Novartis investigational compound LDE225 met primary endpoint in pivotal trial for patients with advanced basal cell carcinoma

Novartis announced today that the pivotal trial of the investigational oral compound LDE225 (sonidegib) in advanced basal cell carcinoma met its primary endpoint of demonstrating an objective response rate among patients within six months of treatment. Objective response included complete response (clinically significant tumor response with complete absence of disease) and partial response (clinically significant tumor shrinkage)^4,5,6.

Basal cell carcinoma is the most common form of skin cancer, accounting for more than 80% of non-melanoma skin cancers, and can be highly disfiguring and life-threatening if it grows^1,2,3. Worldwide incidence of basal cell carcinoma is rising by 10% each year due to factors such as an aging population and increased ultraviolet exposure⁷. Although basal cell carcinoma rarely metastasizes, once it does, it can be associated with significant morbidity⁸.

“For people living with advanced basal cell carcinoma there are currently limited treatment options,” said Alessandro Riva, President, Novartis Oncology ad interim and Global Head, Oncology Development and Medical Affairs. “These results demonstrate the potential for LDE225 to offer a treatment option for this patient population, and we look forward to sharing these data with regulatory authorities worldwide.”

Full study results will be presented at a future scientific meeting.

References

1.   Skin Cancer Foundation. “Basal Cell Carcinoma.”Available at: http://www.skincancer.org/skin-cancer-information/basal-cell-carcinoma. Accessed on February 14, 2014.
2. Skin Cancer Foundation. “Skin Cancer Facts.” Available at: http://www.skincancer.org/skin-cancer-information/skin-cancer-facts. Accessed on February 14, 2014.
3. Rubin AI, Chen EH, Ratner D (2005). Current Concepts: Basal-Cell Carcinoma. N Engl J Med; 353:2262-9.
4. ClinicalTrials.gov. “A Phase II Study of Efficacy and Safety in Patients With Locally Advanced or Metastatic Basal Cell Carcinoma (BOLT)” Available at: http://clinicaltrials.gov/ct2/show/NCT01327053?term=%22LDE225%22+and+%22BOLT%22&rank=1. Accessed on February 14, 2014.
5. National Cancer Institute Dictionary of Cancer Terms. “Complete Response.” Available at: http://www.cancer.gov/dictionary?CdrID=45652. Accessed on February 14, 2014.
6. National Cancer Institute Dictionary of Cancer Terms. “Partial Response.” Available at: http://www.cancer.gov/dictionary?CdrID=45819. Accessed on February 14, 2014.
7. Wong C S M, Strange R C, Lear J T (2003). Basal cell carcinoma. BMJ; 327:794-798.
8. Copcu E, Aktas A. Simultaneous two organ metastases of the giant basal cell carcinoma of the skin. Int Semin Surg Oncol. 2005;2:1-6. Available at: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC544837/. Accessed on February 14, 2014.