Our Mission

Gem Pharmaceuticals, LLC is a biopharmaceutical company focused on the development and commercialization of novel anthracyclines for the treatment of cancer, inflammation, and infectious diseases. The Company was founded in 2003 to exploit discoveries identifying the cause of the lethal cardiotoxicity (heart failure) of major commercial anti-cancer agents called anthracyclines. Gem developed novel chemical synthesis procedures for 5-imino-13-deoxy anthracyclines. 5-imino-13-deoxyanthracyclines cannot be converted into cardiotoxic alcohol metabolites in the heart, or generate toxic reactions through a quinone group. Gem was granted U.S. and foreign patents on these chemical synthesis procedures and on novel 5-imino-13-deoxyanthracycline compounds.

Doxorubicin is a popular and effective anthracycline anticancer drug, but can produce cardiotoxic effects that limit its usefulness and safety. The quinone group in the doxorubicin molecule appears to produce an acute reversible cardiotoxicity, while the alcohol metabolite of doxorubicin (13-hydroxy doxorubicin) appears to produce irreversible dose limiting cardiotoxicity. Gem’s first compound, 13-deoxydoxorubicin, did not form an alcohol metabolite and did not produce long-term irreversible cardiotoxicity in animal models. In a phase II clinical trial in patients with metastatic breast cancer, 13-deoxydoxorubicin showed good anti-cancer activity, indicating that the 13-keto group in doxorubicin is not essential for therapeutic activity. 13-deoxyanthracyclines are expected to retain anti-cancer activity similar to their parent 13-keto compounds.

13-deoxydoxorubicin did show evidence of acute cardiac effects in some patients, as measured by a reduction in ejection fraction. These acute cardiac effects were consistent with the presence of an intact quinone in 13-deoxydoxorubicin. It appears that all anthracyclines that have an intact quinone group will produce some degree of acute cardiotoxicity. All anthracyclines on the market today have an intact quinone group.

From these studies Gem has developed a 5-imino-13-deoxyanthracycline that does not contain a quinone group: 5-imino-13-deoxydoxorubicin (GPX-150). GPX-150 does not appear to have any acute or chronic cardiotoxic effects in pre-clinical studies. GPX-150 also shows good anti-cancer activity in animal models. GPX-150 is presently in a Phase I clinical trial in terminally ill cancer patients to document its excellent safety profile and to determine its maximum tolerated dose. Results to date show that GPX-150 has anticancer activity with little or no significant myelosuppression and no evidence of cardiotoxicity.

Gem has recently obtained patent protection on the combination of GPX-150 and paclitaxel for use in the treatment of cancer. GPX-150 produces a remarkable potentiation of the anticancer effects of paclitaxel.

GPX-150 has further shown potent T-Cell growth inhibition and in-vivo anti-inflammatory effects. Because of the good safety profile of GPX-150, Gem plans to evaluate this compound for topical efficacy and safety in a Phase I trial in psoriasis patients.

Gem’s mission includes developing new 5-imino-13-deoxyanthracyclines to replace all existing anthracyclines for the treatment of cancers. In addition, Gem will develop these drugs for treating inflammatory diseases (rheumatoid arthritis, multiple sclerosis, psoriasis, transplant rejection, etc.) and infectious diseases, and will produce analogs with much greater potency and less susceptibility to drug resistance. Gem will further develop oral formulations of GPX-150.