Clinical Trials

AstraZeneca and Ionis have announced positive results from their phase 3 NEURO-TTRansform trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), showing that the companies’ drug eplontersen met all primary and secondary endpoints at 66 weeks compared to an external placebo group.

At the 66-week point, patients treated with eplontersen showed consistent and sustained benefits on the three co-primary endpoints, which were serum transthyretin (TTR) concentration, neuropathy impairment and quality of life (QoL).

Full results were reported at the Emerging Science Session at the American Academy of Neurology (AAN) 2023 Annual Meeting in Boston, US, and the companies are now seeking approval for the drug in the US, having already had a new drug application accepted by the US Food and Drug Administration (FDA).

Sami Khella MD, chief of the Department of Neurology at Penn Presbyterian Medical Center, professor of Clinical Neurology at the Perelman School of Medicine at the University of Pennsylvania School of Medicine and a principal investigator on the NEURO-TTRansform trial, commented: “In the past, patients with hereditary transthyretin amyloid polyneuropathy usually deteriorated given the limited available treatments. This new study shows eplontersen can halt progression of neuropathy and improve quality of life at 66 weeks when compared to placebo. Today’s important results demonstrate that eplontersen has a consistent and sustained treatment effect and reinforces its potential as an important medicine for the thousands of patients living with this debilitating and fatal disease.”

Mene Pangalos, executive vice president of BioPharmaceuticals R&D at AstraZeneca, added: “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.”