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Latest ONC201 Clinical Efficacy to Be Presented in Oral Abstract Session at ASCO

By: Cancer-News | June 06, 2019 | 17 view(s)

Philadelphia, PA (May 17, 2019) – Oncoceutics, Inc. announced that the latest efficacy data for the use of ONC201 in adult recurrent H3 K27M-mutant glioma will be presented in an Oral Abstract Session at the 2019 Annual Meeting of the American Society of Clinical Oncology (ASCO) in Chicago. In addition, there will be an update on ONC201 in previously irradiated pediatric H3 K27M-mutant glioma that will be presented in a poster session. The oral abstract session presentation, entitled “Single agent ONC201 in adult recurrent H3 K27M-mutant glioma” will describe the clinical experience of ONC201, in adults with recurrent H3 K27M-mutant glioma. It will be presented on Monday, June 3rdfrom 9:24 am to 9:36 am (Abstract 3005) as part of the Developmental Therapeutics and Tumor Biology (nonimmune) session by Isabel Arrillaga, MD, PhD, Associate Clinical Director of Neuro-Oncology at MGH Cancer Center. The presentation will discuss a cohort of adults with H3 K27M-mutant glioma patients who have received ONC201 after failure of available therapies. The results will include assessments of radiographic response, progression-free survival and overall survival, as well as safety and clinical benefit. This presentation was selected for review by a panel of discussants. A second data set to be presented is entitled “ONC201 in previously-irradiated pediatric H3 K27M-mutant glioma,” and will be presented as a poster by Sharon Gardner, MD, Associate Professor in the Department of Pediatrics at NYU Langone, on Saturday, June 1, from 8:00 am to 11:00 am (Abstract 10046, Poster 428). Dr. Gardner will discuss the emerging safety, pharmacokinetics, and outcome data available from the first arm of the first pediatric clinical trial with ONC201. This trial is currently evaluating the compound in children with DIPG, or other forms of H3 K27M-mutant glioma, who have no meaningful treatment options. “As someone who was involved from the very beginning in Oncoceutics’ development programs, I am delighted to see the progress in the development of ONC201 for brain tumors that have challenged our abilities in oncology for a long time”, said Joseph Bertino MD, FAACR, Founding Member of the Scientific Advisory Board of Oncoceutics, Past President of ASCO and AACR, and, amongst the numerous accolades given to him, recipient of the 2018 Award for Lifetime Achievement in Cancer Research. “I am encouraged that the therapeutic concept of addressing GPCRs with imipridones for applications in oncology will materialize as a new paradigm to treat cancer. ONC206 is the next compound from the imipridone portfolio that will enter the clinic, and based on the preclinical profile, including the well characterized binding target and mechanism of action, I expect an efficient translational program.” A team of Oncoceutics team members will be present at the conference. If you have any questions, please contact us atinfo@oncoceutics.com. The abstracts are listed below: 3005. Single agent ONC201 in adult recurrent H3 K27M-mutant glioma. Presented Monday, June 3, 2019.https://meetinglibrary.asco.org/record/172409/abstract 10046. ONC201 in previously-irradiated pediatric H3 K27M-mutant glioma. Presented Saturday, June 1, 2019.https://meetinglibrary.asco.org/record/170980/abstractAbout OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, Musella Foundation, and a series of private and public partnerships. Visit Oncoceutics for more information.

Oncoceutics Invited to Present ONC201 at Public Advisory Committee Meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee on June 20, 2019

By: Cancer-News | May 29, 2019 | 34 view(s)

Philadelphia, PA (May 7, 2019) – Oncoceutics announced that the US Food and Drug Administration invited the company to present information regarding its lead compound ONC201 at the public advisory committee meeting of the Pediatric Oncology Subcommittee of the Oncologic Drugs Advisory Committee on June 20, 2019.   The meeting will provide Oncoceutics with the opportunity to present its clinical data for patients treated with ONC201, including studies for both adult and pediatric patients with gliomas containing the H3 K27M mutation, a disease that has been recognized to be an area of high unmet medical need. Additionally, the meeting will enable Oncoceutics to receive feedback from the subcommittee on issues concerning diseases to be studied, patient populations to be included, and possible study designs in the development of ONC201 for pediatric use.“We are honored and grateful for the opportunity to present to the Pediatric Oncology Subcommittee of the ODAC,” said Wolfgang Oster, MD, PhD, Chief Executive Officer and Chairman of Oncoceutics. “ONC201 has shown biological activity that we hope will translate into the clinic and eventually make a difference in patients’ lives. We look forward to furthering the clinical development of ONC201 with the support and guidance of the FDA.”The Public Advisory Committee Meeting will take place in the Great Room of the FDA’s White Oak campus from 8am to 4:30pm. Oncoceutics will present at 12:30pm. Background materials will be available at least 2 business days before the meeting at https://www.fda.gov/AdvisoryCommittees/Calendar/default.htm, and interested persons from the public may present data, information, or views, orally or in writing, on issues pending before the subcommittee.About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, Musella Foundation, XCures, Cancer Commons, and a series of private and public partnerships. Visit Oncoceutics for more information.

Eon Expert Series + Mark Cuban Press Release

By: EonHealth | May 21, 2019 | 20 view(s)

The Eon Expert Series: Defying Lung Cancer with Eon + Mark Cuban Why does health tech hurt us more than help? We are asking the tough questions. Dallas, TX 5/16/2019   Join us May 20, 2019 at 9 AM CT for an online conversation with Mark Cuban, Dr. Gerard Silvestri, Dr. Peter Mazzone, and Dr. George A. Eapen. The panel will discuss real healthcare technology issues that have led to patients, health care providers and hospitals facing an ever-widening gap between what technology can do and what is currently implemented. Technology has improved processes and become less expensive in every other industry - why not healthcare? It's time to start asking the hard questions! Eon is excited to bring top minds together to answer the tough questions and discuss innovative healthcare solutions and the patient impact they could have. Registration for the webinar is free. We recommend registering as soon as possible, as spots are limited. If you are attending ATS 2019 in Dallas, we are sending one winner backstage to meet Mark and the three panel doctors. Mark Cuban is an entrepreneur, businessman, investor, and writer. Mark has founded multiple technology-based companies such as MicroSolutions and AudioNet. In addition to the owning the Dallas Mavericks, Mark is chairman and CEO of AXS tv, one of ABC's "Sharks" on the hit tv show Shark Tank, and an investor in an ever-growing portfolio of businesses. Gerard Silvestri MD, MS, FCCP is a professor of medicine and a pulmonologist at the Medical University of South Carolina. Dr. Silvestri specializes in the evaluation, management and improvement of outcomes in lung cancer patients. He has experience in evaluating new technologies for the diagnosis and staging of lung cancer. His research includes screening for lung cancer, how patients should be diagnosed and staged with the disease and how to evaluate new technologies needed to diagnose and treat these patients. Peter Mazzone MD, FCCP is a pulmonologist at the Cleveland Clinic where he directs the Lung Cancer Program for the Respiratory Institute and the Lung Cancer Screening Program for the hospital system. He has served within Chest and the American Thoracic Society to lead policy development projects related to quality metrics for the evaluation of lung cancer, components necessary for high-quality lung cancer screening, and the clinical application of molecular biomarkers for lung cancer detection. George A. Eapen MD is currently associated with the Department of Pulmonary Medicine, Division of Internal Medicine at MD Anderson. He received his medical degree from University of Benin Faculty of Medicine and has been in practice for more than 20 years. His areas of interest include lung cancer, bronchoscopy, radiation, and pulmonary medicine. The webinar will be hosted by Eon, a complex management software company out of Denver, Colorado. Determined to create sea change in complex patient management, Dr. Aki Alzubaidi and Christine Spraker, co-CEO’s of Eon, have developed EonDirect which is now the market leader in improving patient care and lowering costs for users across the country. Today, Eon’s mission is to use advanced technology to improve complex patient management of every kind. By collaborating with caregivers, hospitals, and hospital systems, we can improve patient care across the board, saving time, money, and most importantly lives. Together we can defy disease.   Watch the full webinar https://www.oncologytube.com/video/eon-expert-series-mark-cuban/10006248 https://www.youtube.com/watch?v=XzdxvcuuaVY   Eon https://www.eonhealth.com 400 South Colorado Blvd Suite 380 Denver, CO 80246  

Oncoceutics Continues Shift to Fully-Integrated Pharmaceutical Company with Hiring of VP, Clinical Operations

By: Cancer-News | May 20, 2019 | 51 view(s)

  Philadelphia, PA (May 13, 2019) – Oncoceutics announced today that Michael Chiarella, an industry veteran with more than 20 years’ experience at a number of large and small biotech companies, is joining the company as Vice President, Clinical Operations. Mr. Chiarella will be responsible for building out Oncoceutics’ clinical operations in connection with the company’s preparation for an eventual filing of a New Drug Application (NDA) for Oncoceutics’ lead compound, ONC201.ONC201 is currently in Phase II clinical trials for a number of oncology indications, including adult recurrent H3 K27M-mutant high-grade glioma, an indication for which the company has received Fast Track designation from the U.S. Food and Drug Administration (FDA). As the company prepares to transition this Phase II trial into a pivotal program and convert from an early-stage drug discovery and development company into a fully-integrated pharmaceutical company, it will continue to build its clinical operations capabilities.“We are delighted to have an individual with Michael Chiarella’s extensive know-how joining Oncoceutics,” said Lee Schalop, MD, Chief Operating Officer of Oncoceutics. “Michael’s experience in building a completely integrated clinical operations team at companies like Celator Pharmaceuticals and Aton Pharmaceuticals is a perfect fit for Oncoceutics as we grow our clinical operations.”“I am excited to be joining the dedicated and experienced group of individuals at Oncoceutics,” said Mr. Chiarella. “Novel therapies are desperately needed for the treatment of brain cancer, and I look forward to using my experience in the development process to help move ONC201 rapidly forward toward regulatory approval.”About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, The Musella Foundation, and a series of private and public partnerships. Visit Oncoceutics for more information.   ReplyForward  

Study touts new method to reduce cognitive side effects of brain cancer radiation treatment

By: Cancer-News | May 18, 2019 | 30 view(s)

UCI team helps uncover how ‘FLASH radiotherapy’ reduces neural cell damage   Irvine, Calif., May 13, 2019 — In hopeful news for brain cancer patients, researchers from the University of California, Irvine and the University of Lausanne in Switzerland have discovered how an experimental technology called FLASH radiotherapy dramatically reduces the adverse cognitive side effects unleashed by traditional radiation treatments.   “Although more research needs to be done, this has the potential to revolutionize cancer care worldwide,” said Charles Limoli, a professor of radiation oncology in UCI’s School of Medicine and co-author of the study, which appears online this week in Proceedings of the National Academy of Sciences.   Unlike current radiotherapy treatments, which target tumors with low-dose ionizing radiation for several minutes per session and invariably damage surrounding brain tissue, FLASH employs a much higher dose of radiation but only for milliseconds. Overall, the subjects received the same amount of radiation, but FLASH delivered it in a much shorter time frame.   In tests on mice, the quick-burst technique sharply reduced collateral cell damage and thereby reduced the learning and memory problems and mood disorders associated with conventional radiation, according to the study.   Working with Swiss radiobiologist Marie-Catherine Vozenin, who pioneered the technique in 2014, Limoli’s UCI team used laboratory mice and a battery of tests to analyze “the FLASH effect” and decipher how it works. Their conclusion: The split-second duration of FLASH irradiation – delivered 3,000 times faster than conventional treatments – produced lower levels of toxic hydrogen peroxide in brain cells. Limiting that chemical reaction protected the brain from short- and long-term damage, the research found. But FLASH was just as effective against tumors as current treatments.   Although previous animal studies have demonstrated FLASH’s promise as a cancer treatment, this is the first one to show long-term (6-month) cognitive benefits and explain how the method reduces side effects, Limoli said. “Oncology clinics and radiotherapy device manufacturers across Europe and the U.S. are now scrambling to advance this technology,” he added.   It will take about five years to refine the radiotherapy devices and complete the additional studies needed to bring FLASH treatments to human patients, Limoli predicted.   UCI researchers Munjal Acharya, Leila Alikhani, Barrett Allen and Paramvir Singh also contributed to the study, which received support from the National Institute of Neurological Disorders and Stroke, Fond’action, the IRSEC Foundation and an Ecole Normale Superieure de Cachan Fellowship.   About the University of California, Irvine: Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. It’s located in one of the world’s safest and most economically vibrant communities and is Orange County’s second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visit www.uci.edu.   Media access: Radio programs/stations may, for a fee, use an on-campus ISDN line to interview UCI faculty and experts, subject to availability and university approval. For more UCI news, visit news.uci.edu. Additional resources for journalists may be found at communications.uci.edu/for-journalists.    

Research Publications Demonstrate Imipridones Target Mitochondrial Function in Cancer Cells

By: Cancer-News | May 16, 2019 | 54 view(s)

Philadelphia, PA (May 3, 2019) – Oncoceutics, Inc. announced the publication of two scientific research articles demonstrating that members of the imipridone family ONC201 and ONC212 directly activate a mitochondrial protease called caseinolytic protease P (ClpP).One research article, featured on the cover of the journal Cancer Cell, demonstrates that ONC201 and ONC212 hyperactivate ClpP by altering its structural conformation, leading to mitochondrial dysfunction and apoptosis in leukemia. In AML, ClpP is over-expressed in patient samples and its hyperactivation selectively kills cancer cells independent of p53 status, while not affecting normal cells.Another research article, published in the journal ACS Chemical Biology, similarly demonstrates that ONC201 and ONC212 directly bind to and activate ClpP.ClpP is a protease located in the mitochondrial matrix that is overexpressed in tumor cells and plays a central role in mitochondrial protein quality control by degrading misfolded proteins. Modulating the activity of ClpP can cause impaired oxidative phosphorylation that selectively kills cancer cells in vitro and in vivo without affecting normal cells.The two research studies concordantly demonstrate that imipridones are capable of targeting ClpP in tumor cells, which has emerged as a therapeutic target in oncology. ClpP activation by imipridones is consistent with select downstream effects of these compounds that have been previously reported, such as integrated stress response activation and disruption of mitochondrial structure and function in tumor cells.“The unique phenotypic effects of ONC201 and its imipridone family members that have been studied in a broad range of cancers point to a novel mechanism of action,” said Dr. Joshua Allen, PhD, Senior Vice President of R&D at Oncoceutics. “The discovery that ONC201 targets ClpP, in addition to DRD2, expands the biomarker panel for the molecule and other impridones. ClpP activation also helps us interpret downstream effects of imipridones on the mitochondria, as well as their activity in tumor types beyond those that depend on the dopamine pathway.”“ClpP activation in cancer and disruption of the resulting mitochondrial dysfunction by ONC201 provides a mechanism of action that reinforces the basis for its efficacy spectrum across cancer that differentiates from other DRD2 antagonists,” said Dr. Keith Flaherty, MD, Director of Clinical Research at Massachusetts General Hospital and Member of Oncoceutics’ Scientific Advisory Board.About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The lead compound from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from the NCI, FDA, The Musella Foundation, and a series of private and public partnerships. Visit Oncoceutics for more information.   ReplyForward

Composition of Matter Patent and Translational Research Grant issued for ONC213

By: Cancer-News | May 14, 2019 | 55 view(s)

Composition of Matter Patent and Translational Research Grant issued for ONC213Philadelphia, PA (April 30, 2019) – Oncoceutics, Inc. announced that the United States Patent and Trademark Office (USPTO) has issued patent #10,266,533 entitled “7-BENZYL-4-(2-METHYLBENZYL)-2,4,6,7,8,9-HEXAHYDROIMIDAZO [1,2-A]PYRIDO [3,4-E]PYRIMIDIN-5(1H)-ONE, ANALOGS THEREOF, AND SALTS THEREOF AND METHODS FOR THEIR USE IN THERAPY” with an expiration date of January 29, 2036. This patent covers the composition of matter for ONC213, its di-salt formulation, and its use in the treatment of cancer. In addition to the claims related to ONC213, the patent also contains claims for millions of structurally-related imipridones. ONC213 is the fourth molecule in the company’s pipeline of “imipridone” family of anti-cancer small molecules that target G protein-coupled receptors, following ONC201, ONC206 and ONC212. As with other members of the imipridone class, ONC213 has very attractive chemical and biological properties including oral bioavailability, chemical stability and a large therapeutic index. “We are delighted by the decision of the US Patent Office to grant Composition of Matter to the novel molecule ONC213,” said Martin Stogniew, Ph.D., Chief Development Officer of Oncoceutics. “This patent paves the way for future generations of imipridones to enter the clinic and eventually benefit the lives of patients.”ONC213 has demonstrated anti-cancer activity and safety in various preclinical oncology models across hematological malignancies and solid tumors tested in the lab of Principal investigator Dr. Yubin Ge, Associate Professor of Oncology at the Karmanos Cancer Institute and Wayne State University School of Medicine. Results presented at the 61st American Society of Hematology Annual Meeting demonstrated that ONC213 targets leukemic stem cells in patient-derived xenograft mouse models, is well tolerated and combines synergistically with Bcl-2 inhibitor Venetoclax. Dr. Ge recently received a grant from the Kids Without Cancer and the Children’s Hospital of Michigan Foundation to further determine the mechanism of action of ONC213 and enable biomarker selection for clinical studies. “Our team at the Karmanos Cancer Institute is excited by the potential of ONC213 as a new type of cancer therapy,” said Dr. Yubin Ge. “We look forward to continuing development of this novel agent as it makes its way towards the clinic.” About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, i.e. the “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, Musella Foundation, XCures, Cancer Commons, and a series of private and public partnerships. Visit Oncoceutics more information. About Kids Without CancerFounded as Leukemia, Research, Life (LRL) in 1981 and renamed Kids Without Cancer in 2011, this organization has, for the past 36 years, been raising money to fund the pediatric cancer research being conducted at Children’s Hospital of Michigan and its affiliates Wayne State University and the Karmanos Cancer Institute. Kids Without Cancer is the largest contributor of funds to pediatric cancer research at Children’s Hospital of all charities solely dedicated to funding cancer research.

ABC2 Awards Grant to Develop Natural Disease History Benchmark for the Development of ONC201 in Midline Gliomas

By: Cancer-News | April 30, 2019 | 49 view(s)

Philadelphia, PA (April 24, 2019) – Oncoceutics announced today that Accelerate Brain Cancer Cure (ABC2) has awarded a $116,523 grant to a research team at University of California, Los Angeles to perform a natural disease history study of adult midline gliomas, a certain type of aggressive brain tumors. The study will follow patients from first diagnosis through a series of therapeutic interventions that for a subset of patients will include ONC201, an investigational agent in clinical trials for H3 K27M-mutant gliomas that has induced radiographic regressions in some patients.“Novel therapies are desperately needed for treatment of H3 K27M-mutant gliomas,” said Max Wallace, Chief Executive Officer of ABC2. “A dedicated natural history study will open up the path to accelerated approval based on radiographic responses to new treatments such as ONC201 in this rare and immediately life-threatening disease.”ONC201 is the first antagonist of D2-like dopamine receptors DRD2/3 to be developed for clinical neuro-oncology. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to ONC201 for the treatment of adult recurrent H3 K27M-mutant high-grade glioma. Diffuse midline gliomas harboring the histone H3 K27M mutation have a dismal prognosis with a median survival from diagnosis consistently reported to be between 8 and 11 months. The mutation was chosen by the World Health Organization (WHO) as a criterion for WHO grade IV designation, the most severe grade that is associated with a diffuse and aggressive growth pattern. No drug has been approved for H3 K27M-mutant glioma and no investigational therapy has shown clinically meaningful efficacy in this indication. Radiation is typically used in newly diagnosed patients and associated with some benefit before the tumor progresses, leading to the dismal survival rates.  Despite the increased awareness for the prognostic importance of the H3 K27M mutation, dedicated studies of midline gliomas are sparse, particularly in adults. Response criteria that are often applied, e.g. RANO criteria, were developed for supratentorial glioblastoma that have different radiographic features than midline gliomas, including H3 K27M-mutant gliomas. Consequently, the utility of these criteria for many midline glioma patients are confounded by a number of features often found in this disease: minimal or no contrast enhancement, diffuse growth, multifocal dissemination, and sub-centimeter lesions (not measurable as per some criteria). With the disease classifications that consider phenotypic as well as genotypic disease characteristics (e.g. WHO 2016) and patient registries that collect historical data the disease can be studied in more detail. However, many features of midline gliomas are still poorly understood, including tumor growth rates, dissemination patterns, response to therapies, and the relationship of clinical outcomes to these features.“The goal of the study is to quantify the dynamics of how midline gliomas change over time on clinical imaging to better understand the natural progression of this disease and if treatments can alter those dynamics,” said Timothy F. Cloughesy, MD, Clinical Professor and Director of the Neuro-Oncology Program at the University of California, Los Angeles. “The natural disease history could serve as a benchmark for new therapies such as ONC201 and other agents targeting glioma. We are very thankful to ABC2 for their support that will allow us to research this important and unaddressed topic.”About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, Musella Foundation, XCures, Cancer Commons, and a series of private and public partnerships. Visit Oncoceutics for more information.About ABC² (Accelerate Brain Cancer Cure)ABC² is focused on speeding the discovery of a cure for brain cancer. By applying an aggressive, venture funding approach not typically seen in the nonprofit sector, ABC2 closes current gaps in funding in order to catalyze research and rapidly bring new therapies to patients. ABC2 has awarded more than 100 grants totaling $21 million in brain tumor research funding to highly qualified investigators and physician-scientists from 54 institutions and companies. Since its founding in 2001, ABC2 has helped bring 30 different treatments into the clinic. To learn more, visit https://abc2.org/.

NEW DATA SHOW BLUEPRINT® IDENTIFIES ESTROGEN RECEPTOR POSITIVE BREAST CANCER PATIENTS WITH POOR RESPONSE TO ANTI-ESTROGEN THERAPY WHO MAY BENEFIT FROM NEOADJUVANT CHEMOTHERAPY

By: Cancer-News | April 25, 2019 | 38 view(s)

NEW DATA SHOW BLUEPRINT® IDENTIFIES ESTROGEN RECEPTOR POSITIVE BREAST CANCER PATIENTS WITH POOR RESPONSE TO ANTI-ESTROGEN THERAPY WHO MAY BENEFIT FROM NEOADJUVANT CHEMOTHERAPY   ER+ breast cancer patients reclassified as ER+ basal-type demonstrated significant response to chemotherapy underscoring critical role of advanced diagnostic tools   Results published in Nature Publishing Group’s npj Breast Cancer   IRVINE, CALIF., U.S., and AMSTERDAM, NETHERLANDS –24 April, 2019 – Agendia, Inc., a world leader in precision oncology, today announced the publication of study results for BluePrint®, its 80-gene proprietary molecular subtyping test, in Nature Breast Cancer. It is well known that a significant subset of patients diagnosed with early stage, estrogen receptor (ER) positive (ER+) breast cancer appear to be poorly responsive to standard anti-estrogen therapy and may benefit significantly to the addition of chemotherapy. This latest study suggests that use of BluePrint® can more precisely predict tumor response to treatment and clinical outcome.   Published this week, the study, “Estrogen Receptor variants in ER-positive basal-type breast cancers responding to therapy like ER-negative breast cancers” sought to conduct a molecular analysis and clinical follow up of patients that were reclassified as ER+ Basal-type (n=91) in the Neoadjuvant Breast Registry Symphony Trial (NBRST) (n=1072). The results of the analysis showed: ER+ Basal-type patients revealed missing sections from the messenger RNA (mRNA) for the ER gene, ESR1, rendering the translated ER protein non-functional ER+ Basal-type patients experienced a far higher rate of pCR with neoadjuvant chemotherapy than did ER+ Luminal patients (34 vs. 5%) 3-year outcomes were comparable to triple-negative patients   Pat Whitworth, MD, Director, Nashville Breast Center and study author said, “Traditionally, the medical community has been challenged by a certain subtype of ER+ tumors that respond poorly to standard treatment. These “bad actors” read out as ER+ on standard assays even though their ER does not function well in the cell. This study shows that through diagnostic tools like BluePrint®, we now have the ability to predict and identify how a tumor might respond to treatment that goes beyond traditional molecular classifiers, to seeing the fundamental biology of an individual tumor. This diagnostic step is critical to ensuring patients get the right treatment from the start, and helps drive better long-term outcomes.”   In partnership with the Breast Cancer Research Foundation (BCRF), NBRST took breast cancers categorized by traditional pathology measures (immunohistochemistry or IHC) as ER-positive (ER+), and molecularly reclassified them with BluePrint® into one of three subtypes – Luminal, HER2 or Basal – based on gene expression patterns. Thirteen percent of these ER+ patients were reclassified as having Basal-type breast cancer and were observed to have similar clinical outcomes and response to therapy as “triple-negative” breast cancer which typically indicates chemotherapy as standard treatment. Similar to patients with triple-negative breast cancer, these ER+ Basal-type patients experienced a six-fold increase in pathologic complete response (pCR) to neoadjuvant chemotherapy compared to other ER+ patients, which resulted in significant improvement in long-term outcomes.   “What we now understand is that a diagnosis of breast cancer may encompass up to a dozen distinct diseases, all with differing prognoses and treatments. This data offers additional clarity to the oncology community’s understanding of the complexity of breast cancer, and provides further data regarding this clinically important subset of ER+ cancers. Because routine pathology cannot reliably distinguish these cancers, identifying these ER+ Basal-type cancers through subtyping with BluePrint® provides valuable new information for the optimal management of early breast cancer,” added William Audeh, MD, Medical Oncologist and Agendia Chief Medical Officer.   The study is available open access and can be found on-line at https://rdcu.be/bxGN9.   About MammaPrint®   MammaPrint is an in vitro diagnostic medical device, performed as a testing service in a central laboratory, using the 70-gene expression profile of breast cancer tissue samples to assess a patient’s risk for distant metastasis. The device is FDA-cleared and CE-marked, enabling use in the European Union. MammaPrint is indicated for use by physicians as a prognostic marker only, along with other clinical-pathological factors. It is not intended to determine the outcome of disease, nor to suggest or infer an individual patient’s response to therapy. MammaPrint is the only test of its kind recommended for lymph node-negative and lymph node-positive patients by both the American Society of Clinical Oncology (ASCO) and National Comprehensive Cancer Network (NCCN®). The test is also recommended by many other national and international clinical practice guidelines.   About BluePrint®   BluePrint® is an 80-gene complementary gene expression test provided with MammaPrint which allows functional molecular subtyping of a breast cancer sample into three distinct subtypes: Luminal-type, HER2-type and Basal-type, each with marked differences in long-term outcome and response to neoadjuvant chemotherapy.   About Agendia   Agendia is a privately held, leading precision oncology company focused in breast cancer that develops and markets genomic diagnostic products, which help support physicians with their complex treatment decisions. Agendia’s breast cancer tests were developed using an unbiased gene selection by analyzing the complete human genome. The company’s offerings include the MammaPrint Breast Cancer Risk-of-Recurrence Test, and the BluePrint® Molecular Subtyping Test, both on microarray technology, whereas the new MammaPrint BluePrint® Breast Cancer Recurrence and Molecular Subtyping Kit, is on NGS technology. The MammaPrint BluePrint® next-generation sequencing-based kit is a CE-marked device currently available for use in cancer centers in select regions of the world. In addition, Agendia has a pipeline of other genomic products in development. The company collaborates with pharmaceutical companies, leading cancer centers and academic groups to develop companion diagnostic tests in the area of oncology. For more information on Agendia or the MammaPrint and BluePrint® tests, visit www.agendia.com.

Case Report in the Journal of Neurosurgery Highlights Potential of ONC201 in H3 K27M-mutant DIPG

By: Cancer-News | April 09, 2019 | 83 view(s)

Philadelphia, PA (April 9, 2019) –  Oncoceutics announced today the publication of an article entitled "First clinical experience with DRD2/3 antagonist ONC201 in H3 K27M–mutant pediatric diffuse intrinsic pontine glioma: a case report” in the Journal of Neurosurgery (authored by Matthew D. Hall, M.D., MBA). The article summarizes the medical history of a 10-year-old girl with a diffuse intrinsic pontine glioma (DIPG) brain tumor. Following radiation therapy and treatment with ONC201 on a compassionate use basis, she developed near complete resolution of her presenting neurological symptoms for almost one year, enabling her return to school and participation in many normal activities.DIPG is a serious and rare disease with a dismal prognosis and no viable treatment options. It predominantly affects children and young adults, and it is the most common form of brainstem glioma in this age group. DIPG.org, a resource network for the disease, summarizes the disease characteristics as follows: "Currently, outcomes for most patients are poor, with a median survival of less than 1 year from diagnosis. Radiation therapy can shrink tumors, temporarily improving some symptoms and delaying the progression of the disease, but in almost all cases, the tumor continues to grow. So far, clinical trials have not shown that currently available chemotherapy drugs, radiosensitizing drugs (drugs that make tumor cells more likely to be killed by radiation therapy), or biologics (medical products created by biological processes, such as vaccines or gene therapy) benefit patients. Because of their location in the brainstem, DIPGs cannot be removed surgically. New approaches to treating DIPG are urgently needed." ONC201 is an investigational drug developed by Oncoceutics that is being studied in several clinical trials for use against DIPG and other brain tumors. The underlying mechanism of the drug involves the interception of a specific receptor for dopamine, called DRD2. DRD2 is a neurotransmitter that is misused by malignant glioma cells to boost their growth. Tumors that harbor a certain mutation of a highly conserved histone protein, i.e. H3 K27M, are particularly sensitive to ONC201 in spite of the otherwise aggressive growth that this mutation confers. DIPG is one of the tumor types that exhibit a high prevalence of this mutation. The patient described in the article has shown prolonged clinical benefits and is approaching almost two years from diagnosis, although ONC201 was administered only when symptoms progressed after radiation was completed.  This supports further investigation of ONC201 in H3 K27M-mutant gliomas, including DIPG. As a result, Oncoceutics has significantly expanded its pediatric clinical program for ONC201. The company's ongoing pediatric trial for ONC201 in H3 K27M-mutant high-grade gliomas including DIPG (NCT03416530) was extended with additional treatment arms, a pediatric oral solution formulation was introduced, and ONC201 treatment was extended to newly diagnosed DIPG patients with concomitant radiation.The company further implemented an intermediate size Expanded Access Protocol (EAP) to provide access to ONC201 for patients that might benefit from the drug but are not eligible for participation in the ongoing clinical trials. The EAP was made possible by the support of the Food and Drug Administration and their high priority effort to facilitate access to promising medicines for patients with serious or immediately life-threatening diseases or conditions when no comparable or satisfactory alternative therapy options are available (see recent FDA statement). The program is also supported by The Musella Foundation, The Cure Starts Now Foundation, The Michael Mosier Defeat DIPG Foundation, Cancer Commons and xCures. “We are delighted to see a novel concept to treat this horrible disease emerge and show traction in clinical settings,” said Keith Desserich, Chairman of the Board and Co-Founder of The Cure Starts Now. “We have followed the development of ONC201 for some time and are excited about the emerging data in both, children as well as young adults that are expected to become public later in this year. About OncoceuticsOncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds, called “imipridones,” that selectively target G protein-coupled receptors for oncology. The first lead compound to emerge from this program is ONC201, an orally active small molecule DRD2 antagonist. The company is supported by grants from NCI, FDA, Musella Foundation, XCures, Cancer Commons, and a series of private and public partnerships. Visit Oncoceutics.com for more information.About The Musella FoundationThe Musella Foundation For Brain Tumor Research & Information, Inc is a 501(C)3 nonprofit public charity dedicated to helping brain tumor patients through emotional and financial support, education, advocacy and raising money for brain tumor research. Visit virtualtrials.com for more information.About The Cure Starts Now FoundationThe Cure Starts Now Foundation is a 501(c)3 nonprofit organization with international chapters in nearly 40 locations around the world.  It is has funded universal cancer cure strategies starting with cancers such as DIPG and organizes the biennial International DIPG Symposium showcasing innovative research methods. You can learn more at www.thecurestartsnow.org.About Michael Mosier Defeat DIPG FoundationMichael Mosier Defeat DIPG Foundation is committed to finding a cure for brainstem tumors known as diffuse intrinsic pontine gliomas (DIPG).  Nearly every day one child in the United States is diagnosed with DIPG and another child dies from it. The Foundation seeks to make a difference and defeat DIPG both by raising awareness of DIPG and by providing funding for research into effective treatments for DIPG. Visit defeatdipg.org for more information.About Cancer CommonsCancer Commons is a nonprofit collaborative of patients, physicians, and scientists, dedicated to improving patient outcomes by tightly coupling clinical research and care. We arm patients and their physicians with the knowledge they need to achieve the best possible outcomes, help them access the relevant treatments and trials, and track their results to continuously learn. Vistitcancercommons.org for more information.About xCuresxCures is developing an AI-based methodology and platform to run ‘Virtual Trials’, which continuously learn from the clinical experiences of all patients, on all treatments, all the time. Each patient’s treatment regimen is adaptively planned by a ‘Virtual Tumor Board’ to optimize their individual outcome, and these plans are coordinated across the whole patient population to maximize collective learning. Visit xcures.com for more information.