About Heat Shock Proteins (HSPs)

At Orphazyme, our work is focused on the treatment of rare neurodegenerative diseases by harnessing the cell protective properties of the heat shock response, a natural defense mechanism system present in all our cells.

The heat shock response protects cells from the accumulation of misfolded proteins, aggregated proteins, and dysfunctional lysosomes (the cell’s recycling system), which could otherwise lead to toxicity and disease. The heat shock response is generated through the production of HSPs.

About Arimoclomol

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 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, arimoclomol has been studied in seven Phase 1 trials, four Phase 2 trials, and three pivotal Phase 2/3 trial in various protein-misfolding diseases.

If arimoclomol is approved for NPC and additional diseases, we plan to leverage our rare disease commercial infrastructure and expertise to efficiently address those patient populations. We may also opportunistically seek strategic collaborations in certain disease areas or geographies.

About Heat Shock Proteins (HSPs)

About Arimoclomol

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