One pivotal phase 3 trial and many supportive studies form the foundation of the Saforis NDA. The pivotal phase 3 trial of Saforis was completed successfully in 326 patients with breast cancer who were receiving anthracycline- based chemotherapy regimens. The primary endpoint of this trial, defined as a reduction in incidence and severity of oral mucositis, was clearly met. Data indicated that patients receiving Saforis experienced a 22% relative risk reduction of clinically significant oral mucositis compared with placebo. In addition, the incidence of severe oral mucositis was significantly reduced in Saforis patients when compared to placebo. Adverse events observed in patients treated with Saforis were mild in nature and alike to those observed in patients treated with placebo. The most frequently observed adverse events among patients in both the Saforis and placebo arms of this study were vomiting and nausea.

Clinically significant oral mucositis, a most common side effect of mucotoxic cancer therapy, is characterized by painful ulcerations, redness and swelling in the mouth. It is estimated more than 200,000 patients in the US develop significant oral mucositis each year, including up to 40% of patients undergoing standard dose mucotoxic cancer therapy and more than 75% of patients receiving high dose chemotherapy with stem cell transplantation or radiation therapy for head and neck cancer.