Our lead investigational treatment is arimoclomol—a first-in-class heat shock protein (HSP) amplifier:
- A small molecule manufactured in a capsule formulation; designed to be swallowed whole, opened to allow contents to be mixed with soft foods/liquids or delivered through a nasal gastric tube or PEG
- Designed to cross the blood brain barrier to address neurodegenerative manifestations of disease
- Safety data collected from clinical evaluation in more than 500 individuals
The first rare disease for which it is under evaluation is Niemann-Pick disease type C (NPC).
To date, Orphazyme has studied arimoclomol in seven Phase 1 trials, four Phase 2 trials, and one pivotal Phase 2/3 trial in various protein-misfolding diseases.