Derived from a novel chemistry platform and cell-based differential screening, our technology targets cancer while protecting healthy cells.

Therapeutic Focus

Onconova is focused on discovering and developing targeted, small molecule product candidates for the treatment of cancer, including Myelodysplastic Syndromes (MDS).


Onconova is a Phase 3 company with a targeted trial design and several active clinical and pre-clinical programs.

About Onconova Therapeutics

Onconova Therapeutics is a Phase 3 stage biopharmaceutical company focused on discovering and developing drug candidates that treat myelodysplastic syndromes (MDS) and other forms of incurable blood cancers. MDS is a hematological malignancy resulting in bone marrow failure which leads to acute myeloid leukemia (AML) in 30% of patients. There have been no advancements in this indication in more than a decade, and current therapeutic options are not curative and contain side effects. More than 10,000 patients suffer from MDS, representing a significant unmet medical need.

Broad pipeline includes novel drug candidates in
phase 3, early stage clinical and pre-clinical trials


Onconova’s lead product candidate, rigosertib, a small molecule drug candidate, is currently in a pivotal phase 3 study (“INSPIRE”) for the treatment of MDS after failure of a hypomethylating agent. Rigosertib has a unique mechanism of action that targets the Ras cancer gene, one of the most sought after genes in oncology accounting for more than 30% of human cancers. To date, more than 1,100 patients have been treated with rigosertib.


The INSPIRE trial design is informed by prior studies which identified a sub-population in patients where the drug was highly effective. This trial is designed to enroll approximately 225 patients in the United States, Europe, Japan and Australia. An oral form of rigosertib has also been tested in combination with azacitidine in a Phase 1/2 trial for higher-risk MDS (HR-MDS) which showed promising interim data, setting the stage for a pivotal, Phase 3 study. Rigosertib is protected by issued patents and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.


Using a proprietary chemistry platform, Onconova has created a pipeline of targeted anti-cancer agents designed to work against specific cellular pathways that are important in cancer cells, while causing minimal damage to normal cells. Onconova has a broad pipeline with three products in clinical trials including rigosertib, briciclib (in advanced solid tumors refractory to current therapies), reclisib (to treat effects of advanced radiation syndromes, specifically cytopenia) and active pre-clinical programs.

Onconova Therapeutics was incorporated in 1998 in Delaware and began trading on the NASDAQ stock exchange under the ticker symbol ONTX in July, 2013.

Product Pipeline

Onconova is focused on discovering and developing targeted, small molecule product candidates for the treatment of cancer.



IV rigosertib
2nd - line
Oral rigosertib +
1st - line


Phase 1

Phase 2

Phase 3

INSPIRE Pivotal Trial
09 - 08 Phase 2 Trial

Method of Action

Ras Signaling
IV Briciclib
Solid tumors
ON 123300
Dose-escalation Phase 1 Trial
Non-Human Primate Efficacy
Pre-IND Stage
eIF4E targeting
Targeting radiation-induced apoptosis
CDK4/6 Targeting
* Acute Radiation Syndrome