January 18, 2012
Positive Phase III Data on Bayer's Investigational Drug Regorafenib Show Significant Increase in Overall Survival
First presentation of data from CORRECT trial as "Late Breaking Abstract" at the 2012 Gastrointestinal Cancers Symposium of the American Society of Clinical Oncology (ASCO-GI)Berlin, January 18, 2012 - Bayer HealthCare today announced positive data on
its investigational drug regorafenib from the Phase III CORRECT (Colorectal
cancer treated with regorafenib or placebo after failure of standard therapy)
trial that will be presented as a late breaking abstract in an oral abstract
session at the 2012 Gastrointestinal Cancers Symposium of the American Society
of Clinical Oncology (ASCO-GI). The study evaluated regorafenib plus best
supportive care (BSC) versus placebo plus BSC in patients with metastatic
colorectal cancer (mCRC) whose disease has progressed after approved standard
therapies. The study met its primary endpoint of significantly improving
overall survival (OS) by 29% (HR=0.77, p=0.0052). The CORRECT trial also met
two secondary efficacy endpoints, including a significant improvement in
progression-free survival (PFS) (HR=0.49, p<0.000001), and a significant
improvement in the disease control rate (DCR) (p<0.000001). The difference in
the objective response rate (ORR) between the two arms did not reach
statistical significance. These data will be presented at ASCO-GI in San
Francisco, U.S. on Saturday, January 21, 2012, 2:30 PM - 4:00 PM Pacific Time,
Level 3 Ballroom, Moscone Center West (LBA No. 385).
"These data are noteworthy because they demonstrate that regorafenib can
stabilize disease, even at an advanced stage, and prolong life in patients with
metastatic colorectal cancer who have no other treatment options available,"
said Prof. Eric van Cutsem, University Hospital Leuven, Belgium, who shares the
role of the coordinating investigator of the study. "To date regorafenib is the
only oral multi-kinase inhibitor as monotherapy that has demonstrated in a
large Phase III trial the ability to improve clinical outcomes in patients with
advanced refractory colorectal cancer. The data suggest that regorafenib may
provide a new standard of care for the treatment of refractory metastatic
The efficacy analyses showed that patients who were treated with regorafenib
had the following outcomes:
- A median OS of 6.4 months compared to 5.0 months for the placebo group
- A median PFS of 1.9 months compared to 1.7 months for the placebo group (HR=
- A DCR of 44.8% compared to 15.3% for the placebo group (p<0.000001)
- An ORR of 1.0% compared to 0.4% for the placebo group (p=0.188)
The overall safety and tolerability profile for regorafenib was consistent with
results from previous studies. The most common drug-related, treatment-emergent
adverse events (occurring in at least 25% of patients) included fatigue (47.4%
vs. 28.1%), hand-foot skin reaction (46.6% vs. 7.5%), diarrhea (33.8% vs.
8.3%), anorexia (30.4% vs. 15.4%), voice changes (29.4% vs. 5.5%), hypertension
(27.8% vs. 5.9%), oral mucositis (27.2% vs. 3.6%), and rash/desquamation (26.0%
vs. 4.0%) for patients receiving regorafenib as compared to placebo.
The CORRECT trial was unblinded in late 2011 following a pre-planned interim
analysis that determined that the regorafenib arm showed significant
improvement in overall survival, and patients on the placebo arm were offered
treatment with regorafenib.
"We are excited by these results because there is a high unmet medical need for
patients with mCRC and these data show that regorafenib could make a positive
impact on overall survival in patients who currently have no other options,"
said Kemal Malik, MD, Member of the Bayer HealthCare Executive Committee and
Head of Global Development. "We look forward to generating further data for
this compound, including results from our other Phase III trial of regorafenib
in gastrointestinal stromal tumors".
Bayer plans to submit regorafenib for marketing authorization in mCRC in 2012.
About the CORRECT Study
The CORRECT trial is an international, multicenter, randomized, double-blind,
placebo-controlled study that enrolled 760 patients with mCRC whose disease has
progressed after approved standard therapies. The CORRECT study was conducted
in North America, Europe, China, Japan and Australia.
Patients were randomized to receive either regorafenib plus best supportive
care (BSC) or placebo plus BSC. Treatment cycles consisted of 160 mg of
regorafenib (or matching placebo) once daily for three weeks on / one week off
plus BSC. The primary endpoint of this trial was overall survival. Secondary
endpoints included progression-free survival, objective tumor response rate and
disease control rate. The safety and tolerability of the two treatment groups
were also compared.
About Colorectal Cancer
Colorectal cancer (CRC) is a disease in which malignant (cancer) cells form in
the tissues of the colon or rectum. The majority of cancers occurring in the
colon and rectum are adenocarcinomas, which account for more than 90 percent of
all large bowel tumors.
CRC is the fourth most common cancer worldwide, with over one million cases
occurring every year. The mortality rate from CRC is approximately half of its
global incidence. The five-year survival estimate for CRC on average is 55
percent, but is highly variable dependent on the stage of the disease (from 74
percent for patients with Stage I disease to only 6 percent for Stage IV
Regorafenib is an investigational oral multi-kinase inhibitor targeting
angiogenic, stromal and oncogenic kinases. Regorafenib inhibits several
angiogenic VEGF receptor tyrosine kinases which play central roles in tumor
neoangiogenesis and lymphangiogenesis. It also inhibits various oncogenic and
stromal kinases including KIT, RET, PDFGR, and FGFR thereby helping to stop the
proliferation of selected cancer cells. Regorafenib has shown antitumor
activity in preclinical studies by inhibiting tumor growth in multiple
xenograft models via tumor neoantiangiogenic and tumor cell antiproliferative
mechanisms. Based on these results, regorafenib is currently being investigated
in clinical trials for its potential to treat patients with various tumor
Regorafenib is an investigational agent and is not approved by the United
States Food and Drug Administration (FDA), the European Medicines Agency (EMA)
or other health authorities.
Regorafenib was granted Fast Track designation by the U.S. FDA for the
treatment of patients with mCRC who have progressed after approved standard
therapies, as well as for the treatment of patients with metastatic and/or
unresectable gastrointestinal stromal tumors (GIST) whose disease has
progressed despite at least imatinib and sunitinib as prior treatments. Fast
Track is a process designed to facilitate the development, and expedite the
review of drugs to treat serious diseases and fill an unmet medical need.
Regorafenib was granted orphan drug designation by the FDA for the treatment of
patients with GIST. Orphan drug designation aims to encourage the development
of drugs involved in the diagnosis, prevention or treatment of a medical
condition affecting fewer than 200,000 people in the country.
Bayer recently entered into an agreement with Onyx Pharmaceuticals, Inc. under
which Onyx will receive a royalty on any future global net sales of regorafenib
About Bayer HealthCare
The Bayer Group is a global enterprise with core competencies in the fields of
health care, nutrition and high-tech materials. Bayer HealthCare, a subgroup of
Bayer AG with annual sales of EUR 16.9 billion (2010), is one of the world's
leading, innovative companies in the healthcare and medical products industry
and is based in Leverkusen, Germany. The company combines the global activities
of the Animal Health, Consumer Care, Medical Care and Pharmaceuticals
divisions. Bayer HealthCare's aim is to discover and manufacture products that
will improve human and animal health worldwide. Bayer HealthCare has a global
workforce of 55,700 employees (Dec 31, 2010) and is represented in more than
100 countries. Find more information at www.bayerhealthcare.com.
Find more information at www.bayerpharma.com.