Vismodegib in the treatment of patients with metastatic basal cell carcinoma (mBCC) and distant metastases: Survival in the pivotal phase II and phase I studies.

9012 Background: mBCC is rare and poorly characterized. Recent data (McCusker, in press, Eur J Cancer) indicated that between 1981-2011, the median overall survival (OS) of patients (pts) with mBCC and distant metastases was 2.0 years (95% CI 1–2.9) from time of diagnosis, with a 1-year survival probability of 58.6% (95% CI 44.6–72.6). Vismodegib (VISMO) is a first in-class Hedgehog pathway inhibitor approved for adults with mBCC or locally advanced BCC that is inoperable. Here we report OS in a pooled aBCC population of pts with distant metastases from the pivotal phase II and phase I studies in the context of survival estimates from the literature. Methods: 45 mBCC pts with radiographically measurable distant metastases, present at any site, including soft tissue, other than or in addition to regional lymph nodes, were enrolled in the phase I (NCT00607724; Von Hoff, 2009; n=16) and pivotal phase II (NCT00833417; Sekulic, 2012; n=29) trials. Pts received VISMO 150/270/540 mg/d in the phase I and 150 mg/d...