In short
Coramitug (also known as NNC6019-0001) is an investigational monoclonal antibody being studied for transthyretin amyloid cardiomyopathy (ATTR-CM), a heart condition caused by abnormal protein deposits. It has been evaluated in two Phase 2 trials, one completed with 99 participants and one authorised with 89 participants, and is now in a Phase 3 trial with 1,280 participants. Coramitug has no marketing authorisation and is given intravenously as a solution for infusion.
Key points
- Coramitug is a humanized monoclonal antibody that targets misfolded transthyretin protein.
- It is being studied in transthyretin amyloid cardiomyopathy (ATTR-CM), including hereditary and wild-type forms.
- A completed Phase 2 trial enrolled 99 participants and compared two dose levels against placebo.
- A Phase 3 trial (CLEOPATTRA) is authorised and aims to enrol 1,280 participants.
- In the Phase 2 trial, coramitug significantly reduced NT-proBNP levels at 52 weeks compared with placebo.
- Coramitug has orphan designation and is given as an intravenous infusion.
What is coramitug?
Coramitug is a humanized monoclonal antibody — a protein-based medicine designed in the laboratory to recognise and attach to a specific target in the body. It is also known by its development code NNC6019-0001 and was formerly called PRX004. Coramitug is an investigational substance: the product records from the EU Clinical Trials Information System show no marketing authorisation, meaning it is not yet approved as a medicine. The company developing it is Novo Nordisk A/S. Coramitug has received orphan designation, a status given to treatments for rare diseases.
How does coramitug work?
Coramitug is directed against misfolded transthyretin, a protein that can fold incorrectly and build up as amyloid deposits in the heart. By attaching to these abnormal protein forms, coramitug is designed to promote their clearance by the body's immune cells — an approach described as amyloid-depleting therapy. The aim is to remove the amyloid fibrils that damage heart muscle in transthyretin amyloid cardiomyopathy.
Which conditions is coramitug used or studied for?
- Transthyretin amyloid cardiomyopathy (ATTR-CM) — a heart condition where amyloid deposits stiffen the heart muscle, leading to heart failure. The trials include both the hereditary form (ATTRv-CM) and the wild-type form (ATTRwt-CM).
How is coramitug given?
Coramitug is given as a solution for infusion by the intravenous route, meaning it is delivered directly into a vein. In the completed Phase 2 trial, a dose of 60 mg/kg was studied.
What do the clinical trials show?
Three clinical trials of coramitug are registered in the EU Clinical Trials Information System. The completed Phase 2 trial (2023-506824-96-00) enrolled 99 participants with established ATTR-CM and compared two dose levels of coramitug against placebo. Its co-primary endpoints were the change in 6-minute walk test (6MWT) — a measure of how far a person can walk in six minutes — and the change in NT-proBNP, a blood marker of heart strain, from baseline to week 52. In the published Phase 2 trial, treatment with 60 mg/kg coramitug significantly reduced NT-proBNP levels at 52 weeks compared with placebo, though no change was observed in the six-minute walk distance. A second authorised Phase 2 trial (2022-502605-15-00) is assessing long-term safety and tolerability over 156 weeks in 89 participants. The authorised Phase 3 trial CLEOPATTRA (2024-518899-31-00) aims to enrol 1,280 participants and is measuring a composite endpoint of cardiovascular death and recurrent cardiovascular events such as cardiovascular hospitalisation and urgent heart failure visits.
What side effects are known?
In the Phase 2 trial, coramitug was generally well tolerated. The long-term safety trial is specifically measuring the number of treatment-emergent adverse events from baseline up to week 156.