DEVELOPMENT PIPELINE

A pipeline that protects futures

We are building a strong pipeline of targeted therapies designed
specifically for children with cancer. And we’re working hard to develop these medicines with rigor and efficiency to get them to those who need them most.

DEVELOPMENT PIPELINE

A pipeline that protects futures

We are building a strong pipeline of targeted therapies designed
specifically for children with cancer. And we’re working hard to develop these medicines with rigor and efficiency to get them to those who need them most.

Our lead candidate, tovorafenib (DAY101), has the potential to become an approved therapy for the most common childhood brain cancer—pediatric low-grade glioma (pLGG) which is an area of considerable unmet need. And we see potential for tovorafenib to help adults with cancer too.

Our treatments have the potential to save lives, our pipeline aims to protect futures and our commitment aspires to elevate what can be achieved in oncology.

Product Candidate
Indication
  • Pre-CN
  • Ph 1
  • Ph 2
  • Ph 3
Tovorafenib (DAY101) Type II Pan-RAF Inhibitor

Indication: Relapsed pLGG

Progress: Phase 2

Clinical Trial: Enrolling (FIREFLY-1) 1

  • PH2
Enrolling (FIREFLY-1) 1

Indication: Frontline pLGG

Progress: Phase 3

Clinical Trial: Enrolling (FIREFLY-2)

  • PH3
Enrolling (FIREFLY-2)
Pimasertib MEK1/2 Inhibitor

Indication: MAPK-altered solid tumors 3*

Progress: Phase 1

Clinical Trial: Enrolling (FIRELIGHT-1)4

  • PH1
Enrolling (FIRELIGHT-1)4
VRK1 Program VRK1 Inhibitor

Indication: Pediatric and adult cancers 5*

Progress: Pre-Clinical

Clinical Trial:

  • PreCN

1 FIREFLY-1 is a pivotal Phase 2 trial expected to support registration.

2 DAY101 adult monotherapy Phase 1 dose escalation and expansion trial previously completed.

3 Pimasertib Phase 1 dose escalation and expansion trial previously completed.

4 Trial in combination with DAY101.

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

* Includes patients ≥12 years of age.

See our progress in motion.