The 12-month analysis from Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT): A phase II, randomized, double-blind study of sonidegib in patients.

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Presentation transcript:

The 12-month analysis from Basal Cell Carcinoma Outcomes with LDE225 Treatment (BOLT): A phase II, randomized, double-blind study of sonidegib in patients with advanced basal cell carcinoma Reinhard Dummer, MD, Alexander Guminski, MD, PhD, Ralf Gutzmer, MD, Luc Dirix, MD, Karl D. Lewis, MD, Patrick Combemale, MD, Robert M. Herd, MD, Martin Kaatz, MD, Carmen Loquai, MD, Alexander J. Stratigos, MD, Hans-Joachim Schulze, MD, Ruth Plummer, MD, Sven Gogov, MD, Celine Pallaud, PhD, Tingting Yi, PhD, Manisha Mone, PhD, Anne Lynn S. Chang, MD, Frank Cornélis, MD, Ragini Kudchadkar, MD, Uwe Trefzer, MD, John T. Lear, MD, Dalila Sellami, MD, Michael R. Migden, MD Journal of the American Academy of Dermatology Volume 75, Issue 1, Pages 113-125.e5 (July 2016) DOI: 10.1016/j.jaad.2016.02.1226 Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 1 A total of 230 patients with advanced basal cell carcinoma (BCC) were randomized to receive once-daily doses of sonidegib 200 mg (n = 79; locally advanced BCC [laBCC], n = 66; metastatic BCC [mBCC], n = 13) or 800 mg (n = 151; laBCC, n = 128; mBCC, n = 23). All randomized patients were assessed for efficacy. One patient in the sonidegib 800-mg arm did not receive treatment and therefore was not included in the safety analysis. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 2 Objective response rates (ORRs) in patients with locally advanced basal cell carcinoma (laBCC) treated with sonidegib 200 and 800 mg. Best overall response in patients with laBCC treated with sonidegib 200 mg (n = 66) and 800 mg (n = 128) assessed by photography, magnetic resonance imaging, and histology according to basal cell carcinoma–modified Response Evaluation Criteria in Solid Tumors by central (A) and investigator (B) review at the time of the primary and 12-month analyses. Complete and partial responses required confirmation on repeated assessments at visits greater than or equal to 4 weeks apart and biopsy specimens were required to confirm a complete response. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 3 Waterfall plots of best change from baseline in the size of target lesions and of duration of response in patients with locally advanced basal cell carcinoma (laBCC) per central review. Best percentage change from baseline in the sum of the products of perpendicular diameters in target lesion(s) assessed by photography per World Health Organization criteria21 per central review (A). Tumor response was assessed by photography, magnetic resonance imaging, and histology according to basal cell carcinoma–modified Response Evaluation Criteria in Solid Tumors in patients with laBCC, and best overall response is depicted by color. Assessments were excluded from the analysis if percentage change in the size of target lesions was not available or was contradicted by an overall lesion response of unknown. Duration of response by patient evaluated by central review in patients responding to treatment with sonidegib 200 mg (B). Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 4 Images of tumor response in patients with locally advanced basal cell carcinoma (laBCC) treated with sonidegib. A, Cheek of a 49-year-old white male patient with aggressive laBCC treated with sonidegib 200 mg at baseline and week 61. This patient achieved an overall partial response by central and investigator review. B, Nose of a 45-year-old white female patient with aggressive laBCC treated with sonidegib 800 mg at baseline and week 30. This patient achieved an overall response of stable disease by central and investigator review. A partial response was reported at the end of treatment on day 221. Left scalp (C) and back (D) of a 78-year-old white female patient with aggressive laBCC treated with sonidegib 800 mg (baseline and week 21). This patient, who remained on treatment for 590 days, achieved an overall partial response per central and investigator review. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 5 Objective response rates (ORRs) in patients with metastatic basal cell carcinoma (mBCC) treated with sonidegib 200 and 800 mg. Best overall response in patients with mBCC treated with sonidegib 200 mg (n = 13) and 800 mg (n = 23) assessed by computed tomography/magnetic resonance imaging per Response Evaluation Criteria in Solid Tumors v1.122 by central (A) and investigator (B) review at the time of the primary and 12-month analyses. Complete and partial responses required confirmation on repeated assessments at visits greater than or equal to 4 weeks apart. Best overall response of 1 patient changed from partial response to stable disease in the 12-month analysis by central re-review because of identification of a new lesion in a photograph received after the cutoff for the primary analysis (June 28, 2013). Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Fig 6 Advanced basal cell carcinoma. Most common adverse events, irrespective of causality, reported in more than 20% of patients overall. Adverse events reported in patients treated with sonidegib 200 mg (n = 79) and 800 mg (n = 150) were assessed according to the National Cancer Institute's Common Terminology Criteria for Adverse Events version 4.03 guidelines23 and include adverse events occurring while on treatment and within 30 days of study drug discontinuation. A patient with multiple occurrences of an adverse event is counted only once in the category for that event, with the maximum severity rating reported. Adverse events are reported by grade. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Supplemental Fig 1 Waterfall plots of best change from baseline in the size of target lesions in patients with locally advanced basal cell carcinoma (laBCC) per central review in the sonidegib 800-mg arm (A) and per investigator review in the sonidegib 200-mg arm (B) and 800-mg arm (C). Best percentage change from baseline in the sum of the products of perpendicular diameters in target lesion(s) assessed by photography per World Health Organization criteria21 in the 800-mg arm (A) per central review and in the 200-mg arm (B) and 800-mg arm (C) per investigator review. Tumor response was assessed by photography, magnetic resonance imaging, and histology according to basal cell carcinoma–modified Response Evaluation Criteria in Solid Tumors in patients with laBCC, and best overall response is depicted by color. Assessments were excluded from the analysis if percentage change in the size of target lesions was not available or was contraindicated by an overall lesion response of unknown. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions

Supplemental Fig 2 Waterfall plots of best change from baseline in the size of target lesions in patients with metastatic basal cell carcinoma (mBCC) per central review in the sonidegib 200-mg arm (A) and 800-mg arm (B) and per investigator review in the sonidegib 200-mg arm (C) and 800-mg arm (D). Best percentage change from baseline in the sum of the longest diameters in target lesion(s) assessed by all modalities used in the evaluations (ie, computed tomography [CT] or magnetic resonance imaging [MRI]) per Response Evaluation Criteria in Solid Tumors v1.122 in the 200-mg arm (A) and 800-mg arm (B) per central review, and in the 200-mg arm (C) and 800-mg arm (D) per investigator review. Tumor response was assessed by CT/MRI in patients with mBCC, and best overall response is depicted by color. Assessments were excluded from the analysis if percentage change in the size of target lesions was not available or was contradicted by an overall lesion response of unknown. Journal of the American Academy of Dermatology 2016 75, 113-125.e5DOI: (10.1016/j.jaad.2016.02.1226) Copyright © 2016 American Academy of Dermatology, Inc. Terms and Conditions