Trial results for the Phase 2 study (NCT04570332) investigating pembrolizumab in combination with BO-112 for unresectable malignant melanoma were posted on ClinicalTrials.gov on 2025-11-26. The study reported an overall response rate of 25.0% among participants with advanced and/or metastatic melanoma who had progressed on anti-PD-1-containing treatment.
Background
The study investigated BO-112 in combination with pembrolizumab for patients with advanced and/or metastatic melanoma. Participants had previously progressed on anti-PD-1-containing treatment, indicating a population with limited treatment options.
Trial design
The study (NCT04570332) was a Phase 2, single-arm, open-label, adaptive design study. It enrolled 42 participants with advanced and/or metastatic melanoma who had progressed on prior anti-PD-1-containing treatment. The intervention involved administering intratumoral BO-112 in combination with intravenous pembrolizumab. The study aimed to determine preliminary anti-tumor activity and confirm the safety profile of this combination.
Key results
Key measurements from the trial demonstrated anti-tumor activity for the combination of BO-112 and pembrolizumab:
- The Overall Response Rate was 25.0% (percentage of participants).
- The Disease Control Rate was 67.5% (percentage of participants).
- The median Progression Free Survival was 3.71 months.
- The Duration of Response and Overall Survival medians were reported as NA (not available).
Regarding safety, 42 participants experienced Adverse Events and Serious Adverse Events, representing the entire enrolled cohort.
An analysis for the binomial endpoint of Overall Response Rate (ORR) was conducted using a one-sided test. The observed ORR of 25.0% was tested against a null hypothesis of H0: ORR=10%. This analysis yielded a p-value of 0.01, with a 95.0% Confidence Interval for the rate of 12.69% to 41.2%. The minimal statistically significant ORR was 20%.
What this means
The results from this Phase 2 study suggest that the combination of BO-112 with pembrolizumab demonstrates anti-tumor activity in patients with unresectable malignant melanoma who have progressed on prior anti-PD-1 therapy. An overall response rate of 25.0% and a disease control rate of 67.5%, coupled with a statistically significant p-value of 0.01 for the ORR, indicate potential benefit in a challenging patient population. The median progression-free survival of 3.71 months provides further insight into the duration of disease control. These findings warrant further investigation into the role of this combination therapy for advanced melanoma.
Source
The information for this article was sourced from ClinicalTrials.gov, a public database of clinical studies. The trial results for study NCT04570332, titled "BO-112 With Pembrolizumab in Unresectable Malignant Melanoma," were posted on 2025-11-26 on clinicaltrials.gov.