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Celltrion and Teva Announce U.S. FDA Acceptance of Biologics License Application for Proposed Biosimilar to Herceptin® (trastuzumab)

On July 31, 2017 Celltrion, Inc. and Teva Pharmaceutical Industries Ltd. (NYSE and TASE: TEVA) reported that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for CT-P6, a proposed Monoclonal Antibody (mAb) biosimilar to Herceptin1 (INN: trastuzumab) which is used for the treatment of Human Epidermal growth factor Receptor 2 (HER2)-overexpressing breast cancer and metastatic gastric cancer.

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"Following on the heels of our global success with Remsima2 (Inflectra3), our infliximab biosimilar, which has brought affordable and effective biologic treatment to many patients around the world with proven similarity of quality, efficacy and safety to the reference product, we are confident to submit a comprehensive package of quality, nonclinical and clinical data of CT-P6 for FDA review," said Woo Sung Kee, Chief Executive Officer of Celltrion. "If approved, CT-P6 will expand patient access to this important biosimilar treatment option in the U.S."
The BLA for CT-P6 includes data for CT-P6 and reference trastuzumab in terms of efficacy, safety, immunogenicity, pharmacodynamics (PD) and pharmacokinetics (PK). These trials were conducted in over 500 patients in 22 countries.
CT-P6 has been approved by the Korean Ministry of Food and Drug Safety. Celltrion also filed marketing authorization applications for CT-P6 to the European Medicines Agency in October 2016.
"As Celltrion’s North American commercial partner for CT-P6 and CT-P10, a proposed mAb biosimilar to Rituxin2 (rituximab), we look forward to the opportunity to leverage Teva’s strong legacy and U.S. commercial presence in Oncology to bring additional biosimilar treatment options to patients," said Paul Rittman, Senior Vice President and General Manager, Teva Oncology. "With today’s announcement, we are one-step closer to doing so in the U.S. We are very pleased to acknowledge the positive progress in our partnership with Celltrion to help provide these additional options, if approved, to patients living with cancer and other serious diseases."
Celltrion and Teva entered into an exclusive partnership to commercialize CT-P6 and CT-P10 in the U.S. and Canada in October 2016. FDA also accepted for review the Biologics License Application (BLA) for CT-P10, a proposed mAb biosimilar to Rituxan (rituximab) in June 2017.
The BLAs for both CT-P6 and CT-P10 have been accepted for filing by the FDA for standard review, with FDA Regulatory Action expected during the first half of 2018.

Mateon Therapeutics Announces Positive Initial Data from Fifth Cohort of Phase 1b Study of OXi4503 in Relapsed/Refractory AML

On July 31, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported preliminary data from the fifth dose cohort of OX1222, a phase 1b dose-ranging study of OXi4503 in combination with cytarabine in patients with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) (Press release, Mateon Therapeutics, JUL 31, 2017, View Source [SID1234519942]).

Two of four patients had morphological complete remissions after one cycle of treatment with 9.76 mg/m2 of OXi4503. Both patients will receive a second cycle of treatment.

To date, five of 21 study patients in OX1222 have achieved complete remission. At lower doses of OXi4503, the complete remissions occurred following two cycles of treatment, with AML blast reductions noted following one cycle of treatment. In addition to the complete remissions, three other patients in the study experienced meaningful AML blast reductions – two in the third cohort and one in the fourth cohort.

"Every dose of OXi4503 tested in this study has shown encouraging signs of efficacy and a favorable safety profile, with the highest doses showing the earliest and best activity," said William D. Schwieterman, M.D., President and Chief Executive Officer of Mateon. "We continue to be excited about the potential to bring a much needed new treatment option to these very ill patients."

There were no dose-limiting toxicities observed in the fifth cohort and OXi4503 continued to have a favorable safety profile. The most common adverse events (AEs) of any grade across all cohorts include neutropenia, fever, nausea, anemia and diarrhea. Grade 3 or above AEs which were related to treatment include decreased neutrophil count (28%), decreased platelet count (28%), febrile neutropenia (22%), anemia (17%), and decreased white blood cell count (11%).

Mateon is continuing pharmaceutical partnering discussions to secure a partner or additional capital prior to initiating additional clinical studies of OXi4503 in AML.

About Acute Myeloid Leukemia
A devastating form of cancer of the blood and bone marrow, AML is the most common type of acute leukemia in adults and accounts for the greatest number of leukemia deaths in the United States. There is no standard regimen of care for patients who relapse following front-line treatment or have refractory disease. According to the NIH’s National Cancer Institute Surveillance, Epidemiology and End Results (SEER) program, there are an estimated 21,380 new cases of AML and 10,590 deaths expected in 2017 in the United States. AML arises from a clonal hematopoietic stem cell and is characterized by accumulation of malignant myeloblasts in the bone marrow and resulting in ineffective hematopoiesis. AML often responds initially to front-line treatment of conventional cytotoxic chemotherapy, but it often relapses and long-term disease-free survival is low, posing a significant challenge to treat relapsed and/or refractory disease.

About OXi4503
OXi4503 has received Fast Track designation from the U.S. Food and Drug Administration for the treatment of AML. It is a VDA that disrupts tumor vasculature residing within bone marrow while simultaneously targeting malignant myeloid cells. Preclinical data show that OXi4503 disrupts bone marrow endothelial cells which normally protect AML cells from exposure to chemotherapeutic agents. In human xenograft animal models of AML, OXi4503 has demonstrated almost complete elimination of leukemic cells. In other animal models, the combination of OXi4503 and cytarabine has shown a much greater effect against AML than either agent alone.

Kite Files the Industry’s First CAR-T Marketing Authorization Application in Europe for Axicabtagene Ciloleucel

On July 31, 2017 Kite Pharma, Inc. (Nasdaq:KITE), a leading cell therapy company, reported that it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for axicabtagene ciloleucel as a treatment for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) who are ineligible for autologous stem cell transplant (Press release, Kite Pharma, JUL 31, 2017, View Source [SID1234519941]). This application represents the first chimeric antigen receptor (CAR) T-cell therapy submitted to the EMA. Axicabtagene ciloleucel is currently under review by the U.S. Food and Drug Administration (FDA), and the FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2017.

The MAA for axicabtagene ciloleucel is supported by data from the ZUMA-1 trial, which met the primary endpoint of objective response rate (ORR), with 82 percent (p < 0.0001) of patients achieving a response after a single infusion of axicabtagene ciloleucel. At a median follow-up of 8.7 months, 44 percent of patients were in ongoing response, which included 39 percent of patients in complete response (CR). The most common Grade 3 or higher adverse events included cytokine release syndrome and neurologic events, which were generally reversible.

"The MAA submission of axicabtagene ciloleucel marks an important global milestone in the development of engineered T cell therapy," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We are excited to work closely with the EMA, Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) to help bring this potentially transformative therapy to patients in the EU."

Non-Hodgkin lymphoma (NHL) is a type of blood cancer that affects around 93,000 people in Europe every year.i DLBCL is one of the subtypes of NHL that is aggressive or fast growing.ii While many patients can achieve and maintain complete remission after initial treatment, patients who experience relapse or do not respond to initial treatment historically have poor outcomes. The company estimates that approximately 7,800 patients in the EU 5 alone may benefit from CAR-T therapy.

In May 2016, the CHMP and CAT granted access to its newly established Priority Medicines (PRIME) regulatory initiative for axicabtagene ciloleucel in the treatment of patients with refractory DLBCL. Access to the PRIME initiative is granted by the EMA to support the development and accelerate the review of new therapies to treat patients with a high unmet need.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (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. Axicabtagene ciloleucel 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 (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

Diffusion Pharmaceuticals Selects Contract Research Organization for Phase 3 Clinical Trial of Lead Compound TSC for Inoperable GBM Brain Cancer

On July 31, 2017 Diffusion Pharmaceuticals, Inc. (NASDAQ:DFFN), a clinical stage biotechnology company developing novel small molecule therapeutics for cancer and other hypoxia-related diseases, reported that it has selected a premier Clinical Research Organization (CRO) to conduct a Phase 3 clinical trial of Diffusion’s lead molecule, trans sodium crocetinate (TSC), in inoperable glioblastoma multiforme (GBM) patients (Press release, Diffusion Pharmaceuticals, JUL 31, 2017, View Source [SID1234519940]). Diffusion also entered into agreements with top tier partners to manage the MRI imaging, clinical data management, drug supply and other functions related to the trial. Diffusion plans to initiate this Phase 3 clinical trial by the end of 2017.

"After reaching the critical milestone of manufacturing drug product for our Phase 3 trial earlier this month, we have now reached another major milestone with the selection of our full clinical trial team, including a leading global CRO and first-in-class providers of imaging and data management services," said David Kalergis, Diffusion’s Chairman and CEO. "We are pleased to have engaged such skilled and experienced partners for this important endeavor."

Diffusion is now interacting with the FDA on details regarding the design and execution of the planned Phase 3 study. The study will focus on treating newly diagnosed GBM patients who have been judged by their medical team to be inoperable, usually because of the size or location of the tumor. Due to their poor prognosis, these patients are often excluded from participation in other GBM clinical trials. In the Company’s Phase 2 GBM trial, the TSC-treated inoperable group showed a nearly four-fold increase in survival at two years, compared to the control.

Chugai and National Cancer Center Japan Enter into Joint Research Agreement Chugai Joins “MASTER KEY Project”, an industry-academia joint project promoting research and development on rare cancers and genomic medicine

On July 31, 2017 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported the signing of a joint research agreement with the National Cancer Center Japan regarding the "MASTER KEY Project," an industry-academia joint project led by the National Cancer Center Japan (Press release, Chugai, JUL 31, 2017, View Source [SID1234519939]). Chugai will collaborate with the National Cancer Center Japan to promote drugs research and development on rare cancers and genomic medicines.

Rare cancers are defined as cancers with a prevalence of fewer than six cases out of a population of 100,000 persons per year*. As there are an extremely small number of patients and there are more issues related to diagnosis and therapies compared to other types of cancers, there is no consolidated clinical data making it difficult to conduct research, development and clinical studies on these types of cancers.
* View Source (Japanese only)

This project was started in May 2017 with the aim of integrating the advanced know-how, research support functions of the National Cancer Center Japan, and the seeds and development strategies of pharmaceutical companies to build a joint foundation for industry-academia to comprehensively and efficiently promote the development of therapies for rare cancers.

As a top pharmaceutical company in the oncology area, Chugai has participated in Japan’s first industry-academia collaborative nationwide cancer genome screening project, "SCRUM-Japan." Chugai believes that the participation in the "MASTER KEY Project" will further strengthen its collaboration with academia, serving as a first step towards resolving the difficult issues related to rare cancers.

Through participation in the "MASTER KEY Project", Chugai strives to conduct research and development on innovative drugs for rare cancers with limited treatment options and high unmet medical needs.

About the MASTER KEY Project
The MASTER KEY Project (Marker Assisted Selective ThErapy in Rare cancers: Knowledge database Establishing registrY Project) is an industry-academia joint project to promote research and development on rare cancers and genomic medicine led by the National Cancer Center Japan. 11 pharmaceutical companies are participating in the project. The project will conduct registry research to establish a large-scale database that will serve as basic data for research by collecting genomic information, clinical information, and prognosis data on patients with rare cancers. It will also conduct basket trials, a new clinical study method, promoting investigator-initiated or company sponsored clinical trial using drugs suitable for the biomarkers of patient populations with specific biomarkers without restricting or specifying cancer types. The registry clinical research commenced in May 2017.