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LUM
LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function.
LUM001 is a β-catenin modulator that enhances Wnt/β-catenin signaling to promote the expansion and function of intestinal stem cells and barrier function. Used for Short bowel syndrome (SBS).
At a glance
| Generic name | LUM |
|---|---|
| Also known as | lumacaftor, VX-809 |
| Sponsor | Vertex Pharmaceuticals Incorporated |
| Drug class | Tankyrase inhibitor / Wnt signaling modulator |
| Target | Tankyrase (TNKS1/TNKS2) |
| Modality | Small molecule |
| Therapeutic area | Gastroenterology |
| Phase | FDA-approved |
Mechanism of action
LUM001 works by inhibiting tankyrase, which stabilizes β-catenin and activates Wnt signaling in intestinal epithelial cells. This mechanism promotes intestinal stem cell proliferation and differentiation, enhancing intestinal barrier integrity and regeneration. It is designed to treat conditions characterized by intestinal epithelial damage or dysfunction.
Approved indications
- Short bowel syndrome (SBS)
Common side effects
- Diarrhea
- Nausea
- Abdominal pain
- Vomiting
Key clinical trials
- Platform Study to Evaluate the Efficacy and Safety of Anti-malarial Agents in Participants With Uncomplicated Plasmodium Falciparum Malaria (Cohort B2) (PHASE2)
- Investigating the Pharmacology of Tafenoquine in Papua New Guinean Children With Uncomplicated Malaria (PHASE4)
- Feasibility of the LUM Imaging System for Detection of Gastrointestinal Cancers (PHASE1, PHASE2)
- Feasibility of the LUM Imaging System for Peritoneal Surface Malignancies (PHASE1, PHASE2)
- The Remeasure Trial (NA)
- Spinal Stimulation and Mobility Devices (NA)
- Transcripts with Retained H/ACA Box SnoRNA Sequences As Biomarkers for Estrogen Dependence in Lum-B Breast Carcinomas
- Feasibility Study of Intraoperative Detection of Residual Cancer in Breast Cancer Patients (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- LUM CI brief — competitive landscape report
- LUM updates RSS · CI watch RSS
- Vertex Pharmaceuticals Incorporated portfolio CI