In autopsy of failed heart disease study, AstraZeneca raises broader questions about silencer drugs
AstraZeneca's eplontersen trial for transthyretin-mediated amyloid cardiomyopathy failed to meet its primary efficacy endpoint. The trial's failure has raised questions about the effectiveness of silencer drugs for heart disease. The failure also impacts Ionis Pharmaceuticals, which co-developed the drug with AstraZeneca. This development has implications for the future of gene silencer treatments.
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- ✓ AstraZeneca's eplontersen trial for transthyretin-mediated amyloid cardiomyopathy failed to meet its primary efficacy endpoint.
- ✓ The trial's failure has raised questions about the effectiveness of silencer drugs for heart disease.
- ✓ Ionis Pharmaceuticals co-developed eplontersen with AstraZeneca.
- ✓ The failure of the trial impacts the future of gene silencer treatments for heart disease.
What changed
The full data from AstraZeneca's failed cardiomyopathy trial was released, causing stock prices to drop for Ionis Pharmaceuticals and Alnylam.
Live updates
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AstraZeneca's heart disease drug trial failure raises questions about silencer drugs
AstraZeneca's eplontersen trial for transthyretin-mediated amyloid cardiomyopathy failed to meet its primary efficacy endpoint. The trial's failure has raised questions about the effectiveness of silencer drugs for heart disease. The failure also impacts Ionis Pharmaceuticals, which co-developed the drug with AstraZeneca. This development has implications for the future of gene silencer treatments.
Why it matters
The failure of AstraZeneca's eplontersen trial has significant implications for the treatment of transthyretin-mediated amyloid cardiomyopathy, a rare and fatal heart disease. Gene silencer drugs, like eplontersen, aim to reduce the production of a protein that causes the disease. The trial's failure raises questions about the viability of this approach and may impact the development of similar treatments.
What is confirmed
- AstraZeneca's eplontersen trial for transthyretin-mediated amyloid cardiomyopathy failed to meet its primary efficacy endpoint.
- The trial's failure has raised questions about the effectiveness of silencer drugs for heart disease.
- Ionis Pharmaceuticals co-developed eplontersen with AstraZeneca.
- The failure of the trial impacts the future of gene silencer treatments for heart disease.
What to watch next
- Future development plans for eplontersen and similar gene silencer treatments
- Impact on Alnylam's own gene silencer treatment for heart disease
- Regulatory decisions on the approval of silencer drugs for heart disease
confidence 95%Sources used for this update (10)
- STAT — In autopsy of failed heart disease study, AstraZeneca raises broader questions about silencer drugs
- Endpoints News — Future of AstraZeneca’s gene silencer in heart disease uncertain after trial failure
- BioPharma Dive — AstraZeneca, Ionis data spark debate about RNA drugs’ impact on deadly heart disease
- BioSpace — ‘Worse than expected’ Wainua data muddies water for TTR silencers
- News-Medical — CARDIO-TTRansform trial investigating eplontersen fails to meet primary efficacy endpoint
- Medical Xpress — Eplontersen trial fails to meet primary endpoint in transthyretin-mediated amyloid cardiomyopathy
- Yahoo Finance — IONS, AZN Stocks Fall On Full Data From Failed Cardiomyopathy Trial — Jefferies Sees Neutral To Negative Readthrough To ALNY
- Seeking Alpha — Alnylam drops as AstraZeneca posts full trial results for failed heart drug
- STAT — AstraZeneca and Ionis detail surprise failure of heart disease drug, with Alnylam closely watching
- Fierce Pharma — ESC: AZ, Ionis' Wainua post-mortem throws water on silencer-plus-stabilizer approach in ATTR-CM
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