Phase 2 Study of Cemiplimab in Patients with Locally Advanced Basal-Cell Carcinoma After Hedgehog Inhibitor Therapy: Long-Term Follow-Up

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

Alexander J. Stratigos, PhD1; Aleksandar Sekulic, MD2; Ketty Peris, MD3; Oliver Bechter, MD4; Sorilla Prey, MD5; Martin Kaatz, MD6; Karl D. Lewis, MD7; Nicole Basset-Seguin, PhD8; Anne Lynn S. Chang, MD9; Stéphane Dalle, MD10; Almudena Fernandez Orland, MD11; Lisa Licitra, MD12; Caroline Robert, PhD13; Claas Ulrich, MD14; Axel Hauschild, MD15; Michael R. Migden, MD16; Reinhard Dummer, MD17; Suk-Young Yoo, PhD18; Ebony Coates, MHA18; Emmanuel Okoye, MBBS18; Ioannis Bassukas, MD19; Carmen Loquai, MD20; Vincenzo De Giorgi, MD21; Zeynep Eroglu, MD22; Ralf Gutzmer, MD23; Jens Ulrich, MD24; Susana Puig, MD25; Frank Seebach, MD18; Israel Lowy, PhD18; Matthew G. Fury, PhD18

1University of Athens, A. Sygros Hospital, Greece; 2Arizona Mayo Clinic, USA; 3Catholic University Fondazione Policlinico Universitario, Italy; 4University Hospitals, Belgium; 5Bordeaux University Hospital, France; 6SRH Wald-Klinikum Gera GmbH, Germany; 7University of Colorado, USA; 8Hôpital Saint-Louis, France; 9Stanford University, USA; 10Centre Hospitalier Lyon-Sud, France; 11Hospital Universitario Virgen Macarena, Spain; 12Istituto Nazionale dei Tumori, and University of Milan, Italy; 13Gustave Roussy Cancer Center, France; 14Charite-Universitätsmedizin Berlin, Germany; 15University of Kiel, Germany; 16The University of Texas MD Anderson Cancer Center, USA; 17University Hospital Zurich, Switzerland; 18Regeneron Pharmaceuticals, Inc, USA; 19University of Ioannina, Greece; 20University Medical Center Mainz, Germany; 21University of Florence, Italy; 22Moffitt Cancer Center, USA; 23Ruhr University Bochum, Germany; 24University Otto von Guericke Magdeburg, Germany; 25University of Barcelona, Spain

Background: Primary analysis from a phase 2 study demonstrated substantial clinical benefit from cemiplimab with an acceptable safety profile in patients with locally advanced basal-cell carcinoma (laBCC) who discontinued hedgehog inhibitor (HHI) therapy due to progression of disease, intolerance, or no better than stable disease after 9 months (NCT03132636).

Objective: Here, we present the long-term follow-up data at up to 40 months.

Methods: Patients with laBCC received cemiplimab 350 mg intravenously every 3 weeks for up to 93 weeks or until progression of disease. The primary end point was objective response rate (ORR) per independent central review (ICR). Key secondary end points included ORR by investigator assessment, safety and tolerability, duration of response (DOR), progression-free survival (PFS), overall survival (OS), complete response rate, and disease control rate (DCR). DCR was defined as the proportion of patients with complete response, partial response, stable disease, or nonpartial response/nonprogressive disease. Data cutoff was May 20, 2021.

Results: Eighty-four patients were enrolled. Median duration of follow-up was 15.9 months (range, 0.5-39.7). ORR per ICR was 32.1% (95% CI, 22.4-43.2), including 6 complete responses and 21 partial responses. Median DOR was not reached. Kaplan-Meier–estimated proportion with ongoing response at 24 months was 56.6% (95% CI, 29.6-76.6). In responding patients, the median time to response per ICR was 4.3 months (range, 2.1-21.4). DCR was 79.8% (95% CI, 69.6-87.7) per ICR and 88.1% (95% CI, 79.2-94.1) per investigator assessment. Median OS was not reached. Median PFS for all patients was 16.5 months (95% CI, 8.6-21.4). The most common treatment-emergent adverse events (TEAEs) were fatigue (31.0%), diarrhea (23.8%), pruritus (21.4%), asthenia (20.2%), and arthralgia (19.0%); 17.9% of patients discontinued treatment due to TEAEs. Grade ≥3 TEAEs occurred in 52.4% of patients. There were no treatment-related deaths.

Conclusions: Long-term follow-up data up to 40 months showed further efficacy and durability of response with cemiplimab in patients with laBCC who progressed on or were intolerant to HHI. There were no new safety signals. These results support the use of cemiplimab as a treatment option for patients with laBCC after 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 Sameen Yousaf, PhD, of Prime (Knutsford, UK) funded by Regeneron Pharmaceuticals, Inc. and Sanofi.

Previously presented (encore) at the European Association of Dermato Oncology (EADO) 2022 Annual Meeting, April 21-23, 2022, in Seville, Spain.

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Last modified: November 29, 2022

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