AstraZeneca and Ionis build case for amyloidosis drug eplontersen

Title: AstraZeneca and Ionis Build Case for Amyloidosis Drug Eplontersen


AstraZeneca and Ionis Pharmaceuticals are collaborating on the development of a potential game-changer in the treatment of amyloidosis called eplontersen. This drug aims to address the underlying cause of amyloidosis, a rare progressive disease caused by the buildup of amyloid proteins in various organs, leading to organ damage and potentially life-threatening complications. In this blog post, we will focus on the key points surrounding the development of eplontersen and the potential impact it may have in treating amyloidosis.

Key Points:

  1. Understanding Amyloidosis:

Amyloidosis is a rare condition characterized by the accumulation of abnormal amyloid proteins in tissues and organs throughout the body. These amyloid deposits can impair organ function over time, leading to a wide range of symptoms and complications. There are several types of amyloidosis, including AL amyloidosis, ATTR amyloidosis, and AA amyloidosis, each with their unique underlying causes.

  1. The Promise of Eplontersen:

Eplontersen is an investigational drug being developed by AstraZeneca and Ionis Pharmaceuticals specifically for ATTR amyloidosis. ATTR amyloidosis is caused by a faulty version of the transthyretin (TTR) protein, which misfolds and clumps together to form amyloid deposits in various organs. Eplontersen aims to target the production of the faulty TTR protein, potentially slowing or halting the progression of the disease and reducing organ damage.

  1. The Drug Development Process:

AstraZeneca and Ionis are currently conducting clinical trials to evaluate the safety and efficacy of eplontersen for the treatment of ATTR amyloidosis. Early results from phase 2/3 trials have shown promising outcomes, with eplontersen demonstrating the ability to significantly reduce levels of abnormal TTR protein. These positive results are building a strong case for the drug’s potential as a groundbreaking treatment for amyloidosis.

  1. Potential Impact on Patients:

If approved, eplontersen has the potential to transform the treatment landscape for amyloidosis. By targeting the root cause of the disease, rather than simply managing symptoms, eplontersen may offer patients the opportunity for improved outcomes and a higher quality of life. Slowing or halting the progression of amyloid deposits could help preserve organ function and potentially prevent the need for more invasive interventions, such as organ transplantation.

  1. The Future of Amyloidosis Treatment:

The development of eplontersen and the ongoing research into amyloidosis treatment highlight the significant advancements being made in the field. Targeted therapies like eplontersen offer hope for patients with amyloidosis and illustrate the importance of addressing the underlying causes of diseases. As our understanding of amyloidosis and its various forms improves, we can anticipate further developments in treatment options that may pave the way for more effective and personalized approaches to managing this complex disease.


The collaboration between AstraZeneca and Ionis Pharmaceuticals to develop eplontersen brings hope to individuals with amyloidosis, specifically ATTR amyloidosis. By targeting the production of the faulty TTR protein, eplontersen has the potential to slow or halt disease progression and reduce organ damage. The positive outcomes from clinical trials suggest that this investigational drug could be a game-changer in the treatment of amyloidosis, offering improved outcomes and enhanced quality of life for patients. The development of eplontersen represents an exciting advancement in the field of amyloidosis treatment, and it highlights the importance of addressing the underlying causes of diseases to revolutionize patient care.