On December 29, 2021, Ionis Pharmaceuticals announced the completion of a collaboration agreement with AstraZeneca on eplontersen
.
Eplontersen is an investigational antisense oligonucleotide (ASO) therapy developed using Ionis Ligand Conjugated Antisense (LICA) technology to treat both hereditary and non-genetic Sexual TTR amyloidosis (ATTR)
.
The drug is currently in Phase 3 clinical development for the treatment of amyloid TTR cardiomyopathy (ATTR-CM) and amyloid TTR polyneuropathy (ATTR-PN)
.
In ATTR patients, mutant and wild-type TTR proteins form fibrils in human tissues, which in turn disrupt normal tissue function
.
The increase in TTR protein fibrils leads to more tissue damage and disease progression, resulting in reduced quality of life and eventual death
.
Globally, there are an estimated 300,000 to 500,000 ATTR-CM patients, and 1 to 40,000 ATTR-PN patients
.
ATTR-CM is a systemic, progressive and fatal disease that can lead to progressive heart failure and death within four years of diagnosis
.
Inherited ATTR-PN can lead to peripheral nerve damage and movement disorders within five years of diagnosis, and is often fatal within ten years if patients are not treated promptly and effectively
.
The single-stranded DNA molecule of ASO drugs can lead to the degradation of mRNA or change the splicing of mRNA by binding to a specific RNA sequence, so as to achieve the effect of targeting the underlying disease mechanism at the mRNA level and treating the disease
.
By coupling different small molecules to ASOs, Ionis' LICA technology platform can improve the characteristics of ASO drugs in many ways, allowing them not only to be administered by multiple methods, but also to be delivered to different organs and tissues of the human body
.
▲Ionis' new generation LICA technology can deliver ASO to various organs and tissues of the human body by coupling ASO with different ligands (Image source: Ionis official website)
In addition, Ionis also expects hereditary ATTR-PN to be the first indication for eplontersen and is expected to submit a New Drug Application (NDA) to the US FDA by the end of 2022
.
References:
[1] Ionis And Astrazeneca Close Deal To Develop And Commercialize Eplontersen.
Retrieved December 29, 2021, from https://ir.
ionispharma.
com/news-releases/news-release-details/ionis-and-astrazeneca-close-deal -develop-and-commercialize
[2] Astrazeneca And Ionis Close Agreement To Develop And Commercialise Eplontersen.
Retrieved December 29, 2021, from https:// -close-eplontersen-deal.
html
[3] Astrazeneca And Ionis Sign Deal To Develop And Commercialise Eplontersen.
Retrieved December 7, 2021, from https:// -collaborate-on-eplontersen.
html
