Indolent Systemic Mastocytosis

5
Pipeline Programs
3
Companies
6
Clinical Trials
2 recruiting
0
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
1
2
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
583%
Monoclonal Antibody
117%

Competitive Landscape

3 companies ranked by most advanced pipeline stage

AS
AB ScienceFrance - Paris
2 programs
2
MasitinibPhase 3Small Molecule1 trial
MasitinibPhase 3Small Molecule1 trial
Active Trials
NCT04333108UnknownEst. Jun 2025
NCT00814073CompletedEst. Nov 2015
Blueprint Medicines
Blueprint MedicinesMA - Cambridge
3 programs
1
1
ElenestinibPhase 2/3Small Molecule1 trial
AvapritinibPhase 2Small Molecule1 trial
AvapritinibN/ASmall Molecule1 trial
Active Trials
NCT07255638Recruiting80Est. Dec 2028
NCT03731260Active Not Recruiting251Est. Jun 2027
NCT04910685Recruiting534Est. Sep 2032
Allergy Therapeutics
Allergy TherapeuticsUK - West Sussex
1 program
1
sarilumabPhase 2Monoclonal Antibody1 trial
Active Trials
NCT03770273CompletedEst. Jul 2023

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
AB ScienceMasitinib
AB ScienceMasitinib
Blueprint MedicinesElenestinib
Allergy Therapeuticssarilumab
Blueprint MedicinesAvapritinib
Blueprint MedicinesAvapritinib

Clinical Trials (6)

Total enrollment: 865 patients across 6 trials

NCT04333108AB ScienceMasitinib

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment

Start: Jul 2020Est. completion: Jun 2025
Phase 3Unknown
NCT00814073AB ScienceMasitinib

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis

Start: Dec 2008Est. completion: Nov 2015
Phase 3Completed
NCT04910685Blueprint MedicinesElenestinib

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Start: Nov 2021Est. completion: Sep 2032534 patients
Phase 2/3Recruiting
NCT03770273Allergy Therapeuticssarilumab

Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis

Start: Jun 2019Est. completion: Jul 2023
Phase 2Completed
NCT03731260Blueprint MedicinesAvapritinib

(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

Start: Apr 2019Est. completion: Jun 2027251 patients
Phase 2Active Not Recruiting
NCT07255638Blueprint MedicinesAvapritinib

A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany

Start: Dec 2025Est. completion: Dec 202880 patients
N/ARecruiting

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

2 late-stage (Phase 3) programs — potential near-term approvals
2 actively recruiting trials targeting 865 patients
Small Molecule is the dominant modality (83% of programs)
3 companies competing in this space