On December 1, 2014 Celator Pharmaceuticals reported that the independent Data and Safety Monitoring Board (DSMB) for the Company’s Phase 3 clinical study of CPX-351 (cytarabine:daunorubicin) Liposome for Injection versus the conventional cytarabine and daunorubicin treatment regimen (commonly referred to as 7+3) as first-line therapy in older patients with high-risk (secondary) acute myeloid leukemia (AML), has completed a planned safety review and recommended that the study continue as planned without any modifications (Press release Celator Pharmaceuticals, DEC 1, 2014, View Source [SID:1234501036]).

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"We are pleased with the DSMB recommendation to continue with the pivotal Phase 3 study of CPX-351 following this safety analysis of the first 225 randomized patients, out of a total of 309 patients who were enrolled in the study," said Arthur Louie, Chief Medical Officer of Celator Pharmaceuticals. "With the Phase 3 study having recently completed enrollment ahead of schedule, we look forward to the induction response rate analysis, which we expect in the second quarter of 2015."

The DSMB assessment was based on a pre-planned safety analysis on the first 225 randomized patients included in the study with a minimum of 60 days of follow-up. The DSMB will conduct an additional review after all patients become evaluable for safety review.

The Phase 3 study is being conducted in partnership with The Leukemia & Lymphoma Society (LLS) through its Therapy Acceleration Program (TAP), which has supported the development of CPX-351 beginning in Phase 2.

"We are excited with the progress of the Phase 3 study," said Scott Jackson, Chief Executive Officer of Celator Pharmaceuticals. "In addition to the induction response rate data, a secondary endpoint, from the study in the second quarter of next year, we expect the overall survival data, the primary endpoint, in the first quarter of 2016. We also continue to work with investigators to study CPX-351 in other AML patient populations, as well as in other hematologic malignancies, with the hope of providing improved clinical benefits to these patients."

The study (Protocol NCT01696084) enrolled patients between the ages of 60 and 75 who have pathological diagnosis of AML according to WHO criteria with confirmation of:

Therapy-related AML,
AML with a history of myelodysplasia (MDS),
AML with a history of chronic myelomonocytic leukemia (CMMoL), or
De novo AML with karyotypic abnormalities characteristic of MDS.

Patients were randomized 1:1 to receive either CPX-351 (100u/m2; days 1, 3, and 5 by 90 minute infusion) or 7+3 (cytarabine 100mg/m2/day by continuous infusion for 7 days and daunorubicin 60mg/m2 on days 1, 2, and 3). Patients are monitored for all clinical adverse events as well as laboratory evaluations. The primary efficacy endpoint of the study is overall survival. The study is being conducted in the United States and Canada, with more than 40 leading cancer centers participating.

On December 1, 2014 Pharmacyclics reported the acceptance of a Type II variation application for IMBRUVICA (ibrutinib) by the European Medicines Agency (EMA) (Press release Pharmacyclics, DEC 1, 2014, View Source [SID:1234501035]). This submission, filed by strategic partner Janssen-Cilag International NV (Janssen), represents a potential label expansion for IMBRUVICA in the European Union (EU) for the treatment of adult patients with Waldenstrom’s macroglobulinemia (WM), a rare type of B-cell lymphoma for which treatment options are limited in the EU. If approved, IMBRUVICA would be the first label specifically authorized to treat WM.

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IMBRUVICA is a first-in-class, oral, once-daily, therapy being jointly developed and commercialized in the United States (U.S.) by Pharmacyclics and Janssen Biotech, Inc. Janssen affiliates will hold the marketing authorization and market IMBRUVICA in EMEA (Europe, Middle East, Africa), as well as the rest of the world, outside the U.S.

"This additional application in the EU shows the priority and urgency with which we, and our collaboration partner Janssen, pursue the ongoing potential for IMBRUVICA," said Thorsten Graef, M.D., Ph.D., Vice President of Clinical Science, Pharmacyclics. "If approved, IMBRUVICA could address a very important unmet need for patients with WM in Europe, who currently have limited treatment options."

The acceptance of the WM Type II variation submission for IMBRUVICA triggers a $20 million milestone payment to Pharmacyclics under its collaboration agreement with Janssen Biotech, Inc.

The EMA WM filing follows the supplemental New Drug Application submission for IMBRUVICA to the U.S. Food and Drug Administration (FDA), which was submitted by Pharmacyclics in October 2014, for its use in the treatment of patients with WM. Both the FDA and EMA filings were based on data from a Phase II study evaluating the use of IMBRUVICA in WM patients, which was led by Dr. Steven Treon from the Dana-Farber Cancer Institute.

WM is a slow-growing, currently incurable, rare type of B-cell lymphoma for which there are no EU-wide approved drugs. The median age at diagnosis is 63-68 years of age and incidence rates among men and women in the EU are approximately 7.3 and 4.2 per million persons, respectively. WM begins with a malignant change to the B cell, a type of white blood cell (lymphocyte), during its maturation so that it continues to reproduce more malignant B cells.

On Decemebr 1, 2014 ImmunoCellular Therapeutics reported that the European Medicines Agency (EMA) has provided scientific advice supportive of advancing ICT-107 to a registrational phase III program in patients with newly diagnosed glioblastoma (GBM) (Press release ImmunoCellular Therapeutics, DEC 1, 2014, View Source [SID:1234501034]). The EMA guidance is consistent with the positive feedback the Company received from the US FDA relative to the scope, design and endpoints of the program and the inclusion of patients based on HLA and MGMT status. ImmunoCellular intends to finalize the design of the phase III program, ensuring harmony between US and EU trial protocols, with the goal of being in position to initiate the phase III program. The Company now is evaluating options for funding the phase III program, which may enable the initiation in 2015.

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"We appreciate the EMA’s support for conducting a phase III program with ICT-107, and for their detailed guidance on the treatment design and statistical elements of the program," said Andrew Gengos, ImmunoCellular Chief Executive Officer. "The encouragement we have received from both the US and EU regulatory authorities, and from the global neuro-oncology community, increase our confidence in ICT-107’s therapeutic potential for patients with this lethal disease. We intend to complete the design of a high quality phase III program, and to explore appropriate funding alternatives to enable next steps."

Earlier this year, ICT-107 was designated as an Advanced Therapy Medicinal Product by the EU Committee for Advanced Therapies, which should provide access in Phase III to valuable services and incentives offered by the EMA, should the Company conduct the phase III program in the EU.

On December 1, 2014 Eisai reported that it has submitted its application to the regulatory authority in South Korea (Ministry of Food and Drug Safety) for marketing approval of its novel in-house developed anticancer agent lenvatinib mesylate (lenvatinib) as a treatment for progressive radioiodine-refractory differentiated thyroid cancer (Press release Eisai, NOV 30, 2014, View Source [SID:1234501033]). Following the submission of marketing authorization applications in Japan, the United States and Europe, this marks the first time Eisai has submitted a marketing authorization application for lenvatinib in Asia.

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Lenvatinib is an oral molecular targeted agent that selectively inhibits the activities of several different molecules including VEGFR, FGFR, RET, KIT and PDGFR, involved in angiogenesis and tumor proliferation. This potentially makes lenvatinib a first-in-class treatment in thyroid cancer, especially given that it simultaneously inhibits the activity of the three molecules VEGFR, FGFR and also RET via its novel binding mode.

The application submitted for South Korea was based on the positive results from a global Phase III clinical study known as the SELECT (Study of (E7080) LEnvatinib in differentiated Cancer of the Thyroid) trial. (Please refer to the following notes for further details of the trial)

An application seeking the approval of lenvatinib for the indication of thyroid cancer was submitted in Japan in June 2014 as the first in the world, followed by the submission of applications in both the United States and Europe in August 2014. Lenvatinib was granted Orphan Drug Designation for thyroid cancer in by the regulatory authorities in Japan, Europe and the United States. In addition, lenvatinib was also granted an accelerated assessment in Europe by the European Medicines Agency, and granted priority review status in the United States by the U.S. Food and Drug Administration.

The number of patients newly diagnosed with thyroid cancer in 2012 in South Korea was estimated to be 33,000, and in Asia was estimated to be 144,000. Although treatment is possible for most types of thyroid cancer, there are few treatment options available once thyroid cancer has progressed, therefore it remains a disease with significant unmet medical needs.

Eisai is committed to exploring the potential clinical benefits of lenvatinib, and is working to obtain marketing approval in each country in Asia as soon as possible in order to further contribute to patients with thyroid cancer, and their families.