Randomized, Double-Blind Study of Sonidegib (Lde225) in Patients (Pts) with Advanced Basal Cell Carcinoma (Bcc)

ABSTRACT Aim: The BOLT phase 2 study, comparing 2 doses of sonidegib, a hedgehog pathway inhibitor (HhPI), in pts with advanced BCC (aBCC; NCT01327053), met its primary endpoint of objective response rate ≥30% in both arms in analyses of data collected up to 6 mo after randomization of the last pt (June 28, 2013, cutoff; median follow-up [f/u], 13.9 mo; Migden, ASCO 2014). Associations of GLI1 (marker of Hh pathway activation) with clinical outcome (as of June 28, 2013) and updated 12-mo efficacy and safety data (Dec 31, 2013, cutoff; median f/u, 20.0 mo) are presented. Methods: Pts with locally advanced BCC (LaBCC; n = 194) not amenable to curative surgery or radiation or metastatic BCC (m…