Coramitug

Table of contents

• What is coramitug?
• How does coramitug work?
• Which conditions is coramitug used or studied for?
• How is coramitug given?
• What do the clinical trials show?
• What side effects are known?

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.^[5] It is also known by its development code NNC6019-0001 and was formerly called PRX004.^[4] 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.^[1] The company developing it is Novo Nordisk A/S.^[1] Coramitug has received orphan designation, a status given to treatments for rare diseases.^[1]

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.^[4] 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.^[4] 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).^[1][2][3]

How is coramitug given?

Coramitug is given as a solution for infusion by the intravenous route, meaning it is delivered directly into a vein.^[1] In the completed Phase 2 trial, a dose of 60 mg/kg was studied.^[4]

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.^[2] 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.^[2] 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.^[4] A second authorised Phase 2 trial (2022-502605-15-00) is assessing long-term safety and tolerability over 156 weeks in 89 participants.^[1] 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.^[3]

What side effects are known?

In the Phase 2 trial, coramitug was generally well tolerated.^[4] The long-term safety trial is specifically measuring the number of treatment-emergent adverse events from baseline up to week 156.^[1]