On January 15, 2015 Cyclenium Pharma Inc., an emerging pharmaceutical company specializing in the discovery and development of novel drug candidates based on proprietary macrocyclic chemistry and the Institute for Research in Immunology and Cancer — Commercialization of Research (IRICoR) along with Université de Montréal and its Institute for Research in Immunology and Cancer (IRIC) announced today the signing of a collaborative research agreement (Press release, Universite de Montreal, JAN 5/, 2015, View Source [SID1234517247]). The collaboration intends to utilize Cyclenium’s proprietary QUEST Library of next generation macrocyclic molecules and associated hit-to-lead optimization expertise in concert with IRIC’s state-of the-art capabilities in biological target identification, characterization and screening, as well as medicinal chemistry. The objective of the collaboration is to discover and develop new drug candidates in cutting-edge target areas for the treatment of cancer and immunological disorders.

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"We are delighted to have forged this collaboration with IRICoR and the team of leading investigators at IRIC in two of our therapeutic focus areas," stated Helmut Thomas, Ph.D., President & Chief Executive Officerof Cyclenium. "Combining IRIC’s exquisite research expertise in the understanding of novel pharmacological targets with our CMRT drug discovery technology and our joint experience in the medicinal application of macrocyclic compounds and proven development success in the macrocycle arena offers superb potential for success in the search for new pharmaceuticals in oncology and immunology indications."

"We are extremely pleased to be working with Cyclenium," said Michel Bouvier, Chief Executive Officer of both IRIC and IRICoR. "We clearly see multiple areas of synergy between IRIC’s drug discovery, screening, and medicinal chemistry expertise with Cyclenium’s leadership position in macrocycle-based drug development."

On December 30, 2014 Tikcro Technologies Ltd. (OTC PK: TIKRF) reported that it has entered into a research and license agreement with Yeda Research and Development Company Ltd., the technology transfer arm of the Weizmann Institute of Science in Israel (Press release Tikcro, DEC 30, 2014, View Source [SID:1234501263]). This agreement is for the development of new antibodies originating from specified research at the Weizmann Institute of Science addressing identified targets of cancer immune checkpoints.

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Under the agreement, Tikcro will provide funding for further research at the Weizmann Institute of Science to develop certain antibodies selected and verified in pre-clinical trials. The antibodies may have high selectivity and binding qualities towards cancer immune checkpoints. Further research and development will be required to promote such antibodies as therapeutic candidates for immune modulation in oncology.

Tikcro, alone or through sub-licensees, will have the right to obtain the research results and to pursue development through commercialization. The license consideration due from Tikcro to Yeda includes royalties from net sales, sub-license fees and fixed fees linked to clinical and commercial sales milestones.

On December 29, 2014 Ignyta reported that the U.S. Food and Drug Administration (FDA) has granted both orphan drug designation and rare pediatric disease designation for Ignyta’s lead product candidate entrectinib for the treatment of neuroblastoma (Press release Ignyta, DEC 29, 2014, View Source [SID:1234501251]).

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"We are pleased that the FDA has provided us these designations, which highlight the potential for entrectinib to address unmet needs of patients with rare cancers," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "Although Ignyta is intrinsically motivated to continue to aggressively pursue our clinical development program for entrectinib in solid tumors for the benefit of adult and pediatric cancer patients everywhere, we are pleased that the incentives provided by these designations – including the potential for seven years of marketing exclusivity and the potential to obtain a valuable Pediatric Disease Priority Review Voucher from the FDA – can potentially provide additional avenues for creating value for our stockholders."

On December 26, 2014 Chugai Pharmaceutical reported that it obtained approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) on December 26, 2014, for "unresectable melanoma with BRAF mutation," for an anti-cancer agent, BRAF inhibitor, vemurafenib tablets (brand name: Zelboraf Tablet 240mg (hereafter, "Zelboraf") (Press release Chugai, DEC 25, 2014, View Source [SID:1234501253]).
Also, Roche Diagnostics K.K. [Main Office: Minato-ku, Tokyo. President & CEO: Makoto Ogasawara (hereafter, "Roche Diagnostics")] obtained approval from the MHLW for in vitro diagnostics to detect the BRAF mutation, "cobas 4800 BRAF V600 Mutation Test," as a companion diagnostics for Zelboraf on December 2, 2014.

It is necessary to detect BRAF mutation with cobas 4800 BRAF V600 Mutation Test before use of Zelboraf for patients with unresectable melanoma with BRAF mutation. As just described, medical approach to select an appropriate therapy for each patient before administration of a drug is called personalized healthcare. It enables to avoid a treatment unlikely to show efficacy. Furthermore, since a treatment unlikely to be effective is not given, personalized healthcare increase the benefit in terms of medical economics.

It is reported that each year 1,300 to 1,400 patients (Globocan 2012) in Japan are newly diagnosed as malignant melanoma (all stages), and the number has been growing. Of these patients, 26.7 to 41.8% have the BRAF gene mutation).

Chugai and Roche Diagnostics, members of the Roche Group and pioneers in personalized healthcare, are sure that Zelboraf and cobas 4800 BRAF V600 Mutation Test can contribute to the treatment of "unresectable melanoma with BRAF mutation," a disease with poor prognosis and with high unmet medical needs. We will continue to dedicate ourselves to providing treatment options to suit individual patients.

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On December 24, 2014, Genentech reported that the U.S. Food and Drug Administration (FDA) approved a supplemental biologics license application (sBLA) for Gazyva in combination with chlorambucil chemotherapy in people with previously untreated chronic lymphocytic leukemia (CLL) (Press release Genentech, DEC 24, 2014, View Source [SID:1234501261]). The sBLA adds to the label data from Stage 2 of the CLL11 study showing significant improvements with Gazyva plus chlorambucil across multiple clinical endpoints when compared head-to-head with Rituxan (rituximab) plus chlorambucil.

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The approval includes complete response (CR) and minimal residual disease (MRD) data from Stage 2 of the study. Additionally, overall survival (OS) data was added from Stage 1 of the study comparing Gazyva plus chlorambucil to chlorambucil alone.

"Gazyva is the first and only medicine to significantly help people live without their disease worsening when combined with chlorambucil compared to Rituxan and chlorambucil in people with previously untreated chronic lymphocytic leukemia," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "These new data enhance our understanding of the disease and its treatment, and this approval affirms an important treatment option for people with this difficult-to-treat disease."

The sBLA approval updated the Gazyva prescribing information with the following data:

Gazyva plus chlorambucil helped people with previously untreated CLL live nearly a year longer without their disease worsening or death (progression-free survival; PFS) than Rituxan plus chlorambucil (median PFS: 26.7 months vs. 14.9 months, respectively. HR=0.42, 95 percent CI 0.33-0.54, p<0.0001).
Gazyva plus chlorambucil nearly tripled the number of people showing no evidence of disease (CR) compared to Rituxan plus chlorambucil (26.1 percent vs. 8.8 percent, respectively).
Of the people who achieved a complete response with or without complete recovery from abnormal blood cell counts (CR, CRi), 19 percent (18/94) of people in the Gazyva arm compared to 6 percent (2/34) of people in the Rituxan arm were MRD negative in the bone marrow, and 41 percent (39/94) of people in the Gazyva arm compared to 12 percent (4/34) people in the Rituxan arm were MRD negative in the peripheral blood. MRD negative means no residual traces of the cancer were found.
Data from the first stage of the CLL11 study showed that at nearly two years, the rate of death was 9 percent (22/238) for people who received Gazyva plus chlorambucil compared to 20 percent (24/118) for those who received chlorambucil alone (HR=0.41, 95 percent CI 0.23-0.74). The median OS has not yet been reached.

Gazyva can cause serious or life-threatening side effects including: Hepatitis B reactivation, progressive multifocal leukoencephalopathy (PML), infusion reactions, tumor lysis syndrome, infections, and low white blood cell counts. The most common side effects of Gazyva are infusion reactions, low white blood cell counts, low platelet counts, low red blood cell counts, fever, cough, nausea, and diarrhea.

Gazyva, the first medicine approved with the FDA’s Breakthrough Therapy Designation, was approved for use in combination with chlorambucil in people with previously untreated CLL on November 1, 2013. Gazyva, known as Gazyvaro in Europe, was approved by the European Commission for the same indication in July 2014. Gazyva is also being investigated in a broad development program across various types of blood cancers, including multiple Phase III studies in non-Hodgkin’s lymphoma (NHL).

About the CLL11 Study

CLL11 is a Phase III, multicenter, open-label, randomized three-arm study, conducted in cooperation with the German CLL Study Group, in 781 previously untreated people with CLL and co-existing medical conditions. Stage 1 (n=589) compared Gazyva plus chlorambucil to chlorambucil alone and Rituxan plus chlorambucil to chlorambucil alone. Stage 2 (n=663) compared Gazyva plus chlorambucil directly with Rituxan plus chlorambucil. The primary endpoint of the study was PFS with secondary endpoints including overall response rate (ORR), OS, CR, median duration of response, MRD and safety profile. Results from Stage 2 and updated data from Stage 1 were presented in 2013 during the Plenary Scientific Session of the American Society of Hematology (ASH) (Free ASH Whitepaper) meeting and published in the New England Journal of Medicine in 2014.