Philadelphia, PA (September 10, 2018) – Oncoceutics, Inc. announced the receipt of a Small Business Innovation Research (SBIR) Phase IIB Bridge Award from the National Cancer Institute (NCI). The NCI SBIR Bridge Award will allow Oncoceutics to expand and accelerate its clinical trials evaluating ONC201 in patients with a specific type of lethal brain cancer called H3 K27M-mutant glioma.
The awarded project, “Adaptive Clinical Efficacy Evaluation of ONC201 in Recurrent High-Grade Glioma” (grant number 2R44CA192427-04), builds upon the work supported by previous NCI SBIR Fast-Track grant that supported Oncoceutics’ effort demonstrating the activity of ONC201 in high-grade gliomas, including those with the H3 K27M mutation that is commonly present in the midline region of the brain. Each year, the NCI’s SBIR Development Center presents the Phase IIB Bridge Award to a select number of companies based on NCI and external expert review of the scientific merits and commercial potential of the technology. Awardees are required to allocate third-party investment to match the Bridge funding prior to submitting the funding application which occurred in mid-2017. The NCI Bridge Award will provide $3 million to Oncoceutics to advance the clinical development of ONC201 over the next 3 years.
Patients with H3 K27M-mutant glioma often have significant neurological symptoms from their disease and lack proven therapeutic options other than palliative radiotherapy. However, emerging clinical results have shown that some patients treated with single agent ONC201 have stable disease, have had their tumor shrink, and/or have had improvements in neurological symptoms, such as paralyses of peripheral and cranial nerves. This has been observed in adults and children treated with ONC201, including children with diffuse intrinsic pontine glioma (DIPG), a type of high-grade glioma that almost uniformly (80-90%) harbors the H3 K27M mutation.
“We are extremely pleased to see continued support from the NCI for the ONC201 clinical program in H3 K27M-mutant gliomas based on the clinical activity observed in our Phase II study supported by the Fast-Track SBIR grant,” said Patrick Wen, MD, Director, Center For Neuro-Oncology, Dana-Farber Cancer Institute.
“The emerging clinical experience with ONC201 to treat gliomas at our institution and other leading cancer centers around the country is exciting,” added said Yazmin Odia, MD MS, Lead Physician of Medical Neuro-Oncology, Miami Cancer Institute. “The dismal prognosis of midline gliomas and the dearth of therapeutic options means that this therapy could be practice-changing for neuro-oncology. We are eager to follow up on the radiographic and clinical improvements in biomarker-defined patients that we have seen with ONC201 as a single agent in our ongoing clinical trials. Future co-operative group efforts between NRG Oncology and the Children’s Oncology Group to test this drug in children and adults with newly diagnosed H3 K27M-mutant midline gliomas are in the planning process.”
In addition to the support from the NCI, Oncoceutics has also received support from The Musella Foundation, a non-profit organization that helps brain tumor patients through education, advocacy, and financial support. The Musella Foundation has supported Oncoceutics’ development of ONC201 in high-grade gliomas for several years from bench to Phase II clinical trials. The Musella Foundation, in collaboration with Cancer Commons, The Cure Starts Now Foundation, Michael Mosier Defeat DIPG Foundation and xCures, has committed to contribute at least $1 million in hopes of accelerating the development of ONC201.
“Having represented and supported brain tumor patients’ interests for several decades, it has been frustrating to experience the failures of new treatments that have been developed to impact the outcome of this disease,” said Al Musella, Founder and President of the Musella Foundation. “As highlighted in a recent ODAC meeting, the FDA, in collaboration with the NCI, is tasked with determining whether a molecular target is or is not considered substantially relevant to the growth or progression of pediatric cancer. We are excited to work with Oncoceutics that is developing a molecularly targeted agent, ONC201, that demonstrates the potential to advance the concept of Precision Medicine in patients that harbor the H3K27M mutation, a genetic aberration that is considered substantially relevant for the outcome of this disease. We are looking forward to contribute to making this drug available for as many patients as possible.”
Oncoceutics, Inc. is a clinical-stage drug discovery and development company with a novel class of compounds that selectively target G protein-coupled receptors for oncology. The first lead compound to result from this program is ONC201, an orally active DRD2 small molecule antagonist that is well-tolerated and effective against advanced cancers. The company completed a successful Phase I study in solid tumors and has underway a number of Phase I/II and Phase II clinical programs in both solid and hematological malignancies. Oncoceutics and collaborative groups have received more than $7 million in grants over the last two years, including grants from the National Cancer Institute, the U.S. Food and Drug Administration, the Pennsylvania Department of Health, and The Musella Foundation. In addition, outside interest in the company’s portfolio has resulted in several R&D alliance agreements between Oncoceutics and leading comprehensive cancer centers, including The University of Texas MD Anderson Cancer Center and the Fox Chase Cancer Center. The company has established a robust intellectual property position, including several issued patents. Visit oncoceutics.com for more information.
About The Musella Foundation
The 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 Cancer Commons
Cancer 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. Vistit cancercommons.org for more information.
About The Cure Starts Now Foundation
The Cure Starts Now Foundation is a 501(c)3 nonprofit organization with international chapters in nearly 40 locations around the world. It 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 Foundation
Michael 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.
xCures 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.