LatestRoivant's Lung Disease Drug Achieves Mid-Stage Study Goals
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Eplontersen Trial Fails to Meet Primary Goal in Rare Heart Disease Study

Eplontersen Trial Fails to Meet Primary Goal in Rare Heart Disease Study
Heart Disease
Summary

The CARDIO-TTRansform trial investigating eplontersen for transthyretin-mediated amyloid cardiomyopathy did not achieve its primary efficacy endpoint, according to results presented at ESC Congress 2026. This genetic heart condition occurs when abnormal amyloid proteins accumulate in heart tissue, impairing its function. The trial's failure represents a setback for patients with this life-threatening rare disease who have limited treatment options. Researchers will now analyze secondary endpoints and safety data to determine the drug's potential clinical value. The results highlight the ongoing challenge of developing effective therapies for rare cardiac amyloidosis.

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Fetched: August 29, 2026 at 10:01 AM

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