VivoSeq
Oncology

Systemic Mastocytosis

22

Pipeline Programs↗

5

Active Trials↗

13

Companies Involved↗

Systemic Mastocytosis is an emerging landscape with 22 programs across 13 companies. The most active players are Karuna Therapeutics, Inc., a Bristol Myers Squibb company, Cogent Biosciences, Inc., Patara Pharma. 2 therapies are approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

48/100

Pipeline Velocity

6/100

Mech. Concentration

7/100

Completion Rate

82/100

Attrition Rate

12/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

K

Karuna Therapeutics, Inc., a Bristol Myers Squibb company

1 program

Phase 1C

Cogent Biosciences, Inc.

1 program

Phase 2P

Patara Pharma

1 program

Phase 2T

Telios Pharma, Inc.

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2Eisai logo

Eisai

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

79%

Small molecule

19 of 24 classified programs — the dominant modality here.

21%ALL OTHERSMonoclonal antibody28%Protein / enzyme28%Antibody–drug conjugate14%

Small molecule leads; the ring shows all 24 classified programs, each share of the total. Modality classified for 100% of 24 pipeline programs.

Analysis

Trial Status

26

clinical trials

Completed14 · 54%
Unknown status4 · 15%
Recruiting3 · 12%
Terminated3 · 12%
Active, not recruiting2 · 8%

Clinical Trials

Trial Activity

26 trials

(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Recruiting·CCogent Biosciences, Inc.·Phase 2·NCT04996875·140 enrolled
A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany
Recruiting·BBlueprint Medicines·N/A·NCT07255638·80 enrolled
Study of TL-895 in Subjects With Myelofibrosis or Indolent Systemic Mastocytosis
Recruiting·TTelios Pharma, Inc.·Phase 2·NCT04655118·121 enrolled
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
Active, not recruiting·CCogent Biosciences, Inc.·Phase 2·NCT05186753·237 enrolled
(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
Active, not recruiting·BBlueprint Medicines·Phase 2·NCT03731260·251 enrolled
Imatinib in KIT-negative Systemic Mastocytosis
Completed·HHospital Virgen de la Salud·Phase 4·NCT01297777·10 enrolled
Brentuximab Vedotin in Treating Patients With Advanced Systemic Mastocytosis or Mast Cell Leukemia
Completed·—·Phase 2·NCT01807598·10 enrolled
(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies
Completed·BBlueprint Medicines·Phase 1·NCT02561988·86 enrolled
Thalidomide in Treating Patients With Relapsed or Progressive Systemic Mastocytosis
Completed·—·N/A·NCT00769587
Ontak (Denileukin Diftitox) in Patients With Systemic Mastocytosis (SM)
Completed·MM.D. Anderson Cancer Center·Phase 2·NCT00493129·8 enrolled
Interest of Denosumab Treatment in Osteoporosis Associated to Systemic Mastocytosis
Completed·AAssistance Publique - Hôpitaux de Paris·Phase 3·NCT03401060·24 enrolled
Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
Completed·AAB Science·Phase 3·NCT00814073·135 enrolled
Safety and Efficacy of Subcutaneous Sarilumab in Improving the Quality of Life in People With Indolent Systemic Mastocytosis
Completed·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT03770273·21 enrolled
(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
Completed·BBlueprint Medicines·Phase 2·NCT03580655·107 enrolled
Phase 2 Midostaurin in Aggressive Systemic Mastocytosis and Mast Cell Leukemia
Completed·—·Phase 2·NCT00233454·26 enrolled
Page 1 of 2

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)