In short
Eplontersen sodium is an antisense oligonucleotide medicine that reduces production of the transthyretin protein in the liver. It is authorised in the United States under the brand name Wainua for certain forms of hereditary transthyretin-mediated amyloidosis. One registered clinical trial is currently authorised to study eplontersen in combination with another investigational antibody for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), with a planned enrollment of 326 participants in.
Key points
- Eplontersen sodium is an antisense oligonucleotide that lowers transthyretin protein production.
- It is authorised in the United States as Wainua for hereditary transthyretin-related amyloidosis.
- The registered trial is a Phase 2 study in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
- The trial plans to enroll 326 adult participants and is currently authorised but not yet recruiting.
- The main measure is change in peak oxygen consumption during exercise over 52 weeks.
- Eplontersen is given by subcutaneous injection using a pre-filled pen.
What is eplontersen sodium?
Eplontersen sodium is a medicine that belongs to a class of drugs called antisense oligonucleotides. These are short, chemically modified pieces of genetic material designed to bind to a specific messenger RNA and stop it from being used to make a protein. Eplontersen is listed in the United States under the brand name Wainua and is also classified among miscellaneous metabolic agents.
The substance is used in the treatment of hereditary transthyretin-related amyloidosis, a condition in which an abnormal form of the transthyretin protein builds up and damages nerves and other tissues. Eplontersen is not a cure; it works by reducing the amount of the disease-causing protein the body produces.
How does eplontersen sodium work?
Eplontersen is an antisense oligonucleotide. It binds to the messenger RNA that carries the instructions for making transthyretin, a protein produced mainly in the liver. By binding to that RNA, the medicine triggers the cell to break it down before the protein can be made. Less transthyretin in the blood means less of the abnormal protein is available to form amyloid deposits.
In the registered clinical trial, eplontersen is being studied together with ALXN2220, a monoclonal antibody that targets amyloid transthyretin. The trial compares the combination of eplontersen plus ALXN2220 against eplontersen plus placebo, so every participant receives eplontersen as the background treatment.
Which conditions is eplontersen sodium used or studied for?
- Hereditary transthyretin-related amyloidosis: an inherited condition in which abnormal transthyretin protein damages nerves and organs. Eplontersen is authorised in the United States for treating this condition.
- Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM): a form of heart disease caused by amyloid transthyretin deposits in the heart muscle. The registered clinical trial studies eplontersen in this condition.
How is eplontersen sodium given?
Eplontersen is given as a solution for injection under the skin, using a pre-filled pen. The product studied in the trial is Wainzua 45 mg solution for injection in a pre-filled pen for subcutaneous use.
What do the clinical trials show?
One interventional trial is registered for eplontersen in the EU Clinical Trials Information System. It is a Phase 2 study in adult participants with transthyretin-mediated amyloid cardiomyopathy. The trial is currently authorised, meaning it has received regulatory approval to proceed but has not yet reported results.
The study plans to enroll 326 participants. Its main outcome measure is the change from baseline in peak oxygen consumption during a cardiopulmonary exercise test, known as CPET peak VO2, over 52 weeks. This measure reflects how well the heart and lungs deliver oxygen to the body during exercise.
The trial has two arms. One arm receives eplontersen plus ALXN2220, a recombinant human anti-ATTR immunoglobulin G1 monoclonal antibody given intravenously. The other arm receives eplontersen plus placebo for ALXN2220. All participants therefore receive eplontersen as background therapy.
Because the trial is authorised but has not reported results, no efficacy or safety findings from this study are available yet. The registry record describes the design and planned measurements rather than outcomes.
What side effects are known?
The available sources for this page do not provide a list of side effects for eplontersen sodium. The registered trial is still in its authorised stage, and no results have been published in the registry record.