Onyx announces data from regorafenib Phase 3 trial on GIST

Onyx Pharmaceuticals (Nasdaq: ONXX) today announced data from the Phase 3 GRID (GIST – Regorafenib In Progressive Disease) trial evaluating the investigational drug regorafenib, a Bayer compound, in patients with metastatic and/or unresectable gastrointestinal stromal tumors (GIST) whose disease had progressed despite prior treatment with imatinib and sunitinib. The GRID study met its primary endpoint of progression-free survival (PFS) (HR=0.27, p<0.0001). The median PFS was 4.8 months in the regorafenib arm versus 0.9 months in the placebo arm.(1) These data will be presented as a late-breaking abstract in an oral abstract session at the 48th Annual Meeting of the American Society of Clinical Oncology (ASCO) in Chicago, IL (USA) (LBA No. 10008).

"GIST continues to represent an area of unmet patient need, given the aggressive nature of the disease and our limited therapeutic options," said George Demetri, MD, Director, Center for Sarcoma and Bone Oncology, Dana-Farber Cancer Institute, and Chair of the Steering Committee for the GRID trial. "These data from the GRID study indicate that regorafenib may be a potential treatment option for patients who have relapsed after standard therapies."

The most common drug-related, treatment-emergent adverse events (occurring in at least 10% of patients during double-blind treatment) included hand-foot skin reaction (56.1% vs.15.2%), hypertension (48.5% vs. 16.7%), diarrhea (40.9% vs.7.6%), fatigue (38.6% vs. 27.3%), oral mucositis (37.9% vs. 9.1%), alopecia (23.5% vs. 3.0%), hoarseness (22.0% vs. 4.5%), anorexia (20.5% vs. 7.6%), rash, maculopapular (18.2% vs. 3.0%), nausea (15.9% vs. 9.1%), constipation (15.2% vs. 7.6%), myalgia (13.6% vs. 9.1%), and voice alteration (11.4% vs. 3.0%) for patients receiving regorafenib as compared to placebo.

Bayer has submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) for the oral multi-kinase inhibitor regorafenib for the treatment of patients with metastatic colorectal cancer (mCRC). Bayer plans to submit a New Drug Application for regorafenib in GIST in the second half of 2012.