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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 address the lack of new therapies resulting from the traditional drug development model, which has left children with cancer and their families waiting too long for new, life-changing treatments. Children’s cancers continue be a focus 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

FIREFLY-1 (pivotal Phase 2)2

Phase 1 Dose Escalation

Phase 1 Dose Expansion

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

firefly icon
FIREFLY-2 (pivotal Phase 3)

Phase 1 Dose Escalation

Phase 1 Dose Expansion

Phase 2

Phase 3/
Registrational

Approved

Tovorafenib details FIREFLY-2 Clinical Trial

Adenoid Cystic Carcinoma (ACC), adult and pediatric solid tumors

Emiltatug Ledadotin (Emi-Le)

B7-H4-Targeted ADC

Phase 1 Dose Escalation

Phase 1 Dose Expansion

Phase 2

Phase 3/

Registrational

Approved

Potential first-in-class B7-H4-targeted ADC with anti-tumor activity observed in patients living with ACC.

Emi-Le Emi-Le-001 Clinical Trial

Adult and pediatric solid tumors

DAY3014

PTK7 Targeted ADC

Phase 1 Dose Escalation

Phase 1 Dose Expansion

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

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.