AstraZeneca and Ionis’ ATTRv-PN therapy meets trial endpoints

Title: AstraZeneca and Ionis’ ATTRv-PN Therapy Meets Trial Endpoints


Amyloidosis is a rare and debilitating disease that affects multiple organ systems and impairs patients’ quality of life. One form of amyloidosis, hereditary transthyretin-mediated amyloidosis (hATTR), is caused by the accumulation of misfolded proteins in the tissues and organs of affected individuals. In an exciting development, AstraZeneca and Ionis have announced that their ATTRv-PN therapy has met trial endpoints, demonstrating the potential of the therapy to improve patient outcomes in hATTR. In this blog, we will delve into the key points surrounding AstraZeneca and Ionis’ recent trial and the potential impact of ATTRv-PN on the treatment of hATTR.

Key Points:

  1. Understanding hATTR and its Effects:

hATTR is a hereditary form of amyloidosis caused by the abnormal aggregation of transthyretin (TTR) protein in the body. The accumulation of misfolded TTR protein leads to damaging deposits in various organs and tissues, including the heart, nerves, and digestive system. hATTR can cause a range of symptoms, including pain, numbness, tingling, diarrhea, and vomiting, and can lead to organ damage, disability, and reduced life expectancy.

  1. ATTRv-PN Therapy and its Mechanism of Action:

ATTRv-PN is a promising therapy developed by AstraZeneca and Ionis to target TTR accumulation in hATTR. The therapy works by using Ionis’ antisense technology to reduce the production of TTR protein in the liver, slowing the disease’s progression and potentially preventing further organ damage in patients.

  1. Results of the Recent Trial:

The results of the ATTRv-PN Phase 2/3 clinical trial showed promising outcomes for patients with hATTR. The trial met its primary endpoint, demonstrating a significant reduction in the TTR protein levels in patients receiving the therapy compared to those receiving a placebo. The therapy also showed improvement in multiple secondary endpoints, including improvements in neuropathy, quality of life, and gait speed.

  1. Potential Impact on hATTR Treatment:

The successful completion of the Phase 2/3 trial represents a significant milestone in hATTR treatment and offers hope to patients with this debilitating disease. The reduction in TTR protein levels and improvement in neuropathy, gait speed, and quality of life observed in the trial indicate that ATTRv-PN has the potential to slow or halt the progression of hATTR and prevent further organ damage in patients.

  1. Ongoing Research and Collaboration:

While the results of the ATTRv-PN trial are promising, continued research and collaboration are needed to optimize the therapy’s effectiveness and bring it to market. Further clinical trials and collaborations with patient organizations and healthcare professionals can help researchers gain insights into patient experiences and develop more effective approaches to managing hATTR.


hATTR is a debilitating disease that can significantly impair patients’ quality of life and life expectancy. The promising results of AstraZeneca and Ionis’ ATTRv-PN therapy offer hope to patients with this condition, demonstrating the potential to slow or halt the progression of the disease and prevent further organ damage. Continued research and collaboration are crucial to optimize the therapy’s effectiveness and bring novel approaches to managing hATTR to market. The advancements made through this research could offer a lifesaving therapy to millions affected by hATTR worldwide.