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▎Editor of WuXi AppTec Content Team On December 29, 2021, Ionis Pharmaceuticals announced the completion of the cooperation agreement with AstraZeneca on eplontersen
.
Eplontersen is an antisense oligonucleotide (ASO) therapy under development using Ionis ligand-coupled antisense (LICA) technology.
It reduces the production of transthyretin (TTR) to treat hereditary and non-hereditary 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 will form fibrils in human tissues, which will damage the normal function of the tissues
.
The increase of TTR protein fibrils will lead to more tissue damage and disease deterioration, which will reduce the quality of life of patients and eventually die
.
Globally, there are an estimated 300,000 to 500,000 ATTR-CM patients, and 10,000 to 40,000 ATTR-PN patients
.
ATTR-CM is a systemic progressive fatal disease that can cause progressive heart failure and death within four years after diagnosis
.
Hereditary ATTR-PN can cause peripheral nerve damage and dyskinesia within five years after diagnosis.
If the patient does not receive timely and effective treatment, it will usually be fatal within ten years
.
The single-stranded DNA molecules of ASO drugs can cause degradation of mRNA or change the splicing of mRNA by binding to specific RNA sequences, thereby achieving the effect of targeting the underlying disease mechanism at the mRNA level and treating the disease
.
Ionis' LICA technology platform can improve the characteristics of ASO drugs in many ways by coupling different small molecules to ASO.
Not only allows them to be administered in a variety of ways, but also can 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 (picture source: Ionis official website) In addition, Ionis also predicts that genetic ATTR-PN will become eplontersen It is expected to submit a New Drug Application (NDA) to the US FDA before the end of 2022
.
Reference: [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:// releases/2021/astrazeneca-and-ionis-close-eplontersen-deal.
html[3] Astrazeneca And Ionis Sign Deal To Develop And Commercialise Eplontersen.
Retrieved December 7, 2021, from https:// astraz/media-centre/press-releases/2021/astrazeneca-ionis-to-collaborate-on-eplontersen.
htmlDisclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.
This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a treatment recommendation either
.
If you need guidance on the treatment plan, please go to a regular hospital for treatment
.
.
Eplontersen is an antisense oligonucleotide (ASO) therapy under development using Ionis ligand-coupled antisense (LICA) technology.
It reduces the production of transthyretin (TTR) to treat hereditary and non-hereditary 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 will form fibrils in human tissues, which will damage the normal function of the tissues
.
The increase of TTR protein fibrils will lead to more tissue damage and disease deterioration, which will reduce the quality of life of patients and eventually die
.
Globally, there are an estimated 300,000 to 500,000 ATTR-CM patients, and 10,000 to 40,000 ATTR-PN patients
.
ATTR-CM is a systemic progressive fatal disease that can cause progressive heart failure and death within four years after diagnosis
.
Hereditary ATTR-PN can cause peripheral nerve damage and dyskinesia within five years after diagnosis.
If the patient does not receive timely and effective treatment, it will usually be fatal within ten years
.
The single-stranded DNA molecules of ASO drugs can cause degradation of mRNA or change the splicing of mRNA by binding to specific RNA sequences, thereby achieving the effect of targeting the underlying disease mechanism at the mRNA level and treating the disease
.
Ionis' LICA technology platform can improve the characteristics of ASO drugs in many ways by coupling different small molecules to ASO.
Not only allows them to be administered in a variety of ways, but also can 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 (picture source: Ionis official website) In addition, Ionis also predicts that genetic ATTR-PN will become eplontersen It is expected to submit a New Drug Application (NDA) to the US FDA before the end of 2022
.
Reference: [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:// releases/2021/astrazeneca-and-ionis-close-eplontersen-deal.
html[3] Astrazeneca And Ionis Sign Deal To Develop And Commercialise Eplontersen.
Retrieved December 7, 2021, from https:// astraz/media-centre/press-releases/2021/astrazeneca-ionis-to-collaborate-on-eplontersen.
htmlDisclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
.
This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
.
This article is not a treatment recommendation either
.
If you need guidance on the treatment plan, please go to a regular hospital for treatment
.
