Eplontersen Meets Co-Primary Endpoint in Phase 3 Trial for ATTRy-PN


The drug from AstraZeneca and Ionis Pharmaceuticals demonstrates a sustained benefit through 66 weeks for those with hereditary transthyretin-mediated amyloid polyneuropathy.

Pharmacists checking inventory at hospital pharmacy | Image Credit: Jacob Lund - stock.adobe.com

Jacob Lund - stock.adobe.com

Eplontersen from AstraZeneca and Ionis Pharmaceuticals met its co-primary endpoints through 66 weeks for those with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), according to results from the Neuro-TTRansform (NCT04136184) phase 3 trial.

“The positive results from the 66-week analysis of the phase 3 NEURO-TTRansform trial show that eplontersen provided consistent and sustained transthyretin protein reduction and that a substantial number of patients improved in measures of both neuropathy progression and quality of life,” Sami Khella, MD, chief of the department of neurology at Penn Presbyterian Medical Center, said in a statement. “This builds on the favorable 35-week results, which first demonstrated eplontersen’s potential to significantly improve outcomes in this underserved population.”

Investigators found that individuals treated with eplontersen continued to demonstrate a clinically meaningful and statistically significant change of modified Neuropathy Impairment Score +7, a measurement of neuropathic disease progression, and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.

The trial also met its third co-primary endpoint, demonstrating a statistically significant reduction in serum TTR concentration compared with the external placebo group. The reduction was consistent with those reported at 35 weeks.

“These results further underscore eplontersen’s potential to be a best-in-class treatment across all forms of transthyretin-mediated amyloidosis, including polyneuropathy and cardiomyopathy, which can lead to heart failure. With limited treatment options currently available, there is an urgent unmet medical need for new therapies and earlier, accurate diagnosis across the different types of this systemic, progressive and fatal condition,” Mene Pangalos, PhD, FRSB, FMedSci, executive vice president of BioPharmaceuticals R&D at AstraZeneca, said in the statement.

Furthermore, eplontersen continued to demonstrate a consistent safety and tolerability profile.

In the Neuro-TTRansform trial, individuals were compared with the external placebo group, who were from the NEURO-TTR (NCT01737398) registrational trial completed in 2017. The final analysis compared eplontersen with the external placebo group and was completed at week 66 and followed up on the individuals until week 85, when the participants will have the option to transition to the open-label extension study of eplontersen.

AstraZeneca and Ionis are seeking regulatory approval for eplontersen for the treatment of ATTRv-PN in the United States, as well as Europe and other parts of the world.

This month, the FDA accepted a new drug application for eplontersen for the treatment of ATTRv-PN, and granted it orphan drug designation in the United States.

Additionally, eplontersen is being evaluated in the phase 3 CARDIO-TTRansform (NCT04136171) trial for individuals with transthyretin amyloid cardiomyopathy, a systemic progressive and fatal condition that typically leads to heart failure, and often death, within 3 to 5 years after disease onset.

The findings were consistent with the 35-week results announced in June 2022. Both analyses will be presented as an emerging science presentation at the American Academy of Neurology Annual Meeting that takes place between April 22, 2023, and April 27, 2023, in Boston, Massachusetts.


Eplontersen demonstrated sustained benefit in phase 3 trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) through 66 weeks. AstraZeneca. News release. March 27, 2023. Accessed March 28, 2023. https://www.astrazeneca.com/media-centre/press-releases/2023/eplontersen-demonstrated-sustained-benefit-in-phase-iii-trial.html

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