New Pill Targets Rare BRAF and CRAF Tumor Changes

A new pill called tovorafenib is being tested in people with "tough" solid tumors. It targets rare genetic changes in BRAF or CRAF. These changes can push tumors to grow. The drug can reach the brain, which can matter for some cancers. It is selective for these targets. It blocks type II forms of BRAF and CRAF. In a phase two study, patients 12 and older with tumors that came back or did not respond to earlier care were included. The tumors had a BRAF fusion, a CRAF fusion, or a CRAF amplification.

Twenty three patients took part. Eight had melanoma, and fifteen had other solid tumors. The median age was 53, with a range from 21 to 71. About 57 percent had already had at least two prior treatments. Fourteen had a BRAF fusion, six had a CRAF fusion, two had a CRAF amplification, and one had both a CRAF fusion and amplification. Tovorafenib was given at 600 mg once a week in adults. Treatment went on in 28 day cycles. The median time on therapy was 5.3 months, with a range from 0.8 to 22.5 months.

The main goal was to see how many tumors shrank. Ten of 23 patients had a response. That was 43 percent overall. Four of eight melanoma patients responded. That was 50 percent. Six of 15 other tumor patients responded. That was 40 percent. The median time to response was 1.8 months. The median duration of response was 9.2 months. Anemia happened in 39 percent of patients. Itching happened in 30 percent. Raised creatine phosphokinase happened in 26 percent. Rash happened in 26 percent. The side effects were manageable.

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