SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) today announced the enrollment of the first patient in the company's phase 2b clinical trial of SCV-07 for the prevention of oral mucositis (OM) -- a painful, debilitating, and costly toxicity caused by chemoradiotherapy regimens used to treat head and neck cancer.
The study will examine three doses of SCV-07, including two higher doses than those used in the company's recent phase 2a study, to assess the drug's impact on modifying the course of OM in patients with head and neck cancer. In addition, the trial will further evaluate SCV-07's safety and tolerability in this patient population, as well as the role played by specific genetic profiles on patient response to the treatment.
"We are very pleased with how quickly we have been able to advance the SCV-07 OM program from completion of our phase 2a trial to the initiation of this important phase 2b study. This is particularly impressive considering the fact that our scientific team uniquely designed this study protocol to leverage those findings which emerged from our phase 2a trial, including important information related to dosing and efficacy endpoints," stated Friedhelm Blobel, Ph.D., SciClone's President and Chief Executive Officer. "We expect that this upcoming study will provide additional information regarding the potential therapeutic role of SCV-07 in the treatment of OM and, in turn, inform SciClone on the best path for designing a possible pivotal phase 3 trial."
The multicenter, randomized, double-blind, placebo-controlled study will enroll approximately 160 subjects who are receiving standard chemoradiation therapy for treatment of cancers of the head and neck. Subjects will be randomly assigned to one of the trial's four treatment arms: placebo and SCV-07 at doses or 0.1 mg/kg, 0.3 mg/kg and 1 mg/kg. The study's primary efficacy endpoint is the reduction in proportion of subjects with clinically assessed ulcerative OM (WHO Grade greater than or equal to 2) at the time that they have received a cumulative radiation dose of 45 Gy.
The study's secondary endpoints include, among others, incidence and duration of ulcerative and severe (WHO Grade greater than or equal to 3) OM; analgesic use and pain assessments, breaks in radiation or chemotherapy treatment, and unscheduled office or hospital visits.
OM is a common, painful, debilitating complication of cancer treatment, and SciClone estimates that total medical costs may reach around $4.2 billion in the U.S. and $10 billion worldwide in 2010. OM is a condition in which the sensitive cells lining the mouth and throat are damaged by cancer treatments such as chemotherapy (with or without radiation) and become painful mouth sores. Severe OM has been reported to occur in about 50% of patients who receive chemoradiation for the prevention of cancers of head and neck (Sonis, Core Evidence, 2009). Importantly, radiation to the head and neck, especially when it includes the tissues of the mouth, pharynx and hypopharynx, results in significant ulcerative OM in greater than 90% of patients (Manas et al, Clinical Translational Oncology, 2009) and can compromise the patient's ability or willingness to accept a complete course of therapy. Symptoms can include painful ulcers in the mouth and throat, redness and swelling of the gums, dryness and overall soreness in the mouth, and difficulty eating, swallowing, talking and drinking. In addition to the symptoms of OM and its impact on quality of life and continued therapy, mucositis can cause adverse effects on a variety of other health and economic outcomes, such as a risk of serious infection, the need for parenteral nutrition and narcotic analgesia, and increased hospitalization and feeding-tube placement. The National Cancer Institute estimates that 450,000 patients per year in the U.S. suffer from OM during cancer therapy.
About SCV-07
SCV-07 (gamma-D-glutamyl-L-tryptophan) is a small molecule which appears to stimulate the immune system through inhibition of STAT3 signaling and the resulting effects on T-helper 1 cells. SCV-07 has been shown to be efficacious in animal models of immune-sensitive diseases, including prevention of oral mucositis, treatment of cancer and viral infections, and enhancement of response to vaccines.
SCV-07 is protected by composition of matter patents as well as multiple method of treatment patents. SciClone has exclusive worldwide rights to SCV-07 outside of Russia, where the molecule has been approved for stimulation of depressed immune systems.
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