AstraZeneca and Ionis build case for amyloidosis drug eplontersen


AstraZeneca and Ionis Pharmaceuticals are collaborating to develop a potential breakthrough treatment for amyloidosis, a rare and often debilitating disease. The development of the drug, eplontersen, holds promising prospects in addressing this condition, which is characterized by the buildup of abnormal proteins known as amyloids in various organs. In this blog post, we will delve into key points surrounding AstraZeneca and Ionis’ case for eplontersen, shedding light on the potential implications for patients and the advancements made in the field of amyloidosis treatment.

Key Points:

1. Understanding Amyloidosis:

Amyloidosis is a rare disease characterized by the accumulation of amyloid proteins in organs and tissues, which can lead to organ dysfunction. This condition can affect various organs, including the heart, kidneys, liver, and nervous system. Amyloidosis may cause a range of symptoms, depending on the organs involved, and can be life-threatening if left untreated. Hence, there is an urgent need for effective treatments for this complex disease.

2. Collaborative Efforts of AstraZeneca and Ionis:

AstraZeneca and Ionis Pharmaceuticals have joined forces to develop eplontersen as a potential treatment for amyloidosis. The collaboration combines AstraZeneca’s expertise in drug development and commercialization with Ionis’ focus on RNA-targeted therapeutics. This strategic partnership aims to leverage their collective knowledge and resources to advance treatment options for patients suffering from amyloidosis.

3. Targeting the Root Cause of Amyloidosis:

Eplontersen, also known as ION-18, works by targeting the messenger RNA (mRNA) responsible for producing the amyloid precursor protein (APP). By inhibiting the production of APP, eplontersen seeks to reduce the levels of amyloid proteins in affected organs and potentially slow or prevent disease progression. This approach represents a significant step forward in developing a targeted therapy to address the root cause of amyloidosis.

4. Encouraging Clinical Trial Results:

The case for eplontersen is strengthened by encouraging results from clinical trials. Phase 2 studies have demonstrated reductions in amyloid proteins, suggesting that eplontersen has the potential to effectively treat amyloidosis. The positive outcomes observed in these trials have paved the way for further development and testing of the drug, providing hope for patients living with this challenging disease.

5. Potential Implications for Patients:

The development of eplontersen holds significant implications for patients with amyloidosis. If the drug proves effective and gains regulatory approval, it could potentially offer a new treatment modality for managing this rare disease. Eplontersen has the potential to slow or halt disease progression, alleviate symptoms, and improve quality of life for individuals affected by amyloidosis, providing hope for a brighter future.

6. Advancements in Amyloidosis Treatment:

The case for eplontersen represents a broader trend of advancements in amyloidosis treatment. While the disease has traditionally been challenging to treat, recent years have seen significant progress in the development of targeted therapies. The collaboration between AstraZeneca and Ionis showcases the commitment of pharmaceutical companies and researchers to address unmet medical needs in the field of rare diseases, driving innovation, and improving outcomes for patients.


The collaborative efforts of AstraZeneca and Ionis Pharmaceuticals in building a case for eplontersen offer renewed hope for patients with amyloidosis. This potential breakthrough drug, targeting the root cause of the disease by reducing amyloid protein levels, has the potential to transform the treatment landscape for this rare condition. The encouraging clinical trial results and advancements in amyloidosis treatment underscore the dedication to improving the lives of individuals affected by this complex disease. As research continues, patients and healthcare professionals eagerly anticipate the next steps in the development of eplontersen and its potential impact on amyloidosis treatment.