Phase III data at ASCO shows Saforis(TM) helps prevent and treat oral mucositis in cancer patients
Aesgen, Inc. to use these results to complete and submit NDA for Saforis
New Orleans, June 6, 2004 – Aesgen, Inc., a privately-held emerging pharmaceutical company, today announced results from its pivotal Phase III trial that show SaforisTM (L-glutamine) for oral suspension significantly reduced the severity and duration of clinically significant oral mucositis in breast cancer patients receiving standard chemotherapy treatments. These data reinforce earlier study results that show Saforis reduces the severity and duration of oral mucositis and the need for narcotic pain control in a range of cancer patients undergoing chemotherapy or radiation therapy. The study was presented at the 40th Annual Meeting of the American Society of Clinical Oncology (ASCO) by the study's lead investigator, Douglas E. Peterson, DMD, PhD, Professor and Head, Department of Oral Diagnosis, School of Dental Medicine, University of Connecticut Health Center.
Characterized by painful ulcerations, redness, and swelling in the mouth, oral mucositis is one of the most common serious side effects of chemotherapy and radiation therapy, afflicting more than 400,000 adult and pediatric cancer patients in the U.S. every year. However, unlike most other major dose-limiting toxicities of chemotherapy (e.g., neutropenia and anemia), there is no currently approved therapy to prevent or treat oral mucositis. As a result, it remains one of the most debilitating, painful, and costly side effects associated with cancer therapy.
"While moderate and severe cases can threaten the very survival of patients by increasing the risk of serious infections and delaying or reducing cancer treatments, even milder cases can significantly impact quality of life," said Dr. Peterson, co-author of the recently published Mucositis Clinical Practice Guidelines developed by the Multinational Association of Supportive Care in Cancer and the International Society for Oral Oncology. "Despite the devastating toll, an effective and consistent approach to managing oral mucositis has eluded the oncology community to date. Now, with the first clinical practice guidelines recently released, and breakthrough treatments like Saforis being shown effective in rigorous scientific studies, we are confident supportive care for patients with mucositis will soon dramatically improve."
Study Confirms Prevention and Treatment Potential of Saforis
The randomized, double-blind, placebo-controlled, multi-center trial evaluated 2,084 patients receiving at least three cycles of an anthracycline-based chemotherapy regimen. Those patients who experienced clinically significant oral mucositis (WHO Grade 2-4) in a screening cycle (n=326) were randomized to receive either Saforis or placebo in treatment Cycle one and then switched study drug (Saforis patients switched to placebo, placebo patients switched to Saforis) in treatment Cycle two. Saforis reduced the incidence of moderate to severe/life-threatening oral mucositis by 22 percent in treatment cycle one (p=0.0261).
Treatment Cycle two could not be compared to cycle one because of a significant carryover benefit observed in patients treated with Saforis first. The carryover benefit caused patients that crossed over from Saforis to placebo to have 36 percent lower-than-expected incidence of oral mucositis (p =0.0269). This carryover benefit suggests Saforis may protect patients from oral mucositis during subsequent cycles of chemotherapy
The study also demonstrated that Saforis has an excellent safety profile. Side effects associated with Saforis treatment were comparable to, if not slightly better than, placebo.
"This robust trial confirms the efficacy and safety we have seen during the development of Saforis, and it is the final piece we need in order to complete and submit the new drug application to the U.S. Food & Drug Administration under our 'fast track' designation," said Edward Shinal, PhD, President and COO, Aesgen, Inc. "The positive results have also intensified our discussions with potential pharmaceutical marketing partners, increasing our confidence that we will be able to quickly and successfully make Saforis available to patients and their healthcare providers.
Saforis is a patented, topical, oral suspension that utilizes Aesgen's proprietary drug delivery system, UpTecTM, to rapidly deliver over 100 times more L-glutamine directly into chemotherapy or radiotherapy damaged cells of the oral mucosa as compared to conventional L-glutamine. L-glutamine, the active ingredient in Saforis, is the most abundant amino acid in human plasma and has multiple beneficial functions in the human body, including providing energy, nitrogen, and regulation of protein synthesis. The availability of and demand for L-glutamine becomes critical when mucosa has been injured from chemotherapy and radiation. Increased levels of L-glutamine in mucosal tissues prevent damage from cancer treatments and help tissues recover normal function. Until UpTec, there has been no clinically effective way to deliver adequate amounts of L-glutamine to the tissues to prevent mucositis. To date, Saforis has been evaluated in pediatric and adult patients with solid tumors, lymphoma, and hematologic malignancies, who are undergoing chemotherapy, radiation, and/or autologous bone marrow transplantation. Due to the high unmet medical need for effective treatments for oral mucositis and the positive clinical profile, Saforis received Fast Track Designation from the FDA in January of 2003.
Source: Eurekalert & othersLast reviewed: By John M. Grohol, Psy.D. on 21 Feb 2009
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