Abstract 5994
Background
For patients with advanced basal cell carcinoma (BCC), including those with locally advanced BCC (laBCC) and metastatic BCC (mBCC), hedgehog pathway inhibitors (HPIs) are a treatment option. Sonidegib is an HPI approved for use in patients with advanced BCC (Switzerland and Australia) and laBCC (USA and EU), based on data from the BOLT study (NCT01327053). Here we report the duration of response (DOR) at 42 months and quality of life (QoL) results at the 12- and 30-month analyses from BOLT.
Methods
BOLT was a multicenter, randomized, double-blind, phase 2 trial that evaluated sonidegib in treatment-naïve patients with mBCC (n = 36) or laBCC (n = 194) who were not amenable to curative surgery or radiation therapy. Patients were randomized 1:2 to either 200 mg or 800 mg PO QD, and data analyses were performed at 6, 12, 18, 30, and 42 months. The primary endpoint was objective response rate (ORR) and a key secondary endpoint included DOR as assessed by central review. QoL was assessed using the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30) and the associated Head and Neck Cancer Module 35 (EORTC H&N35).
Results
At the 42-month analysis, the median DOR was 26.1 months in laBCC patients treated with sonidegib 200 mg vs 23.3 months in patients treated with 800 mg. The ORR was higher in the 200 mg group vs the 800 mg group (Table). In mBCC patients, the median DOR was 24 months for sonidegib 200 mg and not estimable for the 800 mg group. The ORR was lower in the 200 mg group vs the 800 mg group (Table). During the course of treatment, the QoL for these patients was analyzed, and the majority of patients in both dosage groups had maintenance of or improvement in QoL relative to baseline.Table: 1295P
ORR in sonidegib-treated patients with advanced BCC
| laBCC | mBCC | |||
|---|---|---|---|---|
| 200 mg | 800 mg | 200 mg | 800 mg | |
| n = 66 | n = 128 | n = 13 | n = 23 | |
| ORR % | 56 | 46 | 8 | 17 |
| Complete Response, n (%) | 3 (4.5) | 2 (1.6) | 0 | 0 |
| Partial Response, n (%) | 34 (51.5) | 57 (44.5) | 1 (7.7) | 4 (17.4) |
Conclusions
Data from the 42-months analysis demonstrated that patients receiving sonidegib had a long duration of response. Results from the 12- and 30-month analyses show maintenance and improvement in QoL consistent with that of the primary data observed in BOLT.
Clinical trial identification
NCT01327053 CLDE225A2201.
Legal entity responsible for the study
Novartis Pharmaceuticals Corporation.
Funding
Novartis Pharmaceuticals Corporation.
Editorial Acknowledgement
Medical writing support provided by Adriana Vela, PhD, Medical Exchange International, LLC, New York, NY.
Disclosure
M.R. Migden: Honorarium for advisory boards and consulting: Regeneron, Sun Pharmaceuticals, Novartis.