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Recently, AstraZeneca announced that it has reached a global development and commercialization agreement with Ionis Pharmaceuticals for eplontersen
.
Eplontersen is an antisense oligonucleotide (ASO) therapy under development using Ionis ligand-coupled antisense (LICA) technology.
In ATTR patients, mutant and wild-type TTR proteins will form fibrils in human tissues, such as peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow, 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
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.
▲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)
According to the terms of the agreement, AstraZeneca will pay Ionis an advance payment of US$200 million, and Ionis is also eligible for future potential milestone payments totaling more than US$3.
5 billion
.
It is expected that genetic ATTR-PN will become the first indication for eplontersen, and it is expected to submit a new drug application (NDA) to the US FDA before the end of 2022
Original link: https://
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