Patients randomised into Veregen® arm showed a significantly better clinical response when compared to placebo
Clinical response was measured according to a protocol defined evaluation criteria based on RECIST and responses were categorised into stable disease, partial response, complete response and progressive disease. Clinical responses were measured at 4, 8, 16, 32 and 52 weeks after the start of treatment: 5 patients showed partial response in Veregen® group when compared to only one patient in the placebo group at four weeks; 7 patients showed clinical response in Veregen® group and 3 in the placebo group at eight weeks. At the end of treatment, 16 weeks, 10 patients in Veregen® group responded to treatment: 5 showed complete response; 5 a partial response; 2 patients had stable disease; 1 patient was lost to follow up but responded partially at four weeks. In the placebo group, five patients showed clinical response: 2 complete response, 3 partial response, 4 with stable disease and 1 had progressive disease. Two patients were lost to follow up in the placebo group. At 32 weeks, one patient had progressed in Veregen® treatment arm, who previously had a partial response. This patient continued to have disease progression at 52 weeks follow up. One other patient in the Veregen® treatment arm, who already had a complete response at 16 weeks, recurred at 52 weeks. In the placebo treatment arm, two patients presented with progressive disease, one patient with previously complete response at 16 weeks relapsed, and two further patient with previous complete response and partial response remained disease-free, the remaining patients had stable disease. In the Veregen® group, all patients showed a significant clinical response as opposed to only five patients showing clinical response following treatment (p=0.003) (Figure 1). Patients in the Veregen® group show a decreased time to clinical resolution as compared to placebo, both when clinical resolution is taken to be complete response and complete/partial response (Figure 1).
Biopsies were obtained at 16 and 32 weeks after treatment to evaluate the histological resolution of uVIN and there was no observed difference in best histological response in these two groups. Three patients in each arm showed complete histology resolution; 7 and 6 patients in placebo and active arm, respectively, had persistent disease histologically; 3 and 4 patients in placebo and active arm, respectively, refused post-treatment biopsy. One patient from the placebo group developed early-stage squamous cell carcinoma at 16-week biopsy, she was withdrawn from the study and managed according to our local guideline for vulvar cancer.