2022-06-22| Trials & Approvals

AstraZeneca, Ionis’ ATTR Drug Edges Closer to US Approval With Latest Trial Win

by Joy Lin
Share To

AstraZeneca and Ionis’ eplontersen, a treatment for transthyretin amyloidosis, has met its primary endpoints in the Phase 3 trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTR v-PN), according to an interim analysis. Based on the results, the companies are now eyeing a New Drug Application to the US FDA later this year. 

ATTR v-PN is a progressive disease that leads to peripheral nerve damage with motor disability. Without treatment, it is usually fatal within a decade. Eplontersen is a ligand-conjugated antisense (LICA) medicine designed to reduce the production of the misfolded TTR protein that accumulates in the peripheral nerves and organs and causes the disease. 

“Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide,” said Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca.

“These promising results show eplontersen has the potential to be a new and much needed treatment where limited options exist and significant unmet medical need remains.”

Related article: FDA Approves Alnylam’s Rare Disease RNAi Therapeutic 


Reducing TTR Protein Production and Slowing Disease Progression


The interim analysis, taken at 35 weeks, showed that eplontersen treatment led to a percent reduction in serum transthyretin (TTR) concentration in a statistically significant and clinically meaningful way, thereby reducing TTR protein production. 

The drug also reached its co-primary endpoint of change from baseline in a measure of neuropathic disease progression, versus an external placebo group. 

Patients treated with eplontersen also reported significantly improved quality of life versus placebo. In the trial, the drug showed a favorable safety and tolerability profile with no specific safety signals. 


Over $3 Billion on the Line for Eplontersen


Under terms of a deal established last year, AstraZeneca agreed to pay Ionis $200 million upfront to jointly develop and market eplontersen in the US. Furthermore, AstraZeneca will pay up to $485 million based on the regulatory progress of the drug. If approved, eplontersen could net Ionis up to $2.9 billion in sales milestones, plus royalties.

The deal also gave AstraZeneca rights to develop and market the drug in the rest of the world, except in Latin America.

© All rights reserved. Collaborate with us:
Related Post
Ionis Pharmaceuticals’ Factor XI Reduction Proved Effective in Trial
The First Approved RSV Vaccine Worldwide Belongs to AstraZeneca and Sanofi
AstraZeneca Welcomes Neurofibromatosis And Asthma Treatment Approvals in Japan
Junshi’s PD-1 Antibody Meets Endpoint In Phase 3 Lung Cancer Trial
Moderna Welcomes Phase 3 Win For RSV Vaccine
Moving Beyond COVID With mRNA Technology
Rob Knight and Jing-Yuan Fu Elaborate On Microbiome Research Trends at the 7th Asia Microbiome Conference(AMC)
CARsgen Taps Huadong to Commercialize Multiple Myeloma CAR-T in China
Scientists Study Imaging Probes in First-Ever Amputated Human Limb Model
EMA Plans to Issue Liver Failure Warning for Novartis’ Gene Therapy
Scroll to Top

Create an account with us now to say goodbye to all the pop-ups!