On September 9, 2015 Peregrine Pharmaceuticals, Inc. (NASDAQ:PPHM) (NASDAQ:PPHMP), a biopharmaceutical company focused on developing therapeutics to stimulate the body’s immune system to fight cancer, reported financial results for the first quarter of fiscal year (FY) 2016 ended July 31, 2015, and provided an update on its advancing clinical pipeline and other corporate developments (Press release, Peregrine Pharmaceuticals, SEP 9, 2015, View Source [SID:1234507437]).

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Highlights Since April 30, 2015:
"Over the years, Peregrine’s foundational science and positive clinical results have consistently pointed to bavituximab’s potential as a high-value, next-generation anti-cancer agent," said Steven W. King, president and chief executive officer of Peregrine. "In the last three months, these achievements have compelled others to align with us as we continue to develop bavituximab. In May, Peregrine announced an exciting collaboration with Memorial Sloan Kettering Cancer Center to evaluate combinations of bavituximab with other checkpoint inhibitors and immune stimulatory agents for the purpose of developing new and increasingly effective anti-cancer treatments. Only three months later, we announced a collaboration with AstraZeneca to clinically evaluate bavituximab in combination with AstraZeneca’s investigational anti-PD-L1 immune checkpoint inhibitor, durvalumab (MEDI4736) in multiple solid tumors. These collaborations with world leaders in immuno-oncology speak to the promise of bavituximab and validate our ever-growing enthusiasm for the investigational product. We look forward to advancing both of these programs and completing enrollment of our SUNRISE trial in the next few months."

Clinical Development Highlights
Peregrine and AstraZeneca entered into a cancer immunotherapy clinical trial collaboration to evaluate bavituximab in combination with AstraZeneca’s investigational anti-PD-L1 immune checkpoint inhibitor, durvalumab (MEDI4736). The planned Phase I/Ib trial will evaluate the safety and efficacy of bavituximab in combination with durvalumab in multiple solid tumors. Peregrine is working closely with AstraZeneca to finalize the trial design.

Phase III SUNRISE clinical trial in non-small cell lung cancer (NSCLC) continues to enroll patients and remains on track to complete patient enrollment by end of calendar year 2015.

Peregrine announced plans to expand the bavituximab clinical development program to include a Phase II trial to evaluate the combination of bavituximab and Opdivo (nivolumab), an anti-PD-1 antibody, in previously treated, metastatic NSCLC. This trial is expected to be initiated by the end of calendar year 2015.

Peregrine announced plans to expand the bavituximab clinical development program to include a Phase II/III trial to evaluate bavituximab with chemotherapy combinations in HER2-negative metastatic breast cancer. This trial is expected to be initiated by the end of calendar year 2015.

Supportive Research Highlights
Peregrine and Memorial Sloan Kettering Cancer Center entered into a research agreement to explore the potential of Peregrine’s proprietary PS-targeting antibody platform. The goal of the research is to identify effective treatments combining bavituximab with other checkpoint inhibitors or immune stimulating agents.

New data presented at the International Association for the Study of Lung Cancer’s (IASLC’s) World Conference on Lung Cancer (WCLC) from a translational study of bavituximab demonstrated the ability of bavituximab, alone or in combination with docetaxel, to induce signs of immune activation in non-small cell lung cancer (NSCLC) patient-derived tumor samples, particularly when there was negative PD-L1 expression in the tumor sample. These data further support the potential mechanistic synergies for bavituximab with chemotherapy and checkpoint inhibitors targeting the PD-1/PD-L1 pathway.

Summary data presented at the Combination Immunotherapy Strategies session at the 10th Annual Immunotherapy and Vaccine Summit (ImVacS), highlighted key findings from several recent bavituximab-focused studies including: the potential of bavituximab to shift the tumor microenvironment from immuno-suppressive in which tumors evade immune detection to a state of immune activation in which the immune system recognizes and fights the tumor; bavituximab’s potential to increase the number of activated CD8+ cells in the tumor, which stimulates PD-1 expression, potentially increasing the number of patients able to respond to PD-1 and PD-L1 targeting immunotherapies; and, results from several clinical and preclinical studies in a range of tumor types showing that bavituximab and bavituximab-like antibodies, in combination with conventional therapy, have consistently demonstrated estimated survival curves that plateau.

Data from preclinical studies presented at the 2015 ASCO (Free ASCO Whitepaper) annual meeting demonstrated the ability of the company’s PS-targeting antibodies to significantly increase the prevalence of tumor infiltrating CD8+ T-cells and immune-activating cytokines, while decreasing tumor-promoting macrophages and myeloid cells. These findings highlight the ability of the antibodies to enhance the anti-tumor effects of both chemotherapy and immune checkpoint inhibitors.

Avid Bioservices Highlights
Avid’s new manufacturing suite is fully constructed and the first internal pilot run is currently underway to verify all systems and equipment are properly functioning. Company plans to announce the launch of the new facility in the near term, allowing us to meet our internal manufacturing timelines as well as those of our third-party clients.

Contract manufacturing committed backlog reached $42 million from existing customers covering services to be completed in FY 2016 and into FY 2017.

Corporate Highlights
The European Patent Office (EPO) granted Patent Number 2,269,656, licensed to Peregrine titled "Selected Antibodies Binding to Aminophospholipids and their Use in Treatment, Such as Cancer." The patent covers bavituximab as a composition of matter and for use in therapy, such as for treating cancer including in combination with radiotherapy or chemotherapy, e.g., with docetaxel. This important patent expands upon the company’s intellectual property portfolio, which now numbers more than 140 worldwide issued patents and pending applications for the bavituximab oncology program.

Financial Results
Total revenues for the first quarter of FY 2016 were $9,671,000, compared to $5,496,000 for the same quarter of the prior fiscal year. The increase was primarily attributed to an increase in contract manufacturing revenue generated from Avid Bioservices.
Contract manufacturing revenue from Avid’s clinical and commercial biomanufacturing services provided to its third-party clients for the first quarter FY 2016 were $9,379,000, compared to $5,496,000 for the same quarter of the prior fiscal year. Peregrine expects third-party contract manufacturing revenue for FY 2016 to be between $30 and $35 million. In addition to providing biomanufacturing services to its third-party clients, Avid will continue to support the clinical and potential commercialization of bavituximab.

Total costs and expenses in the first quarter of FY 2016 were $23,425,000, compared to $18,667,000 in the first quarter of FY 2015. This increase was primarily attributable to current quarter increases in research and development expenses associated with the SUNRISE Phase III trial and increases in the cost of contract manufacturing associated with higher reported revenue. For the first quarter of FY 2016, research and development expenses were $13,918,000, compared to $10,201,000 for the first quarter of FY 2015. For the first quarter of FY 2016, cost of contract manufacturing was $4,608,000, compared to $3,583,000 for the first quarter of FY 2015.

Peregrine’s consolidated net loss attributable to common stockholders was $15,101,000, or $0.08 per share, for the first quarter of FY 2016, compared to a net loss attributable to common stockholders of $14,157,000, or $0.08 per share, for the same prior year quarter.

Peregrine reported $59,016,000 in cash and cash equivalents as of July 31, 2015 compared to $68,001,000 at fiscal year ended April 30, 2015.

More detailed financial information and analysis may be found in Peregrine’s Quarterly Report on Form 10-Q, which will be filed with the Securities and Exchange Commission today.

On September 9, 2015 Mirati Therapeutics, Inc. ("Mirati") (NASDAQ: MRTX), an oncology company focusing on genetic and epigenetic drivers of cancer, reported it will present data at the International Association of Lung Cancer (IASLC) 16th World Conference on Lung Cancer on the first non-small cell lung cancer (NSCLC) patient with AXL gene amplification enrolled in the MGCD265 Phase 1b expansion cohort (Filing, 8-K, Mirati, SEP 9, 2015, View Source [SID:1234507446]). Data will be presented showing the patient had a confirmed Partial Response (PR) based on RECIST criteria. Additionally, the Company announced a confirmed PR in a NSCLC patient with MET gene amplification who was enrolled in the MGCD265 expansion cohort.

"Out of four non-small cell lung cancer patients whom have had at least one scan in the ongoing MGCD265 expansion cohort, two patients have RECIST-confirmed PRs. Those PRs, together with tumor regressions seen in all four of these patients, demonstrate the potentially significant clinical benefit of MGCD265 in patients with lung cancer," said Charles M. Baum, M.D., Ph.D., President and CEO, Mirati. "The study is progressing well due to the enthusiasm of the clinical investigators, and this has resulted in increased screening and enrollment at the clinical trial sites. Currently, nine patients with MET or AXL genetic alterations have been enrolled in the study. In light of the dramatic response being presented in the patient with AXL gene amplification at today’s World Conference on Lung Cancer, we felt it was appropriate to provide an interim update on the program. As previously indicated, we will provide a more in-depth update when we have additional data."

NSCLC Patient with Axl Gene Amplification

The male patient was diagnosed with metastatic adenocarcinoma of the lung, with multiple tumors in both lungs which had spread to the lung cavity and lymph nodes. Prior to treatment with MGCD265, he had received multiple chemotherapies, as well as an experimental agent combined with chemotherapy, with the best response being disease progression. After 2 cycles of treatment with MGCD265, tumor imaging showed a PR with a tumor reduction of 42.3% compared to baseline. After 4 cycles of treatment, the PR was confirmed with a tumor reduction of 48.8% based on RECIST criteria. The patient, who remains on study in Cycle 7, also showed improvement in clinical symptoms. Prior to starting treatment with MGCD265, the patient was oxygen dependent. Shortly after treatment with MGCD265, he was off oxygen and able to ride his bike up to seven miles per day.

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"To our knowledge, this is the first reported case of an objective response in a patient with a tumor harboring AXL gene amplification," said Geoffrey Shapiro, Principal Investigator and Director of the Early Drug Development Center, Department of Medical Oncology, Dana-Farber Cancer Institute. "This response, coupled with the patient’s significant symptomatic improvement, provides clinical validation that AXL genomic alterations can result in oncogene addiction in patients with non-small cell lung cancer. We will continue to explore MGCD265, a potent kinase inhibitor, in patients with MET or AXL genomic alterations, in an effort to improve cancer treatment by targeting genetic drivers of cancer."

Data from the study will be presented on September 9, 2015 in an oral presentation titled, "Evaluation of the MET/Axl Receptor Tyrosine Kinase (RTK) Inhibitor MGCD265 in a Patient with Metastatic Non-Small Cell Lung Cancer (NSCLC) Harboring AXL Amplification" by Lynette Sholl, M.D, Assistant Professor, Translational Research Group, Brigham and Women’s Hospital. The presentation is part of the New Kinase Targets session, Treatment of Advance Diseases – NSCLC track (abstract # 3611) from 6:30 – 8:00 PM MT/5:30 – 7:00 PM PT in Colorado Convention Center, Four Seasons Ballroom F3+F4.

Interim Update on the Ongoing MGCD265 Phase 1b Expansion Cohort

MGCD265 is an inhibitor of the MET and Axl receptor tyrosine kinases which, when mutated or amplified, can be drivers of tumor growth. Preclinical data have shown that MGCD265 can potently inhibit tumor cell growth in vitro, and demonstrate marked tumor regression in tumor xenograft models exhibiting MET gene amplification and MET exon 14 deletions.

This multi-national, multi-site, open label, single agent study is designed to evaluate the safety, pharmacokinetics/pharmacodynamics and clinical activity of twice-daily MGCD265 in patients who have failed at least one prior therapy. The study continues to enroll patients with MET or AXL gene alterations. MGCD265 has been well tolerated at the recommended Phase 2 dose, which has demonstrated full inhibition of both MET and Axl tyrosine kinases, and is the only kinase inhibitor that we know of in clinical development that has demonstrated potent and selective inhibition of both MET and Axl.

As of September 1, 2015, 9 patients with genetic alterations in MET or AXL have been enrolled in the expansion cohort, including 7 with NSCLC and 2 with other solid tumors. The Company disclosed that 2 of the 4 NSCLC patients, who are currently evaluable (having had at least 1 on-treatment scan), have confirmed PRs based upon RECIST criteria, including the patient with AXL amplification highlighted above and a patient with MET gene amplification. Both patients remain on study. All 4 of the evaluable NSCLC patients showed clinically significant tumor regressions. Of the 9 patients enrolled, 7 remain on study for up to 8+ months.

About MET and Axl in NSCLC

MET is highly expressed in NSCLC tumors. Extensive preclinical and emerging clinical data indicate that MET is a driver of tumor growth when it is genetically altered by point mutations, exon 14 deletion mutations, and/or gene amplification in a significant fraction (6-7%) of NSCLC patients. MET gene amplification and MET mutations, including exon 14 deletion mutations, each exhibit the key characteristics of driver oncogenes in NSCLC.

Axl is over-expressed in patients with advanced NSCLC and has been associated with poor prognosis. Amplification and rearrangements of the AXL tyrosine kinase gene also appear to be a driver of tumor growth and occur in up to 2% of patients with NSCLC. Preclinical data has shown that dysregulation of Axl is implicated in tumor progression and resistance to standard and targeted cancer therapies. Extensive preclinical and clinical data also indicate that both MET and Axl are important factors in resistance to EGFR inhibitors, as well as the third-generation EGFR inhibitors.

About MGCD265

MGCD265 is a tyrosine kinase inhibitor that potently and selectively targets tumors in patients with driver alterations in MET (gene amplification and mutations) and AXL (gene amplification and rearrangements) that occur in approximately 8% of patients with non-small cell lung cancer (NSCLC). MGCD265 is in the expansion phase of a Phase 1/1b dose escalation study for NSCLC patients with MET or AXL genetic alterations. Genetic alterations in these targets have been implicated as drivers of tumor growth and disease progression in NSCLC, gastroesophageal cancer and other solid tumors. Mirati retains worldwide rights to MGCD265.

On September 9, 2015 RedHill Biopharma Ltd. (Nasdaq:RDHL) (TASE:RDHL) ("RedHill" or the "Company"), an Israeli biopharmaceutical company primarily focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal (GI) diseases, including cancer, reported that the National Cancer Institute (NCI) has awarded a $2 million Small Business Innovation Research Program (SBIR) grant to support the planned Phase II study with YELIVA (ABC294640) for the treatment of refractory or relapsed multiple myeloma (Press release, RedHill Biopharma, SEP 9, 2015, View Source [SID:1234507433]).

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The grant covers a three year period and was awarded to Apogee Biotechnology Corporation ("Apogee") in conjunction with Duke University. RedHill acquired the rights to YELIVA (ABC294640), a proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor, from Apogee in March 2015.

RedHill plans to initiate the Phase II study of YELIVA (ABC294640) for the treatment of refractory or relapsed multiple myeloma by the end of 2015. The open-label, dose escalation Phase II study will be conducted at Duke University Medical Center and is planned to enroll up to 77 patients with refractory or relapsed multiple myeloma who have previously been treated with proteasome inhibitors and immunomodulatory drugs. Dr. Yubin Kang, MD, Associate Professor in the Division of Hematologic Malignancies and Cellular Therapy in the Department of Medicine at Duke University Medical Center, will be the lead investigator for the study, which received Institutional Review Board (IRB) approval from Duke University (DUHS IRB).

The primary objectives of the first portion of the study (Phase Ib) are to assess safety and determine the maximum tolerated dose (MTD) in this group of patients. Secondary objectives include assessment of antitumor activity and determination of the pharmacokinetic (PK) and pharmacodynamic (PD) properties of YELIVA (ABC294640) in refractory or relapsed multiple myeloma patients.

The primary objectives of the second portion of the study (Phase II) are to assess the overall treatment response rate and overall survival. Secondary objectives include evaluating the treatment response of YELIVA (ABC294640) in patients with refractory or relapsed multiple myeloma after three cycles of treatment and evaluation of pharmacodynamic markers.

YELIVA (ABC294640) is a proprietary, first-in-class, orally-administered sphingosine kinase-2 (SK2) selective inhibitor, with anti-cancer and anti-inflammatory activities, targeting multiple oncology, inflammatory and GI indications. SK2 is an innovative molecular target for anti-cancer therapy because of its critical role in catalyzing the formation of the lipid-signaling molecule sphingosine 1-phosphate (S1P), which is known to regulate cell proliferation and activation of inflammatory pathways. By inhibiting SK2, YELIVA (ABC294640) could potentially be effective in treating multiple oncology, inflammatory, and gastrointestinal indications.

RedHill recently initiated a Phase I/II clinical study in the U.S. evaluating YELIVA (ABC294640) in patients with refractory/relapsed diffuse large B-cell lymphoma (DLBCL), primarily in patients with HIV-related DLBCL, also supported by a grant from the NCI Small Business Technology Transfer (STTR) program. A third Phase II clinical study is planned to evaluate YELIVA (ABC294640) as a radioprotectant to prevent mucositis in cancer patients undergoing therapeutic radiotherapy.

The ongoing and planned Phase II studies follow numerous successful pre-clinical studies conducted with YELIVA (ABC294640) in GI, inflammation, radioprotection and oncology models, as well as a Phase I study in patients with advanced solid tumors, supported by grants from the National Cancer Institute (NCI) and the FDA’s Office of Orphan Products Development (OOPD). RedHill recently announced that the last patient has completed the final scheduled follow-up visit in the Phase I study with YELIVA (ABC294640). Preliminary positive data from the Phase I study was presented by Apogee at the November 2013 Molecular Targets and Cancer Therapeutics meeting. The analysis of the study is currently ongoing and top-line results are expected to be announced early in the fourth quarter of 2015. A full analysis and the final Clinical Study Report (CSR) are expected by the end of the year or early 2016.

The studies with YELIVA (ABC294640) are registered on www.ClinicalTrials.gov, a web-based service by the U.S. National Institute of Health which provides public access to information on publicly and privately supported clinical studies.

About YELIVA (ABC294640):

YELIVA (ABC294640) is a first-in-class, proprietary sphingosine kinase-2 (SK2) selective inhibitor, administered orally, with anti-cancer and anti-inflammatory activities, targeting multiple potential oncology, inflammatory and gastrointestinal indications. By inhibiting the SK2 enzyme, YELIVA (ABC294640) blocks the synthesis of sphingosine 1-phosphate (S1P), a lipid that promotes cancer growth and pathological inflammation. YELIVA (ABC294640) was originally developed by U.S.-based Apogee Biotechnology Corp. and completed multiple successful pre-clinical studies in oncology, inflammation, GI, and radioprotection models, as well as the ABC-101 Phase I clinical study in cancer patients with advanced solid tumors. A Phase I/II clinical study evaluating YELIVA (ABC294640) in patients with refractory/relapsed diffuse large B-cell lymphoma (DLBCL) has been initiated in the U.S. The development of YELIVA (ABC294640) was funded to date primarily by grants and contracts from U.S. federal and state government agencies.

(Filing, 10-Q, Peregrine Pharmaceuticals, SEP 9, 2015, View Source [SID:1234507438])

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On September 9, 2015 Varian Medical Systems (NYSE: VAR) reported that it is collaborating with two leading Cape Town universities to launch Africa’s first ‘Access to Care’ program, designed to train cancer caregivers in delivering advanced conformal radiotherapy treatments (Press release, InfiMed, SEP 9, 2015, View Source [SID:1234507423]).

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The three-month course, which will be delivered in conjunction with Cape Peninsula University of Technology and the University of Cape Town’s department of radiation oncology, aims to train and support radiation therapy departments to implement and maintain 3D conformal radiation therapy treatment programs at their hospitals. In many cases, the delegates’ only experience of radiotherapy will have been older cobalt-based 2D treatments.

"As radiation oncology becomes increasingly precise and cancer centers worldwide can offer ever more advanced treatments for their patients, many parts of the developing world are still significantly under-equipped with too few machines to treat their rapidly growing cancer populations," says Michael Sandhu, who heads Varian Oncology Systems’ new global market development team. "Developing regions are starting to invest in new equipment to address this capacity gap but they are often hindered by a lack of qualified staff to plan treatments and run the equipment. ‘Access to Care’ is one of the ways in which Varian, as the global leader in radiation oncology, is seeking to bridge the skills gap between well-equipped countries and developing nations."

The Access to Care program will be offering two courses a year in South Africa and each program will be attended by four teams of radiation oncologists, medical physicists and radiotherapy therapists from participating hospitals from across the African continent. The course includes three weeks of classroom-based training at Groote Schuur Hospital in Cape Town, which is equipped with a virtual linear accelerator and four workstations, followed by a 10-week remote mentorship program. A separate software lab equipped with 10 workstations is available for students to practice contouring and treatment planning – important steps in the radiotherapy process. The classroom is linked to Varian’s virtual education environment hosted in its European headquarters in Cham, Switzerland.

"The training of cancer specialists and their teams enters a new era with the Access to Care program, which is at the leading edge of training globally and will help towards better and safer care of patients with cancer," said Professor Raymond Abratt, retired head of radiation oncology at Groote Schuur Hospital and the University of Cape Town, and current chairperson of the South African Society of Clinical and Radiation Oncologists (SASCRO), at a program launch event that took place in Cape Town today. "Radiation Oncology is an important component of cancer treatment in Africa and I congratulate all those involved in realizing this exceptional facility and the associated training programs."

Access to Care also offers training programs in Vietnam and has plans to commence a similar project in Algeria. "There is some real momentum behind our ‘Access to Care’ training programs and we are excited about the chance to help provide greater access to advanced cancer treatments in developing nations," says Jose-Manuel Valentim, Varian’s director market development in the EMEIA and APAC regions.

Cancer in Africa
According to a 2013 study published in Lancet Oncology, only 23 out of 52 African countries have radiotherapy available for patients. The World Health Organization reports that by 2030 there will be some 1.6 million new cancer cases in Africa each year, resulting in 1.2 million deaths. The most common cancers in Africa are cancers of the cervix, breast, lung, liver and prostate.

Varian has installed more than 100 radiotherapy treatment systems in Africa over the last 25 years. The company recently announced major projects in Algeria, Egypt and South Africa. Varian has also installed equipment in several sub-Saharan nations including Ghana, Angola, Kenya, and Madagascar.