Selpercatinib in <i>RET</i> Fusion–Positive Non–Small Cell Lung Cancer: Final Safety and Efficacy, Including Overall Survival, From the LIBRETTO-001 Phase I/II Trial
Abstract
LIBRETTO-001 (ClinicalTrials.gov identifier: NCT03157128 ) is a registrational phase I/II, single-arm, open-label trial of selpercatinib in RET-dependent cancers. With 19 months of additional follow-up, we report the final efficacy and safety results of selpercatinib in patients with RET fusion–positive non–small cell lung cancer (NSCLC) who had previously received platinum-based chemotherapy (N = 247) or were treatment-naïve (N = 69). The objective response rate (ORR) was 62% for pretreated patients and 83% for treatment-naïve patients. Duration of response (DoR) was 31.6 months for pretreated and 20.3 months for treatment-naïve patients (median follow-up approximately 38 months). Median progression-free survival (PFS) was 26.2 months for pretreated and 22.0 months for treatment-naïve patients (median follow-up approximately 40 months). Median overall survival was 47.6 months in pretreated patients and was not reached in the treatment-naïve group (median follow-up approximately 43 months). At the 3-year landmark estimate, 57% of pretreated and 66% of treatment-naïve patients were alive. Among 26 patients with measurable CNS metastases at baseline, the CNS-ORR was 85% with a CNS-DoR of 9.4 months and CNS-PFS of 11.0 months. The safety profile of selpercatinib was consistent with previous reports. With substantial additional follow-up, selpercatinib continued to show durable responses and intracranial activity, with a manageable safety profile in patients with RET fusion–positive NSCLC.
Article Details
Journal Info
Journal of Clinical Oncology
Lippincott Williams & Wilkins
Authors (13)
Oliver Gautschi
University of Berne and Medical Oncology, Cantonal Hospital of Lucerne, Lucerne, Switzerland
Keunchil Park
Benjamin J. Solomon
Peter MacCallum Cancer Centre, Melbourne, VIC, Australia
Pascale Tomasini
Aix Marseille University – CNRS, INSERM, CRCM; CEPCM – AP-HM Hôpital de la Timone, Marseille, France
Herbert H. Loong
Fahima Dossa, MD, PhD, Department of Surgery, Cedars-Sinai Medical Center, Los Angeles, CA; Chandrajit P. Raut, MD, Department of Surgery, Mass General Brigham, Harvard Medical School, Boston, MA; Andrew J. Wagner, MD, PhD, Department of Medical Oncology, Dana-Farber Cancer Institute, Boston, MA; Robin L. Jones, MD, Sarcoma Unit, The Royal Marsden NHS Foundation Trust and Institute of Cancer Research, London, United Kingdom; Rebecca A. Gladdy, MD, PhD, Department of Surgical Oncology, Mount Sinai Hospital and Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada; Abha A. Gupta, MD, Division of Medical Oncology, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada; Kenneth Cardona, MD, Division of Surgical Oncology, Department of Surgery, Winship Cancer Institute, Emory University, Atlanta, GA; David E. Gyorki, MD, Division of Cancer Surgery, Peter MacCallum Cancer Centre, and Sir Peter MacCallum Department of Oncology, University of Melbourne, Melbourne, VIC, Au...
Filippo de Braud
Koichi Goto
Patrick Peterson
Lilly, Indianapolis
Scott Barker
Eli Lilly and Company, Indianapolis, IN
Katherine Liming
Eli Lilly and Company, Indianapolis, IN
Geoffrey R. Oxnard
Bente Frimodt-Moller
Lilly, Copenhagen
Alexander Drilon
Department of Medicine, Memorial Sloan Kettering Cancer Center