SciClone Pharmaceuticals, Inc. (NASDAQ: SCLN) today announced that researchers have identified two unique gene clusters that differentiated subjects who responded to treatment in the Company’s phase 2a proof of concept study of SCV-07 for the prevention of severe oral mucositis (OM; WHO grades 3-4) in patients with advanced head and neck cancer.
The Company believes that the discovery of these gene clusters may assist in providing the framework for effectively identifying those patients most likely to respond to SCV-07 in future clinical trials based on their individual genomic profile or gene signature. These findings were presented today in a poster presentation at the 4th American Association for Cancer Research (AACR) International Conference on Molecular Diagnostics in Cancer Therapeutic Development.
As part of the Company’s recently completed phase 2a OM study, researchers collected and analyzed RNA samples from patients prior to and at the completion of the trial’s treatment phase. Results from this gene expression analysis demonstrated the strong association of two specific gene clusters with patient response to SCV-07. Consistent with SCV-07’s activity as a modulator of the immune system, these clusters included genes associated with G-protein coupled receptors, signal transducers, glycoproteins and membrane proteins.
“The identification of these specific genetic markers represents an exciting and potentially powerful development in the clinical advancement of SCV-07 for the treatment of oral mucositis,” said Dr. Stephen T. Sonis, speaking in his role as Chief Medical Officer of Biomodels, LLC. Dr. Sonis is also a Clinical Professor of Oral Medicine at Harvard University, and a senior surgeon at Brigham and Women’s Hospital and the Dana Farber Cancer Institute. “The previously reported findings from SciClone’s phase 2a study suggested a consistent trend favoring patients in the trial’s high dose group, and we now also have potentially critical insight into why this compound may be more likely to produce a response in a particular subset of patients. These new biomarker data should prove valuable in our ongoing research related to SCV-07 and we plan to further investigate this genomic profile and its connection to potential responders in future studies.”
In addition to the gene expression differences, researchers demonstrated that SCV-07 treatment was associated with significant differences in levels of several immune-related cytokines thought to be important in the development of OM. Samples from patients treated with high dose SCV-07 were analyzed, and it was determined that levels of MIF (p <0.049), MIP-1 beta (p < 0.049) and VEGF (p < 0.015) were found to be significantly higher compared to placebo, whereas levels of IL-1 alpha (p < 0.045) were found to be significantly lower compared to placebo. The patients in the low dose group did not demonstrate these same changes, consistent with the dose effects seen in the clinical study in which the lower dose did not show the same positive response seen in the higher dose treatment arm. The Company believes that the new information regarding cytokine activity may provide additional insight into the fundamental mechanism of action of SCV-07 in the treatment of OM.
“This phase 2a proof of concept study has provided significant clinical data to support the safety and potential efficacy of SCV-07, as well as the increasingly important area of biomarker identification,” commented Friedhelm Blobel, Ph.D., SciClone’s President and Chief Executive Officer. “Importantly, each of these findings will inform our ongoing clinical development of SCV-07 and we look forward to initiating our phase 2b study in patients with OM in late 2010 or early 2011.”
On May 17, 2010, SciClone announced topline results from the Company’s phase 2a proof of concept study of SCV-07 for the prevention of severe OM in patients with advanced head and neck cancer.
Based on the findings from the phase 2a study and completed discussions with the U.S. Food and Drug Administration, SciClone is planning to initiate a phase 2b study in late 2010 or early 2011. As compared to the completed phase 2a trial, the phase 2b study design is expected to include higher doses of SCV-07 and be adequately powered to demonstrate statistical significance. Additionally, researchers will continue to investigate the role of specific genetic profiles on patient response to SCV-07, as well as the potential link between cytokine activity and SCV-07’s sub-cellular mechanism of action.
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