AstraZeneca Posts Positive Interim Data In Eplontersen Phase III Trial In ATTRv-PN - Quick Facts

(RTTNews) - British drug major AstraZeneca PLC (AZN.L, AZN) announced Tuesday that Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy or ATTRv-PN.

Based on the positive high-level results from interim analysis, AstraZeneca and Ionis (IONS) seek regulatory approval and now plan to file a new drug application or NDA with the U.S. Food and Drug Administration in 2022.

ATTRv-PN is expected to be the first indication for which AstraZeneca and Ionis will seek regulatory approval for eplontersen. The results from the 35-week interim analysis of the trial will be submitted for presentation at a forthcoming medical meeting.

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.

Eplontersen, formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense or LICA investigational medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of ATTR.

The data showed that AstraZeneca and Ionis' eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin or TTR concentration, reducing TTR protein production.

Eplontersen also reached its co-primary endpoint of change from baseline in the modified Neuropathy Impairment Score +7, a measure of neuropathic disease progression, versus external placebo group.

The trial also met its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy showing treatment with eplontersen significantly improved patient-reported quality of life versus external placebo group.

In the trial, eplontersen demonstrated a favourable safety and tolerability profile with no specific concerns.

As part of a global development and commercialisation agreement with Ionis, eplontersen will be jointly developed and commercialised by both companies in the US and will be developed and commercialised in the rest of the world by AstraZeneca, with the exception of Latin America.

Eplontersen was granted Orphan Drug Designation in the US.

It is also currently being evaluated in the CARDIO-TTRansform Phase III trial for amyloid transthyretin cardiomyopathy or ATTR-CM, a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.