1 · Split primary endpoints
SENTRY, RISE UP and PASHA each met one endpoint and missed another, creating regulatory ambiguity.
PASHASENTRYRISE UP→Regulatory uncertainty→Better endpoint design
2 · MRD everywhere
Deep MRD-negativity is becoming the goal across CLL, myeloma and AML.
CLLMyelomaAML→MRD as surrogate→Needs MRD infrastructure
3 · Bispecifics earlier and wider
Bispecifics reached smouldering myeloma, ITP and earlier relapsed myeloma lines.
Smouldering MMITPEarlier RRMM→Earlier and wider use→Risk-benefit scrutiny
4 · Scalable cellular therapy
In vivo and allogeneic CAR-T sidestep the manufacturing bottleneck.
LB2501REVSTAR-123→No bespoke manufacturing→Scalable access
5 · CALR-mutant MPN as targetable
INCA033989 is the first mutation-specific MPN therapy, echoing the BCR-ABL story.
JAK2CALR→Mutation-specific therapy→CALR testing predictive
6 · Targeted autoimmune haematology
wAIHA and ITP now have targeted trial data, validating the FcRn and plasma-cell routes.
wAIHAITP→FcRn / plasma-cell targeting→Licensed targeted era
Not every biologically superior drug improves overall survival.PASHA
MRD is becoming the common language across haematology.CLL and myeloma
Targeting the clone matters more than controlling the count.CALR antibody; ropeginterferon
Cellular therapy is moving from bespoke manufacturing towards scalable delivery.In vivo and allogeneic CAR-T
A smaller spleen is not the same as a patient who feels better.SENTRY
Treating disease earlier raises the bar for proving benefit outweighs harm.ImmunoPRISM