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Arrowhead Nominates ARC-HIF2 As First RNAi Therapeutic Candidate Using Extrahepatic DPC™ Delivery System

On September 11, 2015 Arrowhead Research Corporation (NASDAQ: ARWR), a biopharmaceutical company developing targeted RNAi therapeutics, reported that it has nominated ARC-HIF2 as its first therapeutic candidate delivered using a new Dynamic Polyconjugate (DPC) designed to target tissues outside of the liver (Press release, Arrowhead Research Corporation, SEP 11, 2015, View Source [SID:1234507456]). Arrowhead believes that ARC-HIF2, which uses RNA interference to silence transcription factor hypoxia-inducible factor 2α (HIF-2α), is a promising new candidate for the treatment of clear cell renal cell carcinoma (ccRCC).

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The company will present preclinical data at the European Cancer Congress 2015 (ECC2015) in Vienna on September 27 in a session starting at 16:45 CEST. In a poster titled "HIF-2α targeting with a novel RNAi delivery platform as therapy for renal cell carcinoma," (abstract #353), Arrowhead scientists will show data suggesting that HIF-2α inhibition through RNA interference may significantly impact late stage ccRCC progression. The company is in the process of manufacturing scale up to allow for initiation of IND-enabling studies. Timing for anticipated regulatory submission will be announced in the future.

"Preclinical data using our new extrahepatic DPC delivery system has been very promising. We think the ability to target tissues outside of the liver, including tumors, opens additional opportunities for Arrowhead to develop differentiated RNAi-therapeutics that address numerous diseases without adequate treatment options," said Christopher Anzalone, Ph.D., Arrowhead’s president and chief executive officer. "This is an important milestone for Arrowhead and we look forward to continued development of the DPC delivery platform and product candidates based on it."

ARC-HIF2 is designed to inhibit the production of HIF-2α, which has been linked to tumor progression and metastasis in ccRCC. ARC-HIF2 employs a novel extrahepatic-targeted DPC that comprises a membrane active polymer to promote RNAi trigger endosomal release, an active ligand that targets the DPC to tumor cells, reversible masking to prevent polymer activity prior to cellular uptake, and an RNAi trigger to HIF-2α conjugated directly to the DPC.

Using ARC-HIF2 in a preclinical ccRCC tumor model, mice treated with weekly injections led to greater than 80% knockdown of HIF-2α mRNA in tumors. Furthermore, tumors from treated mice exhibited statistically significant reductions in size and weight, extensive tumor cell death, reduction in the tumor-expressed VEGF-A biomarker, and destruction of the blood vessels feeding the tumors.

Therapies for metastatic ccRCC including agents that target the VEGF/VEGFR or mTor signaling pathways, which are validated cancer targets, have become the standard-of-care and have improved patient outcomes. However, since emergence of resistance to these agents is common, novel therapies targeting alternative pathways are needed for patients with resistant tumors. Arrowhead believes that HIF2α is an attractive target for intervention because over 90% of ccRCC tumors express a mutant form of the Von Hippel-Landau protein that is unable to degrade HIF-2α, leading to its accumulation during tumor hypoxia and promoting tumor growth.

An abstract of the data to be presented at ECC2015 is available on the conference website at www.europeancancercongress.org/.

Kite Pharma Receives Positive Opinion for Orphan Drug Designation in the European Union for KTE-C19, Kite’s Lead Cancer T-Cell Immunotherapy

On September 11, 2015 Kite Pharma, Inc. (Kite) (Nasdaq:KITE) reported that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending KTE-C19 for designation as an orphan medicinal product for the treatment of PMBCL and MCL (Press release, Kite Pharma, SEP 11, 2015, View Source [SID:1234507454]). KTE-C19 is an investigational therapy in which a patient’s T cells are genetically engineered to express a chimeric antigen receptor (CAR) designed to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias. No other product candidate currently has orphan drug designation for the treatment of PMBCL in the EU. Kite previously received orphan drug designation for KTE-C19 for the treatment of diffuse large B‑cell lymphoma (DLBCL) in both the US and the EU.

"The positive opinion for EU orphan designation for PMBCL and for MCL aligns with Kite’s leadership in and broad commitment to delivering innovative therapies that have the potential to transform the lives of cancer patients around the world," said Arie Belldegrun, M.D., FACS, Chairman, President and Chief Executive Officer of Kite. "We are conducting a Phase 1/2 clinical trial of KTE-C19 in patients with refractory, aggressive non-Hodgkin lymphoma, including DLBCL and PMBCL, and plan to report initial topline results from the Phase 1 portion of the trial later this year."

The COMP adopts an opinion on the granting of orphan drug designation, after which the opinion is submitted to the European Commission for endorsement of the opinion. Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU, and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.

BioCancell Receives FDA Fast Track Designation for BC-819 for Treatment of Bladder cancer Patients

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.

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."

NanoString Technologies Expands Immuno-Oncology Portfolio With Commercial Launch of Innovative nCounter RNA:Protein PanCancer Immune Profiling Panel

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.

"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.

8-K – Current report

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.