity modulated radiation therapy (IMRT); G Shukla et al; Friday, Nov. 18; abstract: #RTHP-34 Optimal Patient and Family Education Is Key to Improving the Efficacy of Tumor Treating Fields Plus Chemotherapy in Treatment of GBM; MG Saria et al; Saturday, Nov. 19; abstract: #NIMG-63 Tumor recurrence in areas of lower TTFields Intensity: A reverse simulation case study; J Battiste et al; Saturday, Nov. 19; abstract: #NIMG-29 Late-Breaking Oral Presentation Prospective multi-center phase III trial of Tumor Treating Fields together with temozolomide compared to temozolomide alone in patients with newly diagnosed glioblastoma; R Stupp et al; Friday, Nov. 18

On November 7, 2016 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported a corporate update and reported its financial results for the third quarter ended September 30, 2016 (Press release, Portola Pharmaceuticals, NOV 7, 2016, View Source;p=RssLanding&cat=news&id=2220288 [SID1234516691]).

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"We continue to focus on obtaining regulatory approval for our two lead programs in the United States and EU. Both programs have the potential to become the standard of care in two growth areas of thrombosis that have a high unmet medical need and limited treatment options. If approved, betrixaban, our oral Factor Xa inhibitor, would be the first anticoagulant indicated for extended VTE prophylaxis in the over 24 million medically ill patients admitted to the hospital annually in the G7. AndexXa, our much anticipated Factor Xa inhibitor antidote, would be the first agent approved to treat the growing number of patients admitted to the hospital with life-threatening anticoagulant-related bleeding. We have made important progress toward bringing both betrixaban and AndexXa to market. We submitted an NDA for betrixaban in the United States and plan to submit an MAA in the EU," said Bill Lis, chief executive officer of Portola. "Also, we continue to engage in positive, productive dialogue with the FDA to resolve outstanding questions regarding the Complete Response Letter for AndexXa, most of which are focused on manufacturing."

Recent Achievements, Upcoming Events and Milestones

Betrixaban – an oral Factor Xa inhibitor anticoagulant in development for the prevention of venous thromboembolism (VTE) in acute medically ill patients; designated Fast Track status by the U.S. Food and Drug Administration (FDA)

Submitted a New Drug Application (NDA) to the FDA seeking approval to market betrixaban for extended-duration prophylaxis of VTE in acutely ill medical patients with risk factors for VTE
Plan to submit a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) by year-end
Three abstracts on results of sub-analyses of the Phase 3 APEX Study conducted by the PERFUSE Study Group were accepted for oral and poster presentations at the upcoming American Heart Association Scientific Sessions 2016
AndexXa (andexanet alfa) – a Factor Xa inhibitor antidote in development for patients treated with a Factor Xa inhibitor when reversal of anticoagulation is needed due to life-threatening bleeding or when urgent surgery is required; designated a Breakthrough Therapy and an Orphan Drug by the FDA

Submitted an MAA, which completed the validation period and was accepted for review by the EMA
Continued productive dialogue with the FDA regarding the Complete Response Letter; Portola currently plans to resubmit the BLA in the second quarter of 2017
Presented a preliminary analysis of interim data from the ongoing Phase 3b/4 ANNEXA-4 Study in patients with acute major bleeding in a Late-Breaking Science Hot Line session at the European Society of Cardiology 2016 Congress with simultaneous publication online by The New England Journal of Medicine
Plan to present data from the ongoing Phase 2 study of andexanet alfa reversal of betrixaban in an oral presentation at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting 2016
Cerdulatinib – an oral, dual Syk/JAK inhibitor in development to treat resistant or relapsed hematologic cancer patients

Continued to enroll patients in a Phase 2a study evaluating the safety and efficacy of cerdulatinib in patients with relapsed/refractory B-cell malignancies who have failed multiple therapies
Two abstracts were accepted for presentation at the upcoming ASH (Free ASH Whitepaper) Annual Meeting 2016
Third Quarter 2016 Financial Results
Collaboration revenue earned under Portola’s collaborations with Bristol-Myers Squibb Company and Pfizer, Bayer Pharma and Janssen Pharmaceuticals, and Daiichi Sankyo was $9.3 million for the third quarter of 2016 compared with $2.9 million for the third quarter of 2015. The increase in revenue was primarily due to $2.5 million received upon achievement of a milestone from the Bayer and Janssen Phase 3 agreement, $2.5 million received upon achievement of a milestone from the Daiichi Sankyo Phase 3 agreement, and incremental revenue of $2.0 million from three collaboration and license agreements executed in the first quarter of 2016 to develop and commercialize andexanet alfa in Japan.

Total operating expenses for the third quarter of 2016 were $100.8 million compared with $58.5 million for the same period in 2015. Total operating expenses for the third quarter of 2016 included $7.8 million in stock-based compensation expense compared with $6.1 million for the same period in 2015.

Research and development expenses were $87.2 million for the third quarter of 2016 compared with $48.4 million for the third quarter of 2015. The increase in R&D expenses was primarily due to a $27.3 million impairment charge for AndexXa manufacturing expenses that Portola prepaid to CMC Biologics. These expenses were intended to be credited against future batch manufacturing costs for the Line C, or 6×2,000 liter, manufacturing process. This impairment charge was triggered by the Company’s decision to focus on Line A/B manufacturing at CMC Biologics and Gen 2 manufacturing at Lonza, and suspend Line C manufacturing at CMC Biologics.

Selling, general and administrative expenses for the third quarter of 2016 were $13.6 million compared with $10.1 million for the same period in 2015 as the Company increased headcount to support its growth and increased commercial launch preparation activities for AndexXa in advance of the PDUFA date in August 2016.

For the third quarter of 2016, we reported a net loss of $92.9 million dollars, or a net loss per share of $1.64, compared with a net loss of $55.2 million dollars, or a net loss per share of $1.05, for the same period in 2015. Net loss for the third quarter of 2016 included the $27.3 million impairment charge. Shares used to compute net loss per share attributable to common stockholders were approximately 56.5 million for the third quarter of 2016 compared with approximately 52.6 million for the same period in 2015.

As of September 30, 2016, cash, cash equivalents and investments totaled $274.6 million compared with cash, cash equivalents and investments of $460.2 million as of December 31, 2015.