On June 1, 2016 Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs (miR), reported that it has expanded its clinical trial collaboration agreement with GSK (NYSE: GSK) for the development of RG-101, Regulus’ wholly-owned, GalNAc-conjugated anti-miR that targets miR-122 (Press release, Regulus, JUN 1, 2016, View Source [SID:1234512943]). In the expanded collaboration, the companies plan to conduct a multi-centered, randomized, dose-ranging Phase II study evaluating the combination of RG-101 and GSK’s long-acting parenteral ("LAP") formulation of GSK2878175 as a potential single-visit cure in patients chronically infected with HCV. This study will be conducted outside the United States and is planned to begin in the fourth quarter of 2016. Based on predicted enrollment rates, interim results from this expanded collaboration should be available in the second half of 2017, enabling a potential initiation of a pivotal study in late 2017. As with the initial collaboration, both parties will share equally in the costs associated with the study. Neither Regulus nor GSK has any further obligations or commitments to each other beyond this expanded clinical collaboration agreement.

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"We are very pleased to expand our working collaboration with GSK. This is an important next step to advance the scientific understanding of the potential for a combination therapy to achieve a single-visit cure for HCV," said Paul Grint, M.D., President and CEO of Regulus. "The market research conducted to date indicates that a potential single visit cure would be a highly preferred product profile to existing regimens."

Zhi Hong, Senior Vice President and Head of the Infectious Diseases Therapy Area, GSK, commented, "We are excited about the potential of this combination to provide people living with HCV a new treatment option that could be delivered in a single visit. Together with Regulus, we are taking another step forward to proving this novel concept."

Earlier this year, pursuant to the initial GSK-Regulus clinical trial collaboration agreement, Regulus began enrolling patients in an open-label Phase II clinical trial combining RG-101 and GSK2878175 for the treatment of HCV to evaluate the potential to achieve sustained viral responses post treatment with a single subcutaneous administration of 4 mg/kg of RG-101 in combination with daily oral administrations of 20 mg of GSK2878175 for up to 12 weeks in treatment-naïve patients chronically infected with HCV genotypes 1 and 3. Regulus and GSK anticipate reporting interim results from this study by year-end.

About microRNAs

The discovery of microRNAs in humans during the last decade is one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 800 microRNAs have been identified in the human genome, and over two-thirds of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNA expression, or function, has been shown to be significantly altered or dysregulated in many disease states, including oncology, fibrosis, metabolic diseases, immune-inflammatory diseases and HCV. Targeting microRNAs with anti-miRs, chemically modified, single-stranded oligonucleotides, offers a unique approach to treating disease by modulating entire biological pathways and may become a new and major class of drugs with broad therapeutic application.

On June 1, 2016 BRCA Share, a public-private BRCA gene datashare initiative, reported the public release of a large collection of new data on genetic variants in the BRCA1 and BRCA2 genes (Filing, 8-K, LabCorp, JUN 1, 2016, View Source [SID:1234512944]). Mutations of these genes raise the risk of breast, ovarian and other cancers. The findings are to be presented today at the 6th International Biennial Meeting of Human Variome Project Consortium (HVP6) held at UNESCO headquarters in Paris.

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BRCA Share was co-founded by Quest Diagnostics and the French National Institute of Health and Medical Research (Inserm) in April 2015, with Laboratory Corporation of America Holdings (LabCorp) as the first commercial participant. The goal of the initiative is to share clinical, genetic, epidemiological and biological data on BRCA variants, particularly variants of uncertain significance, in order to improve the quality of laboratory diagnostics to better predict which individuals are at risk of developing hereditary breast and ovarian cancers, and to accelerate research on BRCA gene mutations. BRCA Share builds on a BRCA gene-data curation process developed by Inserm, Institut Curie, and Unicancer Genetic Group (UGG) with associated University Hospital Centers, using data developed over a decade of BRCA patient testing by the 16 UGG laboratories in France together with the same associated University Hospital Centers.

This new release adds variants from Quest Diagnostics and LabCorp, two clinical testing laboratory companies operating primarily in the United States, to the collection previously developed by Inserm through the Universal Mutation Database (UMD) project in France. The BRCA Share database now contains over 6,200 total BRCA variants, an increase of nearly 30% compared to the previous Inserm UMD database. Of these variants, 334 are newly identified pathogenic or likely pathogenic, increasing by about 20% the total number of pathogenic or likely pathogenic variants to 1,826.

The data include 375 BRCA gene variants whose role in cancer risk was previously uncertain. Of these, 93% are now classified as neutral or likely neutral, while the remaining 7% are now classified as pathogenic or likely pathogenic. Neutral gene variants are generally considered benign, or non-disease-causing, while pathogenic variants are gene mutations that increase an individual’s cancer risk.

The findings are significant because they will contribute to well-informed patient management strategies. A patient with a pathogenic BRCA gene test result may consider options to reduce the risk of developing cancer in the future, including increased screening or prophylactic mastectomy or oophorectomy (removal of breasts or ovaries). Participating commercial laboratories notify physicians of changes in classifications for affected patients.

The BRCA Share Initiative is intended to help scientists, physicians and laboratory experts improve the interpretation of BRCA gene mutations for patient testing and cancer research. With the release of the new set of variants and clinical data, BRCA Share is the largest database providing access to high-quality BRCA genetic data that has been researched and curated to determine clinical significance. Other public BRCA data initiatives collect a mix of curated and uncurated BRCA variant information from participating laboratories. Data curation is an essential step in maximizing the likelihood that data is clinically appropriate and actionable. Variants that are curated have been individually researched and categorized to determine the likelihood that they confer increased cancer risk. Advances in scientific knowledge may lead to new variant discoveries and reclassifications.

"We created BRCA Share to accelerate BRCA science and bring clarity to BRCA patient testing, and in short order, that’s exactly what this initiative has done," said Charles (Buck) Strom, M.D., Ph.D., FAAP, FACMG, HCLD, vice president, genetics and genomics, Quest Diagnostics. "This doesn’t just benefit patients of Quest, or LabCorp, or Inserm’s participating labs. This initiative benefits anyone in the global medical community seeking robust analysis of BRCA genetics based on shared pooling of clinical-grade data and expertise."

"The promise of BRCA Share was to significantly improve BRCA diagnostics and enhance patient care and treatment," said Marcia Eisenberg, Ph.D., chief scientific officer of LabCorp Diagnostics. "BRCA Share has already delivered on that promise, and it will help physicians and patients make more informed monitoring and treatment decisions that can improve health and improve lives."

"BRCA Share demonstrates that public-private data sharing collaborations, funded by commercial parties, can hasten advances in medical research that will benefit patients. This first experience encourages Inserm Transfert to continue its development strategy on health databases whenever it is relevant with industrial partners," said Pascale Augé, Ph.D., CEO, Inserm Transfert.

"In little more than a year, this initiative has provided more than 1,300 new variants and has lifted the veil of uncertainty from 375 variants and identified 334 likely cancer risk variants," added Prof. Christophe Béroud, Pharm.D., Ph.D., leader of the "Genetics and Bioinformatics" research team, Inserm/Aix Marseille University (AMU) UMR_S910.

Research entities and individuals with a research-only focus on BRCA — including physicians and patients — can participate in BRCA Share at no charge. The new data is available, beginning today, at View Source and View Source . Commercial laboratories may participate in BRCA Share by paying an annual fee to Inserm determined on a sliding scale to fund research and administrative expenses.

Since the launch of BRCA Share, nearly 1,000 scientists from 49 countries have registered to access BRCA Share for research purposes.

To participate, BRCA Share commercial laboratory members must submit their BRCA variant data, which Inserm’s variant team then researches and curates. When a variant is curated and/or reclassified, Inserm notifies BRCA Share members and uploads the new data into BRCA Share. BRCA Share’s participating laboratories may then begin to use the new data to inform decisions about patient test results.

Members of BRCA Share are free to also share their BRCA data with other datashare initiatives.

On May 31, 2016 Blaze Bioscience, Inc., the Tumor Paint Company, a biotechnology company focused on guided cancer therapy, reported that the company’s Senior Vice President of Development, Dennis Miller, Ph.D., will present at the American Society of Surgical Oncology (ASCO) (Free ASCO Whitepaper) 2016 Annual Meeting, taking place in Chicago, IL on June 3-7, 2016 (Press release, Blaze Bioscience, MAY 31, 2016, View Source [SID:1234512904]).

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The poster presentation, titled "Phase 1 Dose Escalation and Expansion Safety Study of BLZ-100 in Pediatric Subjects with Primary Central Nervous System Tumors," will highlight the company’s first study in the pediatric population.

Details of the poster presentation are as follows:
Trials in Progress Presentation – Pediatric Oncology Session
Date: Monday, June 6, 2016
Time: 8:00 AM – 11:30 AM CDT
Location: Hall A Poster
Number: 274b
Abstract Number: TPS10584

About BLZ-100
BLZ-100 is the first product candidate from Blaze’s Tumor Paint platform and consists of an Optide (optimized peptide) and a fluorescent dye, which emits light in the near-infrared range. Tumor Paint products are designed to provide real-time, high-resolution intraoperative visualization of cancer cells, potentially enabling more precise, complete resection of cancer throughout surgery. Preclinical utility of Tumor Paint technology has been demonstrated in a wide range of cancer types. BLZ-100 is an investigational agent currently in multiple Phase 1 proof-of-concept clinical studies to evaluate the safety and imaging characteristics of BLZ-100 in solid tumors, including brain, breast, lung, prostate, and colorectal cancer. More details about on-going trials are available at www.blazebioscience.com or www.clinicaltrials.gov.

On May 31, 2016 Blue Earth Diagnostics Ltd., a molecular imaging diagnostics company, reported that the U.S. Food and Drug Administration (FDA) has approved Axumin (fluciclovine F 18) injection, a novel molecular imaging agent indicated for use in positron emission tomography (PET) imaging to identify suspected sites of prostate cancer recurrence in men who have elevated blood levels of prostate specific antigen (PSA) following prior treatment (Press release, Blue Earth Diagnostics, MAY 31, 2016, View Source [SID:1234512906]). Axumin is the first FDA-approved F18 PET imaging agent indicated for use in patients with suspected recurrent prostate cancer.

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Prostate cancer is the second leading cause of cancer death in men. While most primary prostate cancer can be successfully treated, recurrence occurs in up to one-third of patients. Recurrent disease is typically detected by a rise in PSA levels but often the location and extent of the disease cannot be detected by conventional imaging. Of those who experience biochemical recurrence, approximately one-third develop metastatic prostate cancer. Axumin was developed to target the increased amino acid transport that occurs in many cancers, including prostate cancer. It is labeled with the radioisotope F18, enabling it to be visualized in the body with PET imaging.

"FDA approval of Axumin is a major milestone for Blue Earth Diagnostics, and we hope also for patients and their physicians," said Jonathan Allis, D. Phil., CEO of Blue Earth Diagnostics Ltd. "Axumin is our first approved product and we believe that it will benefit patients who are affected by biochemically recurrent prostate cancer. Axumin will be increasingly available in coming months through the national radiopharmacy network of our exclusive U.S. commercial manufacturer and distributor, Siemens’ PETNET Solutions."

"Approximately 180,000 new cases of prostate cancer are expected to be diagnosed in the United States in 2016, and between 20 to 30 percent of patients receiving primary therapy will develop biochemically recurrent disease," said Brian F. Chapin, M.D., Assistant Professor, Department of Urology, The University of Texas MD Anderson Cancer Center. "There is a need for clinical imaging techniques that can detect and localize suspected recurrent prostate cancer to facilitate the most appropriate patient management decision. Current commercially available imaging techniques have some limitations in terms of identifying recurrent tumors, which may impact subsequent patient management decisions. Additionally, many patient care options for men with suspected recurrent prostate cancer have uncertain benefits that may not justify the risk of side effects. New imaging procedures that can provide reliable information can be useful tools for effective patient management and care."

"An imaging agent with sufficient diagnostic performance to adequately detect and localize recurrent prostate cancer can provide referring physicians with actionable information to guide biopsy and inform management decisions for their patients," said David M. Schuster, M.D., a Georgia Research Alliance Distinguished Cancer Scientist, Associate Professor of Radiology and Imaging Sciences, and Director of the Division of Nuclear Medicine and Molecular Imaging at Emory University School of Medicine. "The fluciclovine molecule in Axumin was originally developed at Emory by Mark Goodman, Ph.D., and detects the upregulation of amino acid transport that occurs in prostate cancer and can potentially identify recurrent prostate cancer more reliably than conventional imaging techniques. The product will be convenient for patients and imaging facilities, as it can be made widely available and the entire imaging procedure can typically be completed in less than 30 minutes."

The FDA-approved prescribing information provides summaries from two clinical studies of Axumin, including an evaluation of Axumin images from 105 patients by three independent readers who were unaware of the clinical details of each patient or whether the biopsy of the prostate gland was positive or negative for cancer. On average, a correct image finding was identified in 77% of patients (range: 75%-79%). For cancer outside the region of the prostate, a correct image finding for cancer was identified in an average of 90% of patients (range: 88%-93%). The results seem to be affected by PSA levels with, in general, lower PSA levels in patients with negative scans than in those with positive scans. In patients with PSA levels ≤ 1.78 ng/mL, 15 of 25 had a positive scan, with 11 confirmed as positive by histology; 71 of 74 patients with PSA levels > 1.78 ng/mL had a positive scan, of which 58 were confirmed as positive (see full U.S. prescribing information at www.axumin.com).

Axumin will be commercially available through the national radiopharmacy network of the company’s exclusive U.S. commercial manufacturer and distributor, Siemens’ PETNET Solutions. Initial commercial production of Axumin is underway at certain regional radiopharmacies, and increasingly broader availability is planned in coming months.

Both Emory University and inventor Goodman, Professor of Radiology and Imaging Sciences and Director of the Radiopharmaceutical Discovery Lab at Emory, are eligible to receive royalties for this technology.

About AxuminTM (fluciclovine F 18)

Axumin (fluciclovine F 18) injection is a novel product indicated for use in positron emission tomography (PET) imaging to identify suspected sites of prostate cancer recurrence in men. Recurrence of prostate cancer is suspected by an increase in prostate specific antigen (PSA) levels following initial therapy. PET imaging with Axumin may identify the location and extent of such recurrence. Axumin was developed to enable visualization of the increased amino acid transport that occurs in many cancers, including prostate cancer. It consists of a synthetic amino acid that is preferentially taken up by prostate cancer cells compared with surrounding normal tissues, and is labeled with the radioisotope F18 for PET imaging. Axumin was approved by the U.S. Food and Drug Administration on May 27, 2016 following Priority Review, and is the first product commercialized by Blue Earth Diagnostics, which licensed the product from GE Healthcare. The molecule is being investigated by Blue Earth Diagnostics for other potential cancer indications, such as glioma.

Indication and Important Safety Information About Axumin

INDICATION

Axumin (fluciclovine F 18) injection is indicated for positron emission tomography (PET) imaging in men with suspected prostate cancer recurrence based on elevated blood prostate specific antigen (PSA) levels following prior treatment.

IMPORTANT SAFETY INFORMATION

Image interpretation errors can occur with Axumin PET imaging. A negative image does not rule out recurrent prostate cancer and a positive image does not confirm its presence. The performance of Axumin seems to be affected by PSA levels. Axumin uptake may occur with other cancers and benign prostatic hypertrophy in primary prostate cancer. Clinical correlation, which may include histopathological evaluation, is recommended.
Hypersensitivity reactions, including anaphylaxis, may occur in patients who receive Axumin. Emergency resuscitation equipment and personnel should be immediately available.
Axumin use contributes to a patient’s overall long-term cumulative radiation exposure, which is associated with an increased risk of cancer. Safe handling practices should be used to minimize radiation exposure to the patient and health care providers.
Adverse reactions were reported in ≤ 1% of subjects during clinical studies with Axumin. The most common adverse reactions were injection site pain, injection site erythema and dysgeusia.
To report suspected adverse reactions to Axumin, call 1-855-AXUMIN1 (1-855-298-6461) or contact FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Full Axumin prescribing information is available at: www.axumin.com.

About Prostate / Recurrent Prostate Cancer

Prostate cancer is the second leading cause of cancer death in men. While most primary prostate cancer can be successfully treated, the disease recurs in up to one-third of patients. In some patients recurrent disease is detectable only by a rise in prostate specific antigen (PSA) levels, yet the location of the recurrence cannot consistently be located by conventional imaging, severely limiting treatment guidance for these patients.

About Positron Emission Tomography (PET) Imaging

Positron emission tomography (PET) is an imaging test that uses a special type of scanner in conjunction with a radiolabeled tracer (a molecular imaging agent) to visually examine biochemical processes in the body. PET scan images depict biological function and are complementary with technologies which show anatomical information, such as computed tomography (CT) scans or magnetic resonance imaging (MRI).

On May 31, 2016 Novocure (NASDAQ:NVCR) reported that the United States Food and Drug Administration (FDA) has approved its investigational device exemption (IDE) application to initiate the METIS trial. METIS is a multi-center, phase 3, pivotal, open-label study of radiosurgery with or without Tumor Treating Fields (TTFields) for 1-10 brain metastases from non-small cell lung cancer (NSCLC) (Press release, NovoCure, MAY 31, 2016, View Source [SID:1234512907]). 270 patients will be randomized 1:1 to receive either TTFields delivered at an output frequency of 150kHz with supportive care or supportive care alone after radiosurgery. The primary endpoint of the METIS trial is time to first cerebral progression. Secondary endpoints include, among others, time to neurocognitive failure, overall survival and radiological response rate following study treatments.

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TTFields are low-intensity, alternating electric fields delivered to the region of a tumor. TTFields exert forces on key electrically charged molecules essential to the mitotic process by which all cells divide. Interference with the normal functioning of these key molecules leads to cell death through multiple pathways. Treatment with TTFields, delivered via Optune, is currently approved in the United States and European Union for newly diagnosed and recurrent glioblastoma and in Japan for recurrent glioblastoma. METIS will be Novocure’s first phase 3 pivotal trial outside of glioblastoma. Novocure has ongoing or completed phase 2 pilot trials in brain metastases, non-small cell lung cancer, pancreatic cancer, ovarian cancer and mesothelioma.

"Novocure remains focused on increasing Optune adoption for glioblastoma and on developing TTFields for a variety of additional solid tumors," said Asaf Danziger, Chief Executive Officer. "We look forward to initiating a phase 3 pivotal trial in brain metastases given the unmet medical need. With the FDA IDE approval in hand, we are now working closely with trial sites and institutional review boards to open sites and enroll patients as quickly as possible."

About Brain Metastases

Metastatic cancer is cancer that has spread from the place where it first started to another place in the body. The exact incidence of brain metastases is unknown because no national cancer registry documents brain metastases, but it has been estimated that 98,000 to 170,000 new cases are diagnosed in the United States each year. Brain metastases cause an estimated 20% of all cancer deaths in the United States annually.

Tumor Treating Fields (TTFields) are not approved for the treatment of brain metastases by the U.S. Food and Drug Administration. The safety and effectiveness of TTFields therapy for brain metastases has not been established.