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Gem Pharmaceuticals Presents Promising Phase 2 Data for GPX-150 in Sarcoma Patients at the 2016 ASCO Annual Meeting
Clear evidence of pharmacologic activity and an attractive safety profile

BIRMINGHAM, Ala., June 7, 2016 /PRNewswire/ -- Gem Pharmaceuticals announced preliminary Phase 2 results of its lead drug candidate, GPX-150, in sarcoma patients this week at the annual meeting of the American Society of Clinical Oncology (ASCO).  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. 

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The ongoing, open-label safety and efficacy study enrolled a total of 22 sarcoma patients in the front-line setting.  Disease stabilization or partial response was seen in 9 patients (41%), and participating clinical investigators considered overall tolerability to be better than would typically be expected for treatment using doxorubicin.  Importantly, there has been no statistical difference in the heart function of the patients (as measured by left ventricular ejection fraction) between baseline and final measurement; zero cases of irreversible cardiotoxicity have been seen to date.  Because of the excellent safety profile, GPX-150 may be able to be administered without regard to cumulative dose.  Three patients still remain on drug, with one such subject having continued dosing for more than a year. 

"Based on the preclinical and human data generated thus far, GPX-150 holds the potential to be the anthracycline that we have long sought that is devoid of chronic cardiotoxicity," said Brian Van Tine, M.D., Ph.D., a sarcoma specialist at the Washington University School of Medicine in St. Louis and a clinical investigator in the trial.  "Anthracyclines in general and doxorubicin in particular remain a workhorse in our chemotherapeutic regimen – and widespread use will continue despite, and potentially in combination with, today's emerging immuno-oncology regimens."

The poster, entitled "A phase 2 trial of 5-imino-13-deoxydoxorubicin (GPX-150) in metastatic and non-resectable soft tissue sarcomas," will be posted on Gem's website; in addition, these data were featured at an oral Sarcoma Discussion Session during the ASCO meeting.

Soft tissue sarcoma (STS) 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 received Orphan Drug Designation for the use of GPX-150 in the treatment of this indication from the U.S. Food and Drug Administration in December 2015.

About Gem Pharmaceuticals 
Gem Pharmaceuticals is a clinical-stage biopharmaceutical company developing proprietary anthracycline derivatives specifically designed to eliminate the critical irreversible cardiotoxicity side effect of this powerful class of chemotherapeutics while maintaining their well-documented anti-cancer efficacy.  In so doing, Gem seeks to transform traditional broad-spectrum cytotoxic drugs into modern – and biochemically targeted – anti-cancer agents that hold the potential for higher and/or longer-term dosing and correspondingly improved therapeutic utility.  Gem believes that its lead non-cardiotoxic anthracycline compound (GPX-150) has the potential to attain significant market penetration by replacing the existing use of doxorubicin and other anthracyclines; by expanding the oncology uses of anthracyclines; and by broadening anthracycline use beyond cancer.  For additional information about Gem, please visit  More information about the Phase 2 clinical trial of GPX-150 in soft tissue sarcoma patients can be found at

Arthur Klausner, CEO 


SOURCE Gem Pharmaceuticals