BIRMINGHAM, Ala., Jan. 6, 2016 /PRNewswire/ — Gem Pharmaceuticals announced today that the U.S. Food and Drug Administration has granted orphan drug designation to GPX-150, the Company’s lead product candidate that is currently undergoing a Phase 2 clinical trial in patients suffering from soft tissue sarcoma. This study is fully enrolled, with final safety and efficacy data expected to be available by Q-4 2016.
Orphan drug designation is granted for novel drugs to treat rare medical diseases or conditions that affect less than 200,000 people in the United States. The designation qualifies the sponsor for various incentives, including seven years of market exclusivity after the drug’s approval, tax credits for clinical research costs, and reductions in FDA application fees.
“This orphan drug designation represents an important regulatory advance as we further our development of GPX-150, which has demonstrated intriguing pharmacological activity in sarcoma patients in our ongoing, open-label, Phase 2 clinical study,” said Arthur Klausner, CEO of Gem Pharmaceuticals.
Soft tissue sarcoma (STS) includes a group of malignancies that affect tissues such as fat, muscles, nerves, and tendons. According to the American Cancer Society, approximately 12,000 new cases of STS occur in the U.S. annually, with about 5,000 deaths.
About Gem Pharmaceuticals
Gem Pharmaceuticals is a clinical-stage biopharmaceutical company developing proprietary anthracycline derivatives specifically designed to eliminate the critical 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 visitwww.gempharmaceuticals.com. More information about the Phase 2 clinical trial of GPX-150 in soft tissue sarcoma patients can be found at www.clinicaltrials.gov.