The denominator first
The peer-reviewed phase 1 gave oral GFH375 to 74 patients with KRAS G12D tumours. Primary endpoints were safety, the maximum tolerated dose and the phase 2 dose. None of 22 dose-escalation patients had a dose-limiting toxicity; the maximum tolerated dose was not reached; the recommended phase 2 dose was written as 600 mg once daily.[1]
One grade 5 treatment-related event was reported as septic shock, 1.4 percent. Grade 3 or higher treatment-related events sat at 27/74, 36.5 percent. Those figures fill the safety denominator. They do not fill the missing comparison arm.[1]
Response from a single arm
In pancreatic ductal adenocarcinoma the confirmed objective response rate was 35.1 percent, 13/37. Median duration of response was 5.6 months, median progression-free survival 5.5 months, median overall survival 9.9 months. In non-small cell lung cancer the confirmed response rate was 35.7 percent, 5/14; median progression-free survival 5.5 months, median overall survival 13.4 months.[1]
Those rates come from a heavily treated, selected phase 1 funnel. There is no randomisation, no concurrent control, and no prespecified historical success bar in the text. A replication would be a comparative phase 2 at the same dose holding a similar magnitude.[1]
The signal bar
The paper says the phase 2 expansion is ongoing under NCT06500676. That does not mean 13/37 has been confirmed. What would count is a confirmed response at the same 600 mg dose, tied to a comparison or to a bar declared in advance.[1]