While most cases of BCC can be managed by surgery, some progress to advanced stage where surgery is inappropriate. Vismodegib, first-in-class Hedgehog-pathway inhibitor, has demonstrated efficacy in advanced BCC. STEVIE is a safety study of vismodegib in advanced BCC. Patients received oral vismodegib 150mg, once-daily until progressive disease, unacceptable toxicity or withdrawal. Safety assessed by Common Terminology Criteria for Adverse Events (AEs) v4.0. Overall response rate assessed according to Response Evaluation Criteria in Solid Tumours, (v1.1). Recruitment is ongoing. This analysis (cutoff-17 May 2012) included 150 patients with locally advanced (n=138) or metastatic (n=12) BCC with potential for ≥3-month follow-up. Locally advanced patients had lesions considered inoperable-54.3% or surgery contraindicated-45.7%. Median treatment duration-144 days. Most common treatment emergent AEs (TEAEs; ≥20% of patients) included muscle spasms-53.3%, alopecia-42.7%, dysgeusia-36.0%, ageusia-27.3%, and asthenia-26.7%. Most TEAEs were mild/moderate severity. Serious TEAEs occurred in 22 patients (14.7%). Patients discontinued treatment (25.3%) due to AEs (n=10). Deaths were due to disease progression (n=2) or AEs considered by investigators unrelated to study drug. Initial preliminary best overall response was confirmed for 124 patients; 19.4%-complete responders, 55.6%-partial responders, 21.8%-stable disease and 3.2%-progressive disease. This interim analysis from STEVIE confirms a similar vismodegib safety profile to ERIVANCE BCC.
Acknowledgment: This study was funded by Roche-Genentech. Support for medical writing assistance was provided by F. Hoffmann-La Roche.