What question did this study set out to answer?

To assess the efficacy and safety of ficerafusp alfa combined with pembrolizumab versus placebo plus pembrolizumab in HPV-negative R/M HNSCC.

May 30, 2026

A multicenter, randomized, double-blind, phase 2/3 study of ficerafusp alfa (BCA101) or placebo in combination with pembrolizumab for first-line treatment of HPV-negative, PD-L1–positive, recurrent or metastatic (R/M) head and neck squamous cell carcinoma (HNSCC): FORTIFI-HN01.

Puntos clave

To assess the efficacy and safety of ficerafusp alfa combined with pembrolizumab versus placebo plus pembrolizumab in HPV-negative R/M HNSCC.
Randomized, double-blind, placebo-controlled trial with 2:1 randomization after determining the optimal biological dose.
Participants received either ficerafusp alfa or placebo alongside pembrolizumab.
Primary endpoints included objective response rate (ORR) and overall survival, with interim analyses planned.
Ficerafusp alfa demonstrated promising efficacy in early phases, setting the stage for further evaluation in phase 3.
The trial plans to enroll approximately 650 subjects to evaluate outcomes comprehensively.
Safety and tolerability are being monitored throughout treatment, with regular patient assessments every 6-9 weeks.

Resumen

TPS6129 Background: HPV-negative HNSCC is an aggressive disease characterized by high rates of recurrence, metastasis, and resistance to standard treatments. Most HPV-negative HNSCC tumors overexpress tumorigenic factors EGFR and TGF-β. In a phase 1/1b trial (NCT04429542), ficerafusp alfa demonstrated promising efficacy and a manageable safety profile in first-line R/M HNSCC. FORTIFI-HN01 (NCT06788990) is an ongoing randomized, double-blind, placebo-controlled, phase 2/3 trial designed to assess the efficacy and safety of ficerafusp alfa combined with pembrolizumab vs placebo plus pembrolizumab in patients with PD-L1-positive first-line R/M HPV-negative HNSCC. Methods: Eligible patients must have histologically confirmed R/M HNSCC with primary lesions in the oral cavity, larynx, or hypopharynx, or HPV-negative OPSCC confirmed by central laboratory testing. Additional eligibility criteria include no prior systemic therapy for R/M disease, PD-L1-positive tumor (CPS ≥1), measurable disease per RECIST v1.1, and ECOG performance status 0 or 1. The phase 2 objective was to determine the optimal biological dose (OBD) of ficerafusp alfa through an integrated analysis of safety, tolerability, PK, PD, and efficacy. Following OBD determination (1500 mg QW), the trial transitioned seamlessly into the phase 3 portion with 2:1 randomization (ficerafusp alfa:control). Randomization is stratified by PD-L1 CPS (1-19 vs ≥20) and disease extent (local/regional recurrence only, distant metastasis only, or both). Patients receive pembrolizumab (200 mg IV every 3 weeks for up to 35 cycles) and either ficerafusp alfa or placebo IV QW until disease progression or unacceptable toxicity. Tumor imaging occurs every 6 weeks during the first year and every 9 weeks thereafter. The primary endpoints are objective response rate (ORR) per RECIST v1.1 (blind independent committee review) and overall survival. An interim analysis evaluating ORR is planned. Secondary endpoints include safety, duration of response, progression free survival, clinical benefit rate, and patient-reported outcomes. The trial is actively recruiting, with planned enrollment of ~650 subjects. Clinical trial information: NCT06788990 .

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