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Amgen Files for Early Approval of its Breakthrough Leukemia Drug


Today, Amgen announced that it has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) seeking approval for its leukemia drug.

Specifically, the company submitted the application for its investigational cancer immunotherapy blinatumomab for treatment of adult patients with Philadelphia-negative (Ph-) relapsed/refractory B-precursor acute lymphoblastic leukemia (ALL), a rapidly progressive cancer of the blood and bone marrow. Amgen’s blinatumomab has been granted orphan drug designation and awarded with breakthrough therapy designation from the FDA, intended to expedite review of the drug.

“We look forward to working with regulatory authorities to make blinatumomab available for adult patients with acute lymphoblastic leukemia, who experience high relapse rates and have limited treatment options,” said Sean E. Harper, MD, executive vice president of Research and Development at Amgen. “The filing for blinatumomab brings us a step closer to first realizing the potential of BiTE® technology and represents our commitment to evaluating this novel approach in a broad range of difficult-to-treat cancers.”

Blinatumomab is an investigational bispecific T cell engager (BiTE) antibody construct. BiTE® antibody constructs represent an innovative immunotherapy approach that helps the body’s immune system targeting cancer cells.

Amgen’s application includes data from a mid-stage trial of adult patients with Ph- relapsed/refractory B-precursor ALL treated with blinatumomab, which successfully met its primary endpoint, with 43 percent of patients achieving complete remission or complete remission with partial hematologic recovery within two cycles of treatment with blinatumomab. Results were presented at the American Society of Clinical Oncology (ASCO) meeting.

“Currently, there is no broadly accepted standard treatment regimen for adult patients with relapsed or refractory ALL,” said Anthony S. Stein, MD, clinical professor, Hematology/Oncology at City of Hope. “Blinatumomab has the potential to significantly advance treatment options for patients living with this difficult-to-treat disease, and the BLA submission marks an important step toward achieving this goal.”

ALL progresses rapidly and affects immature blood cells. Worldwide, the disease accounts for more than 12 percent of leukemia cases. Among the 42,000 people diagnosed with ALL worldwide, 31,000 will die from the disease. Patients with ALL have abnormal white blood cells, also known as lymphocytes, that crowd out healthy white blood cells, red blood cells and platelets, leading to infection, anemia (fatigue), easy bleeding and other serious side effects.

Source: Amgen

Last updated: 9/22/14; 11:15am EST

Study Finds PSA Screening Guidelines Have Little Effect

PSA screening

A new study found that the guidelines recommending that elderly men should not be routinely screened for prostate cancer have been minimally effective at best.

The study, led by researchers at Henry Ford Hospital and published as a research letter online in JAMA Internal Medicine, focused on the use of prostate-specific antigen (PSA) to test for prostate cancer. The researchers found an estimated 17 million men age 50 or older without a history of prostate cancer or prostate problems who reported undergoing PSA screening.

“We found that the effect of the guidelines recommending against the routine screening of elderly men in particular has been minimal at best,” said Jesse Sammon, D.O., a researcher at Henry Ford’s Vattikuti Urology Institute and lead author of the study.

During the first decade after approval from the US Food and Drug Administration, PSA testing was credited with a significant improvement in five-year cancer survival rates. However, its use for routine screening is controversial due to concerns that the test often provides false-positives, leading individuals who do not have a prostate malignancy to undergo treatment that they do not need. The individuals undergoing treatment suffer from side effects as impotence and urinary incontinence that could be avoided. A few years ago, the debate caused the US Preventative Services Task Force to recommend against PSA screening in any age group.

“But in the time since, nationwide patterns of PSA screening were largely unknown,” said Dr. Sammon. “We sought to examine those patterns to determine the effects of the most recent USPSTF recommendation.”

The researchers at Henry Ford got their data from the 2012 Behavioral Risk Factor Surveillance System, which is the world’s largest continuously conducted health survey. The study group was analyzed by age, race and/or ethnicity, education, income, residence location, insurance status, access to regular health care and marital status.

They found that higher rates of screening were most strongly associated with access to regular health care, followed by an income greater than $75,000, college education, health insurance and those between the ages of 70 and 74. The next highest rate of screening, which followed by only a fraction of a percentage point, was in men ages 65 to 69. Those between the age of 50 and 54 were found to be the least likely to report PSA screening, even though several professional medical organizations have previously recommended screening for that age group.

Additionally, researchers analyzed self-reported PSA screening across the US and found the highest rate in Hawaii and the lowest in New Hampshire, with reported rates of 59.4 percent and 24.5 percent, respectively.

“Looking at rates of colorectal and breast cancer screening, state-by-state and regional variability is expected, but not to the pronounced extent that was found for PSA screening,” Sammon explained. “This was another concerning and surprising study finding. It’s alarming that the prevalence of PSA screening can double from one state to the next.”

The researchers said that their findings likely reflect both the considerable disagreement among experts and the conflicting recommendations on PSA screening.

“Taken together, those results suggest that national guidelines have had a limited effect on clinical practice among health care providers,” said Dr. Sammon.

Source: Henry Ford Health System

Last updated: 9/19/14; 2:50pm EST 

Boehringer Ingelheim Licenses CureVac's Investigational Cancer Vaccine

Boehringer Ingelheim

Boehringer Ingelheim has gained exclusive global rights to CureVac’s investigational therapeutic cancer vaccine, in a deal worth up to about $600 million.

The companies made a joint announcement today regarding the exclusive global license and development collaboration focused on CureVac’s CV9202, a novel investigational mRNA vaccine, in early clinical development for the treatment of lung cancer.

Boehringer Ingelheim said that it will start clinical investigation of the vaccine in at least two different lung cancer settings, including in combination with afatinib in patients with advanced metastatic epidermal growth factor (EGFR) mutated non-small cell lung cancer (NSCLC) and in combination with chemo-radiation therapy in patients with unresectable stage III NSCLC. Under the deal, Boehringer Ingelheim will pay an upfront fee of about $45 million to CureVac and roughly $555 million in prospective milestones.

“At Boehringer Ingelheim we are proud of our commitment to help the treatment of cancers with a high medical need. In our collaboration with CureVac, we will investigate combining existing treatments with the approach of sustained activation of the immune system. With this we hope to be able to develop new treatments and further expand our broad pipeline in lung cancer,” said Professor Klaus Dugi, Chief Medical Officer, Boehringer Ingelheim.

CureVac’s mRNA-based technology represents a novel approach in cancer treatment. For the first time, mRNA could be optimized to mobilize the patient’s own immune system to fight the tumor with a specific immune response elicited through the RNActive vaccine. CV9202 is a combination of mRNA molecules coding for six antigens that are overexpressed in lung cancer. The vaccine is designed to induce an immune response against the tumor. CureVac’s CV9202 and the preceding RNActive cancer vaccine CV9201 tested in initial clinical trials by CureVac demonstrated clinical safety and activity in generating immune responses against all anti-tumor antigens.

“This collaboration is extremely relevant for CureVac because, as a biotech enterprise, we rely on collaboration with strong partners for the clinical development and commercialization of our compounds. Cancer immunotherapy represents one of the biggest innovations in cancer treatment of recent times and we are delighted to now be working with Boehringer Ingelheim. The out-licensing and clinical development of our promising therapeutic vaccine CV9202 represents the logical next step in developing this novel treatment for cancer patients and the significant commitment from Boehringer Ingelheim underscores the relevance of the mRNA technology,” said Ingmar Hoerr, co-founder and CEO of CureVac GmbH.

Source: Boehringer Ingelheim

Last updated: 9/19/14; 11:25am EST

Gilead's Experimental Pancreatic Cancer Drug Fails its Mid-Stage Trial

Gilead Sciences, Inc.

Today, Gilead Sciences, Inc. announced that its investigational pancreatic cancer failed to meet the primary endpoint in its mid-stage study.

The company reported results from a Phase II study evaluating its simtuzumab, an investigational inhibitor of lysl oxidase-like (LOXL2), in combination with gemcitabine for patients with previously untreated advanced pancreatic cancer. The results show that adding simtuzumab to gemcitabine did not significantly increase progression-free survival (PFS), the primary endpoint of the study, compared to placebo plus gemcitabine.

In the Phase II trial, 236 patients with advanced pancreatic cancer received intravenous gemcitabine plus either intravenous simtuzumab or placebo in cycles of 28 days. Median PFS for the simtuzumab 200 mg, simtuzumab 700 mg and placebo group was 3.5 months, 3.7 months and 3.7 months, respectively. The difference in PFS between the simtuzumab and placebo arms was not statistically significant. Expected gemcitabine-related toxicities included anemia, thrombocytopenia, neutropenia and nausea. There was no difference in adverse events between patients taking simtuzumab versus placebo.

“Although simtuzumab did not provide clinical benefit in difficult-to-treat advanced pancreatic cancer patients in this study, we continue to explore simtuzumab in other areas of unmet medical need, with ongoing clinical trials in colorectal cancer, myelofibrosis and serious fibrotic lung and liver diseases,” said Norbert Bischofberger, PhD, Gilead’s Executive Vice President of Research and Development and Chief Scientific Officer.

Simtuzumab is an investigational monoclonal antibody that is highly selective for LOXL2, an enzyme that modifies the extracellular matrix by promoting the cross-linking of collagen fibers. LOXL2 is throught to play an important role in tumor progression and metastasis and in the development of fibrotic diseases. The drug is being studied in multiple ongoing Phase II trials for treatment of therapeutic areas including colorectal cancer, myelofibrosis, idiopathic pulmonary fibrosis, and liver fibrosis caused by non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis (PSC).

Source: Gilead Sciences, Inc.

Last updated: 9/17/14; 4:15pm EST

Abcodia and OncoCyte Partner for Breast Cancer Diagnostic


Abcodia has partnered with OncoCyte Corporation, a subsidiary of BioTime, Inc., to develop a new test for early detection of breast cancer.

The companies announced that they will collaborate on the development of OncoCyte’s blood-based PanC-Dx test. PanC-Dx is a class of non-invasive cancer diagnostics based on OncoCyte’s proprietary set of cancer markers, which were discovered by company scientists through an analysis of broad gene expression patterns in numerous cancer types. PanC-Dx is currently being evaluated in four clinical studies, sponsored by OncoCyte, evaluating the test in bladder, breast and lung cancer.

Abcodia has exclusive commercial access to a unique longitudinal biobank of over five million serum samples collected through the UK Collaborative Trial for Ovarian Cancer Screening. The biobank was derived from over 200,0000 initially healthy individuals, 50,000 of whom provided samples annually for up to ten years. Since first recruited, more than 3,700 participants have been diagnosed with breast cancer, which generates opportunities to assess serum biomarker changes that occurred years prior to diagnosis of breast cancer was made.

The collaboration will begin with an initial study, which according to the companies, will be completed by the end of this year. Under the current agreement, OncoCyte will test the performance of its proprietary PanC-Dx cancer markers in detecting breast cancer in a set of patient samples selected from Abcodia’s biobank. If the initial study’s outcome is promising, future studies could proceed and expand into the use of a larger cohort to assess OncoCyte’s PanC-Dx cancer markers in a case-controlled longitudinal design. Due to the large number of samples in the biobank, it may be possible to conduct a very large study of PanC-Dx much more quickly than otherwise would be possible, potentially expediting the broad commercialization of the test. The test’s ability to detect the absence, presence and development of early breast cancer will be considered in determining the intended use for PanC-Dx and the regulatory approval pathway that the company will pursue. As part of the initial collaboration, OncoCyte retails all rights to develop and market its proprietary breast cancer diagnostic products.

Early detection of cancer and its precursors is associated with improved outcomes for patients. The National Cancer Institute (NCI) recommends screening mammograms every one to two years in women over 40 years and older, while the American Cancer Society and the National Comprehensive Cancer Network both recommend screening mammography every year starting at age 40. The NCI estimates that approximately 20 percent of all breast cancers are not detected by mammography during annual screening, which indicates there is an unmet need for a breast cancer screening test with superior specificity and sensitivity when compared to standard screening mammography. PanC-Dx does not expose those undergoing the test to radiation, and could be indicated for all women regardless of age, and could be performed during the course of regular care with a familiar physician at low cost.

“A high-performing, blood-based breast cancer screening test would have multiple potential uses and users. Ultimately, our breast-cancer test could be used in conjunction with all screening mammography in order to detect breast cancer early and with a high degree of certainty. Working with Abcodia and using their unique set of longitudinal pre-diagnosis breast cancer samples will be of great value in generating robust validation data to support broader user adoption of our breast cancer diagnostic. We look forward to collaborating with them on this important product,” said OncoCyte’s Chief Executive Officer Joseph Wagner, PhD.

Source: Abcodia Ltd.

Last updated: 9/15/14; 2:35pm EST