On December 1, 2015 Following an independent Data Monitoring Committee (DMC) meeting, OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported that its Phase 3 AFFINITY trial is continuing based on the pre-planned interim futility analysis of the intent-to-treat (ITT) population (Press release, OncoGenex Pharmaceuticals, DEC 1, 2015, View Source [SID:1234508379]). In the final safety review, no new safety issues were identified by the DMC. Both the DMC and OncoGenex remain blinded to all analyses and final results are expected in the second half of 2016, depending on timing of the event-driven final analysis.

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AFFINITY is designed to evaluate whether the investigational treatment custirsen, when combined with cabazitaxel, improves survival in men with metastatic castrate-resistant prostate cancer (CRPC) whose disease has progressed following treatment with docetaxel. The final AFFINITY efficacy analysis is designed to show a survival benefit with 85 percent power based on a hypothesized hazard ratio of 0.75.

Separately, an analysis of a prospectively defined subpopulation of men in the AFFINITY trial who had multiple poor prognostic risk factors revealed that the combination of custirsen and cabazitaxel did not meet the rigorous criteria required to demonstrate an improvement in overall survival (hypothesized hazard ratio ≤0.69, one-sided p value ≤0.015). This subpopulation was identified and evaluated based on a retrospective analysis of a previous Phase 3 trial of men with similar clinical features who experienced a reduced risk of death when custirsen was added to chemotherapy. In addition, OncoGenex pursued the evaluation of this subpopulation, independent of the ITT, in order to obtain an expedited approval for these patients with more aggressive disease.

"The overall survival endpoint of the entire AFFINITY trial remains clinically meaningful and its target hazard ratio of 0.75 is attainable with sufficient power to demonstrate a benefit from custirsen for men who are battling advanced prostate cancer," said Scott Cormack, President and CEO of OncoGenex. "We designed the AFFINITY trial so that the final analysis of the ITT population could stand alone as a Phase 3 submission to regulatory agencies regardless of the outcome of the smaller subgroup."

As part of the Phase 3 development program for custirsen, OncoGenex continues with its ENSPIRIT clinical trial in patients with non-small cell lung cancer (NSCLC). The trial is evaluating the ability of custirsen, in combination with docetaxel treatment as second-line chemotherapy, to extend survival in patients with NSCLC. Based on current enrollment projections, ENSPIRIT results could be available in the second half of 2016.

The company also expects several additional clinical trial milestones in 2016 with its other lead product candidate apatorsen, which include:

Announcing results for the primary progression-free survival endpoint of the Phase 2 Spruce trial in advanced NSCLC
Announcing results of the Phase 2 Borealis-2 trial in patients with metastatic bladder cancer
Completing enrollment in the Phase 2 Pacific trial in metastatic CRPC

Conference Call Details
OncoGenex will host a conference call at 4:30 p.m. Eastern Time today, Tuesday, December 1, 2015, to discuss today’s news. A live event will be available on the Investor Relations section of the OncoGenex website at www.OncoGenex.com. Alternatively, visitors may access the live conference call by dialing (877) 606-1416 (U.S. & Canada) or (707) 287-9313 (International). A webcast replay will be available approximately two hours after the call and will be archived on www.OncoGenex.com for 90 days.

About the AFFINITY Trial
The Phase 3 AFFINITY trial is an international, randomized, open-label study designed to evaluate whether custirsen, when combined with cabazitaxel, has the potential to improve survival outcomes for metastatic CRPC patients whose disease has progressed following treatment with docetaxel. The two primary objectives of the study are overall survival in the ITT population and overall survival in men who had two or more pre-defined clinical prognostic features of metastatic CRPC.

Both groups received cabazitaxel in combination with weekly custirsen or cabazitaxel alone, and treatment continued until disease progression, unacceptable toxicity or completion of 10 cycles. The AFFINITY trial enrolled 630 men with metastatic CRPC at 95 sites throughout North America, Europe, Russia and Australia.

For more information on the AFFINITY trial, please visit ClinicalTrials.gov (NCT01578655).

About prostate cancer
More than 220,000 new cases of prostate cancer are diagnosed each year and prostate cancer is the second leading cause of cancer-related deaths in the United States among men. Approximately 50% of patients with clinically localized prostate cancer are estimated to progress despite initial treatment. Metastatic CRPC often spreads to the bone, making it difficult for some men to perform even the simplest daily activities, like standing up and walking around. Prostate cancer deaths are usually the result of metastatic CRPC, which has a median survival of less than two years.

On Dec. 1, 2015 MabVax Therapeutics Holdings, Inc. (OTCQB: MBVX), a clinical-stage oncology drug development company, reported it has filed an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA) for the Company’s lead fully human antibody product HuMab 5B1 as a therapeutic agent (Press release, MabVax, DEC 1, 2015, View Source [SID:1234508375]). Subject to FDA acceptance, MabVax plans to initiate the Phase I clinical trial early in 2016.

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The planned Phase I trial will evaluate the safety, tolerability and pharmacokinetics of HuMab 5B1 as a single agent or in combination with the current standard of care chemotherapy regimen in subjects with metastatic pancreatic cancer. The first cohort of patients to be enrolled in the planned clinical trial will be enrolled in a traditional dose escalation regimen to assess safety and determine the optimal dose of the antibody. A second patient cohort will establish the safety and optimized dose of the antibody when administered with standard of care chemotherapy and a third patient cohort will be administered the optimized dose of antibody as a single agent for the treatment of patients with advanced cancer.

David Hansen, MabVax’s President and Chief Executive Officer, said, "The filing of the first of two planned INDs for our novel HuMab 5B1 antibody is a significant achievement for MabVax. Pending FDA acceptance of the IND, we will begin the dose escalation portion of this Phase I trial as early in 2016 as possible and anticipate reporting on the early safety assessment and determination of a maximum tolerated dose in mid-year 2016. Achievement of this important interim milestone will enable us to move into the combination therapy and monotherapy portions of the trial where we could learn much more about the pharmacological effects of this new therapy. The milestone could also have a positive impact on our future commercial and corporate development activities. We currently anticipate having full enrollment of all three patient cohorts sometime before the end of 2016."

MabVax plans to file a second IND application this month for its HuMab 5B1-based PET imaging agent and, subject to FDA acceptance, will begin this Phase I trial as early as possible in 2016. When the antibody is combined with a radio-label as a novel PET imaging agent, the 89Zr-HuMab 5B1 product has demonstrated high image resolution of tumors in established xenograft animal models, making it attractive as a potential companion diagnostic for the HuMab 5B1 therapeutic product.

"We believe the data generated in the early portions of these two Phase I trials will help demonstrate the initial safety, targeting specificity, and utility of the HuMab 5B1 antibody in this devastating disease," added Mr. Hansen. "We are excited about the potential applicability of our dual-product development approach in other cancers with HuMab 5B1, as well as with follow-on antibodies under development at MabVax."

About HuMab 5B1:

MabVax’s HuMab 5B1 antibody is fully human and was discovered from the immune response of cancer patients vaccinated with an antigen-specific vaccine during a Phase I trial at Memorial Sloan Kettering Cancer Center. In preclinical research, the 5B1 antibody has demonstrated high specificity and affinity, and has shown potent cancer cell killing capacity and efficacy in animal models of pancreatic, colon and small cell lung cancers. The antigen the antibody targets is expressed on more than 90% of pancreatic cancers making the antibody potentially broadly applicable to most patients suffering from this type of cancer.

On December 01, 2015 Advaxis, Inc. (NASDAQ:ADXS), a clinical-stage biotechnology company developing cancer immunotherapies, reported that the European Medicines Agency (EMA) granted Orphan Drug Designation for ADXS-HER2 for the treatment of osteosarcoma (Press release, Advaxis, DEC 1, 2015, View Source [SID:1234508369]).

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"Receiving Orphan Drug Designation from the EMA is another significant step forward for Advaxis as we continue to advance ADXS-HER2," said Daniel J. O’Connor, CEO of Advaxis. "We recently initiated our first-in-human study of our lead Lm Technology immunotherapy product for HER2 expressing solid tumors and we hope to expand that trial into four HER2 expressing tumor types, including breast, gastric, esophageal and osteosarcoma."

Orphan Drug Designation in the EU is granted to drugs or biologics that treat a life-threatening or chronically debilitating rare disease affecting fewer than five in 10,000 individuals in the European Union. Products receiving orphan drug designation are eligible to receive market exclusivity for a period of up to ten years, as well as development incentives such as regulatory and protocol assistance and scientific advice.

About HER2 Expressing Solid Tumor Cancers
Human epidermal growth factor receptor 2 (HER2) is overexpressed in a percentage of solid tumors such as breast, gastric, bladder, brain, pancreatic, ovarian and pediatric bone cancer (osteosarcoma). The American Cancer Society estimates that in 2015 in the United States alone there will be 231,840 new cases of invasive breast cancer; 24,590 new cases of gastric cancer; 74,000 new cases of bladder cancer; 22,850 new cases of brain/spinal cancer; 48,960 new cases of pancreatic cancer; 21,290 new cases of ovarian cancer; and 207 new cases of pediatric osteosarcoma. HER2 expression is associated with more aggressive disease, increased risk of relapse and decreased overall survival, and is an important target for immunotherapy.

About ADXS-HER2
ADXS-HER2 is an Lm Technology immunotherapy product candidate being developed by Advaxis to target HER2 expressing cancers. ADXS-HER2 has received orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of osteosarcoma. Advaxis is developing ADXS-HER2 for both human and animal health, and has seen encouraging data in canine osteosarcoma, which is considered a model for human osteosarcoma. Advaxis has licensed ADXS-HER2 and three other immunotherapy constructs to Aratana Therapeutics, Inc. for the development of pet therapeutics.

On December 1, 2015 Adaptimmune Therapeutics plc (Nasdaq: ADAP), a leader in the use of TCR engineered T-cell therapy to treat cancer, and Seattle-based Universal Cells Inc., a genome editing company developing universal donor stem cells, reported that they have entered into a collaboration and exclusive license agreement for the development of allogeneic T-cell therapies (Press release, Adaptimmune, DEC 1, 2015, View Source [SID:1234508368]).

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With Universal Cells’ proprietary gene editing technology, Adaptimmune intends to develop affinity enhanced donor T-cells that are universally applicable. The enhanced T-cell technology involves selective engineering of cell surface proteins (TCRs and class I and class II HLA proteins), without the use of nucleases, to develop universal T-cell products. Adaptimmune and Universal Cells are planning to develop these off-the-shelf allogeneic affinity-enhanced T-cell therapeutics to treat large patient populations.

"This collaboration marks another step towards our goal of providing innovative immunotherapeutics to patients suffering from cancer," said Dr. Helen Tayton-Martin, Adaptimmune’s Chief Operating Officer.
"Our proprietary platform for TCR identification, affinity enhancement and safety testing is already best in class, and we set high standards for collaborations. We believe that Universal Cells’ platform for generating universal donor cells is also best in class and provides us with a great opportunity to test the feasibility of a longer term allogeneic product, thus allowing large numbers of patients to be treated from a single cell line."

"We are very excited about working with Adaptimmune. By partnering with the world leader in TCR engineered T-cell immunotherapies, we are poised to develop a scalable, safe, and efficacious product with the potential to revolutionize cancer immunotherapy," said Claudia Mitchell, Chief Executive Officer of Universal Cells. "This partnership will combine Universal Cells’ nuclease-free genome editing platform with Adaptimmune’s unique expertise in TCR engineering to develop a first-in-class therapeutic product based on our universal donor cells."

Under the terms of the agreement, Universal Cells will grant to Adaptimmune an exclusive, sublicensable, worldwide license to use, sell, supply, manufacture, import, and develop products and services utilizing Universal Cells’ technology within the T-cell immunotherapy field. Universal Cells will receive an upfront license and start-up fee of $5.5 million, and will be eligible for up to $41 million in milestone payments for certain development and product milestones. Universal Cells would also receive a profit-share payment for the first product, and royalties on sales of other products utilizing its technology.

On December 1, 2015 ImmunoCellular Therapeutics, Ltd. ("ImmunoCellular") (NYSE MKT: IMUC) reported the establishment of an agreement with the European Organisation for Research and Treatment of Cancer (EORTC) for the phase 3 registrational trial of ICT-107 in patients with newly diagnosed glioblastoma (GBM) (Press release, ImmunoCellular Therapeutics, DEC 1, 2015, View Source [SID:1234508367]).

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The EORTC contributed to the trial design and will assist ImmunoCellular Therapeutics in facilitating accrual of patients for this trial in Europe.

Prof. Michael Weller of the University Hospital Zurich, Chair of the EORTC Brain Tumor Group, and coordinator of this study commented "The EORTC is a leader in enabling multi-disciplinary approaches to investigate strategic therapeutic questions through clinical and translational research. We aim to improve the understanding of disease and help advance innovative therapies in cancer care for the benefit of patients. We are pleased to collaborate with ImmunoCellular to conduct the phase 3 registrational trial of ICT-107, a potentially promising cancer immunotherapeutic approach to treating brain cancer."

"Working with the EORTC is a key step in our strategy to establish a network of relationships within the clinical oncology community to support the conduct of the ICT-107 Phase 3 trial," said Andrew Gengos, ImmunoCellular’s Chief Executive Officer. "For several months, we have been working with prominent cancer cooperative groups in the US and Europe to refine and finalize the trial design, gain their endorsement and secure their help in executing a high quality trial. With EORTC’s support, we believe that we can access large numbers of affiliated trial sites which will facilitate patient accrual. We anticipate patient enrollment in the phase 3 trial to begin within the next few weeks."