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

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.

Author

Joy Lin

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