Our Focus

Orphazyme is a late-stage biopharmaceutical company pioneering the heat shock protein (HSP) response for the treatment of neurodegenerative orphan diseases. We have clinical trial programs underway in four areas to explore the potential of our lead investigational treatment, arimoclomol.

Heat shock proteins, such as heat shock protein 70 (HSP70), are a group of endogenous stress-response proteins that protect against cellular toxicity caused by misfolded proteins, protein aggregation, and lysosomal dysfunction. Our product candidate arimoclomol is designed to selectively amplify HSPs.

The U.S. Food and Drug Administration (FDA) has accepted our new drug application (NDA) for arimoclomol for the treatment of Niemann-Pick disease Type C (NPC) and designated it for priority review. We submitted a marketing authorization application to the European Medicines Agency (EMA) for NPC in November 2020.

We also have a registrational trial ongoing in amyotrophic lateral sclerosis (ALS) and intend to advance arimoclomol into pivotal-stage clinical development in neurological Gaucher disease. On March 29, 2021, the Company reported topline results from the Phase 2/3 trial in IBM; arimoclomol did not meet the primary and secondary endpoints in the Phase 2/3 trial in IBM. Additional analyses are being performed and the Company expects to present further information at an upcoming scientific forum.

ARIMOCLOMOL: PIPELINE-IN-A-PRODUCT POTENTIAL

Scroll over the pipeline chart to read more about each disease area.

DESIGNATIONS
STAGE OF DEVELOPMENT
ANTICIPATED MILESTONES
Orphan Drug
Fast Track
BTD**
PC
Ph1
Ph2
Ph3
Filed
Anticipated Milestones
Niemann-Pick disease type C*

DESIGNATIONS

Orphan Drug
Fast Track
BTD**
Orpha Drug
Orpha Drug
Orpha Drug
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
Ph 2/3 (data reported)
ANTICIPATED MILESTONES

U.S. FDA accepted New Drug Application (NDA) for review; Submitted MAA (EU) 2020

Niemann-Pick disease type C (NPC) is a rare progressive neurodegenerative disease. It belongs to the family of lysosomal storage diseases. Niemann-Pick disease type C can be difficult to recognize, which leads to diagnostic delays. There are no approved treatments in the United States for Niemann-Pick disease type C.
Gaucher disease***

DESIGNATIONS

None

Orphan Drug
Fast Track
BTD**
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
Ph 2 (top-line
data reported)
ANTICIPATED MILESTONES

Gaucher disease is a rare, inherited lysosomal storage disorder causing certain sugar-containing fats to abnormally accumulate in the lysosomes of cells, especially within cells of the blood system and nerve cells, thereby affecting organs such as the brain, bone marrow, spleen, and liver. The typical systemic symptoms of Gaucher disease, which can appear at any age, include an abnormally enlarged liver and/or spleen and low levels of circulating red blood cells and platelets. Currently no treatments are available that can cross the blood brain barrier to help reduce neurological symptoms.
Amyotrophic lateral sclerosis

DESIGNATIONS

Orphan Drug
Fast Track
BTD**
Orpha Drug
Orpha Drug
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
Ph 3 (registrational)
ANTICIPATED MILESTONES

Top-line results
H1 2021

Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurological disease. ALS attacks neurons responsible for controlling voluntary muscles—resulting in muscle weakness in limbs—and impacts speaking, chewing, swallowing, and breathing, leading to progressive disability and eventually death—typically from respiratory failure and aspiration pneumonia. Currently, there are limited treatment options available.
Inclusion body myositis

DESIGNATIONS

Orphan Drug
Fast Track
BTD**
Orpha Drug
Orpha Drug
STAGE OF DEVELOPMENT
PC
Ph1
Ph2
Ph3
Filed
Ph 2/3 (registrational)
ANTICIPATED MILESTONES

Top-line results reported on March 29, 2021; did not meet primary and secondary endpoints in IBM

Inclusion body myositis (IBM) is a progressively debilitating muscle-wasting disease. IBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability. There are no approved treatments for IBM.
*Early-access program is underway at multiple sites; **Breakthrough Therapy Designation (BTD);
***Type 1 and Type 3 Gaucher disease.
Rare pediatric disease designation