Onconova Therapeutics, a Phase 3 stage biopharmaceutical company focused on discovering and developing novel small molecule drug candidates to treat cancer, with a primary focus on Myelodysplastic Syndromes (MDS), today announced the establishment of a collaborative, multi-institutional research and clinical program to evaluate rigosertib in pediatric RASopathies, a group of rare syndromes, which, together, are among the most common genetic conditions in the world, according to the RASopathies.Net. The program will generate supportive non-clinical data and obtain early clinical experience in the pediatric setting with rigosertib, Onconova’s lead clinical candidate.
Rigosertib in currently being evaluated in a global Phase 3 trial (INSPIRE) for MDS patients after failure of therapy with Hypomethylating Agents (HMAs). A Phase 2 trial of oral Rigosertib combined with Azacitidine is aimed at patients with MDS and AML.
The RASopathies are developmental syndromes usually caused by mutations that alter the RAS subfamily and mitogen-activated protein kinases that control signal transduction. Together, the RASopathies represent a group of neurodevelopmental syndromes affecting more than 1 in 1000 individuals, according to RASopathies.Net.
Reflecting Onconova’s focus on MDS and Myeloproliferative Neoplasms (MPNs), the Company will initially prioritize Juvenile Myelomonocytic Leukemia (JMML), a pediatric, typically germline, disease that shares characteristics of adult MDS and MPNs. JMML is a well-described RASopathy affecting children, which is incurable without an allogenic hematopoietic stem cell transplant. In addition, Onconova will collaborate with the National Cancer Institute on a broad clinical trial for pediatric patients with RASopathies.
“We are advancing research into one of the most important pediatric genetic syndromes, as we work together with families, clinicians and scientists to foster collaborative research efforts. Our program is based on mechanistic rationale, clinical activity in adult marrow diseases and sound safety data. By leveraging our focus in MDS and MPNs, we expect to further advance approaches to studying rigosertib in a variety of RAS associated diseases,” said Steven Fruchtman, M.D., Onconova’s Chief Medical Officer.
Dr. Fruchtman will present findings highlighting approaches for studying rigosertib in RAS associated diseases on Sunday, July 30th, at the 5th International RASopathies Symposium, organized by RASopathies.Net and held at the Renaissance Hotel in Orlando, Florida.
A copy of the presentation, “Strategies to RASopathies and JMML,” can be accessed by visiting “Scientific Presentations” in the Investors section of Onconova’s website.
Onconova is also hosting a Key Opinion Leader Breakfast Symposium for investors in New York City on Wednesday, October 11, 2017, to further highlight RASopathies. Dr. Elliot Steigholtz from the University of California San Francisco, and Dr. Bruce Gelb from Mount Sinai, along with Dr. Fruchtman, will present on novel approaches in this evolving area. The Company will disclose further details regarding the Symposium in the coming months.
Filed Under: Drug Discovery