Phase 3 Myelodysplastic Syndromes Trial Reaches Enrollment Milestone

According to a story from, the biopharmaceutical company Onconova Therapeutics, Inc. recently announced that they have successfully surpassed the 75 percent milestone for enrollment in the company’s Phase 3 clinical trial. This clinical trial will test the company’s investigational product candidate rigosterib as a treatment for high risk cases of myelodysplastic syndromes. Onconova is focused on developing small molecule drugs for treating various forms of cancer.

About Myelodysplastic Syndromes

Myelodysplastic syndromes are a type of blood cancer in which developing blood cells remain immature and fail to transform into usable blood cells. Risk factors for this disease include exposure to radiation, chemotherapy, benzene, xylene, and Agent Orange. Family history is also a risk, as are certain genetic disorders such as Down syndrome. In an individual case, it is rare for the direct cause to be identified. Myelodysplastic syndromes rarely present with symptoms initially, but it can eventually present with anemia, neutropenia, thrombocytopenia, cell abnormalities, chromosome abnormalities, enlarged spleen and/or liver, easy bleeding and bruising, and infections. The disease also has the potential to evolve into acute myeloid leukemia. Treatment may include bone marrow transplant, stem cell transplant, blood products, and certain chemotherapy agents. Outcomes in this disease ranges widely and can depend on a number of factors. To learn more about myelodysplastic syndromes, click here.

About The Trial

The meeting of this milestone is a significant step towards the maintaining the schedule the Onconova has planned for this trial. This study will be open label and international in scope and is intended to measure the tolerability, safety, and effectiveness of rigosertib as a single agent for patients with high risk disease. Enrollment for the trial is expected to be finished later in 2019. This trial will also include patients under age 82 that have received treatment with a hypomethylating agent and have seen their myelodysplastic syndromes progress, relapse, of have otherwise failed to respond. The primary endpoint for this trial is overall survival. The trial is expected to include a total of 360 patients.

About Rigosertib

Rigosertib is a small molecule drug that that is designed to target RAS effector pathways. The drug is being developed in both intravenous (IV) and orally available forms. The testing of this therapy in myelodysplastic syndromes is ongoing.

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