Phase 3 efficacy readout for AYVAKIT in second-line GIST. AYVAKIT is currently approved in PDGFRA D842V-mutant GIST and indolent/advanced systemic mastocytosis. Watch: PFS vs. sunitinib comparator.
What’s at stake
AYVAKIT (avapritinib) is a highly selective oral kinase inhibitor developed by Blueprint Medicines that targets activating mutations in KIT and PDGFRα — the dominant oncogenic drivers in gastrointestinal stromal tumors (GIST) and systemic mastocytosis. The drug fits precisely into the ATP-binding pocket of these mutant kinases to block the aberrant signaling driving tumor growth and mast cell accumulation. AYVAKIT is approved for PDGFRA Exon 18 D842V-mutant GIST — a subset previously insensitive to imatinib — and for advanced and indolent systemic mastocytosis; Blueprint is studying avapritinib versus sunitinib in second-line GIST in an ongoing Phase 3 trial.
No primer in glossary yet.
- PFSendpointProgression-Free Survival
Time from randomization until tumor progression or death. Mid-term oncology endpoint; correlates with overall survival in many tumor types.
How Appr readouts in Oncology - Solid have landed.
Reference, not prediction. We surface the historical record so you can read it yourself. We never extrapolate to the upcoming event.
Competitive landscape
| Drug | Company | Modality | Mechanism | Phase | Next catalyst |
|---|---|---|---|---|---|
| REC-994 | RXRX | small molecule | oxidative stress modulator (CCM) | READOUT · Nov 26 |
Prior BPMC reactions to Readout events
| Date | Headline | Outcome | 1W | 1M | 6M |
|---|---|---|---|---|---|
| Jan 2026 | AYVAKIT — Phase 3 Topline — Mixed Result | negative | -14.2% | -39.5% | -19.4% |
| Aug 2024 | AYVAKIT — Phase 2b Topline Met Primary Endpoint | mixed | +19.1% | +22.9% | +31.5% |
Disclosure trail
- Mar 25, 2026·1mo agopinned · highest confidenceHIGH confPRMONTHtop claimSEP2026
“PIONEER, the Phase 3 study of AYVAKIT in second-line GIST, is expected to deliver topline data in September 2026.”
conf 89%via llm