We are building a strong pipeline of targeted therapies designed specifically for children and others with cancer. And we’re working hard to develop these medicines with rigor and efficiency to get them to those who need them most.
Approved
Therapeutic Area: BRAF-altered
Relapsed pLGG
Progress: Phase 2
Clinical Trial: FIREFLY-1 (pivotal Phase 2)
Therapeutic Area: Frontline
RAF-altered pLGG
Progress: Phase 3
Clinical Trial: FIREFLY-2 (pivotal Phase 3)
Therapeutic Area: MAPK-altered
solid tumors †
(combination with tovorafenib)
Progress: Phase 1
Clinical Trial: FIRELIGHT-1††
Therapeutic Area: Pediatric and adult solid tumors
Progress: Pre-Clinical
Clinical Trial:
Therapeutic Area: Pediatric and adult cancers
Progress: Pre-Clinical
Clinical Trial:
1 OJEMDA has received accelerated approval by the U.S. Food and Drug Administration.
† Pimasertib Phase 1 dose escalation and expansion trial previously completed.
†† Includes patients ≥12 years of age.
VRK1 Program is a research collaboration and license agreement with Sprint Bioscience for exclusive worldwide rights to a research-stage program targeting VRK1.
DAY301 is a license agreement with MabCare Therapeutics for exclusive worldwide rights, excluding Greater China, for MTX-13, a novel ADC targeting PTK7.
