Cell Therapy News/Articles, page-18009

https://ir.syndax.com/news-releases/news-release-details/syndax-and-incyte-announce-positive-topline-results-pivotal#:~:text=The%20trial%20achieved%20its%20primary,50%25%2C%20respectively%20(95%25

Did they get approval based on just a Phase II trial which had a primary of response to the treatment, not improvement in outcome? Appreciate over 200 patients but also interesting was the results were worse for patients that received higher doses, plus high number of adverse reactions compared to MSB alternative.

extract below;
The global Phase 2 AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD whose disease had progressed after two prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1 mg/kg every two weeks or 3 mg/kg every four weeks. The trial's primary endpoint is the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints include duration of response, percent reduction in daily steroids dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale. For more information about AGAVE-201, visit https://clinicaltrials.gov/ct2/show/NCT04710576.