Trial shows AstraZeneca-Ionis drug shown to be effective in treating rare nerve disease


Detailed results from a Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy showed AstraZeneca and Ionis’ eplontersen showed positive results.

The condition is caused by aging or genetic mutations that affect nerves and affects about 10,000 worldwide. The disease leads to nerve damage with motor (limb) disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

The results were presented in an Emerging Science Session at the American Academy of Neurology (AAN) 2023 Annual Meeting in Boston.

At 66 weeks, patients treated with eplontersen had consistent and sustained benefits in slowing the disease and showed no adverse effects.

Mene Pangalos, executive vice president, of BioPharmaceuticals R&D, AstraZeneca, said: “Without treatment, hereditary transthyretin-mediated amyloid polyneuropathy is a relentlessly progressive disease. These results show that eplontersen sustains reduced transthyretin levels and improves neuropathy progression and quality of life consistently across a substantial number of patients. We are confident in eplontersen’s potential to be a much-needed and differentiated treatment option for patients living with all types of this devastating disease, which can also lead to heart failure.”


As part of a global development and commercialization agreement, AstraZeneca and Ionis are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the US and plan to seek regulatory approval in Europe and other parts of the world. The US Food and Drug Administration accepted a New Drug Application for eplontersen for the treatment of ATTRv-PN.3 Eplontersen was granted Orphan Drug Designation in the US.

Eplontersen is currently being evaluated in a late-stage trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), progressive a fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset. About 7,000 people annually are diagnosed with ATTR-CM.

Ionis is based in Carlsbad, CA. AstraZeneca, based in the United Kingdom, employs 1,500 in northern Delaware in non-research positions.

The two companies earlier signed an agreement worth a potential $3.6 billion to Ionis based on R&D and sales milestones. Treatment with a current drug used to transthyretin-mediated amyloid polyneuropathy runs upwards of half a million dollars annually