Onconova Therapeutics (ONTX)

Onconova Therapeutics is a late-clinical stage biotech company focused on the development of Ras-targeted rigosertib as a potential treatment of high risk myelodysplastic syndromes (MDS). Although the previous rigosertib in high risk MDS Phase III (ONTIME) trial failed to meet its primary endpoint, we believe the company has identified several previously undetected prognostic factors. By leveraging them in the design of the current Phase III trial (INSPIRE) as a potential 2nd-line treatment in high risk MDS patients who were refractory to prior hypomethylating agents (HMAs); this could substantially increase the probability of success. In addition, the incorporated interim analyses provide greater flexibility for targeting more specific patient cohorts, and thereby enhancing the chance of success. The ongoing Phase III trial (INSPIRE) of intravenously administered rigosertib in HMA refractory high risk MDS could have interim and top-line readouts in 2H17 and 2018  which would be a major part of a critical catalyst-rich period over the next 18 months. ONTX is also developing an oral rigosertib to be used in combination with a HMA, azacitidine, as a potential 1st-line treatment in high risk MDS. The combination regimen demonstrated promising results in a Phase II trial and the company is working on the final study design for a Phase III trial, which could possibly start in 2018. ONTX also has an earlier stage product candidate, ON 123300, which has the potential for treating CDK4/6 overactive tumors and is under preclinical study.