Novartis LDE225 For Basal Cell Carcinoma Met Primary Endpoint
Novartis this week announced that its trial for investigational oral compound LDE225 (sonidegib) met its primary endpoints. Novartis is studying the compound for treatment of advanced basal cell carcinoma.
The compound was evaluated in the Phase II, randomized, double-blind BOLT (Basal cell carcinoma Outcomes inLDE225 Trial) study. LDE225’s safety and efficacy was evaluated in two oral dose levels of 200 mg and 800 mg with locally advanced or metastatic basal cell carcinoma. “The primary endpoint was the proportion of patients achieving an objective response rate, defined as a confirmed complete response and partial response as their best overall response per modified RECIST criteria, within six months of starting treatment with LDE225. Key secondary endpoints of the study included assessing the duration of tumor response and the rate of complete response,” the company stated in a press release.
LDE225 is an oral, investigational, selective smoothened inhibitor being assessed for treatment of several diseases including myelofibrosis, leukemia and solid tumors. Smoothened (SMO) molecules regulate the hedgehog (Hh) signaling pathway critical in stem cell maintenance and tissue repair. LDE225 is currently under clinical development and has no established safety and efficacy profile as of present.
Alessandro Riva, President, Novartis Oncology ad interim and Global Head, Oncology Development and Medical Affairs, said, “For people living with advanced basal cell carcinoma there are currently limited treatment options. 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.”
Basal cell carcinoma accounts for more than 80% of non- melanoma skin cancers and is the most common form of skin cancer. The disease is highly disfiguring and can turn life-threatening if it progresses without treatment. Basal cell carcinoma rarely metastasizes, but can be associated with morbidity once it does. The incidence of basal cell carcinoma is rising by 10% every year worldwide due to increased ultraviolet exposure and aging populations, among other factors.
Source:
http://www.novartis.com/newsroom/media-releases/en/2014/1762942.shtml