On January 16, 2015 Peregrine Pharmaceuticals reported on the presentation of clinical data related to the company’s immuno-oncology development program and its lead investigational immunotherapy drug candidate bavituximab at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (Press release Peregrine Pharmaceuticals, JAN 16, 2015, View Source [SID:1234501385]). This conference is being held January 15-17, 2015 at the Moscone West Convention Center in San Francisco, California.

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The clinical presentation titled: "A Phase II Study of Bavituximab and Sorafenib in Advanced Hepatocellular Carcinoma (HCC)" will be presented this afternoon by Adam Yopp, M.D., assistant professor of surgery at the University of Texas Southwestern Medical Center Dallas, Texas.

In this single-center, single-arm, open-label investigator-sponsored trial (IST), 38 patients with advanced HCC received bavituximab (3mg/kg) weekly and sorafenib (400 mg) twice daily until disease progression or toxicity. Data show that the combination of bavituximab and sorafenib is associated with an improved time to progression (TTP) of 6.7 months, a disease specific survival (DSS) of 8.7 months, a disease control rate (DCR) of 58% (22 out of 38 patients) and a 4-month progression-free survival (PFS) of 62%. Two patients (5%) achieved a partial response according to Response Evaluation Criteria In Solid Tumors (RECIST). The secondary endpoint of median overall survival (OS) was 6.2 months. The combination of bavituximab and sorafenib was well-tolerated in patients with advanced HCC with no indications of autoimmune adverse events that have been seen with other checkpoint immunotherapies.

"These clinical outcomes of time to progression, disease control rate and 4-month progression-free survival are quite encouraging, especially in this heavily pretreated patient cohort with very poor prognosis due to their unfavorable disease biology including a high rate of macrovascular invasion," said Dr. Yopp. "I was also pleased to see an extended tail in the survival curve that is typical of emerging immunotherapies for cancer. The positive data from this study should be considered as rationale for future randomized trials to further evaluate the potential of bavituximab in liver cancer."

"These data, along with recently reported translational data from this study, continue to build the knowledge base for the bavituximab clinical program and, in particular, highlight the potential immunotherapeutic synergies of bavituximab and sorafenib. We agree with Dr. Yopp that these data warrant further clinical evaluation of this combination in later stage clinical trials," said Joseph Shan, vice president of clinical and regulatory for Peregrine. "We continue to build value in the bavituximab program across multiple programs including the execution of the SUNRISE Phase III trial in second-line, non-small cell lung cancer and from data generated from this and other investigator-sponsored trials as well as other ongoing clinical trials. We look forward to new clinical collaborations with the goal of further exploring the utility of bavituximab in combination with other immuno-oncology drugs."

Liver IST Clinical Translation Data

Recently presented translational data of six patients from this trial show that half of the patients evaluated had an increase in tumor fighting immune cells following one cycle of bavituximab treatment, similar to what has been shown for PS-targeting antibodies in multiple preclinical cancer models. In addition, the increase in immune response was associated with patients that remained on study treatment for longer time periods, suggesting the possibility of a clinically meaningful anti-tumor immune response. Three of the six patients evaluated had increased infiltration of activated tumor-fighting T-cells (CD8) into the tumor microenvironment which correlated with a prolonged time to disease progression. In addition, these responding patients initially expressed lower levels of PD-1 positive cells, an established marker of T-cell activation and disease outcome, prior to the initiation of therapy that was followed by a measurable rise post bavituximab treatment.

On January 15, 2015 Amgen reported on new data from the Phase 2 PEAK and Phase 3 PRIME studies that support the first-line use of Vectibix (panitumumab) in combination with FOLFOX, an oxaliplatin-based chemotherapy regimen, in patients with wild-type RAS (absence of exons 2, 3, or 4 KRAS or NRAS mutations) metastatic colorectal cancer (mCRC) (Press release Amgen, JAN 15, 2015, View Source;p=RssLanding&cat=news&id=2008116 [SID:1234501347]). The data will be presented during a poster session at the 2015 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium taking place in San Francisco from January 15 to 17.

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In an exploratory analysis from the PEAK study (abstract #660), treatment with Vectibix compared to bevacizumab (Avastin) resulted in a significantly higher proportion of patients with earlier tumor shrinkage at week eight (64 percent vs. 45 percent, respectively; 95 percent CI, p=0.0232), and among responding patients, a significantly longer duration of response (11.4 vs. 8.5 months, respectively; 95 percent CI, p=0.0142) and greater depth of response (65 percent vs. 46 percent, respectively; p=0.0007). Overall response rates (ORR) appeared to be similar between Vectibix and bevacizumab. This is consistent with observed overall survival (OS) and progression-free survival (PFS) rates, and with data previously reported. The safety profile of Vectibix was consistent with previously reported studies.

"These analyses help deepen our understanding of how Vectibix works when added to a standard first-line chemotherapy for the treatment of wild-type RAS metastatic colorectal cancer," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Our Vectibix clinical trial program continues to yield new insights regarding biomarkers and drug sequencing."

While the primary analysis from PEAK showed similar ORR between the Vectibix- and bevacizumab-based regimens, this exploratory analysis demonstrates that Vectibix produces early, sustained anti-tumor activity, which may in part explain the OS and PFS benefits seen with Vectibix versus bevacizumab in this trial.

A separate analysis from the Phase 3 PRIME study (abstract #537), demonstrated that there were no significant differences in quality of life among patients treated with Vectibix plus FOLFOX versus FOLFOX alone despite the incidence of adverse events associated with each treatment regimen. The quality of life analysis included a scale that assessed mobility, self-care, usual activities, pain/discomfort and anxiety/depression.

Colorectal cancer is the third most common cancer found in both men and women in the U.S. and is the second leading cause of cancer deaths. Approximately 1.2 million cases of colorectal cancer are expected to occur globally each year.

On January 12, 2015 Tokai Pharmaceuticals reported that it has entered into an agreement with the Johns Hopkins University related to the development of a companion diagnostic to determine the AR-V7 status of patients with castration-resistant prostate cancer (CRPC) for use with the Company’s lead product, galeterone, which is in development for the treatment of AR-V7 positive metastatic CRPC (Filing 8-K , Tokai Pharmaceuticals, JAN 15, 2015, View Source [SID:1234501348]).

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Under the agreement, the Company has obtained an exclusive, worldwide license from the Johns Hopkins University to patent applications and know-how covering an assay that has been used to determine the AR-V7 status of prostate cancer patients.

AR-V7 positive prostate tumors express a truncated form of the androgen receptor (AR). These truncated ARs are missing the C-terminal end of the receptor that contains the ligand binding domain, which is known as C-terminal loss. The AR splice variant AR-V7 is the most prevalent of the splice variants that cause C-terminal loss.

Clinical data from a prospective trial at the Johns Hopkins University as well as retrospective analyses of studies at MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center have shown that AR-V7 specifically and C-terminal loss generally is associated with poor responsiveness to Zytiga (abiraterone acetate) and Xtandi (enzalutamide), two commonly used oral therapies for metastatic CRPC. The Company believes that galeterone has the potential to treat patients with AR-V7 based on data from preclinical studies and retrospective data in patients with C-terminal loss from the Company’s Phase 2 ARMOR2 trial of galeterone.

"We are pleased to have formalized the agreement with the Johns Hopkins University to support our ongoing AR-V7 companion diagnostic program. This license adds to the intellectual property around galeterone and is a critical milestone in the development of the companion diagnostic that will be used in our 148 patient Phase 3 ARMOR3-SV trial scheduled to begin in the first half of this year," stated Jodie Morrison, president and chief executive officer of Tokai Pharmaceuticals. "It is our hope that future availability of a companion diagnostic will allow prostate cancer patients and their physicians to make more informed decisions regarding their care."

In addition, the Company announced that it has entered into an agreement with Qiagen N.V. under which Qiagen will develop a non-invasive companion diagnostic utilizing an array of novel technologies for use with galeterone. The agreement formalizes an existing collaboration under which work has been ongoing.

On January 14, 2015 NantCell, LLC, a subsidiary of NantWorks, LLC, focusing on the discovery and development of immunology based innovative treatments for diseases through cell-based treatments at the molecular level, reported that it has entered into a licensing agreement with Amgen Inc. for AMG 479 (ganitumab), previously in Phase 3 development (Press release, NantWorks, JAN 14, 2015, View Source [SID:1234511990]). Under the agreement, NantCell acquired the exclusive rights to develop and commercialize worldwide, excluding Japan, AMG 479, a fully human monoclonal antibody that targets Type 1 insulin-like growth factor receptor (IGF-1R), a promising target for cancer therapy. Financial terms were not disclosed.

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"We are pleased to enter into this agreement with Amgen," said Dr. Patrick Soon-Shiong, founder and chief executive officer of NantCell. "We acquired an immuno-oncology compound in late-stage development, with the potential to address a number of cancers affecting significant patient populations. This month marks the 10 year anniversary of the FDA approval of Abraxane, the first protein-based chemotherapy delivery vehicle now approved in multiple countries worldwide for breast, lung and pancreatic cancer. It is our belief that the future of cancer care will involve combination therapy with low dose, metronomic use of multiple chemotherapeutic agents, but combined also with immuno-oncology molecules, or with engineered killer cells targeted at the proteomic profile of the specific tumor, regardless of the anatomical type. The development of antibody molecules such as IGF-1R will require next generation sequencing at the proteomic level with a deep level of molecular interrogation to establish the appropriate combination with other drugs."

Patients entering clinical trials conducted by NantCell would be identified after a comprehensive "omic" analysis from DNA, RNA and protein, and treated on the resulting molecular profile to maximize clinical outcome and minimize side effects.

"We are pleased to collaborate with NantCell and NantWorks in their mission to establish a new level of quantitative predictability of patient selection," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Their integrated suite of technologies may help to improve patient selection and further clinical development of AMG 479."

Scientific and medical research suggest that IGF-1R plays a role in the development and progression of many cancers, possibly due to its anti-apoptotic properties, which allow cancerous cells to resist the cytotoxic properties of chemotherapeutic drugs or radiation therapy. The novel IGF-1R antibody inhibits cancer cell proliferation through disruption of the P13K/Akt and MAPK pathways. Signaling through IGF-1R plays an important role in the regulation of cell growth and survival, and has been shown to be a critical promoter of anchorage independent growth, a well-recognized mechanism for malignancy.

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About NantCell

NantCell, a wholly-owned subsidiary of NantWorks, LLC, is an immuno-oncology company focused on the discovery of innovative antibody, T cell and NK cell based treatments by developing molecularly targeted therapeutics, based on the proteomic profile of the patient’s tumor, independent of the cancer’s anatomical type. Dr. Patrick Soon-Shiong, the creator of Abraxane and the founder of the nab technology platform established NantCell to develop a pipeline of human antibodies and inhibitors of proteins which drive tumor growth and pursue Chimeric Receptor Antigen platforms in both T and NK cells. NantCell’s mission is to make obsolete the standard method of clinical trial design of "trial and error" and replace it with a level of quantitative predictability based on both the genomic and proteomic profile performed a priori. The Company will tap into comprehensive "omic" analytic tools and "big data" generated from supercomputing to develop molecularly designed drugs in this era of genomics and proteomics and identify patients and their tumor signature at the most granular cellular, DNA and protein levels. Patients entering clinical trials would be identified after a comprehensive "omic" analysis from tissue to cell to DNA to RNA to protein to peptide to drug, and tested based on this molecular profile to maximize clinical outcome and minimize side effects. For more information please visit www.nanthealth.com and follow Dr. Soon-Shiong on Twitter@solvehealthcare.

About NantWorks

NantWorks, LLC, founded by renowned physician scientist and inventor of the first human nanoparticle chemotherapeutic agent Abraxane, Dr. Patrick Soon-Shiong, is the umbrella organization for the following entities: NantHealth, NantMobile, NantMedia, NantOmics, NantBioScience, NantCell, NantPharma, NantCapital and NantCloud. Fact-based and solution-driven, each of NantWorks’ division entities operates at the nexus of innovation and infrastructure.

The core mission of NantWorks is convergence: to develop and deliver a diverse range of
technologies that accelerates innovation, broaden the scope of scientific discovery, enhance
groundbreaking research, and improve healthcare treatment for those in need. NantWorks is building an integrated fact-based, genomically-informed, personalized approach to the delivery of care and the development of next generation diagnostics and therapeutics.

On January 14, 2015 Roche licenses additional EGFR pathway-related intellectual property to QIAGEN(Press release Hoffmann-La Roche, JAN 14, 2015, View Source [SID:1234501342]).

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Roche (SIX: RO, ROG; OTCQX: RHHBY) reported that the company has entered into an agreement with QIAGEN that includes a provision of non-exclusive licenses to recently granted Roche patents pertaining to the detection of mutations in the EGFR pathway (including in the KRAS gene). Financial details were not disclosed.

The licenses apply to testing products which detect these mutations using molecular techniques including PCR, next generation sequencing (NGS) and other applications to aid in identification of cancer patients eligible for treatment with certain tyrosine kinase inhibitors. The licenses can be applied to existing and future products.

"As a leader in molecular assay development, we are pleased to provide licenses to the applicable patents so that existing and new tests can support patient treatment decisions," said Paul Brown, Head of Roche Molecular Diagnostics. "Ensuring that assays that utilize Roche proprietary information are fully licensed is a key business strategy for us."

"We are pleased with the agreement, which expands our existing intellectual property portfolio covering more than 35 biomarkers and our deep intellectual property estate in EGFR-related testing, including KRAS- testing", said Dr. Achim Ribbe, Vice President Corporate Business Development Licensing. "As a global leader in personalized healthcare, QIAGEN is working with numerous pharmaceutical companies to develop and market molecular companion diagnostics that can help improve patient outcomes and better utilize healthcare resources."