ACT001

Phase 2Small molecule

ACT001 is one of 6 small molecule programs targeting Diffuse Intrinsic Pontine Gliomas (DIPG). It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Diffuse Intrinsic Pontine Gliomas (DIPG)Recruiting

Recurrent GlioblastomaRecruiting

Diffuse Intrinsic Pontine Gliomas (DIPG)Phase 2

Recruiting

Recurrent GlioblastomaPhase 2

Recruiting

Competition

Competitive Landscape

7 competitors in Recurrent Glioblastoma

#DrugCompanyPhaseTrials

Keytruda

Merck & Co.

Phase 2

2 2

Avastin

Roche

Phase 2

2 3

Tecentriq

Roche

Phase 2

2 4

Cabometyx

Bristol-Myers Squibb

Preclinical

1 5

ACT001

NNationwide Children's Hospital

Phase 2

1 6

LMP744

NNational Cancer Institute (NCI)

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2027

Phase 2Recruiting

Recurrent Glioblastoma

NCT06894225

Q3 2035

Phase 2Recruiting

Diffuse Intrinsic Pontine Gliomas (DIPG)

NCT06838676

Clinical

Trials by Indication[1]

Glioblastoma Multiforme1 trial

A Study to Evaluate Safety and Efficacy of ACT001 and Anti-PD-1 in Patients With Surgically Accessible Recurrent Glioblastoma Multiforme

Unknown status·AAccendatech USA Inc.·Phase 1/2·NCT05053880·48 enrolled

Recurrent Glioblastoma1 trial

Proof-of-Concept Study of ACT001 in Adult Patients With Recurrent Glioblastoma Harbouring STAT3-High Signature

Recruiting·NNational Neuroscience Institute·Phase 2·NCT06894225·12 enrolled

Diffuse Intrinsic Pontine Gliomas (DIPG)1 trial

A Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

Recruiting·NNationwide Children's Hospital·Phase 2·NCT06838676·60 enrolled

Sources

References

[1]
ClinicalTrials.gov — 3 trial records for ACT001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — ACT001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for ACT001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Nationwide Children's Hospital company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: NNationwide Children's Hospital

ACT001

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q2 2027

Q3 2035

Trials by Indication[1]

Glioblastoma Multiforme1 trial

Recurrent Glioblastoma1 trial

Diffuse Intrinsic Pontine Gliomas (DIPG)1 trial

References

Drug Info

Quick Facts

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