Trial results for the Phase 2 study (NCT05814666) evaluating danvatirsen in combination with pembrolizumab for recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC) were posted on ClinicalTrials.gov on 2026-01-30. The study, which was terminated, showed that 7 participants in the combination arm achieved confirmed objective response rate (ORR) compared to 3 in the pembrolizumab alone arm, while 37 participants in the combination arm experienced adverse events compared to 10 in the monotherapy arm.
Background
The study investigated the activity and safety of danvatirsen in combination with pembrolizumab in patients with HNSCC. HNSCC is a type of cancer that starts in the squamous cells that line the moist surfaces inside the head and neck.
Trial design
The Phase 2, open-label, randomized, controlled study (NCT05814666) enrolled 69 participants with recurrent/metastatic HNSCC. The trial compared danvatirsen in combination with pembrolizumab against pembrolizumab alone as first-line treatment. Two-thirds of patients were randomized to receive the combination, and one-third received pembrolizumab alone. The study aimed to evaluate the efficacy and safety of the treatments. The trial was ultimately TERMINATED.
Key results
Key measurements from the trial included confirmed objective response rate (ORR), duration of response (DOR), disease control rate (DCR) & complete response (CR) rate, and the number of participants with adverse events.
- For Confirmed ORR:
- Danvatirsen Plus Pembrolizumab arm: 7 Participants
- Pembrolizumab arm: 3 Participants
- For Number of Participants With Adverse Events as Assessed by CTCAE v5.0:
- Danvatirsen Plus Pembrolizumab arm: 37 Participants
- Pembrolizumab arm: 10 Participants
- For DOR:
- Danvatirsen Plus Pembrolizumab arm: Median DOR was 6.36 Months (95% Confidence Interval: not provided).
- Pembrolizumab arm: Median DOR was NA.
- For DCR & CR Rate:
- Overall DCR & CR Rate (combining DCR and CR): 21 Participants in the Danvatirsen Plus Pembrolizumab arm vs 12 Participants in the Pembrolizumab arm.
- Disease Control Rate (DCR): 15 Participants in the Danvatirsen Plus Pembrolizumab arm vs 9 Participants in the Pembrolizumab arm.
- Complete Response (CR) Rate: 1 Participant in the Danvatirsen Plus Pembrolizumab arm vs 0 Participants in the Pembrolizumab arm.
A Fisher Exact analysis for an unspecified outcome yielded a p-value of 0.6039.
What this means
The results of this Phase 2 study in recurrent/metastatic HNSCC indicate that while the combination of danvatirsen and pembrolizumab showed a numerically higher confirmed ORR compared to pembrolizumab alone, this difference was not statistically significant (p-value of 0.6039). Furthermore, the combination arm was associated with a higher number of participants experiencing adverse events. The termination of the trial suggests that the combination did not demonstrate a sufficiently favorable risk-benefit profile to warrant further investigation in this setting.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT05814666, titled "Activity and Safety of Danvatirsen and Pembrolizumab in HNSCC," were posted on 2026-01-30 on clinicaltrials.gov.