2022-06-22| Trials & Approvals

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

by Joy Lin
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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.

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