Phase 3 NEURO-TTRansform study on WAINUA™ (eplontersen) data to be presented at 2024 ISA

Ionis Pharmaceuticals, in collaboration with AstraZeneca, is set to present new subgroup analyses from their Phase 3 NEURO-TTRansform study on WAINUA™ (eplontersen) at the 2024 International Symposium on Amyloidosis in Rochester, Minnesota. WAINUA, approved by the FDA in December 2023, is designed for treating hereditary transthyretin-mediated amyloidosis polyneuropathy (ATTRv-PN) in adults. The study aims to demonstrate the drug's impact on neuropathy impairment and quality of life across various patient segments.

Key presentations scheduled for May 29, 2024, include:

1. Neuropathy Impairment and Nutritional Status:
- Presenting Author: Jonas Wixner
- Focus on how eplontersen affects neuropathy and nutritional status in ATTRv-PN patients.

2. Exploratory Analysis in V30M TTR Variant Patients:
- Presenting Author: Julian D. Gillmore
- Analysis of eplontersen’s efficacy in patients with both early-onset and late-onset disease.

3. Treatment Effect in Male and Female Patients:
- Presenting Author: Márcia Waddington Cruz
- Examines the drug's impact across genders.

As part of a global agreement, AstraZeneca and Ionis are working to commercialize WAINUA in the U.S. and seek regulatory approvals in Europe and other regions. Additionally, eplontersen is being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This condition typically results in progressive heart failure and high mortality within three to five years of onset. The CARDIO-TTRansform study involves over 1,400 patients, making it the largest study of its kind.

About WAINUA™ (eplontersen):
WAINUA is a Ligand-Conjugated Antisense (LICA) medicine that inhibits the production of the transthyretin (TTR) protein. It is approved in the U.S. for treating hereditary transthyretin-mediated amyloidosis polyneuropathy in adults.

Safety Information:
Patients using WAINUA should supplement with vitamin A due to reduced serum vitamin A levels caused by the treatment. The most common adverse reactions include decreased vitamin A levels and vomiting.

NEURO-TTRansform Study:
This global, open-label, randomized trial assesses eplontersen's efficacy and safety in ATTRv-PN patients at 35, 66, and 85 weeks. The final analysis compared eplontersen to an external placebo group at week 66, with patients continuing treatment until week 85. Post-study options included an open-label extension or a 20-week post-treatment evaluation.

About Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN):
This condition results from the accumulation of misfolded TTR protein in peripheral nerves, causing progressive nerve damage and motor function loss. Misfolded TTR also accumulates in major organs, leading to compromised function and disability within five years, often fatal within a decade.

Ionis Pharmaceuticals:
For over three decades, Ionis has developed innovative medicines for serious diseases. The company currently markets five medicines and leads in neurology, cardiology, and other high-need areas. Ionis pioneers RNA-targeted therapies and gene editing, continually advancing in these fields to deliver life-changing treatments.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!