Interim Analysis of Phase 2 Results for Cemiplimab in Patients with Metastatic Basal Cell Carcinoma (mBCC) who Progressed on or are Intolerant to Hedgehog Inhibitors (HHIs)
Bibliographic record
Abstract
Basal cell carcinoma (BCC) is the most common type of skin cancer 1 and ultraviolet exposure is a major risk factor. 2• Surgery is a curative option for most patients, but systemic therapy is indicated for a small percentage of patients who develop advanced BCC. 3• Vismodegib is a hedgehog signalling pathway inhibitor (HHIs) that is approved for treatment of patients with metastatic BCC (mBCC) or locally advanced BCC (laBCC) who are not candidates for curative surgery or curative radiotherapy.Sonidegib is another HHI that is approved for the treatment of laBCC only.• There are no available data for patients who progress on or are intolerant to HHIs.• Cemiplimab is a fully human antibody, derived using VelocImmune technology, 4-5 which is a high-affinity, highly potent, hinge-stabilized, immunoglobulin G4 monoclonal antibody directed against programmed cell death-1 (PD-1).6• Cemiplimab has recently been shown to have profound clinical activity as monotherapy in first-line non-small cell lung cancer with ≥50% PD-ligand 1 expression.7• Cemiplimab is approved for treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.8• In a pivotal Phase 2 study of patients with advanced BCC who discontinued HHI therapy due to disease progression, intolerance, or no better than stable disease after 9 months, cemiplimab became the first systemic therapy to show clinical benefit in patients with laBCC after HHI therapy, with estimated duration of response (DOR) exceeding 1 year in 85% of responders.9-Cemiplimab produced an objective response rate (ORR) of 31% in patients with laBCC after treatment with HHI therapy; the safety profile was acceptable and consistent with that previously reported for cemiplimab in other tumor types.9• Here, we present the prespecified interim analysis of the mBCC cohort from the pivotal Phase 2 study (NCT03132636). Objectives• The primary objective is ORR by independent central review (ICR).• Secondary objectives include ORR according to investigator review; duration of progression-free survival (PFS) by central and investigator review; overall survival (OS); complete response rate by central review; and safety and tolerability of cemiplimab.• Interim analysis included patients with the opportunity to be followed for approximately 57 weeks to provide an ORR with 95% confidence interval (CI). Methods• This is a Phase 2, non-randomized, multi-center study of cemiplimab in patients with either mBCC or laBCC (Figure 1).
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.007 | 0.004 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.003 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.002 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.003 |
| Insufficient payload (model declined to judge) | 0.006 | 0.001 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".