WAYNE, N.J., Feb. 4, 2011 /PRNewswire/ — Bayer HealthCare
Pharmaceuticals Inc. announced today that its investigational
compound regorafenib (BAY 73-4506) has been granted orphan drug
status for the treatment of patients with gastrointestinal stromal
tumors (GIST) by the U.S. Food and Drug Administration (FDA).

Regorafenib is an investigational agent and is not approved by
the FDA, the European Medicines Agency (EMA) or other Health
Authorities.

“This is an important step in the overall development process
for this investigational compound,” said Kemal Malik, MD, Head of
Global Development and member of the Bayer HealthCare Executive
Committee.

In January, Bayer began enrolling patients in a randomized,
double-blind, placebo-controlled Phase III study of regorafenib
plus best supportive care versus placebo plus best supportive care
for subjects with metastatic and/or unresectable gastrointestinal
stromal tumors (GIST) whose disease has progressed despite prior
treatment with at least imatinib and sunitinib (NCT01271712). The
trial is estimated to enroll 170 patients, who will be randomized
in a 2:1 ratio to receive either regorafenib or placebo. Subjects
receiving placebo who experience disease progression may be offered
open-label regorafenib treatment (cross-over option).

The primary endpoint of this trial is progression-free survival
(PFS), and secondary endpoints include overall survival (OS), time
to progression (TTP), disease control rate (DCR), tumor response
rate (RR), duration of response (DOR), and safety. All patients
will enter the Survival Follow-Up Period upon discontinuation of
study treatment, during which assessment of survival status will be
performed every three months.

For information about this study, please visit www.clinicaltrials.gov
(NCT01271712).

Orphan Drug Designation in the United States

In the United States, the Orphan Drugs Act (OD

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