AstraZeneca and Ionis have reported that the Phase III NEURO-TTRansform clinical trial of eplontersen in hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) patients met the co-primary endpoints.

A LICA investigational therapy, Eplontersen is intended to lower the production of transthyretin, or TTR protein, for treating all ATTR types.

The open-label, international, randomised trial is analysing the safety and efficacy of eplontersen in ATTRv-PN patients. 

The percent variation from baseline in serum TTR concentration and variation in the modified Neuropathy Impairment Score +7 (mNIS+7) versus the external placebo arm at week 35 were the co-primary endpoints in the interim analysis of the trial. 

mNIS+7 is a measure of neuropathic disease progression.

Findings showed that eplontersen attained a statistically significant and clinically meaningful change from baseline for serum TTR concentration, lowering the production of TTR protein.

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Furthermore, eplontersen met the other co-primary endpoint of change from baseline in mNIS+7 compared to the external placebo arm.

High-level findings also showed that the trial met the secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN).

Eplontersen treatment substantially boosted the patient-reported quality of life compared to the external placebo arm. 

In addition, it was found to have a favourable safety and tolerability profile without any particular concerns in the trial.

AstraZeneca BioPharmaceuticals R&D executive vice-president Mene Pangalos said: “Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide. 

“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.”

Based on the latest interim assessment data, AstraZeneca and Ionis intend to seek regulatory approval for eplontersen and submit a new drug application with the US Food and Drug Administration this year. 

Under a development and commercialisation agreement, eplontersen will be co-developed and co-marketed by both the companies in the US while AstraZeneca will develop and market it globally, excluding Latin America.