GIST

5
Pipeline Programs
3
Companies
14
Clinical Trials
1 recruiting
2
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
2
0
1
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
3100%
+ 2 programs with unclassified modality

On Market (2)

Approved therapies currently available

Blueprint Medicines
AYVAKITApproved
avapritinib
Blueprint Medicines
Kinase Inhibitor [EPC]oral2020
Takeda
ICLUSIGApproved
ponatinib hydrochloride
Takeda
oral2012

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Blueprint Medicines
Blueprint MedicinesMA - Cambridge
3 programs
1
1
avapritinibPhase 3Small Molecule1 trial
AvapritinibN/ASmall Molecule1 trial
Patient treated by Avapritinib in real lifeN/A1 trial
Active Trials
NCT03862885Approved For Marketing
NCT04927260Unknown25Est. Dec 2024
NCT03465722Completed476Est. Sep 2021
Takeda
TakedaTOKYO, Japan
1 program
1
1
ICLUSIG(Ponatinib)Phase 2Small Molecule5 trials
Active Trials
NCT07224100Recruiting33Est. Jul 2028
NCT04501614Terminated11Est. Jul 2024
NCT06945146Completed65Est. Sep 2024
+2 more trials
Biogen
BiogenCAMBRIDGE, MA
1 program
1
BIIB021Phase 22 trials
Active Trials
NCT00618319Completed20Est. Apr 2010
NCT00412412Completed34Est. Apr 2011

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
TakedaPonatinib
Blueprint Medicinesavapritinib
TakedaPonatinib
TakedaPonatinib
TakedaPonatinib
TakedaPonatinib
TakedaPonatinib
BiogenBIIB021
TakedaPonatinib
TakedaPonatinib
BiogenBIIB021
Blueprint MedicinesPatient treated by Avapritinib in real life
TakedaPonatinib

Clinical Trials (14)

Total enrollment: 1,765 patients across 14 trials

NCT03589326TakedaPonatinib

A Study of Ponatinib Versus Imatinib in Adults With Acute Lymphoblastic Leukemia

Start: Oct 2018Est. completion: Jul 2027245 patients
Phase 3Active Not Recruiting
NCT03465722Blueprint Medicinesavapritinib

(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

Start: Mar 2018Est. completion: Sep 2021476 patients
Phase 3Completed
NCT02627677TakedaPonatinib

A Study Comparing Ponatinib and Nilotinib in Participants With Chronic Myeloid Leukemia

Start: Dec 2015Est. completion: Jan 202144 patients
Phase 3Terminated
NCT07224100TakedaPonatinib

Dose-Adjusted EPOCH With or Without Rituximab Plus Ponatinib for the Treatment of Newly-Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia/Lymphoma

Start: Mar 2026Est. completion: Jul 202833 patients
Phase 2Recruiting
NCT02467270TakedaPonatinib

Ponatinib in Participants With Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML) to Characterize the Efficacy and Safety of a Range of Doses

Start: Jun 2015Est. completion: Apr 2025283 patients
Phase 2Completed
NCT01874665TakedaPonatinib

A Phase 2 Trial of Ponatinib in Participants With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor

Start: Jun 2013Est. completion: Jul 201645 patients
Phase 2Completed
NCT01207440TakedaPonatinib

Ponatinib for Chronic Myeloid Leukemia (CML) Evaluation and Ph+ Acute Lymphoblastic Leukemia (ALL)

Start: Sep 2010Est. completion: Jan 2019449 patients
Phase 2Completed
NCT00618319BiogenBIIB021

An Open-Label, 18FDG-PET Pharmacodynamic Assessment of the Effect of BIIB021 in Subjects With Gastrointestinal Stromal Tumors (GIST)

Start: Feb 2008Est. completion: Apr 201020 patients
Phase 2Completed
NCT04501614TakedaPonatinib

A Study of Ponatinib With Chemotherapy in Children, Teenagers, and Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia

Start: Feb 2021Est. completion: Jul 202411 patients
Phase 1/2Terminated
NCT01667133TakedaPonatinib

A Study of Ponatinib in Japanese Participants With Chronic Myeloid Leukemia (CML) and Ph+ Acute Lymphoblastic Leukemia (ALL)

Start: Aug 2012Est. completion: Aug 201835 patients
Phase 1/2Completed
NCT00412412BiogenBIIB021

CNF2024 (BIIB021) HER2- (QD) HER2+ (BIW w/Herceptin) PK/PD Study

Start: Dec 2007Est. completion: Apr 201134 patients
Phase 1Completed
NCT03862885Blueprint MedicinesAvapritinib

Early Access Program (EAP) for Avapritinib in Patients With Locally Advanced Unresectable or Metastatic GIST

N/AApproved For Marketing
NCT04927260Blueprint MedicinesPatient treated by Avapritinib in real life

French Long Term Registry With Longitudinal Follow up of PDGFRA D842V-GIST Patients

Start: Mar 2021Est. completion: Dec 202425 patients
N/AUnknown
NCT06945146TakedaPonatinib

Genomics Study in CML Patients With Ponatinib Treatment

Start: Sep 2020Est. completion: Sep 202465 patients
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 1,765 patients
Small Molecule is the dominant modality (100% of programs)
3 companies competing in this space