On September 10, 2015 BioCancell Ltd. (TASE: BICL), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies to treat cancer-related diseases, reported that the United States Food and Drug Administration (FDA) has granted Fast Track designation to BC-819 for use in bladder cancer patients (Press release, BioCancell Therapeutics, SEP 10, 2015, View Source [SID:1234507457]). BC-819 is being developed as a treatment for non-muscle-invasive bladder cancer (NMIBC), and will enter two Phase III confirmatory studies in the first half of 2016. The FDA Fast Track designation has been granted for both of BC-819’s planned Phase III indications: for patients who have failed treatment with BCG (the current standard of care) and for patients who are unresponsive or intolerant to BCG treatment and will be treated with BC-819 as a monotherapy.

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The FDA Fast Track program is designed to expedite the development and review of drugs that demonstrate the potential to address unmet medical needs by treating serious or life-threatening conditions. Companies that receive Fast Track designation are allowed to submit sections of their final marketing application (BLA) on a rolling basis as data becomes available, expediting the FDA review process. They also benefit from more frequent interactions with the FDA review team, including meetings to discuss the drug’s development plan to support potential drug approval.

Jonathan Burgin, Chief Executive Officer of BioCancell, stated, "It is encouraging that BC-819 has received FDA Fast Track designation. This is an important step towards initiating two Phase III studies in 2016, and we look forward to the opportunity to work closely with the FDA as we further the development of BC-819 as a potential new therapy for bladder cancer patients."

On September 10, 2015 NanoString Technologies, Inc. (NASDAQ:NSTG), a provider of life science tools for translational research and molecular diagnostic products, reported the commercial launch of the nCounter RNA:Protein PanCancer Immune Profiling Panel (Press release, NanoString Technologies, SEP 10, 2015, View Source [SID:1234507452]). The panel can be analyzed on the full line of nCounter Analysis Systems, including the new nCounter SPRINT Profiler, utilizing breakthrough technology, which simultaneously measures both RNA and Protein expression through multiplexed digital counting. Data generation from an early access program has demonstrated that the technology is robust, reproducible and sensitive, detecting both RNA and protein with as few as 150,000 cells in a single reaction.

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"Developing new cancer therapies and molecular diagnostics requires extracting all relevant biological information from clinical samples that are often too small to characterize using multiple different assays, such as small biopsies from recurrent tumors," said Martin McIntosh, Ph.D. of the Fred Hutchinson Cancer Research Center. "We now routinely quantify RNA and cell-surface protein abundance for these and other types of samples using the NanoString digital barcoding technology because we have found the information highly relevant, and even catalytic, in so many areas."

The RNA:Protein PanCancer Immune Profiling Panel from NanoString is the first example of an entirely new category of multiplexed digital assays, enabling the next generation of tumor profiling experiments. Most cancer biologists already perform RNA and protein expression analysis using separate methodologies when sufficient tumor sample is available. However, in many cases the analysis has been limited to RNA due to lack of sample. The RNA:Protein PanCancer Immune Profiling Panel offers a potential solution to this problem, by enabling simultaneous measurement of RNA and protein in small amounts of tumor sample—with 800 RNA and protein measurements in as few as 150,000 cells.

This initial immuno-oncology focused RNA:Protein Profiling Panel builds on the success of the company’s existing PanCancer Immune Profiling Panel for gene expression analysis. The new panel combines the measurement of 30 proteins with 770 RNA measurements to create a targeted immuno-oncology solution. The RNA measurements include genes indicated in the Hallmarks of Cancer, first described in seminal papers by Hanahan and Weinberg (Hanahan, D., and Weinberg, R.A. (2000). Cell 100, 57-70 and Hanahan, D., and Weinberg, R.A. (2011). Cell 144, 646-674), which identify immune cell types, cancer antigens, checkpoint blockades and key immune pathway genes for both innate adaptive and humoral immune responses. The protein measurements focus on cell surface and immune checkpoint targets indicated in the Cancer Immunity Cycle, first described by Chen and Mellman ( Chen DS, Mellman I. Immunity. 2013;39:1-10).

"NanoString is proud to be at the forefront of this new product category, providing our customers with the ability to measure gene and protein expression in a single experiment," said Joseph M. Beechem, Ph.D., Senior Vice President of Research & Development at NanoString Technologies. "We believe that our initial RNA:Protein Profiling Panel will become a powerful tool for immuno-oncology researchers, particularly those focused on drug development, where an understanding of protein interactions is critical."

About NanoString Technologies, Inc.

NanoString Technologies provides life science tools for translational research and molecular diagnostic products. The company’s nCounter Analysis System has been employed in life sciences research since it was first introduced in 2008 and has been cited in over 800 peer-reviewed publications. The nCounter Analysis System offers a cost-effective way to easily profile the expression of hundreds of genes, proteins, miRNAs, or copy number variations, simultaneously with high sensitivity and precision, facilitating a wide variety of basic research and translational medicine applications, including biomarker discovery and validation. The company’s technology has also been applied to diagnostic use. The Prosigna Breast Cancer Prognostic Gene Signature Assay, together with the nCounter Dx Analysis System, is FDA 510(k) cleared for use as a prognostic indicator for distant recurrence of breast cancer.

For more information, please visit www.nanostring.com.

On September 10, 2015 Champions Oncology, Inc. (CSBR), engaged in the development of advanced technology solutions and services to personalize the development and use of oncology drugs, reported its financial results for the first quarter ended July 31, 2015 (Filing, 8-K, Champions Oncology, SEP 10, 2015, View Source [SID:1234507451]).

First Quarter and Recent Business Highlights:

• Completed uplisting to NASDAQ Capital Market
• Filed patent applications to develop humanized mice to be used in immune-oncology
• Announced additions to management team

Joel Ackerman, Champions Oncology CEO, stated, "We continue to make progress in building the company. As the recognition of the value of patient derived xenografts grows, we are being presented with many opportunities to expand our platform into new areas. We are pursuing many of these opportunities in areas including immune oncology and clinical applications for our TOS customers."

Financial Results

For the first quarter of 2015, revenue was $2.8 million, as compared to $1.9 million for the three months ended July 31, 2014, an increase of $892,000 or 47.6%. Total operating expenses for the first quarter 2015 were $5.7 million, as compared to $5.6 million for the three months ended July 31, 2014, an increase of $78,000 or 1.4%.

For the first quarter of 2015, Champions reported a loss from operations of $2.9 million as compared to a loss from operations of $3.7 million for the three months ended July 31, 2014. Excluding stock-based compensation of $775,000 and $808,000 for the three months ended July 31, 2015 and 2014, Champions recognized a net loss of $2.1 million and $2.7 million respectively.

Operating Results

Personalized Oncology Solutions (POS):

POS revenue was $485,000 and $341,000 for the three months ended July 31, 2015 and 2014, respectively, an increase of $144,000 or 42.2%. Core revenue from its Champions TumorGraft technology platform decreased $16,000 or (4.9%). This decrease is due to a 21.6% decline in implant revenue offset by a 6.3% increase in panel revenue. Non-core revenue increased $160,000.

POS cost of sales was $661,000 and $757,000 for the three months ended July 31, 2015 and 2014, respectively, a decrease of $96,000 or (12.7%). For the three months ended July 31, 2015 and 2014, gross margins for POS were (36.3%) and (122%), respectively. The improvement in gross margin is attributed to the increase in higher margin, non-core revenue and cost reductions in the core business.

Translational Oncology Solutions (TOS):

TOS revenue was $2.3 million and $1.6 million for the three months ended July 31, 2015 and 2014, respectively, an increase of $700,000, or 48.8%. The increase is the result of increased bookings in prior quarters due to the expansion of the TOS sales team and the growth of the platform.

TOS cost of sales was $1.6 million and $965,000 for the three months ended July 31, 2015 and 2014, respectively, an increase of $647,000, or 67%. For the three months ended July 31, 2015 and 2014, gross margin for TOS was 31% and 38.6%, respectively.

Research and development expense was $1.1 million and $1.4 million for three months ended July 31, 2015 and 2014, respectively, a decrease of $300,000, or (22.7%). The decrease is due to lower one-time expenses in genomic characterization of our Champions TumorGraft Bank. Sales and marketing expense for both the three months ended July 31, 2015 and 2014 was $1 million. General and administrative expense for the three months July 31, 2015 and 2014 was $1.3 million and $1.46 million, respectively a decrease of $145,000, or (9.9%). The decline is mainly due to a decrease in stock based compensation expense of $105,000.

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On September 10, 2015 Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, reported oral and poster presentations at the 16th World Conference on Lung Cancer (WCLC) which took place September 6 – 9, 2015, in Denver, CO.

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Four oral presentations included results from a Phase 2 clinical trial of VS-6063 (defactinib) in advanced refractory KRAS-Mutated Non-Small Cell Lung Cancer (NSCLC) and presentations on preclinical studies of VS-6063 and VS-5584 in Small-Cell Lung Cancer (SCLC) and Mesothelioma. Two poster presentations detailed trial designs for Verastem’s ongoing COMMAND trial in mesothelioma and Phase 1 dose-escalation study of VS-5584 in advanced solid tumors.

Verastem presented these clinical and preclinical data at WCLC in support of its programs targeting cancer stem cells (CSCs) through inhibition of the focal adhesion kinase (FAK; VS-6063) and PI3K/mTOR (VS-5584) signaling pathways. Research on the FAK and PI3K/mTOR signaling pathways has revealed critical roles for each in determining CSC survival and disease progression. CSCs represent a subpopulation of cancer cells that have tumor-initiating capability, are particularly resistant to chemotherapy and can mediate tumor recurrence both locally and at metastatic sites.

Details and links to the data presentations at WCLC are below:

Oral Presentations
Title: Phase 2 study of defactinib, VS-6063, a focal adhesion kinase (FAK) inhibitor, in patients with KRAS mutant non-small cell lung cancer (NSCLC)
Session info: Mini oral 30: New Kinase Targets; Track: Treatment of Advanced Diseases – NSCLC
Link to slides from the oral presentation: http://bit.ly/R3M6wc

Title: The cancer stem cell inhibitors VS-6063 (defactinib) and VS-5584 exhibit synergistic anticancer activity in pre-clinical models of mesothelioma
Session info: Oral session 40: Biology 1; Track: Thymoma, Mesothelioma and Other Thoracic Malignancies
Link to slides from the oral presentation: http://bit.ly/R3M6wc

Title: Targeting Cancer Stem Cells in Small Cell Lung Cancer
Session info: Mini oral 27: Biology and other issues in SCLC; Track: Small Cell Lung Cancer
Link to slides from the oral presentation: http://bit.ly/R3M6wc

Title: FAK inhibitor VS-6063 targets mesothelioma cancer stem cells: Rationale for maintenance therapy after conventional chemotherapy
Session info: Mini oral 38: Biology and Prognosis; Track: Thymoma, Mesothelioma and Other Thoracic Malignancies
Link to slides from the oral presentation: http://bit.ly/R3M6wc
Poster Presentations

Title: COMMAND: A Phase 2 randomized, double-blind, study of defactinib (VS-6063) as maintenance therapy in malignant pleural mesothelioma
Poster #: P3.08.014
Session info: Thymoma, Mesothelioma and Other Thoracic Malignancies – Mesothelioma
Link to copy of the poster presentation: http://bit.ly/R3M6wc

Title: A Phase 1 dose escalation study of VS-5584, a PI3K/mTOR inhibitor, administered with VS-6063, a focal adhesion kinase inhibitor, in mesothelioma
Poster #: P2.08.008
Session info: Thymoma, Mesothelioma and Other Thoracic Malignancies – Mesothelioma
Link to copy of the poster presentation: http://bit.ly/R3M6wc

About VS-6063
VS-6063 (defactinib) is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research has demonstrated that FAK activity is critical for the growth and survival of cancer stem cells. VS-6063 is currently being studied in the registration-directed COMMAND trial in mesothelioma (www.COMMANDmeso.com), a "Window of Opportunity" study in patients with mesothelioma prior to surgery, a Phase 1/1b study in combination with paclitaxel in patients with ovarian cancer, a trial in patients with Kras-mutated non-small cell lung cancer and a trial evaluating the combination of VS-6063 and VS-5584 in patients with relapsed mesothelioma. VS-6063 has been granted orphan drug designation for use in mesothelioma in the U.S. and EU.

About VS-5584
VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a dose escalation trial of VS-5584 in patients with advanced solid tumors as a single agent and a combination trial of VS-5584 and VS-6063 in patients with relapsed mesothelioma. VS-5584 has been granted orphan drug designation for use in mesothelioma in the U.S. and EU.

On September 10, 2015 DelMar Pharmaceuticals, Inc. (OTCQX: DMPI) ("DelMar" and the "Company"), a biopharmaceutical company focused on the development and commercialization of new cancer therapies, reported updated clinical data from its Phase I/II study of lead product candidate VAL-083 (dianhydrogalactitol), in patients with refractory glioblastoma multiforme (GBM), at GBM2015: 2nd International Symposium on Clinical and Basic Investigation in Glioblastoma in Toledo, Spain (Press release, DelMar Pharmaceuticals, SEP 10, 2015, View Source [SID:1234507445]).

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The Company’s data was presented in a poster entitled, "Update on Phase I/II study of VAL-083 (dianhydrogalactitol) in patients with recurrent malignant glioma."

DelMar is conducting a multicenter Phase I/II clinical study with VAL-083 in patients with recurrent GBM. Eligible GBM patients must have failed both Avastin (bevacizumab) and Temodar (temozolomide) unless either of these therapies was contraindicated. (ClinicalTrials.gov Identifier NCT01478178). Data from the Phase I dose-escalation portion of the study was presented at ASCO (Free ASCO Whitepaper). Dose limiting toxicity was observed at a dose of 50mg/m2/day; no drug-related severe adverse events were reported and myelosuppression was mild at doses ≤40mg/m2/day. Preliminary analysis suggested a dose-dependent and clinically meaningful survival benefit in GBM patients whose tumors had progressed following standard treatment with temozolomide, radiotherapy, bevacizumab and a range of salvage therapies.

"We view the trend toward a meaningful survival benefit, and at doses that were well tolerated by patients, as highly promising for the potential of VAL-083 to offer a new treatment alternative for GBM patients. Our subsequent and more detailed analysis of data from the Phase I portion of this trial continues to support these observations," stated Jeffrey Bacha, DelMar’s president and CEO.

The sub-group analysis for patients receiving up to 5 mg/m2 daily x 3 every 21 days (low dose) versus those patients receiving 30mg/m2 or 40mg/m2 (high dose) was presented today at the GBM2015 meeting. In summary, the data indicated:

Patients in the low dose sub-group had a median survival of 5 months versus median survival of 9 months for patients in the high dose sub-group following initiation of VAL-083 treatment (p = 0.04).

Increased survival was observed at 6, 9 and 12 months following initiation of treatment in the high-dose sub-group compared to the low dose sub-group.

Survival was not correlated with screening Karnofsky performance status (R2KPS = 0.03) or subject age at screening (R2AGE = 0.01).

Survival was not correlated with the localization of either the initial or recurrent lesion.

A longer time to progression following front-line temozolomide or second line bevacizumab treatment was not correlated with a survival benefit following initiation of VAL-083 therapy.

All patients had failed standard treatments and one or more salvage therapies prior to initiation of VAL-083 (median number of prior therapies = 3 for both the low and high dose sub-groups). No commonalities in prior therapy were observed to correlate with survival.

Survival was not correlated with MGMT expression in either the low dose or high dose sub-group.
"These analyses show a diminished possibility that factors, other than the proposed clinical activity of VAL-083, are responsible for the observed dose-dependent survival benefit following treatment," stated Mr. Bacha.

The Company has initiated a Phase II expansion cohort for the refractory GBM study at a dose of 40mg/m2 with approximately 14 patients expected to be enrolled. The purpose of the Phase II expansion cohort is to gain additional information about the safety and efficacy of VAL-083 at the 40mg/m2 dose prior to advancement into registration-directed Phase II/III clinical trials.

To date, 20 patients have been screened and eight (8) patients have initiated treatment with VAL-083 at the 40mg/m2 dose.
To further explore the therapeutic window of VAL-083, three (3) patients have also initiated treatment at an interim dose of 45mg/m2.

The Phase II expansion cohort may be continued at the higher 45mg/m2 dose if safety data warrants.
Further information on the trial design can be found on the company’s website at View Source

"We are pleased with the momentum of our enrollment in the trial. We look forward to presenting further data from the Phase II expansion study as we continue activities geared toward the initiation of a registration-directed Phase II/III clinical trial over the course of the next several months," added Mr. Bacha.

The Company’s poster presentation from GBM2015 may be found on DelMar’s website under View Source

About VAL-083
VAL-083 is a "first-in-class," small-molecule chemotherapeutic. In more than 40 Phase I and II clinical studies sponsored by the U.S. National Cancer Institute, VAL-083 demonstrated safety and efficacy in treating a number of cancers including lung, brain, cervical, ovarian tumors and leukemia. VAL-083 is approved in China for the treatment of chronic myelogenous leukemia (CML) and lung cancer and has received orphan drug designation in Europe and the U.S. for the treatment of gliomas.

DelMar is currently studying VAL-083 in a multi-center Phase I/II clinical trial for patients with refractory GBM in accordance with the protocol that has been filed with the U.S. Food and Drug Administration (FDA) at five clinical centers in the United States: Mayo Clinic (Rochester, MN); UCSF (San Francisco, CA) and three centers associated with the Sarah Cannon Cancer Research Institute (Nashville, TN, Sarasota, FL and Denver, CO). As a potential treatment for glioblastoma, VAL-083’s mechanism of action appears to be unaffected by the expression of MGMT, a DNA repair enzyme that is implicated in chemotherapy resistance and poor outcomes following front-line treatment with Temodar (temozolomide).