Onconova Enrolls First Patient in Europe for Phase 3 INSPIRE Trial of Rigosertib in Higher-Risk Myelodysplastic Syndromes

On March 21, 2016 Onconova Therapeutics, Inc. (NASDAQ:ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, reported the enrollment of the first European patient in Salzburg, Austria for the Phase 3 INSPIRE trial for IV rigosertib as a treatment for higher-risk myelodysplastic syndromes (HR-MDS) following failure of hypomethylating agent (HMA) therapy (Press release, Onconova, MAR 21, 2016, View Source [SID:1234509771]). The first patient in this global trial was enrolled at the MD Anderson Cancer Center in December 2015.

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"We are excited to expand enrollment for this important trial to Europe," said Dr. Steven Fruchtman, Chief Medical Officer of Onconova. "The recent publication in Lancet Oncology of results of the ONTIME study provides support for the present trial and highlights the urgent unmet medical needs of patients following failure of front-line treatment with HMAs. There are no approved therapies for HR-MDS patients whose disease has failed treatment with an HMA, and the clinical community is looking for novel drugs to address this treatment gap."

The INSPIRE trial is a multi-center, randomized controlled Phase 3 study to assess the efficacy and safety of IV rigosertib in HR-MDS patients who had progressed on, or failed to respond to, or relapsed, following previous treatment with HMAs. The trial will enroll approximately 225 patients randomized at a 2:1 ratio into two treatment arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival and an interim analysis is anticipated.

The INSPIRE trial is actively screening patients at multiple sites in the United States and Europe, and additional sites are being initiated globally. Onconova’s collaboration partner in Japan and Korea, SymBio Pharmaceuticals, Ltd., intends to begin enrolling patients in Japan shortly.

About INSPIRE

The INternational Study of Phase III IV RigosErtib, or INSPIRE, is based on guidance received from the U.S. Food and Drug Administration and European Medicines Agency and derives from the findings of the ONTIME Phase 3 trial. INSPIRE is a multi-center, randomized controlled study to assess the efficacy and safety of IV rigosertib in HR-MDS patients who had progressed on, failed to respond to, or relapsed after previous treatment with an HMA within the first nine months of initiation of HMA treatment. This time frame optimizes the opportunity to respond to treatment with an HMA prior to declaring treatment failure, as per NCCN Guidelines.1 The trial will enroll approximately 225 patients randomized at a 2:1 ratio into two treatment arms: IV rigosertib plus Best Supportive Care versus Physician’s Choice plus Best Supportive Care. The primary endpoint of INSPIRE is overall survival and an interim analysis is anticipated. Full details of the INSPIRE trial, such as inclusion and exclusion criteria, as well as secondary endpoints, can be found on clinicaltrials.gov (NCT02562443).

About Rigosertib

Rigosertib is a small molecule inhibitor of cellular signaling and acts as a Ras mimetic. These effects of rigosertib appear to be mediated by direct binding of the compound to the Ras-binding domain (RBD) found in many Ras effector proteins, including the Raf kinases and PI3K. The therapeutic focus for rigosertib is myelodysplastic syndromes (MDS), a group of bone marrow disorders characterized by ineffective formation of blood cells that often converts into acute myeloid leukemia (AML). Clinical trials for rigosertib are being conducted at leading institutions in the United States, Europe, and the Asia-Pacific region. Rigosertib is protected by issued patents (earliest expiry in 2026) and has been awarded Orphan Designation for MDS in the United States, Europe and Japan.