Novartis releases positive results of Phase II trial of BCC drug sonidegib

The trial met its primary endpoint of showing an objective response rate among patients within six months of treatment.

The objective response comprised of complete response (clinically significant tumor response with complete absence of disease) and partial response (clinically significant tumor shrinkage).

BCC is the most common form of skin cancer, accounting for over 80% of non-melanoma skin cancers, and can be highly disfiguring and life-threatening if it grows.

It is claimed that BCC rarely metastasizes or kills and once it does, it can be associated with significant morbidity.

Novartis president, ad interim & global head, Oncology Development and Medical Affairs Alessandro Riva 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," Riva said.

The randomized, double-blind BOLT (Basal cell carcinoma Outcomes in LDE225 Trial) Phase II trial was designed to evaluate the safety and efficacy of two oral dose levels of LDE225 (200mg and 800mg) in patients with locally advanced or metastatic BCC, which are subtypes of advanced basal cell carcinoma.

Primary endpoint of the trial 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.

Major secondary endpoints included evaluating the duration of tumor responseand the rate of complete response, while other secondary endpoints included progression-free survival, time to tumor response and overall survival.

Image: Novartis AG headquarters in Basel. Photo: courtesy of Andrew