Onconova Therapeutics Inc. (NASDAQ: ONTX) announced the submission of an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for IV rigosertib as a treatment for higher-risk myelodysplastic syndromes (HR-MDS) after failure of hypomethylating agent (HMA) therapy. Upon clearance, the IND enables Onconova to initiate a randomized, controlled pivotal Phase 3 trial in patients with HR-MDS that have failed prior HMA therapy.
The primary endpoint of this study will be overall survival, and an interim analysis is anticipated. This randomized trial of approximately 225 patients will be conducted at about 100 sites globally. The enrollment for this trial is expected to begin later in the year, though the ability to conduct the trial as planned will require additional financing.
For some background on Onconova, it is a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer. Onconova’s clinical and preclinical stage drug development candidates are derived from its extensive chemical library and are designed to work against specific cellular pathways that are important in cancer cells, while causing minimal damage to normal cells.
Ramesh Kumar, Ph.D., president and CEO of Onconova, said:
We are pleased to achieve this important milestone, which positions rigosertib back on an approval track in HR-MDS. We plan to submit Clinical Trial Applications (CTAs) in several European countries shortly. Onconova anticipates initiating enrollment in the new Phase 3 study in the second half of 2015.
So far Onconova has had a weak performance in the market. Shares are down nearly 40% year to date and over 60% in the past 52 weeks.
Shares of Onconova were up nearly 33% at $2.63 on Friday afternoon. The stock has a consensus analyst price target of $5.00 and a 52-week trading range of $1.52 to $5.78.