Primary Analysis of Phase 2 Results for Cemiplimab in Patients with mBCC Who Progressed on or Were Intolerant to Hedgehog Inhibitors

November 2022 Vol 13, No 11 —November 17, 2022

Karl D. Lewis, MD1; Ketty Peris, MD2,3; Aleksandar Sekulic, MD4; Alexander J. Stratigos, PhD5; Lara Dunn, MD6; Zeynep Eroglu, MD7; Anne Lynn S. Chang, MD8; Michael R. Migden, MD9; Suk-Young Yoo, PhD10; Kosalai Mohan, PhD10; Ebony Coates, MHA, MSJ10; Emmanuel Okoye, MBBS10; Jean-François Baurain, PhD11; Oliver Bechter, MD12; Axel Hauschild, MD13; Marcus O. Butler, MD14; Leonel Hernandez-Aya, MD15; Lisa Licitra, MD16,17; Rogerio I. Neves, MD, PhD18; Emily S. Ruiz, MD, PhD19; Frank Seebach, MD10; Israel Lowy, MD, PhD10; Priscila Goncalves, MD10; Matthew G. Fury, MD10

1Department of Medicine-Medical Oncology, University of Colorado School of Medicine, Aurora, CO, USA; 2Department of Medicine and Translational Surgery, Dermatology, Università Cattolica del Sacro Cuore, Rome, Italy; 3Department of Medical and Surgical Sciences, Dermatology, Fondazione Policlinico Universitario A Gemelli-IRCCS, Rome, Italy; 4Department of Dermatology, Mayo Clinic, Scottsdale, AZ, USA; 5First Department of Dermatology-Venereology, National and Kapodistrian University of Athens, School of Medicine, Andreas Sygros Hospital, Athens, Greece; 6Department of Medicine, Head and Neck Medical Oncology, Memorial Sloan Kettering Cancer Center, New York, NY, USA; 7Department of Cutaneous Oncology, Moffitt Cancer Center, Tampa, FL, USA; 8Dermatology Department, Stanford University School of Medicine, Redwood City, CA, USA; 9Departments of Dermatology and Head and Neck Surgery, The University of Texas MD Anderson Cancer Center, Houston, TX, USA; 10Regeneron Pharmaceuticals, Inc., Tarrytown, NY, USA; 11Department of Medical Oncology, Cliniques Universitaires Saint-Luc and Université Catholique de Louvain, Brussels, Belgium; 12Department of General Medical Oncology, University Hospitals, Leuven, Belgium; 13Department of Dermatology, University Hospital Schleswig-Holstein (UKSH), Kiel, Germany; 14Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University of Toronto, Toronto, Ontario, Canada; 15Division of Medical Oncology, Department of Medicine, Washington University School of Medicine, St Louis, MO, USA; 16Department of Medical Oncology Head and Neck Cancer, Istituto Nazionale dei Tumori, Milan, Italy; 17Department of Oncology and Hemato-Oncology, University of Milan, Milan, Italy; 18Division of Plastic Surgery, Penn State Milton S. Hershey Medical Center, Hershey, PA, USA; 19Department of Dermatology, Brigham and Women’s Hospital, Harvard Medical School, Boston, MA, USA

Background: Cemiplimab is the first treatment approved in the United States (as cemiplimab-rwlc) for patients with metastatic basal-cell carcinoma (mBCC) or locally advanced disease (laBCC) after hedgehog inhibitor (HHI) treatment or for whom an HHI is not appropriate. Cemiplimab provided substantial clinical benefit and an acceptable safety profile in patients with laBCC who discontinued HHI therapy due to progressive disease (PD), intolerance, or no better than stable disease (SD) after 9 months (NCT03132636).

Objective: Here, we present the primary analysis of the mBCC cohort.

Methods: Patients with mBCC (nodal or distant) post-HHI treatment received cemiplimab 350 mg intravenously every 3 weeks for up to 93 weeks or until PD. The primary end point was objective response rate (ORR) by independent central review (ICR). Key secondary end points included safety and tolerability, ORR per investigator (INV) assessment, duration of response (DOR), progression-free survival (PFS), overall survival (OS), and complete response (CR) rate (data cutoff date: May 20, 2021).

Results: Fifty-four patients were enrolled (70.4% male; median age 63.5 years [range, 38-90]; Eastern Cooperative Oncology Group performance status 0 [66.7%] and 1 [33.3%]). Median duration of follow-up was 8.4 months (range, 1.5-36.2). ORR per ICR was 24.1% (95% CI, 13.5-37.6), with 1 CR and 12 partial responses (PRs). ORR per INV was 25.9% (95% CI, 15.0-39.7), with 2 CRs and 12 PRs. Among responders, median time to response per ICR was 4.0 months (range, 2.0-10.5). Estimated median DOR per ICR was 16.7 months (95% CI, 9.8-not evaluable). Disease control rate was 63.0% (95% CI, 48.7-75.7) per ICR and 70.4% (95% CI, 56.4-82.0) per INV. Median OS was not reached. Median PFS per ICR was 8.3 months (95% CI, 4.2-15.9). The most common treatment-related adverse events were fatigue (37.0%), diarrhea (14.8%), pruritus (13.0%), hyperthyroidism (9.3%), and arthralgia (9.3%), as well as hypothyroidism, asthenia, constipation, and maculopapular rash (7.4% each). There were no treatment-related deaths.

Conclusions: Until the current study, patients with mBCC who had progressed on or showed intolerance to first-line HHI therapy had no standard second-line options. Cemiplimab provided clinically meaningful antitumor activity, including durable responses, and an acceptable safety profile in this patient population. These results complement those of the laBCC cohort and suggest that patients with mBCC may benefit from treatment with cemiplimab following first-line HHI therapy or for whom HHI therapy is not appropriate.

Funding: Regeneron Pharmaceuticals, Inc. and Sanofi. Medical writing and editorial support were provided by Daniel Himmel, PhD, of Prime (Knutsford, UK) funded by Regeneron Pharmaceuticals, Inc. and Sanofi.

Previously presented (encore) at the American Association for Cancer Research (AACR) 2022 Annual Meeting, April 8-12, 2022, in New Orleans, LA, USA.

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Last modified: November 29, 2022

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