Gem Pharmaceuticals announced today that the initial soft tissue sarcoma (STS) patients have been enrolled into the Company's Phase 2 clinical trial. This open-label study will assess the efficacy and safety of Gem's lead compound, GPX-150 (an investigational medication), in approximately 30 adults as first-line therapy for advanced or metastatic disease.
GPX-150 is a proprietary analog of the widely used chemotherapeutic doxorubicin, where the doxorubicin molecule has been modified at two locations in an effort to decrease the cardiotoxic side effect that limits the maximum cumulative dose of doxorubicin that can be safely administered. In the trial, GPX-150 will be infused every three weeks for up to 48 weeks, which is twice as long as the standard 24-week limit for doxorubicin administration. Gem is exploring the likelihood that this longer duration of treatment with GPX-150 will produce an improved therapeutic effect when compared to that seen historically with doxorubicin treatment. The study's primary endpoint is progression-free rate at 12 months.
Soft tissue sarcoma includes a group of malignancies that affect tissues such as fat, muscles, nerves, and tendons. With approximately 12,000 new cases of STS occurring in the U.S. annually, Gem is planning to apply to the U.S. Food and Drug Administration for Orphan Drug Designation for the use of GPX-150 in the treatment of this indication.
"I was impressed by the degree of disease stabilization shown by GPX-150 in several late-stage sarcoma patients during the Phase 1 safety trial at our facility, so I'm indeed excited to be participating in this Phase 2 study," said Mohammed Milhem, M.D., Deputy Director for the Holden Comprehensive Cancer Center at the University of Iowa in Iowa City. The additional clinical sites for the study are the Robert H. Lurie Comprehensive Cancer Center of Northwestern University in Chicago, Washington University School of Medicine in St. Louis, and Penn State Hershey Cancer Institute.
SOURCE Gem Pharmaceuticals