This study was conducted in subtype of sarcoma called leiomyosarcoma, and leiomyosarcomas are one of the more common sarcoma subtypes, probably accounting for about 10-15% of all sarcomas. For patients with advanced or metastatic leiomyosarcoma the treatment options are limited. Systemic therapies can include anthracycline and gemcitabine based schedules as well as pazopanib, trabectedin, and dacarbazine. But there’s still a huge unmet need for novel, effective, and well-tolerated systemic therapies for these patients.

I think one of the major things that we really need is a better way to identify which patient with leiomyosarcoma is going to benefit from each one of these individual treatments.

What was the methodology and what were the findings?

This was a randomised phase III trial open to patients with advanced or metastatic leiomyosarcoma. Patients had received one or more prior lines of therapy, including an anthracycline-based schedule. They had to have measurable disease by RECIST 1.1 and have a performance status of zero or one and be over the age of 18 years.

Patients were randomised to receive either catequentinib 12mg/day, two weeks on, one week off, or placebo. That randomisation was 2:1, so two to catequentinib and one to placebo. The primary endpoint to the trial was progression-free survival by blinded independent central radiology review, and secondary endpoints included objective response rate, overall survival, and duration of response.

111 patients were randomised, 75 were randomised to catequentinib, and 36 to placebo. There was a statistically significant difference in progression free survival between the two arms, so median progression free survival for catequentinib was 3.42 months compared to 1.41 months for placebo. The hazard ratio was 0.536.

What are the clinical implications of these findings?

One of the major points regarding catequentinib is that it is very well tolerated with a really good toxicity profile. Because of this one of the major points is that it could potentially be used as a combination therapy for patients with metastatic leiomyosarcoma.

I suppose the major thing is could we do further trials of combination therapy with catequentinib to try and improve the outcome of patients with metastatic leiomyosarcoma.