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Ignyta Receives Orphan Drug Designation in the European Union for Entrectinib for the Treatment of Neuroblastoma

On December 8, 2015 Ignyta, Inc. (Nasdaq: RXDX), a precision oncology biotechnology company, reported that the European Medicines Agency (EMA) has granted orphan drug designation for Ignyta’s product candidate entrectinib for the treatment of neuroblastoma (Press release, Ignyta, DEC 8, 2015, View Source [SID:1234508488]).

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"We are pleased that the EMA has provided this designation for entrectinib, adding to our orphan designations in the U.S. for the treatment of neuroblastoma and the treatment of TrkA-, TrkB-, TrkC-, ROS1- or ALK-positive non-small cell lung cancer and colorectal 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."

About Entrectinib

Entrectinib is a novel, orally available, selective tyrosine kinase inhibitor targeting tumors that harbor activating alterations to NTRK1/2/3 (encoding TrkA/ TrkB/TrkC), ROS1 or ALK. Entrectinib is the most potent Trk inhibitor in the clinic, without undesirable off-target activity, and the only Trk inhibitor with clinically demonstrated activity against CNS metastases. This product candidate is in a Phase 2 clinical trial called STARTRK-2, which is the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases." The trial is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types and molecular targets.

Eagle Pharmaceuticals Achieves Milestone to Receive $15 Million Payment for Approval of BENDEKA

On December 8, 2015 Eagle Pharmaceuticals, Inc. ("Eagle" or "the Company") (Nasdaq:EGRX) reported that it has achieved the milestone which entitles the Company to receive a $15 million payment from Teva Pharmaceuticals Industries Ltd. resulting from the U.S. Food and Drug Administration ("FDA") approval of BENDEKA, (bendamustine hydrochloride) injection, a liquid, low-volume (50 mL) and short-time 10-minute infusion formulation of bendamustine for the treatment of patients with chronic lymphocytic leukemia ("CLL") and for the treatment of patients with indolent B-cell non-Hodgkin lymphoma ("NHL") that has progressed during or within six months of treatment with rituximab or a rituximab-containing regimen (Press release, Eagle Pharmaceuticals, DEC 8, 2015, View Source [SID:1234508487]).

The Company will receive a 20% royalty on net sales of BENDEKA. In addition, Eagle may earn an incremental step-up royalty upon the achievement of future milestones.

"We look forward to Teva’s successful commercialization of this new treatment option. We believe the $15 million milestone payment triggered by approval, coupled with future royalties of 20% on Teva’s net sales of BENDEKA, will expedite Eagle’s ability to deliver long-term, sustainable growth," stated Scott Tarriff, President and Chief Executive Officer of Eagle Pharmaceuticals.

BENDEKA was granted Orphan Drug Designations for both CLL and indolent B-cell NHL.

Under the February 2015 exclusive license agreement for BENDEKA, Teva is responsible for all U.S. commercial activities for the product including promotion and distribution.

Promising data from Phase I/II IIT and Compassionate Use Programme with dendritic cell vaccines in AML presented at ASH Annual Meeting 2015

On December 8, 2015 Medigene AG (MDG1, Frankfurt, Prime Standard) reported that early data from two independent clinical programmes in patient groups with acute myeloid leukaemia (AML) receiving dendritic cell (DC) vaccines, prepared according to technologies licensed and developed by Medigene, show an excellent safety profile and the capacity to induce T cell responses in elderly patients unable to undergo stem cell transplantation (Press release, MediGene, DEC 8, 2015, View Source [SID:1234508484]).

Two posters were presented at the 57th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in Orlando, FL, USA, detailing early clinical results of patients with acute myeloid leukaemia (AML) treated with these next-generation DC vaccines. One poster, entitled "Next-Generation Dendritic Cell Vaccination in Postremission Therapy of AML: Results of a Clinical Phase I Trial", included data from an ongoing Phase I/II investigator initiated trial (IIT) under the direction of Prof. Marion Subklewe of the Ludwig-Maximilians-Universität (LMU) in Munich, Germany. In particular, results were presented regarding the six patients included in a Phase I proof-of-concept study who have completed vaccination lasting up to 26 weeks. The second poster entitled "AML Patients in Minimal Residual Disease Vaccinated with a Novel Generation of Fast Dendritic Cells Expressing WT-1 and PRAME Mount Specific Immune Responses That Relate to Clinical Outcome" included results from four patients treated with DCs from 5 to 16 months so far in an ongoing Compassionate Use Programme[1] under the direction of Prof. Gunnar Kvalheim at Oslo University Hospital (OUH) in Norway.

Links to the poster abstracts:
Phase I/II IIT of LMU: View Source
Compassionate Use Programme of OUH: View Source

Prof. Marion Subklewe, Professor of Internal Medicine with special Focus on Cellular Immunotherapy at the Ludwig-Maximilian University Großhadern, Munich, explains: "Upon completion of our Phase I trial, we have obtained the first evidence that use of WT-1 and PRAME as vaccine antigens can be validated through detection of T cell responses in various patients analysed to date".

Prof. Gunnar Kvalheim, Head of Department of Cellular Therapy at the Oslo University Hospital comments on his findings so far: "We are optimistic that these results pave the way for DC vaccines as a new therapy option for patients that have high risk for disease relapse and do not qualify for stem cell transplantation. The feasibility to make good quality DC vaccines from heavily pretreated patients and the capacity of our patients to make T cell responses to one or both antigens are important early findings."

Prof. Dolores J. Schendel, Chief Scientific Officer of Medigene AG, summarizes the findings from the two ASH (Free ASH Whitepaper) reports: "We are pleased with the new information that could be derived from the preliminary assessments of the ten AML patients receiving next-generation DC vaccines in these ongoing independent studies. It was feasible to manufacture high quality DCs according to our technology that led to detectable immune responses in different patients to one or both leukaemia-associated antigens. The rapidity with which T cell responses were detected in some patients speaks to the good immunizing capacity of the DCs. These observations support the approach implemented in our own company-sponsored DC vaccine trial that was launched in March of this year at OUH."

Nemucore Medical Innovations Options Clinical-Stage Aurora Kinase Inhibitor GSK1070916 From Cancer Research Technology (CRT)

On December 7, 2015 Nemucore Medical Innovations, Inc., a privately held, clinical-stage biopharmaceutical company dedicated to the development of therapies targeting multi-drug resistant cancers with a special emphasis on highly lethal women’s cancers, reported the completion of an option agreement with Cancer Research Technology Ltd (CRT), the commercial arm of Cancer Research UK, for the exclusive license of worldwide commercial rights to GSK1070916 (now designated NMI-900 by Nemucore), a potent Aurora B/C kinase inhibitor targeting a broad range of cancers (Press release, Nemucore Medical Innovations, DEC 7, 2015, View Source [SID1234563930]).

"We are thrilled to be able to build on the excellent foundational clinical research conducted by Cancer Research UK, and continue the development of this innovative and very promising anticancer therapeutic," said Timothy P. Coleman, Ph.D., Chairman, Chief Executive Officer and President of Nemucore. "Based on its unique properties and pharmaceutical profile, we believe NMI-900 has best-in-class potential as a breakout therapy for treating women’s and other cancers associated with high mortality rates that have already been demonstrated to be intractable to conventional therapeutics."

NMI-900 is a potent ATP-competitive inhibitor of Aurora B kinase that has demonstrated high affinity for Aurora B, a significantly slower dissociation rate compared to its peers, potent anti-proliferative activity in multiple cancer cell lines, and minimal effects on non-proliferating normal human cells. In 2014, Cancer Research UK’s Centre for Drug Development successfully completed a Phase 1/2a trial of NMI-900. In this trial, NMI-900 elicited response in 61% of patients with no remaining standard therapies available to them across a wide variety of advanced and/or metastatic solid tumors. NMI-900 was well tolerated, with the most prevalent adverse event presenting as predictable and treatable neutropenia. NMI-900 was developed by Cancer Research UK’s Centre for Drug Development in partnership with GSK, under the Clinical Development Partnerships (CDP) initiative. This initiative, a joint effort launched by Cancer Research UK and Cancer Research Technology Ltd, provides a simple route for companies to progress oncology agents that would not otherwise be developed, and increase the number of clinical trials being undertaken for the treatment of cancer.

Dr. Keith Blundy, CEO of Cancer Research Technology commented, "We’re very pleased that Nemucore plans to take this promising new drug candidate and develop it through more clinical trials so that it has a greater chance of reaching patients who are in urgent need of new treatment options, sooner. The drug forms part of our Clinical Development Partnerships initiative, and is one of twelve drugs on the scheme that are moving out of the lab into clinical trials – something that wouldn’t have been possible otherwise."

Nemucore expects to initiate a Phase 2b clinical trial of NMI-900 in patients with advanced, platinum-resistant ovarian cancer in mid-2016 based on the supportive preclinical and early clinical trial results. As part of their clinical development and commercial strategy, the Company is concurrently developing a companion diagnostic with the Medical Prognosis Institute to identify patients most likely to respond to NMI-900. Nemucore expects to investigate the efficacy of NMI-900 in the treatment of EGF receptor-positive non-small cell lung cancer (NSCLC), myelodysplastic syndrome (MDS) and other difficult-to-treat cancers in the future.

Walloon Government supports the development of iTeos proprietary pipeline to develop novel therapeutics targeting the immune tumor micro-environment

On December 7, 2015 iTeos Therapeutics SA, the drug discovery company for immunomodulators,reported that it has received a €2.94 million non-dilutive funding from the Walloon Region of Belgium to expand its preclinical drug discovery pipeline targeting new immunotherapies for the tumor micro-environment (Press release, iTeos Therapeutics, DEC 7, 2015, View Source [SID:1234513306]).

"This grant will allow us to expand our proprietary pipeline by adding a program to develop small molecule inhibitors for a target involved in T cell anergy". said Christophe Quéva Ph.D., chief scientific officer of iTeos. "It will complement our growing portfolio of small molecules and antibody approaches aimed at stimulating certain immune responses against cancers."

"We are very pleased to continue to receive strong support for our preclinical research from the Walloon Region. In parallel to our strategic collaboration with Pfizer, which was announced in 2014, we are expanding the Company’s drug candidate pipeline where such grant support is pivotal for our research efforts to discover our own diversified and innovative set of drug candidate programs." said Michel Detheux Ph.D., chief executive officer of iTeos.