Clinical Trials

Date: 2017-07-25

Type of information: Initiation of development program

phase:

Announcement: initiation of development program

Company: Onconova Therapeutics (USA - PA)

Product: rigosertib

Action mechanism: kinase inhibitor/phosphoinositide 3-kinase (PI3K) inhibitor. Rigosertib is a small molecule inhibitor of cellular signaling and acts as a Ras mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the Ras-binding domain (RBD) found in many Ras effector proteins, including the Raf kinases and PI3K. The initial therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials for rigosertib are being conducted at leading institutions in the United States, Europe, and the Asia-Pacific region. Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.

Disease: pediatric RASopathies (juvenile myelomonocytic leukemia,

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

Latest news:

• • On July 25, 2017, Onconova Therapeutics announced the establishment of a collaborative, multi-institutional research and clinical program to evaluate rigosertib in pediatric RASopathies. The program will generate supportive non-clinical data and obtain early clinical experience in the pediatric setting with rigosertib. Rigosertib in currently being evaluated in a global Phase 3 trial (INSPIRE) for myelodysplastic syndromes  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. Onconova 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.

Is general: Yes