Rishabh Jain, Medical Oncologist at AIIMS, shared a post on X:
“A new potential option is emerging for lower-risk MDS.
Ofirnoflast (HT-6184), a first-in-class oral NEK7/NLRP3 inflammasome modulator, has received FDA Fast Track Designation following encouraging Phase 2 results.
Key findings:
- 67% hematologic improvement rate
- 56% achieved RBC transfusion independence (≥8 weeks)
- Median duration of transfusion independence: 28 weeks
- Median Hb rise: +4.6 g/dL No treatment-related serious adverse events
Notably, activity was observed across molecular subgroups, including patients with and without SF3B1 mutations or del(5q).
Could targeting inflammasome biology become the next major advance in lower-risk MDS after ESA and luspatercept failure?”
Other articles featuring Rishabh Jain on OncoDaily.