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 new drug for amyloidosis called Eplontersen. This condition occurs when abnormal proteins called amyloids accumulate in various organs or tissues, causing inflammation and damage. The drug is designed to target the genetic basis of the disease and reduce the levels of amyloid protein. In this blog post, we will explore the key points surrounding the drug’s development and its potential impact on patients living with amyloidosis.

Key Points:

  1. Understanding Amyloidosis:
    Amyloidosis is a rare and complex disease characterized by the accumulation of amyloid proteins in various organs and tissues throughout the body. These abnormal proteins can lead to inflammation, tissue damage, and organ failure.
  2. The Need for a New Treatment:
    At present, there are limited treatment options available for patients with amyloidosis. The current treatments are often limited in their effectiveness and do not address the underlying genetic abnormalities that lead to the accumulation of amyloid proteins.
  3. Eplontersen – The New Drug:
    Eplontersen is a new drug developed by AstraZeneca and Ionis Pharmaceuticals that targets the production of the amyloid protein by reducing the levels of transthyretin (TTR), a protein responsible for transporting thyroid hormones throughout the body. The drug works by binding to the messenger RNA (mRNA) that produces TTR, thus blocking its production.
  4. Positive Results from Clinical Trials:
    Initial results from clinical trials of Eplontersen have been positive. The drug has been shown to significantly reduce the levels of TTR in both healthy volunteers and patients with familial amyloidosis. This reduction has led to a reduction in amyloid deposits and improvements in patients’ symptoms.
  5. Future Prospects:
    The success of the clinical trials has led to increased optimism that Eplontersen could become a significant new treatment option for patients with amyloidosis. Further research is needed to validate the drug’s efficacy and safety. However, researchers are hopeful that the drug could help slow or halt the progression of the disease and ultimately improve patients’ quality of life.
  6. Collaborative Efforts:
    The collaboration between AstraZeneca and Ionis Pharmaceuticals highlights the importance of partnerships in developing new and effective treatments for rare diseases. By combining their expertise, the companies have been able to develop Eplontersen and progress its development towards becoming a viable treatment option for amyloidosis.


The development of Eplontersen represents a significant step forward in the fight against amyloidosis. The drug’s ability to target the genetic abnormalities that lead to the accumulation of amyloid proteins and reduce their levels offers new hope for patients with this rare disease. The success of clinical trials and ongoing research highlights the benefits of collaboration and underscores the importance of continued investment in the development of new treatments for rare diseases like amyloidosis. As researchers continue to explore and refine the use of Eplontersen, we can expect greater insights into the drug’s potential impact on those affected by this debilitating condition.