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 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 Bristol-Myers Squibb (NYSE:BMY) reported that their oncology team set off on the first leg of the Coast 2 Coast 4 Cancer Ride, a 19-day bike relay that will involve over 80 employees riding a combined total of nearly 2,900 miles, from today’s start on the Oregon Coast to the Jersey Shore, to show their support for the cancer community while raising funds for cancer research (Press release, Bristol-Myers Squibb, SEP 10, 2015, View Source [SID:1234507440]). Bristol-Myers Squibb will match donations raised by the riders, dollar-for-dollar up to $500,000, to support Stand Up To Cancer, whose collaborative "Dream Teams" of scientific researchers are working together to accelerate cancer research and to provide innovative treatment to patients faster.

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"This is the second consecutive year that Bristol-Myers Squibb employees are making this remarkable effort for Coast 2 Coast 4 Cancer. Most of our cyclists are riding for people they know whose lives have been touched by this disease, and the teams’ dedication reflects our commitment to helping cancer patients across the country," said Teresa Bitetti, senior vice president, U.S. Oncology, Bristol-Myers Squibb. "The strength and passion of our employees make it possible for us to strive toward changing the way cancer is treated, and those same qualities inspire us to reach the Coast 2 Coast 4 Cancer finish line on behalf of the cancer community."

In addition to the Coast 2 Coast 4 Cancer Ride, Bristol-Myers Squibb has a significant history of providing support to Stand Up To Cancer to advance cancer research, awareness and patient care. "We are very impressed by the Bristol-Myers Squibb employees who are cycling this year and are grateful for their support of the Stand Up To Cancer movement," said Stand Up To Cancer Co-Founder Katie Couric. "Their commitment to our dream of making every person diagnosed with cancer a survivor is inspiring for all of us, and we are proud to be a part of the larger Coast 2 Coast 4 Cancer team."

Last year, Coast 2 Coast 4 Cancer riders raised $359,000 for Stand Up To Cancer research and this year’s ride is even more ambitious, with six teams of 11-14 riders each, covering approximately 450 miles per team. "I am riding for my mom who battled cancer. She was my best friend and if I have achieved anything in this life, it is because of her. This race is about the patients we serve, their families, their doctors and their nurses who fight every day for them," said E. G. "Bubba" Klugh, Bristol-Myers Squibb, Little Rock, AR. "The training leading up to our ride has been strenuous, but the discipline required is part of what makes Coast 2 Coast 4 Cancer so meaningful to the riders because we are riding for patients. We would all ride as far as it takes – for them."

For more information on how you can show your support for Coast 2 Coast 4 Cancer, visit www.cancerbikeride.org.

On September 10, 2015 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies,reported that the Farrah Fawcett Foundation honored Advaxis with the Foundation’s inaugural "Medical Visionary Angel Award" on September 9, 2015, at the Wallis Annenberg Center for the Performing Arts in Beverly Hills, Calif (Press release, Advaxis, SEP 10, 2015, View Source [SID:1234507441]). The event benefitted the Farrah Fawcett Foundation / Stand Up To Cancer (SU2C) research team on HPV-related cancers and celebrated the life and legacy of actress Farrah Fawcett and her commitment to fund research for a cure for anal cancer, which took her life in 2009.

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Advaxis also announced plans to commence enrollment this fall of a Phase 2 clinical study of Advaxis’s lead investigational Lm Technology immunotherapy, axalimogene filolisbac (ADXS-HPV), in metastatic anal cancer. The study is to be called FAWCETT (Fighting Anal-Cancer with CTL Enhancing Tumor Therapy), in honor of Farrah Fawcett, and will be the company’s second Phase 2 study of axalimogene filolisbac in anal cancer. The two-part, single-arm, open-label study is designed to evaluate the efficacy and safety of axalimogene filolisbac as monotherapy in patients with human papillomavirus (HPV)-associated metastatic anal cancer who have received at least one prior treatment regimen for advanced disease.

"We would like to thank the Farrah Fawcett Foundation for this honor," said Daniel J. O’Connor, President and Chief Executive Officer of Advaxis. "While there is still so much work to be done in this space, we are unwavering in our commitment to bring new treatments to market in anal cancer and other underserved HPV-associated cancers."

The FAWCETT study will enroll approximately 31 patients in Part A and approximately 24 patients in Part B. The primary efficacy endpoints include overall response rate and progression-free survival, and the secondary endpoints include evaluation of overall response rate and the safety/tolerability of the dose. Further information about the Phase 2 study can be found on ClinicalTrials.gov, using Identifier NCT02399813.

The Farrah Fawcett Foundation’s "Medical Visionary Angel Award" is intended for companies that have demonstrated an outstanding commitment to innovative research in anal cancer and HPV-related cancers. Advaxis was selected for the award due to the company’s collaboration with Brown University Oncology Group on a Phase 1/2 clinical study conducted by Howard Safran, M.D., investigating axalimogene filolisbac in HPV-associated locally advanced anal cancer. The preliminary data showed treatment with axalimogene filolisbac indicated a clinical complete response and no recurrence in all 10 patients who completed the treatment regimen.

"When the Farrah Fawcett Foundation first invested in the cancer research at Brown University, we did so with great enthusiasm, knowing the work of Dr. Safran and appreciating the collaboration with Advaxis," said Alana Stewart, President and CEO of the Farrah Fawcett Foundation. "Now we are proud to honor Advaxis for their continued research on underserved cancers and breakthrough discoveries in the treatment of anal cancer and other HPV-associated cancers."

The Brown University Oncology Group study has the potential to transition into a pivotal Phase 2/3 clinical trial in collaboration with the Radiation Therapy Oncology Group (RTOG) Foundation and NRG Oncology to evaluate the safety and efficacy of axalimogene filolisbac with or without chemotherapy and radiation as adjuvant treatment in high-risk, locally advanced anal cancer.

About the Farrah Fawcett Foundation

The mission of the Farrah Fawcett Foundation is to provide funding for cutting edge cancer research, to support prevention and awareness, and to help those struggling with cancer today. Farrah Fawcett was diagnosed with anal cancer in 2006 and established the Foundation before her death in 2009. For more information, visit www.thefarrahfawcettfoundation.org and follow them on Facebook.

About Anal Cancer

Anal cancer is a fairly rare form of cancer in the United States, but the number of new anal cancer cases has been rising for years. The risk of being diagnosed with anal cancer in one’s lifetime is about 1 in 500. According to the American Cancer Society, approximately 7,270 new cases of anal cancer were diagnosed and about 1,010 people died of the disease in 2014.

About Axalimogene Filolisbac

Axalimogene filolisbac (ADXS-HPV) is Advaxis’s lead Lm Technology immunotherapy candidate for the treatment of HPV-associated cancers and is in clinical trials for three potential indications: invasive cervical cancer, head and neck cancer, and anal cancer. In a completed randomized Phase 2 study in recurrent/refractory cervical cancer, axalimogene filolisbac showed apparent prolonged survival, objective tumor responses, and a manageable safety profile alone or in combination with chemotherapy, supporting further development of the company’s Lm Technology.

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).