On October 11, 2016 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that the first patient has been enrolled in the global phase 3 QuANTUM-First study evaluating the oral FLT3-ITD inhibitor quizartinib in patients with newly-diagnosed FLT3-ITD-positive (+) acute myeloid leukemia (AML) (Press release, Daiichi Sankyo, OCT 11, 2016, View Source [SID:SID1234515762]).

QuANTUM-First is a randomized, double-blind, placebo-controlled study evaluating quizartinib in combination with induction and consolidation chemotherapy and as maintenance monotherapy in patients with newly-diagnosed FLT3-ITD+ AML. The primary endpoint of the study is event-free survival. Secondary endpoints include overall survival, complete remission rate, composite complete remission rate and the percentage of subjects achieving a complete remission with no evidence of minimal residual disease.

Approximately 30 percent of patients with AML have a genetic mutation called FLT3-ITD, which is associated with more aggressive disease, resulting in increased relapse rate and reduced overall survival compared to those without this mutation.1FLT3-ITD mutations are more common than FLT3-TKD mutations, which occur in approximately 10 percent of AML patients.1There is controversy as to whether FLT3-TKD mutations carry as poor a prognosis as FLT3-ITD mutations.1Currently, there are no approved targeted treatments for FLT3-ITD+ AML, with little change in the treatment of AML for the past 30 years.2

"It is well established that patients with FLT3-ITD mutated AML have an overall worse prognosis compared to those without this specific mutation," said Harry Erba, MD, PhD, Chair of the QuANTUM-First Steering Committee and Professor of Medicine and Director of the Hematologic Malignancy Program at the University of Alabama at Birmingham. "In this study we are evaluating whether adding quizartinib to standard first-line chemotherapy will help delay or prevent relapse, which in turn may impact overall survival in patients with FLT3-ITD+ AML."

"Given the high unmet need in FLT3-ITD+ AML, we are moving forward with a comprehensive clinical development program investigating the role of quizartinib in multiple lines of treatment including induction and consolidation chemotherapy, maintenance therapy and salvage therapy," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "Additionally, we also are looking to combine quizartinib with other investigational agents in our pipeline such as our MDM2 and BRD4 inhibitors where science suggests combining different mechanisms of action may help improve outcomes."

QuANTUM-First is expected to enroll more than 500 patients between 18 and 75 years of age in the Americas, Europe and Asia-Pacific. More information about the study is available at ClinicalTrials.gov or www.QuantumFirstStudy.com.

About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is the most common type of acute leukemia accounting for about 33 percent of all new cases of leukemia.3 An aggressive blood and bone marrow cancer, AML causes uncontrolled growth and accumulation of malignant white blood cells that fail to function normally and interfere with the production of normal blood cells. 3 The five-year survival rate of AML is approximately 26 percent, which is the lowest of all leukemias.3

About Quizartinib
Quizartinib is an investigational oral small molecule that potently and selectively inhibits FLT3-ITD (FMS-like tyrosine kinase-3-internal tandem duplication), which is a growth driver of abnormal cells that contribute to the development of AML.4 Quizartinib has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of AML. Quizartinib also has been granted Fast Track Designation by the FDA for the treatment of relapsed/refractory AML. Quizartinib has not been approved by any regulatory authority for uses under investigation.
In addition to QuANTUM-First, a global, randomized, open-label, phase 3 study called QuANTUM-R is evaluating quizartinib as monotherapy in patients with FLT3-ITD+ AML who are refractory to or have relapsed after first-line treatment with or without hematopoietic stem cell transplant (HSCT). The primary objective of QuANTUM-R is to determine whether quizartinib prolongs overall survival compared to salvage chemotherapy, and the secondary objective is to determine event-free survival. The trial is expected to enroll approximately 363 patients age 18 or older in North America, Europe and Asia-Pacific. More information about QuANTUM-R is available at www.QuantumRStudy.com or ClinicalTrials.gov.

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On October 12, 2016 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and the Dana-Farber Cancer Institute reported a preclinical research collaboration focused on lung cancer (Press release, Daiichi Sankyo, OCT 11, 2016, View Source [SID:SID1234515767]).

A team of scientists led by Pasi A. Jänne, M.D., Ph.D., Director, Lowe Center for Thoracic Oncology, Dana-Farber Cancer Institute; Scientific Director, Belfer Center for Applied Cancer Science; and, Professor of Medicine, Harvard Medical School will partner with Daiichi Sankyo on the development of a translational pharmacology package with unique experimental animal and patient derived xenograft models that were established at Dana-Farber Cancer Institute. The collaboration will leverage the translational medicine expertise of the Belfer Center. Financial terms of the agreement were not disclosed.

"We are excited to enter into this unique partnership with Daiichi Sankyo. Working together we can accelerate the development of new therapeutic strategies for patients with lung cancer," said Dr. Jänne.
"Despite several recent significant advances in the treatment of lung cancer with EGFR mutations,many patients still die of this disease, hence our urgency and obligation to pursue swiftly great science," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "By partnering with scientists at Dana-Farber, we are looking to better understand drivers of disease as well as initial and secondary resistance to established targeted treatments with the ultimate goal of identifying a potential new treatment for patients with lung cancer."

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On October 11, 2016 Delcath Systems, Inc. (NASDAQ: DCTH), an interventional oncology Company focused on the treatment of primary and metastatic liver cancers, reported that five new clinical trial sites in the U.S. have been activated in the Company’s global FOCUS Phase 3 clinical trial for patients with hepatic dominant ocular melanoma (the FOCUS Trial) (Press release, Delcath Systems, OCT 11, 2016, View Source;p=RssLanding&cat=news&id=2210776 [SID:SID1234515730]). These accredited centers join several other prestigious centers as active participants in the FOCUS Trial. Currently, Delcath now has a total of 8 cancer centers in the U.S. open for patient enrollment in the FOCUS Trial.

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The new participating centers are:

Ohio State University Comprehensive Cancer Center
MD Anderson Cancer Center
Thomas Jefferson University Sidney Kimmel Cancer Center
University of Chicago Comprehensive Cancer Center
University of Maryland Greenebaum Cancer Center
"We are pleased to add these respected cancer research institutions to our FOCUS Trial, and to expand the regional coverage available to patients in the U.S. with ocular melanoma liver metastases," said Jennifer K. Simpson, Ph.D., MSN, CRNP, President and CEO of Delcath. "Interest in the FOCUS Trial has been strong, and we expect to achieve our goal of activating approximately 30 centers in both the U.S. and Europe in the coming months."

About the FOCUS Trial
The FOCUS Trial is a global Phase 3 clinical study evaluating the safety, efficacy and pharmacokinetic profile of the Company’s Melphalan/HDS system versus best alternative care in 240 patients with ocular melanoma liver metastases. The FOCUS Trial’s primary endpoint is a comparison of overall survival between the two study arms; secondary and exploratory endpoints include progression-free survival, overall response rate and quality-of-life measures. The FOCUS Trial is being conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA). The SPA provides agreement that the Phase 3 trial design adequately addresses objectives that, if met, would support the submission for regulatory approval of Melphalan/HDS.

On October 11, 2016 Celsion Corporation (NASDAQ:CLSN), a leading oncology drug development company, reported a collaboration with the Children’s Research Institute to conduct a clinical study of ThermoDox, Celsion’s heat activated liposomal encapsulation of doxorubicin, in combination with magnetic resonance-guided high intensity focused ultrasound (MR-HIFU) to treat relapsed or refractory solid tumors in children and young adults (Press release, Celsion, OCT 11, 2016, View Source [SID:SID1234515733]). This investigator-sponsored Phase I clinical study is being partially funded by the National Institutes of Health and is expected to commence in the fourth quarter of 2016.

"Even with the use of intensive therapy, the prognosis for children diagnosed with metastatic sarcoma and recurrent solid tumors remains poor and has not improved over the past three decades," stated Dr. Nicolas Borys, Celsion’s chief medical officer. "Recent advances in the use of non-invasive MR-HIFU coupled with novel therapies such as ThermoDox have demonstrated the clear potential to overcome the challenges to treating pediatric malignancies by enabling safer, more tolerable targeted therapies with the potential to change cancer treatment paradigms."

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The trial targeting treatment of childhood sarcomas will be carried out as a multi-disciplinary collaboration among Celsion, the research groups of Dr. AeRang Kim, MD, PhD at the Children’s National Medical Center – Department of Hematology/Oncology, and Dr. Brad Wood and Dr. Rosandra Kaplan at the National Institutes of Health.

"Celsion’s experience in combining ThermoDox with HIFU, a non-invasive next generation heating technology, supports this very important research in childhood cancers. From a safe dose, ThermoDox’s proven ability to deliver high concentrations of an effective chemotherapy directly to a heated tumor makes it an ideal candidate for a trial involving children and young adults," said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. "This study will further define ThermoDox’s potential in combination with ultrasound-induced hyperthermia, and highlight potential applications of ThermoDox in combination with a broad range of heating technologies that could address an even larger population of patients."

ThermoDox is currently in late stage clinical trials in primary liver cancer and recurrent chest wall breast cancer. It is positioned for use with multiple heating technologies, and has the potential for applications in the treatment of other forms of cancer including metastatic liver and non-muscle invading bladder cancers.

About ThermoDox
Celsion’s most advanced program is a heat-mediated, tumor-targeting drug delivery technology that employs a novel heat-sensitive liposome engineered to address a range of difficult-to-treat cancers. The first application of this platform is ThermoDox, a lyso-thermosensitive liposomal doxorubicin (LTLD), whose novel mechanism of action delivers high concentrations of doxorubicin to a region targeted with the application of localized heat at 40°C, just above body temperature. ThermoDox has the potential to address a broad range of cancers.

Celsion’s LTLD technology leverages two mechanisms of tumor biology to deliver higher concentrations of drug directly to the tumor site. In the first mechanism, rapidly growing tumors have leaky vasculature, which is permeable to liposomes and enables their accumulation within tumors. Leaky vasculature influences a number of factors within the tumor, including the access of therapeutic agents to tumor cells. Administered intravenously, ThermoDox is engineered with a half-life to allow significant accumulation of liposomes at the tumor site as these liposomes recirculate in the blood stream. In the second mechanism, when an external heating device heats tumor tissue to a temperature of 40°C or greater, the heat-sensitive liposome rapidly changes structure and the liposomal membrane selectively dissolves, creating openings that can release a chemotherapeutic agent directly into the tumor and into the surrounding vasculature. Drug concentration increases as a function of the accumulation of liposomes at the tumor site, but only where the heat is present. This method damages only the tumor and the area related to tumor invasion, supporting more precise drug targeting.

On October 11,2016 Kite Pharma, Inc. (Nasdaq:KITE) reported that it will hold an Investor Day on October 18, 2016 in New York where the company will share updates on its advancing pipeline of chimeric antigen receptor (CAR) and T cell receptor (TCR) product candidates, next generation research and development programs, and KTE-C19 launch readiness. The event and live webcast will begin at 12:00pm Eastern Time (Press release, Kite Pharma, OCT 11, 2016, View Source [SID:SID1234515737]). The live audio webcast can be accessed through the Events and Presentations section under the Investors tab of Kite’s website at www.kitepharma.com. Following the live audio webcast, a replay will be available on Kite’s website for approximately 30 days.

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About KTE-C19

Kite Pharma’s lead product candidate, KTE-C19, is an investigational therapy in which a patient’s T cells are engineered to express a CAR to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. KTE-C19 has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.