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ONCURIOUS NV Announces European Commission Confirmed Orphan Medicinal Product Designation for TB-403 for Medulloblastoma

On January 17, 2017 ONCURIOUS NV, an emerging oncology company focused on the development of innovative orphan drugs for the treatment of pediatric tumors, reported that the European Commission has confirmed the orphan drug designation for TB-403 for medulloblastoma (Press release, Oncurious, JAN 17, 2017, View Source [SID1234517434]). Today’s decision by the EC follows the earlier positive opinion issued by the European Medicine Agency (EMA).

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TB-403 is a humanized monoclonal antibody against placental growth factor (PlGF). PlGF is expressed in several types of cancer, including medulloblastoma. In medulloblastoma patients, high expression of the PlGF receptor neuropilin 1 has been shown to correlate with poor overall patient survival.

An orphan drug designation is for "medicines to be developed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or very serious".

Today’s confirmation by the EC follows an earlier in-depth review and positive opinion on the drug candidate by the EMA Committee for Orphan Medicinal Products (COMP). The COMP gathers a group of experts chosen on the strength of their qualifications and a number of patient organization representatives. Altogether, the COMP assesses the potential incremental benefits of a drug candidate versus existing treatments.

TB-403 is currently being evaluated in a Phase I/IIa clinical trial for treatment of medulloblastoma, a rare, life-threatening pediatric cancer.

The clinical trial is being conducted in partnership with the Neuroblastoma and Medulloblastoma Translational Research Consortium (NMTRC). The Consortium is a collaboration of 25 US academic medical centers, teaching hospitals and other entities, with the purpose of facilitating and conducting collaborative research activities and investigations of new treatments for neuroblastoma, medulloblastoma and other pediatric cancers. NMTRC is headquartered in Grand Rapids, MI, USA.

ONCURIOUS nv is working with BioInvent International AB as a development partner for this project.

Patrik De Haes, MD, Executive Chairman of ONCURIOUS nv comments
"We are very happy with today’s news. Gaining this orphan-drug designation is an important validation of the TB-403’s scientific rationale

Xcovery Joins National Cancer Institute (NCI) Formulary to Help Expedite Cancer Clinical Trials

On January 17, 2017 Xcovery, a developer of targeted therapeutics for cancer, reported their participation in the NCI Formulary, a public-private partnership between the National Cancer Institute (NCI), part of the National Institutes of Health (NIH), and pharmaceutical and biotechnology companies to expedite the use of agents in clinical trials (Press release, Xcovery, JAN 17, 2017, View Source [SID1234517433]). The partnership, which launched last week with fifteen targeted agents from six pharmaceutical companies, will seek to alleviate the lengthy process to develop new therapies for patients, and respond to the call for greater collaboration within the industry made by Vice President Biden’s Cancer Moonshot Initiative.

"Collaboration among pharmaceutical companies with the NCI and the NIH will speed up the development of novel therapies and benefits for patients down the line," said Lieming Ding, M.D., Chairman of Xcovery. "Xcovery has focused on developing drugs with reduced toxicity, which allows for combination therapies. We’re excited to join the NCI Formulary by providing our ALK inhibitor, ensartinib, to the broad research community for additional development opportunities, particularly in combination therapy programs."

Research indicates that combination therapies, which include drugs with different mechanisms of action impacting cancer cells in multiple ways, provide an improved clinical benefit and can decrease the risk of relapse. The establishment of the NCI Formulary will enable the NCI to facilitate and streamline the arrangements for access to and use of pharmaceutical agents from multiple organizations, allowing for further research on various therapeutic opportunities. Following Xcovery’s approval, investigators will be able to obtain and test ensartinib in new preclinical or clinical studies, including combination studies with formulary agents from different companies.

About Ensartinib

Ensartinib (X-396) is a potent anaplastic lymphoma kinase (ALK) inhibitor currently in a global Phase 3 trial in ALK positive non-small cell lung cancer (NSCLC) patients. Besides ALK, ensartinib also potently inhibits TRKA fusions, TRKC, ROS1, EphA2, and c-MET.

The German National Center for Tumor Diseases (NCT) Heidelberg and Protagen AG Investigate Responsiveness to Checkpoint Inhibitors in Melanoma

On January 17, 2017 The German National Center for Tumor Diseases (NCT) Heidelberg and Protagen AG reported a collaboration to utilize Protagen’s SeroTag technology to identify biomarkers that predict therapy responsiveness and the detection of immune-related adverse events (irAEs) in melanoma patients treated with checkpoint inhibitors (Press release, Protagen, JAN 17, 2017, View Source [SID1234517430]).

Checkpoint inhibitors offer great potential for the treatment of many indications, including melanoma. Yet, only a subset of patients respond favorably to such treatment and it is not currently possible to predict who will benefit from the therapy in clinical routine. In addition, checkpoint inhibitors also trigger immune-related Adverse Events (irAEs) and even the onset of autoimmune diseases. Through this collaboration, Protagen and NCT will utilize Protagen‘s proprietary immune system profiling platform to predict response, monitor patients and detect immune related adverse events.

PD Dr. Jessica Hassel from the DermatoOncologic department of the Department of Dermatology and the NCT commented: "Checkpoint inhibitors offer exciting potential to cure cancer patients. However, at least half of the patients with a metastasized melanoma do not benefit long-term. Response rates can be increased via combination therapies such as a combination of ipilimumab and nivolumab, but such combinations also significantly increase the risk of suffering from sometimes serious irAEs, which have prevalence as high as 60% in these patients. To overcome the challenges posed by irAEs and to better select the appropriate therapy for each patient, we must learn more about the immune system status of these patients in general and their production of specific autoantibodies. Utilizing Protagen’s SeroTag platform enables this insight and we look forward to this collaboration."

Dr. Peter Schulz-Knappe, Protagen’s Chief Scientific Officer, added: "Our unique SeroTag technology has already proven that it can be used to define homogeneous patient subgroups in autoimmune diseases with the potential to predict treatment response. Based on the link between immuno-oncology and autoimmune disease, it is a natural extension to apply our profiling approach to checkpoint inhibitors to address some of the most challenging questions in this field. We feel privileged that Dr. Hassel and the NCT share this view and we are excited about our collaboration."

Agenus announces collaboration with the National Cancer Institute to evaluate the combination of Pembrolizumab (Keytruda®, Merck & Co., Inc.) with Prophage™ (Agenus Inc.) autologous vaccine

On January 17, 2017 Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with a pipeline of immune checkpoint antibodies and cancer vaccines, reported a clinical trial collaboration with the National Cancer Institute (NCI) (Press release, Agenus, JAN 17, 2017, View Source [SID1234517428]). The double-blind, randomized controlled Phase 2 trial will evaluate the effect of Agenus’ personalized autologous vaccine candidate, Prophage (HSPPC-96), in conjunction with Merck’s pembrolizumab on the overall survival rate of patients with newly diagnosed glioblastoma (ndGBM). The trial will be conducted by the Brain Tumor Trials Collaborative (BTTC), a consortium of top academic centers led by Dr. Mark Gilbert, Chief of the Neuro-Oncology Branch at the NCI Center for Cancer Research.

The trial aims to assess the efficacy of PD-1 targeted checkpoint blockade in combination with a heat-shock protein based vaccine candidate in an indication in which this vaccine has shown improved progression-free survival, as presented at ASCO (Free ASCO Whitepaper) 2015. It is a two-arm trial with one arm receiving pembrolizumab as a monotherapy and a second arm receiving both Prophage and pembrolizumab in combination. Forty-five patients will be randomly assigned to each arm.

Under this collaboration, Agenus will supply Prophage, Merck will provide pembrolizumab (Keytruda) and NCI and BTTC member sites will recruit patients and conduct the trial.

About Prophage
Prophage is an individualized autologous vaccine candidate derived from proteins extracted from the patient’s tumor. The vaccine candidate consists of heat shock protein peptide complexes that include the chaperone gp-96 (HSPPC-96) naturally bound to tumor protein fragments. Administration of HSPPC-96 bearing the precise antigenic fingerprint of the patient’s particular cancer represents an effective immune education strategy that enhances recognition of a tumor as ‘non-self’ leading to a potent anti-tumor immune response.

In a Phase 2 study of Prophage monotherapy, patients with ndGBM exhibited an improved progression-free and overall survival as compared to historical control with standard of care.

About KEYTRUDA (pembrolizumab)
KEYTRUDA is a humanized monoclonal antibody that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells. KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma; for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have high PD-L1 expression (TPS ≥50%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations and upon disease progression on or after platinum-containing chemotherapy in patients with NSCLC whose tumors express PD- L1 (TPS ≥1); and for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy. KEYTRUDA is administered as an intravenous infusion every three weeks for the approved indications.

For Safety and Prescribing Information for KEYTRUDA (pembrolizumab), please see View Source

Tesaro Announces Opening of Niraparib Expanded Access Program for U.S. Patients With Ovarian Cancer

On January 17, 2017 TESARO, Inc. (NASDAQ:TSRO), an oncology-focused biopharmaceutical company, reported the opening of an expanded access program (EAP) in the United States for the investigational PARP inhibitor, niraparib (Press release, TESARO, JAN 17, 2017, View Source [SID1234517426]). Through this program, niraparib is being made available for eligible women with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer following a complete or partial response to platinum-based chemotherapy. Healthcare professionals can learn more about the niraparib EAP by visiting www.niraparibEAP.com. An EAP for niraparib in Europe is planned to open in the first half of 2017, and will be initiated on a country-by-country basis.

Expanded access programs enable patients with serious or life-threatening illnesses who do not otherwise qualify for participation in a clinical trial, and for whom there are no comparable or satisfactory alternate therapies, to access investigational medicines.

"Ovarian cancer is the fifth most frequent cause of cancer death among women in the United States, yet there have been few advances in the treatment of ovarian cancer in over a decade," said Mary Lynne Hedley, Ph.D., President and COO of TESARO. "The niraparib EAP will provide a mechanism by which eligible women with ovarian cancer may benefit from access to this investigational therapy, which has been accepted for priority review by the U.S. FDA."

About the Niraparib Expanded Access Program
The niraparib EAP is a program for women with recurrent, ovarian, fallopian tube, or primary peritoneal cancer following a complete or partial response to platinum. This EAP is being administered on behalf of TESARO by the Idis Managed Access division of Clinigen Group plc. U.S. based healthcare professionals seeking more information about the niraparib EAP can call Idis Managed Access at 1-877-768-4303 or email [email protected] for further details. Patients who are interested in enrolling in the niraparib EAP should speak with their physician to understand if niraparib is an appropriate option. Niraparib is an investigational agent and, as such, has not been approved by the U.S. Food and Drug Administration (FDA) or any other regulatory agencies in any markets. Additional information about the niraparib EAP, including a list of Frequently Asked Questions, is available at www.niraparibEAP.com.

An EAP for niraparib in Europe is planned to open in the first half of 2017, and will be initiated on a country-by-country basis.

About Niraparib
Niraparib is an oral, once-daily PARP inhibitor that is currently being evaluated in three pivotal trials. TESARO is building a robust niraparib franchise by assessing activity across multiple tumor types and by evaluating several potential combinations of niraparib with other therapeutics. The ongoing development program for niraparib includes a Phase 3 trial in patients who have received first-line treatment for ovarian cancer (the PRIMA trial), a registrational Phase 2 trial in patients who have received multiple lines of treatment for ovarian cancer (the QUADRA trial), and a Phase 3 trial for the treatment of patients with germline BRCA-mutated, metastatic breast cancer (the BRAVO trial). Several combination studies are also underway, including trials of niraparib plus pembrolizumab and niraparib plus bevacizumab. Janssen Biotech has licensed rights to develop and commercialize niraparib specifically for patients with prostate cancer worldwide, except in Japan.

The niraparib New Drug Application (NDA) has been accepted for priority review by the FDA and is supported by data from the ENGOT-OV16/NOVA trial, a double-blind, placebo-controlled, international Phase 3 study that enrolled 553 patients with recurrent ovarian cancer following complete or partial response to their most recent platinum-based chemotherapy. The full results of the ENGOT-OV16/NOVA trial were presented in detail at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress in Copenhagen on October 8, 2016 by Dr. Mansoor Raza Mirza, M.D., Medical Director of the Nordic Society of Gynecologic Oncology (NSGO) and principal investigator. These data were simultaneously published in the New England Journal of Medicine.

Niraparib is an investigational agent and, as such, has not been approved by the U.S. FDA, European Medicines Agency (EMA), or any other regulatory agencies.

About Ovarian Cancer
Approximately 22,000 women are diagnosed each year with ovarian cancer in the United States, and more than 65,000 women are diagnosed annually in Europe. Ovarian cancer is the fifth most frequent cause of cancer death among women. Despite high response rates to platinum-based chemotherapy in the second-line advanced treatment setting, approximately 85% of patients will experience recurrence within two years. If approved, niraparib may address the difficult "watchful waiting" periods experienced by patients with recurrent ovarian cancer in between cycles of platinum-based chemotherapy.