adrabetadex
Phase 1- Indications
- Niemann-Pick Type C1 disease
Company intelligence / Profile preview
Mandos Health
Los Angeles, California, USAmandoshealth.com ↗
- Ownership
- Private
- Development stage
- Phase 1
01
Overview
Mandos Health leads the global development of adrabetadex for NPC1 after acquiring it from Mallinckrodt. The drug remains investigational as prior studies did not result in FDA approval. The company continues expanded access programs, collaborates with patient registries for real-world evidence, and plans further regulatory engagement.
- Therapeutic areas
- Metabolic Disorders
- Modalities
- Small molecules (adrabetadex/VTS‑270)
- Industry
- Biotech
02
Drug pipeline
1 asset03
Partnerships
Mallinckrodt Pharmaceuticals/Vtesse LLC – asset transfer/collaboration during transition of adrabetadex clinical responsibilitiesInternational Niemann-Pick Disease Registry (INPDR) – collaboration for real-world evidence generation using registry data to inform development strategy
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