VivoSeq
Rare Disease

Pompe Disease

35

Pipeline Programs↗

11

Active Trials↗

20

Companies Involved↗

Pompe Disease is a moderately active landscape with 35 programs across 20 companies. The most active players are Denali Therapeutics Inc., Kenvue Brands, Maastricht Radiation Oncology. 5 therapies are approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

56/100

Pipeline Velocity

15/100

Mech. Concentration

20/100

Completion Rate

79/100

Attrition Rate

15/100

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.

D

Denali Therapeutics Inc.

1 program

Phase 1K

Kenvue Brands

1 program

Phase 1M

Maastricht Radiation Oncology

1 program

Phase 1P

Parkedale

1 program

Phase 1G

Glaxo Dba Glaxosmithkline

1 program

Phase 1Pfizer logo

Pfizer

1 program

Phase 1

Pipeline

Programs by Phase

Preclinical7

Avalglucosidase Alfa

Sanofi

Filgrastim

Eastern Cooperative Oncology Group

Alglucosidase Alfa

Genzyme, a Sanofi Company

Genetic: GC301

Seventh Medical Center of PLA General Hospital

AT2221

Amicus Therapeutics

Phase 112

RG6026

Roche

Acetaminophen

Kenvue Brands

Rapamycin

Maastricht Radiation Oncology

Diphenhydramine

Parkedale

Lidocaine

MEDRx USA, Inc.

Phase 28

Clenbuterol

University of Copenhagen

VAL-1221

Valerion Therapeutics, LLC

S-606001

Shionogi

Raav1-CMV-GAA (study Agent) Administration

University of Florida

Recombinant Human Acid Sphingomyelinase

Sanofi

Phase 33

Cipaglucosidase Alfa

Amicus Therapeutics

Miglustat

Johnson & Johnson

BMN 333

BioMarin Pharmaceutical

Approved5

Methotrexate

Alnylam Pharmaceuticals

Rituximab

Roche

Immune Globulin

CSL Behring

Bortezomib

Shilpa Grover, MD, MPH

Salbutamol

GSK

Analysis

Modality Breakdown

72%

Small molecule

23 of 32 classified programs — the dominant modality here.

28%ALL OTHERSProtein / enzyme722%Monoclonal antibody13%Gene therapy13%

Small molecule leads; the ring shows all 32 classified programs, each share of the total. Modality classified for 95% of 39 pipeline programs.

Analysis

Trial Status

54

clinical trials

Completed30 · 56%
Terminated7 · 13%
Active, not recruiting5 · 9%
Recruiting4 · 7%
Unknown status3 · 6%
Not yet recruiting2 · 4%
Other3 · 6%

Clinical Trials

Trial Activity

54 trials

A Global Prospective Observational Registry of Patients With Pompe Disease
Recruiting·AAmicus Therapeutics·N/A·NCT06121011·500 enrolled
A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
Recruiting·DDenali Therapeutics Inc.·Phase 1·NCT07354724·32 enrolled
Electrophysiological Study of the Phrenic Nerve and Diaphragm in Pompe Disease: Retrospective and Prospective Cohort Analysis
Recruiting·IIRCCS National Neurological Institute "C. Mondino" Foundation·N/A·NCT07664930·20 enrolled
A Phase 2, Multicenter, Randomized, Placebo-controlled, Double-blind Study to Investigate the Safety, Pharmacodynamics, and Preliminary Efficacy of S-606001 as an Add-on to Enzyme Replacement Therapy in Patients With Late-onset Pompe Disease
Recruiting·Shionogi logoShionogi·Phase 2·NCT07123155·45 enrolled
AAV2/8-LSPhGAA (ACTUS-101) in Late-Onset Pompe Disease
Active, not recruiting·AAskBio Inc·Phase 1·NCT03533673·7 enrolled
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)
Active, not recruiting·Sanofi logoSanofi·Phase 4·NCT05164055·17 enrolled
A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa
Active, not recruiting·Sanofi logoSanofi·Phase 2·NCT03019406·22 enrolled
PROPEL-2: Cipaglucosidase + Miglustat in Late-Onset Pompe
Active, not recruiting·Amgen logoAmgen·Phase 3·NCT05700001·175 enrolled
PROPEL: Cipaglucosidase alfa + Miglustat in Pompe
Active, not recruiting·Amgen logoAmgen·Phase 3·NCT04681717·123 enrolled
Higher Dose of Alglucosidase Alpha for Pompe Disease
Not yet recruiting·TTaipei Veterans General Hospital, Taiwan·N/A·NCT05017402·36 enrolled
Treatment Frequency Reduction in Pompe Disease
Not yet recruiting·EErasmus Medical Center·Phase 4·NCT06575829·10 enrolled
Extension Study of Long-term Safety and Efficacy of Myozyme in Patients With Pompe Disease Who Were Previously Enrolled in Genzyme Sponsored Enzyme Replacement Therapy (ERT) Studies
Completed·Sanofi logoSanofi·Phase 2·NCT00763932·7 enrolled
Safety and Efficacy of Albuterol in Individuals With Late-onset Pompe Disease
Completed·—·Phase 1/2·NCT01885936·16 enrolled
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
Completed·BioMarin Pharmaceutical logoBioMarin Pharmaceutical·Phase 1/2·NCT01230801·22 enrolled
Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Completed·UUniversity of Florida·Phase 1/2·NCT00976352·9 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)