Ignyta Receives Orphan Drug Designation from FDA for Entrectinib for the Treatment of Molecularly Defined Subsets of Colorectal Cancer

On February 17, 2015 Ignyta, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for Ignyta’s lead product candidate entrectinib for the treatment of TrkA-positive, TrkB-positive, TrkC-positive, ROS1-positive or ALK-positive colorectal cancer (Press release Ignyta, FEB 17, 2015, View Source [SID:1234501634]).

“We are pleased to receive from the FDA this orphan drug designation for colorectal cancer, our third orphan designation in addition to neuroblastoma and non-small cell lung cancer,” said Jonathan Lim, M.D., Chairman and CEO of Ignyta. “Entrectinib has the potential to address unmet needs of patients with rare cancers, and we will continue to aggressively pursue our clinical development program for entrectinib in solid tumors for the benefit of these patients and to create value for our stockholders.”

Takeda Announces Phase 3 MONET-A Study Evaluating Motesanib (AMG 706) in Patients with Advanced Non-Squamous Non-Small Cell Lung Cancer Does Not Meet Primary Endpoint

On February 17, 2015 Takeda Pharmaceutical reported that the primary endpoint of progression-free survival (PFS) was not met in the Phase 3 MONET-A study of patients with stage IV non-squamous non-small cell lung cancer (NSCLC) who were randomized to treatment with investigational motesanib in combination with paclitaxel and carboplatin compared to placebo in combination with paclitaxel and carboplatin (Press release Takeda, FEB 16, 2015, View Source [SID:1234501638]). As a result,Takeda has elected to terminate the MONET-A trial, and will report on the full trial results once available. Motesanib is an investigational, orally administered small molecule antagonist of vascular endothelial growth factor receptors 1, 2 and 3, platelet driven growth factor receptors and stem cell factor receptor.

Takeda is working with trial investigators to ensure patients who participated in the study
will receive appropriate therapies.

About the MONET-A Study
MONET-A is a multinational Phase 3, randomized, placebo-controlled, double-blind study examining the efficacy and safety of motesanib when administered with paclitaxel and carboplatin. Patients (n=401) with stage IV or recurrent non-squamous NSCLC were randomized in a 1:1 ratio to either motesanib in combination with paclitaxel and carboplatin or placebo in combination with paclitaxel and carboplatin.
Patients included in the study had a confirmed diagnosis of stage IV non-squamous NSCLC, no prior chemotherapy, molecularly-targeted therapy or immunotherapy (for metastatic disease) and other outlined eligibility criteria.

The primary efficacy endpoint was PFS. Secondary endpoints included overall survival (OS), objective response rate (ORR), duration of response, safety and pharmacokinetics of motesanib and its metabolites when the compound was administered in combination with paclitaxel and carboplatin.
The MONET-A study was initiated among patients in Japan, South Korea, Taiwan and Hong Kong, following the completion of a previous Phase 3 trial, MONET1, which compared motesanib combined with paclitaxel and carboplatin to chemotherapy alone in patients with non-squamous NSCLC. Although the MONET1 trial did not meet its primary objective of demonstrating a statistically significant improvement in OS, a pre-planned analysis revealed significant and consistent findings in the Asian population for ORR, PFS and OS, providing rationale for further investigation.

U.S. FDA APPROVES ANTICANCER AGENT LENVIMA TM (LENVATINIB MESYLATE) AS TREATMENT FOR RADIOACTIVE IODINE-REFRACTORY D CANCER

On February 16, 2015 Eisai reported that its U.S. subsidiary Eisai Inc. has received approval of its in-house developed novel anticancer agent Lenvima (lenvatinib mesylate) as a treatment for locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (RAI-R DTC) from the U.S. Food and Drug Administration (FDA) (Press release Eisai, FEB 15, 2015, View Source [SID:1234501629]). Lenvima was granted priority review status by the FDA, and was ultimately approved six months from the submission of the New Drug Application in August 2014, two months ahead of the FDA priority review action date. This marks the first country in the world where the agent has received marketing authorization.

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Lenvima is an orally administered molecular targeted agent that selectively inhibits the activities of several different molecules including VEGFR, FGFR, RET, KIT and PDGFR. In particular, the agent simultaneously inhibits VEGFR, FGFR and also RET which are especially involved in tumor angiogenesis and proliferation of thyroid cancer. Furthermore, Lenvima has been confirmed through X-ray crystal structural analysis to be the first compound to demonstrate a new binding mode (Type V) to VEGFR2, and exhibits rapid and potent inhibition of kinase activity, according to kinetic analysis.

The approval was based on the results of a multicenter, randomized, double-blind, placebo-controlled Phase III study (the SELECT study) of 392 patients with progressive RAI-R DTC. In the study’s primary endpoint of progression-free survival (PFS), Lenvima demonstrated a statistically significant extension in PFS compared to placebo (p<0.001; median PFS in the Lenvima group: 18.3 months, median PFS in the placebo group: 3.6 months; Hazard Ratio (HR) 0.21 [99% CI: 0.14-0.31]). In addition, Lenvima demonstrated a statistically significant improvement in response rate (sum of complete and partial responses) compared to placebo (p<0.001; Lenvima: 64.8% vs placebo: 1.5%). In particular, complete response was observed in 1.5% (4 patients) of the Lenvima group and zero in the placebo group. The most common Lenvima treatment-related adverse events of any grade, which occurred in more than 40% of patients in the Lenvima group, were hypertension (67.8%), diarrhea (59.4%), fatigue or asthenia (59.0%), decreased appetite (50.2%), weight loss (46.4%) and nausea (41.0%).

Currently, the agent is undergoing regulatory review in Japan and the EU, as well as Switzerland, South Korea, Canada, Singapore, Russia, Australia and Brazil, and Lenvima was also granted accelerated assessment in the EU. Eisai will continue to file applications seeking regulatory approval for the agent in countries around the world, and the company will market the agent in those countries where approval has been received. Furthermore, Eisai is conducting a global Phase III trial of Lenvima in hepatocellular carcinoma as well as Phase II studies of Lenvima in several other tumor types such as renal cell carcinoma and non-small cell lung cancer.

The number of patients newly diagnosed with thyroid cancer in 2012 in the United States was estimated to be approximately 52,000. 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. In addition to providing Lenvima as a new treatment option for thyroid cancer, Eisai is committed to exploring the potential clinical benefits of Lenvima in order to further contribute to patients with cancer, and their families.

10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

Vertex Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing 10-K , Vertex Pharmaceuticals, FEB 13, 2015, View Source [SID1234501631]).

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10-K – Annual report [Section 13 and 15(d), not S-K Item 405]

HedgePath Pharmaceuticals has filed a 10-K – Annual report [Section 13 and 15(d), not S-K Item 405] with the U.S. Securities and Exchange Commission (Filing 10-K , HedgePath Pharmaceuticals, FEB 13, 2015, View Source [SID1234501630]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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