IB1001

Phase 3Small molecule

MMedexus Pharma, Inc.

IB1001 is one of 38 small molecule programs targeting GM2 gangliosidosis. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

GM2 GangliosidosisActive, not recruiting

Niemann-Pick DiseaseActive, not recruiting

Hemophilia BActive, not recruiting

Ataxia-TelangiectasiaActive, not recruiting

GM2 GangliosidosisPhase 2

Active, not recruiting

Niemann-Pick DiseasePhase 2

Active, not recruiting

Hemophilia BPhase 3

Active, not recruiting

Ataxia-TelangiectasiaPhase 2

Active, not recruiting

Competition

Competitive Landscape

42 competitors in Hemophilia B

#DrugCompanyPhaseTrials

Recombinant Factor IX Coagulation

Pfizer

Approved

2 2

IB1001

MMedexus Pharma, Inc.

Phase 3

3 3

Translarna

BENEFIX

6 5

Recombinant Factor IX

BBaxalta now part of Shire

Phase 3

1 6

Factor Ix

CSL Behring

Phase 3

3 7

Rfix

Pfizer

Phase 3

3 8

Nonacog Alfa

Pfizer

Phase 3

3 9

Rix-FP

CSL Behring

Phase 3

3 10

Rfixfc

BBioverativ Therapeutics Inc.

Phase 3

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

Clinical

Trials by Indication[1]

Hemophilia B3 trials

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Subjects With Hemophilia B

Withdrawn·CCangene Corporation·Phase 3·NCT02048111

Study of Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects With Hemophilia B

Terminated·MMedexus Pharma, Inc.·Phase 3·NCT01271868·9 enrolled

Study of Recombinant Factor IX Product, IB1001, in Subjects With Hemophilia B

Completed·MMedexus Pharma, Inc.·Phase 2/3·NCT00768287·77 enrolled

GM2 gangliosidosis1 trial

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

Completed·IIntraBio Inc·Phase 2·NCT03759665·30 enrolled

Niemann-Pick disease1 trial

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Completed·IIntraBio Inc·Phase 2·NCT03759639·33 enrolled

Ataxia-telangiectasia1 trial

N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)

Terminated·IIntraBio Inc·Phase 2·NCT03759678·17 enrolled

Sources

References

[1]
ClinicalTrials.gov — 6 trial records for IB1001. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — IB1001 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for IB1001. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Medexus Pharma, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: MMedexus Pharma, Inc.

IB1001

Status by Indication[3][1]

Competitive Landscape

Trials by Indication[1]

Hemophilia B3 trials

GM2 gangliosidosis1 trial

Niemann-Pick disease1 trial

Ataxia-telangiectasia1 trial

References

Drug Info

Quick Facts

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