In the first, Alexander M.M. Eggermont, Director General of the Gustave Roussy Cancer Campus Grand Paris in Villejuif, France, and investigators from 23 countries across the world, found that giving a one-year course of 18 doses of the immunotherapy drug pembrolizumab (Keytruda) significantly reduced the risk of the cancer returning for patients with stage III melanoma who were at high risk of recurrence after surgery (Press release, EORTC, APR 16, 2018, View Source [SID1234525394]). Patients with stage III melanoma have metastatic disease in one or more regional lymph nodes. Giving a dose of 200 milligrams of pembrolizumab every three weeks after surgery for up to a year significantly reduced the risk of recurrence for these patients the investigators found.

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Pembrolizumab works by blocking a protective mechanism of cancer cells, thus allowing the immune system to destroy them. Of the 1,019 patients recruited to the double-blind trial, 514 were randomised to pembrolizumab, and the others to placebo. For all those randomised to pembrolizumab, the 12-month recurrence-free survival rate was 75.4 percent, compared with 61.0 percent for all those randomised to placebo. The estimated hazard ratio was 0.57, indicating that risk of recurrence or death was reduced by 43 percent in patients randomised to pembrolizumab as compared to those randomised to placebo.

After a median follow-up of 1.25 years, 135 of the 514 patients randomised to pembrolizumab and 216 of the 505 patients randomised to placebo had been diagnosed with recurrent disease or had died. Patients randomised to placebo who had recurrence were offered access to pembrolizumab. "This cross-over design is unique in the world of adjuvant trials in melanoma and will permit us to analyse if adjuvant therapy with pembrolizumab right after surgery is better or not than treating only those who relapse and start treatment at relapse," says Eggermont. "We hope that these data will lead to regulators in the United States and Europe approving pembrolizumab as a new treatment option for these patients."
The trial results are published in the New England Journal of Medicine.
Abstract no CT001 : Pembrolizumab versus placebo after complete resection of high-risk stage III melanoma: Efficacy and safety results from the EORTC 1325-MG/Keynote 054 double-blinded phase III trial

In the second trial, investigators from eight European countries, headed by Patrick Schöffski from KU Leuven, Belgium, found that treatment with a targeted cancer drug achieved complete or partial tumour shrinkage in 50% of patients with inflammatory myofibrobastic tumour (IMFT), a very rare type of soft tissue sarcoma. Soft tissue sarcomas in themselves are very rare, accounting for just 1% of all solid tumour diagnoses, and IMFT so uncommon that there are no reliable statistics for its incidence or for mortality rates.
Many IFMTs have rearrangements of the ALK gene. Recently discovered as a target for cancer therapies, the ALK gene can be oncogenic in three ways – by forming a fusion gene with any of several other genes, by gaining additional copies or through mutations of the actual DNA code for the gene itself.
Schöffski and colleagues therefore set out to see whether the ALK inhibitor crizotinib might be a potential treatment for patients with IMFT. "Inflammatory myofibroblastic tumours are usually resistant to conventional chemotherapy and radiotherapy, so patients with unresectable or locally recurring disease have few treatment options," he says.
Because IMFT is so rare, only 35 patients with a local diagnosis of inflammatory myofibroblastic tumour could be recruited to the trial. Twenty of these patients were confirmed centrally to have the disease and received crizotinib.
Nineteen patients were evaluable for response. The objective response rate among the 12 evaluable patients with ALK-positive IFMTs who received crizotinib was 50 percent (95% confidence interval : 21.1-78.9%); two had a confirmed complete response and four had a confirmed partial response. Among the seven evaluable patients with ALK-negative IFMTs, the rate was 14.3 percent (95% confidence interval: 0.0-57.9%). In the group of patients with ALK-positive IFMT, crizotinib activity met the pre-specified response rate criteria set by the protocol.
« Limitations of the trial include that it is a noncomparative, single-arm study with a relatively small number of patients. Given the disease prevalence, a more definitive, randomised, comparative trial would not be possible, » says Schöffski.
« However, our study highlights how identifying the genetic drivers of a rare type of cancer can be used to find a new precision medicine for patients who otherwise have few treatment options," he adds. "The inclusion in our trial of a group of patients with ALK-negative inflammatory myofibroblastic tumors provides valuable insight into the selectivity of crizotinib and our understanding of this rare disease, even if we cannot formally compare the outcomes for the ALK-positive and -negative groups."
The trial results are published in The Lancet Respiratory Medicine.
Abstract no CT045 :Prospective precision medicine trial of crizotinib (C) in patients (pts) with advanced, inoperable inflammatory myofibroblastic tumor (IMFT) with and without ALK alterations: EORTC phase II study 90101 "CREATE"

On April 16, 2018 Biological Dynamics, a molecular diagnostics company dedicated to improving global health outcomes by empowering global communities with low-cost, accessible cancer diagnostics, reported that new data on the company’s novel technology (ACE) will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2018 Annual Meeting, at McCormick Place Convention Center on April 13 – 18 in Chicago (Press release, Biological Dynamics, APR 16, 2018, View Source [SID1234525413]).

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Abstracts from two studies examining the application of the company’s technology as an isolation platform for a novel class of biomarkers, such as extracellular vesicles (EVs), and as a cell-free DNA (cfDNA)-based diagnostic assay, will be presented:

Novel AC Electrokinetic Platform for Rapid Isolation and Characterization of Extracellular Vesicles from NSCLC Patients
Presented by Raj Krishnan, Ph.D., CEO of Biological Dynamics, on April 16 at 1:00 p.m. CT in Section 44. (Late-breaking abstract # LB-174)
Diagnostic Application of Novel ACE Technology: Treatment Response Monitoring via Quantification of Cell-Free DNA (cfDNA) in Plasma from Late-Stage Cancer Patients
Presented by Robert Kovelman, Ph.D., Biological Dynamics’ Sr. Director of Assay Development and Clinical Affairs, on April 17 at 8:00 a.m. CT in Section 27. (Abstract # 3666)
Biological Dynamics announced on Friday, April 13, the addition of two new members to its Board of Directors. Irwin Jacobs, founding Chairman and CEO Emeritus of Qualcomm and Chairman Emeritus of the Salk Institute for Biological Studies, and Martin J. Wygod, founder of Medco Containment Services Inc. and former Chairman of WebMD Health Corp., have joined Biological Dynamics’ Board of Directors. (Read more here.)

On April 16, 2018 Innovation Pharmaceuticals (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, is pleased to report additional information from the Company’s successfully completed Phase 2 clinical trial of Brilacidin-OM (see NCT02324335) for the indication of decreasing the incidence of Severe Oral Mucositis (SOM) (WHO Grade ≥3) in Head and Neck Cancer (HNC) patients receiving chemoradiation (Press release, CellCeutix, APR 16, 2018, View Source [SID1234525812]).

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These additional data align with previously released Brilacidin-OM results showing a risk reduction in the incidence of SOM, including up to an 80.3% risk reduction in the incidence of SOM among patients receiving more aggressive chemotherapy. Other previously released results indicate Brilacidin-OM also delayed onset of SOM. The Company is developing Brilacidin-OM under FDA Fast Track designation as a convenient, and clearly differentiated, therapy aimed to decrease incidence of SOM.
Initial Instance Duration of SOM was defined as the number of days from initial WHO Grade ≥3 during radiation therapy to the first WHO Grade 2 or lower OM Grade. Overall Duration of SOM was defined as the number of days from initial WHO Grade ≥3 during radiation therapy to the day prior to the next OM assessment after the last WHO Grade ≥3 during/after radiation therapy. Note: 50th percentiles are from Kaplan-Meier analysis. Patients who did not experience SOM have duration set to 0.

Previously, the Company reported statistically significant results showing Brilacidin-OM reduced the incidence of SOM in HNC patients receiving cisplatin administered in a high-dose regimen (80-100 mg/m2), approximately every 21 days. For the Modified Intent-to-Treat (mITT) population, Brilacidin-OM in the high-dose chemotherapy regimen reduced the incidence of SOM by 65.0% ([incidence control- incidence active]/incidence control) as compared with placebo (Brilacidin: 25.0%; placebo: 71.4%; p=0.0480). For the Per Protocol (PP) population, Brilacidin-OM in the high-dose chemotherapy regimen similarly reduced the incidence of SOM by 80.3% as compared with placebo (Brilacidin: 14.3%; placebo: 72.7%; p=0.0249).

Exploratory Endpoint: Unplanned Office Visits, Emergency Department Visits, and/or Hospital Admissions Due to OM

Positive OM assessment endpoints are additionally supported by zero (0) of the patients in the Brilacidin-OM group having unplanned office visits, ED visits, or hospital admissions due to OM, compared to four (4) patients in the placebo group.

Other Study Observations

Regardless of the oral sites irradiated (at least two sites from: buccal mucosa, floor of mouth, ventral/lateral tongue, and soft palate), the incidence by patient of Severe OM on Brilacidin-OM relative to placebo was consistently reduced.

Across cumulative radiation dose intervals, patients in the Brilacidin-OM group consistently reported less often feeling the sensation "swollen" (approximately half of that reported for the placebo group). "Burning" sensation also was reported consistently less frequently in the Brilacidin treatment group.

Patients in the Brilacidin-OM group appeared to trend more favorably over the course of chemoradiation treatment according to Eastern Cooperative Oncology Group (ECOG) Performance Status—a common set of criteria used in oncology trials to assess debility.

Management Comments

"Drug makers, the world-over, have spent decades and enormous sums in both money and resources trying to develop an effective OM drug in a bid to address dire patient needs as well as capture a tremendous market opportunity," commented Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "Yet, there is currently no drug approved to treat, let alone prevent, severe OM in patients with Head and Neck Cancer. Most Pharmas currently conducting OM trials target shortening the duration of severe OM as their primary endpoint, not reduction of incidence, like we did. The Brilacidin-OM Phase 2 trial met its primary objective and its key secondary objectives. As we continue to analyze subset data, we are extremely enthusiastic about observed trends. Hundreds of thousands of patients would benefit from a preventative OM treatment and we’re excited that Brilacidin-OM may one day provide these patients a much-needed breakthrough treatment option."

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On April 15, 2018 AstraZeneca and Merck & Co., Inc., Kenilworth, N.J., US (Merck: known as MSD outside the US and Canada) reported that presented data from the Phase III OlympiAD trial showing the final overall survival (OS) results for Lynparza (olaparib) in metastatic breast cancer at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, US, 14-18 April 2018 (Press release, AstraZeneca, APR 15, 2018, View Source [SID1234525367]).

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The trial compared Lynparza with chemotherapy (physician’s choice of capecitabine, eribulin or vinorelbine) for patients with germline BRCA-mutated (gBRCAm) HER2-negative metastatic breast cancer and met its primary endpoint of progression-free survival (PFS).

Results at AACR (Free AACR Whitepaper) include updated findings from the secondary endpoint of overall survival (OS). While the trial was not powered to demonstrate a statistically-significant difference, the median OS was 19.3 months in patients treated with Lynparza and 17.1 months for patients treated with chemotherapy (HR 0.90; 95% CI 0.66-1.23; p=0.513). At the final OS data cut-off (64% maturity), nearly 13% of patients remained on Lynparza and no patients remained on chemotherapy.

Sean Bohen, Executive Vice President, Global Medicines Development and Chief Medical Officer at AstraZeneca, said: "OlympiAD is the first Phase III trial to demonstrate disease control with a PARP inhibitor in BRCA-mutated HER2-negative metastatic breast cancer. While the trial was not powered to show overall survival compared to chemotherapy, the results are another encouraging marker in the use of Lynparza for this patient population."
Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "For patients and physicians, these results are meaningful in that they support the progression-free survival endpoint – which showed that patients treated with Lynparza gained seven months chemotherapy-free time – and reinforce the importance of identifying BRCA status to optimise metastatic breast cancer management."
When analysing the predefined subgroups, the results were consistent with the overall analysis, which did not show a statistically-significant difference between arms. The greatest difference was seen in patients who had not received chemotherapy in the metastatic setting with a median difference in OS of 7.9 months with Lynparza (HR 0.51; 95% CI 0.29-0.90; nominal p=0.02; median 22.6 vs 14.7 months).

The safety profile of Lynparza remained consistent with the primary analysis, indicating no relevant cumulative toxicity with extended exposure. Serious adverse events (Grade >3) were reported in 38% of patients who received Lynparza vs 49.5% of patients in the chemotherapy arm.

These results build on previously reported findings, which demonstrated Lynparza significantly improved PFS (HR 0.58; 95% CI 0.43-0.80; p=0.0009 median 7.0 vs 4.2 months) and showed benefit beyond initial disease progression, prolonging time to second progression or death (PFS2) by 3.9 months (HR 0.57; 95% CI 0.40-0.83; p=0.003 median 13.2 months vs 9.3 months). Previously reported findings also showed Lynparza doubled objective response rates (52% [95% CI 44-60] vs 23% [95% CI 13-35]) and improved quality-of-life scores. The data from the OlympiAD trial can be found in the 10 August 2017 issue of the New England Journal of Medicine.
In January 2018, Lynparza was approved by the US FDA for the treatment of metastatic breast cancer, based on the OlympiAD data. A Type II variation application was recently validated by the European Medicines Agency for Lynparza in gBRCAm HER2-negative metastatic breast cancer.

A Phase III trial (n=1800), OlympiA, is evaluating Lynparza as an adjuvant treatment in patients with gBRCA HER2-negative breast cancer, with results expected in 2020. The trial is powered to assess potential benefit in OS.
Lynparza is approved in around 60 countries for advanced ovarian cancer and has treated more than 20,000 patients globally. It has the broadest clinical development programme of any PARP inhibitor and AstraZeneca and MSD are working together to bring Lynparza to more patients across multiple cancers.
NOTES TO EDITORS
About OlympiAD
OlympiAD is a global, randomised, open-label, multi-centre Phase III trial of 302 patients, assessing the efficacy and safety of Lynparza tablets (300 mg twice daily) compared to chemotherapy (physician’s choice of capecitabine, eribulin or vinorelbine). 205 patients were randomised to receive Lynparza and 97 patients were randomised to receive chemotherapy.
Patients in the OlympiAD trial had germline BRCA-mutated, HER2-negative (hormone receptor-positive or triple negative) breast cancer and received Lynparza for treatment in the metastatic setting. Prior to enrolment, 71% of patients had received no more than two previous chemotherapy treatments for metastasised breast cancer and 28% of patients had received prior platinum-based chemotherapy. Also enrolled were patients with HR+ breast cancer who had received at least one endocrine therapy (adjuvant therapy or therapy for metastatic disease) and had disease progression during therapy, unless they had disease for which the endocrine therapy was considered inappropriate.

The primary endpoint was PFS. Secondary endpoints included OS, time to second progression or death, objective response rate, health-related quality of life and safety and tolerability.
About Metastatic Breast Cancer

PRs, ERs and HER2 receptors may be expressed on breast cancer cells. A patient’s breast cancer will test either negative or positive for these three receptors. If a tumour tests positive for PR and/or ER, it is considered hormone-receptor positive. If a tumour tests negative for all three receptors, it is considered triple negative. These receptors indicate which hormones or other proteins may be promoting growth of the cancer.
Metastatic Breast Cancer (MBC) is the most advanced stage of breast cancer (Stage IV), and occurs when cancer cells have spread beyond the initial tumour site to other parts of the body, outside of the breast and nearby lymph nodes.

Despite the increase in treatment options during the past three decades, there is currently no cure for patients diagnosed with MBC and only 26.9% of patients survive for five years after diagnosis. Thus, the primary aim of treatment is to slow progression of the disease for as long as possible, improving, or at least maintaining, a patient’s quality of life.

Breast cancer is the most common cancer in women, with an estimated 1.67 million new cases diagnosed worldwide in 2012 alone – one in four of all cancer cases. Approximately 30% of women who are diagnosed with early breast cancer will go on to develop advanced disease.

About BRCA Mutations
BRCA1 and BRCA2 are human genes that produce proteins responsible for repairing damaged DNA and play an important role maintaining the genetic stability of cells. When either of these genes is mutated, or altered, such that its protein product either is not made or does not function correctly, DNA damage may not be repaired properly and cells become unstable. As a result, cells are more likely to develop additional genetic alterations that can lead to cancer.

About Lynparza
Lynparza was the first in class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells. Specifically, in vitro studies have shown that Lynparza-induced cytotoxicity may involve inhibition of PARP-enzymatic activity and increased formation of PARP-DNA complexes, resulting in DNA damage and cancer cell death.
Lynparza, which has the broadest clinical development programme of any PARP inhibitor, is being investigated in a range of DDR-deficient tumour types, and is the foundation of AstraZeneca’s industry-leading portfolio of compounds targeting DDR mechanisms in cancer cells.
About the AstraZeneca and MSD Strategic Oncology Collaboration
In July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialise Lynparza, the world’s first PARP inhibitor and potential new medicine selumetinib, a MEK inhibitor, for multiple cancer types. The collaboration is based on increasing evidence that PARP and MEK inhibitors can be combined with PD-L1/PD-1 inhibitors for a range of tumour types. Working together, the companies will develop Lynparza and selumetinib in combination with other potential new medicines and as a monotherapy. Independently, the companies will develop Lynparza and selumetinib in combination with their respective PD-L1 and PD-1 medicines.
About AstraZeneca in Oncology

AstraZeneca has a deep-rooted heritage in Oncology and offers a quickly growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020 and a broad pipeline of small molecules and biologics in development, we are committed to advance Oncology as one of AstraZeneca’s Four Growth Platforms focused on lung, ovarian, breast and blood cancers. In addition to our core capabilities, we actively pursue innovative partnerships and investments that accelerate the delivery of our strategy as illustrated by our investment in Acerta Pharma in haematology.
By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DDR and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and one day eliminate cancer as a cause of death.

On April 15, 2018 Blueprint Medicines Corporation (NASDAQ:BPMC), a leader in discovering and developing targeted kinase medicines for patients with genomically defined diseases, reported the online publication of preclinical and clinical proof-of-concept data for BLU-667 in Cancer Discovery, an American Association for Cancer Research (AACR) (Free AACR Whitepaper) journal (Press release, Blueprint Medicines, APR 15, 2018, View Source;p=RssLanding&cat=news&id=2342579 [SID1234525368]). Designed and developed by Blueprint Medicines, BLU-667 is a potent and highly selective inhibitor targeting oncogenic RET fusions and mutations, which are key drivers across multiple cancers, including subsets of patients with non-small cell lung cancer (NSCLC) and medullary thyroid cancer (MTC).

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The manuscript reports detailed preclinical data characterizing the potency and selectivity of BLU-667 against oncogenic RET variants and resistant mutants and anti-tumor activity in multiple solid tumor models. In addition, four patient vignettes from the ongoing Phase 1 ARROW clinical trial describe clinical responses in patients with RET-KIF5B-altered NSCLC and medullary thyroid cancer (MTC) harboring multiple RET mutations, including patients who had progressed on prior multi-kinase therapy
.
"The publication of our work in Cancer Discovery highlights BLU-667’s compelling preclinical profile and preliminary clinical activity in patients with RET-altered cancers and further demonstrates the power of Blueprint Medicines’ scientific platform," said Erica Evans, Ph.D., Senior Director of Biology at Blueprint Medicines and the senior author of the paper. "The published data show BLU-667 has the potential to deliver anti-tumor activity and meaningful clinical responses, regardless of tumor type, RET alteration or prior therapy. Coupled with the initial results from the ongoing Phase 1 ARROW clinical trial that will be presented today at the AACR (Free AACR Whitepaper) Annual Meeting, these data support the rapid development of BLU-667 in patients with RET-altered cancers."

RET has long been recognized as an oncogene that drives multiple cancers. However, there are currently no approved selective RET inhibitors, and RET-targeted treatment is limited to non-selective multi-kinase therapies that can have significant off-target toxicities and limited efficacy. BLU-667 was specifically designed by Blueprint Medicines to target oncogenic RET fusions and mutations, including predicted resistance mutations, with the goal of providing durable clinical responses to patients with RET-altered cancers.
Key highlights included:

In vitro studies show BLU-667 has 10- to 10,000-fold increased potency against oncogenic RET variants and resistant mutants over approved multi-kinase inhibitors. In addition, BLU-667 has 20-fold increased potency against RET-KIF5B fusions, the most common RET alteration in patients with NSCLC, compared to the investigational multi-kinase inhibitor RXDX-105.

Additional in vitro studies show BLU-667 is 88-fold more selective for RET over VEGFR-2, which when inhibited can result in dose-limiting toxicities. Overall, BLU-667 is 100-fold more selective for RET over 96 percent of 371 kinases tested.

In vivo studies show BLU-667 potently inhibits the growth of NSCLC, MTC and colorectal tumors in RET-driven disease models, including models harboring multi-kinase inhibitor-resistant mutants.
Four patient vignettes from the ongoing Phase 1 ARROW clinical trial show that BLU-667 significantly inhibits RET signaling and induces durable clinical responses in patients with RET-altered NSCLC and MTC without notable off-target toxicity, providing clinical validation for selective RET targeting.
The paper, titled "Precision targeted therapy with BLU-667 for RET-driven cancers," was published online in Cancer Discovery on April 15, 2018.
About BLU-667

BLU-667 is an orally available, potent and highly selective inhibitor designed to target RET fusions, mutations and predicted resistance mutations. Blueprint Medicines is developing BLU-667, an investigational medicine, for the treatment of patients with RET-altered NSCLC, MTC and other solid tumors. BLU-667 was discovered by Blueprint Medicine’s research team leveraging its proprietary compound library, and Blueprint Medicines retains worldwide development and commercialization rights for BLU-667.
About RET-Altered NSCLC, MTC and Other Solid Tumors
RET activating fusions and mutations are a key disease driver in multiple cancers, including NSCLC and MTC. RET fusions are implicated in approximately 1-2% of patients with NSCLC, while RET mutations are implicated in approximately 60% of patients with MTC and 10% of papillary thyroid cancer. In addition, genomic analyses published by scientists at Blueprint Medicines have identified RET fusions at low frequencies in colon and breast cancer. Currently, there are no approved therapies that selectively target RET-driven cancers, though there are several approved multi-kinase inhibitors with RET activity being evaluated in clinical trials. Thus far, clinical activity attributable to RET inhibition has been uncertain for these inhibitors, likely due to insufficient inhibition of RET and off-target toxicities.