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A bold ambition

Advancing first- or best-in-class medicines for people of all ages.

Our goal is to take aim at the gaps that have left patients and their families behind.

We were founded to flip the script on traditional drug development to address the innovation gap that left children and their families waiting too long for new, potentially life-changing treatments. Children’s cancers continue be a priority as we expand with equal intensity to identifying and developing the next breakthrough therapeutics for serious childhood and adult diseases in urgent need of new solutions.

BRAF-altered relapsed pLGG

Tovorafenib3

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FIREFLY-1 (pivotal Phase 2)2

Pre-clinical

Phase 1

Phase 2

Phase 3/

Registrational

Approved

Tovorafenib is an investigational, oral, brain-penetrant, highly-selective type II RAF kinase inhibitor designed to target a key enzyme in the MAPK signaling pathway, which is being investigated in primary brain tumors.

OJEMDA™ (tovorafenib)1

Type II RAF Inhibitor

OJEMDA™ brand name in U.S.1

Ex-U.S. Rights:

Tovorafenib details FIREFLY-1 Clinical Trial Learn about OJEMDA™

Frontline RAF-altered pLGG

Tovorafenib3

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FIREFLY-2 (pivotal Phase 3)

Pre-clinical

Phase 1

Phase 2

Phase 3/
Registrational

Approved

Tovorafenib details FIREFLY-2 Clinical Trial

Adult and pediatric solid tumors

DAY3014

PTK7 Targeted ADC

Pre-Clinical

Phase 1

Phase 2

Phase 3/

Registrational

Approved

Day301 is a potentially first-in-class antibody drug conjugate (ADC) that has shown antitumor activity in preclinical models.

DAY301 DAY301-001 Clinical Trial

Adult and pediatric cancers

VRK1 Program5

VRK1 Inhibitor

Pre-clinical

Phase 1

Phase 2

Phase 3/

Registrational

Approved

The safety and efficacy of investigational agents and/or investigational uses of approved products have not been established.

  • 1 OJEMDA has received accelerated approval by the U.S. Food and Drug Administration
  • 2 FIREFLY-1 is an open-label, pivotal Phase 2 trial.
  • 3Ex-U.S. license agreement with Ipsen to develop and commercialize OJEMDA (tovorafenib) outside the U.S.

  • 4DAY301 is a license agreement with MabCare Therapeutics for exclusive worldwide rights, excluding Greater China, for MTX-13, a novel ADC targeting PTK77.

  • 5VRK1 Program is a research collaboration and license agreement with Sprint Bioscience for exclusive worldwide rights to a research-stage program targeting VRK1.