Onconova Therapeutics Files European Clinical Trial Applications for Global Phase 3 Trial for IV Rigosertib in Higher-Risk Myelodysplastic Syndromes

Onconova Therapeutics, Inc. ONTX, a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, today announced that it has filed Clinical Trial Applications (CTAs) with the United Kingdom, German and Austrian regulatory agencies for IV rigosertib as a treatment in higher-risk myelodysplastic syndromes (HR-MDS) patients after failure of a hypomethylating agent (HMA) therapy. Upon clearance of the CTAs and the recently updated U.S. IND submission, Onconova intends to initiate a single randomized controlled pivotal Phase 3 trial, designated 04-30 or "INSPIRE", in patients with HR-MDS whose prior therapy with an HMA has failed. Additional filings in other European countries and in Japan are expected to follow shortly. "These filings follow our recent updated IND submission to the U.S. FDA," said Ramesh Kumar, Ph.D., President and CEO of Onconova. "Together, acceptance of these regulatory filings will allow Onconova to initiate a single global pivotal trial for IV rigosertib in HR-MDS. We anticipate enrollment in the new Phase 3 study will begin in the second half of 2015." Per a development and licensing agreement with Baxalta (formerly the BioScience business of Baxter International Inc.), which grants Baxalta commercialization rights to rigosertib in the European Union and other countries in Europe, the company has elected to and Baxalta will fulfill its obligation under the agreement to pay for half of the costs for the trial of rigosertib in HR-MDS up to a specified cap. Onconova has partnered with SymBio Pharmaceuticals, Ltd. for clinical development of rigosertib in Japan and Korea. SymBio plans to participate in the global Phase 3 trial by enrolling patients in Japan, which should accelerate the timing of regulatory filings in Japan and Korea. "We completed a Phase 1 trial for oral rigosertib (SyB C-1101) in relapsed or refractory MDS last June and expect to complete the ongoing Phase 1 trial for IV rigosertib (SyB L-1101) in the treatment of relapsed or refractory HR-MDS patients this October. After consulting with the PMDA (Japanese Pharmaceuticals and Medical Devices Agency), it is our plan to participate in the global 04-30 trial, and to begin enrolling patients in Japan for this unmet medical need in HR-MDS," added Mr. Fuminori Yoshida, President and CEO of SymBio. "Onconova is appreciative of the continued collaborations with Baxalta and SymBio to advance the INSPIRE Trial," continued Dr. Kumar. "We look forward to working closely with our partners in the development of IV rigosertib in HR-MDS in Europe and in Japan." The INSPIRE Trial will enroll HR-MDS patients who had progressed on, or failed to respond to, previous treatment with an HMA. The primary endpoint of this study is overall survival, and an interim analysis is anticipated. This randomized trial of approximately 225 patients will be conducted at about 100 sites globally. Enrollment in this trial is expected to begin later this year.