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Regulus Expands Clinical Trial Collaboration with GSK

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.

8-K – Current report

On June 1, 2016 Nektar Therapeutics (Nasdaq: NKTR) reported that it has entered into an agreement with Daiichi Sankyo Europe for Nektar’s investigational drug therapy, ONZEALD (etirinotecan pegol, NKTR-102), which has completed a Phase 3 clinical trial (the BEACON study) in patients with advanced breast cancer (Filing, 8-K, Nektar Therapeutics, JUN 1, 2016, View Source [SID:1234512942]). The agreement grants Daiichi Sankyo Europe exclusive rights to market ONZEALD in Europe (EEA), Switzerland and Turkey. Nektar Therapeutics will retain rights to ONZEALD in the United States and the rest of the world.

Under the terms of the agreement, Nektar Therapeutics is entitled to an upfront payment of $20 million as well as an additional $60 million in milestone payments, based upon the achievement of European regulatory milestones and European sales of ONZEALD. Nektar is also entitled to significant double-digit royalties on net sales in Europe.

"This new collaboration with Daiichi Sankyo Europe allows Nektar to advance ONZEALD to a potential conditional approval and availability in Europe as early as next year, and also enables us to retain ownership of the drug in the U.S. and rest of world," said Howard W. Robin, President and Chief Executive Officer of Nektar Therapeutics. "We are pursuing conditional approval for ONZEALD based on highly promising data from our Phase 3 BEACON clinical trial in the pre-specified subgroup of patients with advanced breast cancer who have a history of brain metastases. A diagnosis of brain metastases in women with advanced breast cancer is devastating and there are no therapies approved to treat this specific patient population."

Nektar plans to submit an MAA filing in June 2016 seeking conditional approval from the European Medicines Agency (EMA) for the use of ONZEALD in the treatment of patients with advanced breast cancer and brain metastases. On May 26, 2016, the Committee for Medicinal Products for Human Use (CHMP) granted an accelerated assessment procedure for the planned ONZEALD filing, which provides for an accelerated MAA review timeline.

Nektar will be responsible for sponsoring and funding the confirmatory trial which will support the Marketing Authorization Application (MAA) filing for ONZEALD in Europe. The data from the confirmatory trial can be used by Nektar for a potential U.S. new drug application (NDA) filing for ONZEALD.

Breast cancer is the most frequently diagnosed cancer in women worldwide with nearly 1.7 million new cases diagnosed in 2012.1 There are approximately 250,000 newly-diagnosed cases of breast cancer in the United States and 470,000 in Europe each year. 1 Approximately 10-30 percent of patients with advanced breast cancer are also diagnosed with brain metastases. 2

Nektar’s planned MAA filing is based upon data from a subgroup of patients from the completed BEACON study of single-agent ONZEALD in patients with advanced breast cancer. In this subgroup of 67 patients who also had a history of brain metastases, treatment with single-agent ONZEALD resulted in an improvement in median overall survival (OS) of 5.2 months compared to treatment with a single-agent chemotherapy of physician’s choice (TPC) (10 months vs. 4.8 months, P < 0.01). TPC included a choice of ixabepilone, vinorelbine, gemcitabine, eribulin or a taxane. In the planned primary analysis for the overall patient population in the BEACON study, ONZEALD median OS was 2.2 months longer than TPC (12.4 months vs. 10.3 months, P= 0.08).3 In the overall patient population in the BEACON study, fewer patients in the ONZEALD arm had grade 3 or worse adverse events (AEs) than those in the TPC arm (204 [48%] vs. 256 [63%]; p<0·0001).3 The most common grade 3 and above AEs observed with ONZEALD were diarrhea (9.6%), neutropenia (9.6%), anemia (4.7%) and fatigue (4.5%). The most common grade 3 and above AEs observed with TPC were neutropenia (30.8%), anemia (4.7%), and dyspnea (4.4%).

In order to satisfy the EMA’s requirement for additional controlled data with the MAA for conditional approval, Nektar will sponsor a global, randomized Phase 3 trial of ONZEALD in approximately 350 patients with advanced breast cancer and brain metastases. The trial will compare ONZEALD to TPC and the primary endpoint in the trial will be OS. The trial will include a pre-specified interim analysis for OS which is to be conducted after 130 events have been observed in the trial. The U.S. Food and Drug Administration has also reviewed the Phase 3 study design with the Statistical Analysis Plan, and indicated the trial could serve as a potential registrational study by Nektar for purposes of seeking approval of ONZEALD to treat this patient population in the U.S.
The EMA may grant conditional marketing authorization when the potential treatment addresses a severely debilitating disease with an unmet medical need, has a positive benefit to risk profile, and the benefits to public health of its immediate availability outweigh the risks inherent in the fact that additional data are still required. Ongoing or new studies must be completed with the objective of confirming that the benefit to risk balance is positive. A conditional approval granted by the EMA is renewed on an annual basis until all obligations have been fulfilled, at which point a full approval may be granted by the EMA.

For additional terms and conditions of the licensing agreement between Nektar and Daiichi Sankyo Europe, please refer to the Current Report on Form 8-K filed today with the Securities and Exchange Commission.

About ONZEALD (etirinotecan pegol) (formerly NKTR-102)

ONZEALD is the first long-acting topoisomerase I inhibitor with an extended half-life and a unique structure that is designed to concentrate the drug in tumors. In patients, ONZEALD leads to greatly prolonged plasma SN38 exposure compared with irinotecan (elimination half-life of 37 days compared with 2 days) yet peak SN38 concentrations are at least 5- to 10-times less, which may also result in a favorable tolerability profile. ONZEALD was evaluated in a Phase 3, open-label, randomized, multicenter study (the BEACON study) that enrolled 852 women with locally recurrent or metastatic breast cancer, who have previously been treated with an anthracycline, taxane and capecitabine therapies.

Infinity Announces Launch Of Disease Awareness Campaign For Hematologic Malignancies

On June 1, 2016 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported the launch of a mechanism of disease (MOD) awareness initiative designed to educate community and academic hematologists/oncologists about the role of the tumor microenvironment in patients with certain hematologic malignancies, or blood cancers (Press release, Infinity Pharmaceuticals, JUN 1, 2016, View Source;p=RssLanding&cat=news&id=2173919 [SID:1234512925]). With more research emerging about the role of the tumor microenvironment in hematologic malignancies, Infinity developed this campaign to contribute to the emerging knowledge of this scientific concept and its clinical relevance. The initiative will be showcased in the Infinity booth (#22415) at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place in Chicago from June 3 – June 7, 2016. Additional information and resources on the tumor microenvironment are available on a newly launched website: www.manipulatedTME.com.

In B-cell malignancies, the tumor microenvironment is made up of healthy, non-cancerous cells that are recruited by malignant (cancerous) B-cells to promote tumor survival.[1] The tumor microenvironment is thought to support key characteristics of cancer, including disease progression, resistance to cell death and sustained proliferation.[2]

"Research suggests that the tumor microenvironment may provide protection and support for malignant B-cells that hinder treatment effectiveness, resulting in poor responses to treatment," said Nathan Fowler, M.D., Associate Professor, Department of Lymphoma/Myeloma at the University of Texas MD Anderson Cancer Center. "As hematologists and oncologists continue to conduct research and develop treatment plans for patients living with incurable B-cell malignancies, this online resource may provide insights as to why patients continue to relapse."

The campaign artwork is centered around a sinister character, representing a malignant B-cell, who is supported by cells in the tumor microenvironment, such as macrophages, endothelial cells, and immune cells. The B-cell character is shown to have manipulated these healthy cells to form the tumor microenvironment, a network of support and protection for malignant B-cells that may enable tumor growth and proliferation.

Learn more about the MOD campaign at www.manipulatedTME.com.

Delcath Announces Launch of CHEMOSAT at the HM Sanchinarro University Hospital in Madrid

On June 1, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), a specialty pharmaceutical and medical device company focused on oncology with an emphasis on the treatment of primary and metastatic liver cancers, reported that the General and Digestive Surgery team at HM Sanchinarro University Hospital, led by Emilio Vicente, M.D. and Yolanda Quijano, M.D., has successfully launched the hospital’s Hepatic Chemosaturation Program (Press release, Delcath Systems, JUN 1, 2016, View Source;p=RssLanding&cat=news&id=2173899 [SID:1234512920]). The Sanchinarro team successfully performed three procedures with Delcath’s proprietary Melphalan Hydrochloride for Injection for use with the Delcath Hepatic Delivery System (CHEMOSAT or Melphalan/HDS). The team at HM Sanchinarro is using the CHEMOSAT procedure in peripheral cholangiocarcinoma, neuroendocrine tumours and colorectal liver metastases. HM Sanchinarro University Hospital is the second center in Spain to offer the treatment.

"This is an exciting approach for the treatment of liver tumors," said Drs. Vicente and Quijano. "We believe that the administration of high-dose melphalan directly to the liver has great promise in the treatment of a variety of tumor types, and the associated side effects are also minimized and more manageable. In recent years, the successful development of this percutaneous procedure has been a breakthrough, as it significantly reduces the technique’s morbidity rate. We are very excited about adopting this new procedure at HM Sanchinarro University Hospital."

Jennifer K. Simpson, Ph.D., MSN, CRNP, President and CEO of Delcath Systems Inc., said, "We are extremely pleased that the HM Sanchinarro Hospital has commenced its Chemosaturation Program with our CHEMOSAT Delivery System. HM Sanchinarro is one of the leading cancer centers in Spain, both in terms of treatment and research. This underscores how CHEMOSAT continues to be increasingly accepted as an effective therapy for patients with liver metastasis who would otherwise have few, if any, treatment options."

Atreca to Present at the Jefferies 2016 Global Healthcare Conference

On June 1, 2016 Atreca, Inc., a biotechnology company focused on developing novel therapeutics based on a deep understanding of the human immune response, reported that Tito Serafini, Ph.D., President, Chief Executive Officer, and Co-‐Founder, will present at the Jefferies 2016 Global Healthcare Conference on Wednesday, June 8, 2016 at 3:00 p.m. Eastern Time in New York, NY (Press release, Atreca, JUN 1, 2016, View Source [SID1234522966]). Dr. Serafini will provide an overview of Atreca and business update.