RE: The 7% ORR Problem14 Jan 2026 12:56
Sure, Actuate therapeutics:
Primary Endpoint -
Best Overall Response Rate (ORR) per RECIST v1.1: The trial aimed to detect a 25% ORR (with >5 responders out of 32 patients providing 84.7% power to rule out a 10% ORR).
Result: ORR was 9.4% (95% CI: 2-25%), with 3 partial responses (PRs) and no complete responses. All responses occurred in the non-ACC subgroup (18% ORR in non-ACC with immune priming).
Met?: No, the primary endpoint was not met in the overall population.
Secondary Endpoints
Progression-Free Survival (PFS): Median PFS was 6.4 months (95% CI: 2.3-8.8 months), with a 12-month PFS rate of 43.6% in ACC vs. 16.7% in non-ACC.
Overall Survival (OS): Not reported in the available data.
Duration of Response (DoR): Median DoR was 6.9 months (range: 2.2-7.3 months) among responders.
Safety and Tolerability: The combination was generally manageable. Treatment-related adverse events (TRAEs) included anemia (69%), nausea (50%), and neutropenia (44%). Grade 3+ TRAEs occurred in 59% of patients (e.g., anemia 19%, neutropenia 13%), with one grade 4 event (neutropenia 3%). Discontinuations due to toxicity were low (6%), and there were no treatment-related deaths.
Met?: Secondary endpoints were not framed with predefined success criteria like the primary endpoint, so they were not formally "met" or "not met."
______________________
Christina has stated numerous times that Avacta will be using PFS as their primary endpoint and it's already far outshone the 6.4m we can say above here.