HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Summary
This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in participants with ATTR amyloidosis with cardiomyopathy.
Timeline
- Start
- 2019-11-26
- Primary completion
- 2024-05-08
- Completion
- 2026-12-02
Outcome
Met primary endpoint
registry analysis (superiority test); DB Period: Placebo vs DB Period: Vutrisiran; p =0.0118; Hazard Ratio (HR) 0.718 (95% CI 0.555 to 0.929); Modified Andersen-Gill Model NCT04153149 ↗
registry analysis (superiority test); DB Period: Placebo vs DB Period: Vutrisiran; p =0.0162; Hazard Ratio (HR) 0.672 (95% CI 0.487 to 0.929); Modified Andersen-Gill Model NCT04153149 ↗
paper Vutrisiran treatment led to a lower risk of death from any cause and recurrent cardiovascular events than placebo (hazard ratio in the overall population, 0.72; 95% confidence interval PMID 39213194 ↗
release “Reported Positive Topline Results from HELIOS-B Phase 3 Study of Vutrisiran, Achieving Statistical Significance on Primary and All Secondary Endpoints” alnylam.com ↗
release “HELIOS-B Phase 3, randomized, double-blind, placebo-controlled multicenter global study which met all 10 of its primary” alnylam.com ↗
release “HELIOS-B Phase 3, randomized, double-blind, placebo-controlled multicenter global study, which met all 10 of its pre-specified primary and secondary endpoints” alnylam.com ↗
release “Presented new analyses from the HELIOS-B Phase 3 trial of vutrisiran in patients with ATTR-CM. Vutrisiran reduced the risk” alnylam.com ↗
release “In this censored monotherapy population, patients treated with vutrisiran demonstrated a statistically significant 32% reduction in the risk of the primary composite endpoint” alnylam.com ↗
release “Late-Breaking Prespecified Subgroup Analysis of HELIOS-B Demonstrates that Vutrisiran Provided Consistent Clinical Benefit Across All-Cause Mortality and Recurrent Cardiovascular Events” alnylam.com ↗
Publications
- Fontana M, Algalarrondo V, Garcia-Pavia P, Maurer MS, Gillmore JD, Cappelli F, Kolachana K, Gao X, Jadhav S, Mehrotra N, Robbie G, Badri P. Vutrisiran-Mediated Knockdown of Transthyretin in Patients with ATTR Amyloidosis. Clin Pharmacokinet. 2026 May 27. doi: 10.1007/s40262-026-01651-3. Online ahead of print.
- Endo J, Minamino T, Oh J, Tahara N, Tsujita K, Izumi C, Okumura T, Onoue K, Kitaoka H, Chen C, Chan-Daniels A, Kim D. Vutrisiran in East Asian Patients With Transthyretin Amyloidosis With Cardiomyopathy: Post-Hoc Analysis of HELIOS-B. JACC Asia. 2026 May 18:S2772-3747(26)00192-4. doi: 10.1016/j.jacasi.2026.03.031. Online ahead of print.
- Claggett BL, Fontana M, Vaduganathan M, Hamatani Y, Maurer MS, Gillmore JD, Solomon SD. Timing of Mortality Benefit in Outcomes Trials in Transthyretin Amyloidosis. J Am Coll Cardiol. 2026 Feb 10;87(5):522-529. doi: 10.1016/j.jacc.2025.09.1512. Epub 2025 Sep 25.
- Jering KS, Fontana M, Lairez O, Longhi S, Azevedo O, Morbach C, Bender S, Jay PY, Vest J, Bulwer BE, Prasad N, Solomon SD, Skali H. Effects of vutrisiran on cardiac structure and function in patients with transthyretin amyloidosis with cardiomyopathy: secondary outcomes of the HELIOS-B trial. Nat Med. 2025 Oct;31(10):3560-3568. doi: 10.1038/s41591-025-03851-z. Epub 2025 Aug 6.
- Maurer MS, Berk JL, Damy T, Sheikh FH, Gonzalez-Costello J, Morbach C, Delgado D, Bondue A, Azevedo O, Poulsen SH, Jankowska EA, Yang L, Bender S, Eraly SA, Jay PY, Vest J, Fontana M. Impact of Vutrisiran on Cardiac Biomarkers in Patients With Transthyretin Amyloidosis With Cardiomyopathy From HELIOS-B. J Am Coll Cardiol. 2025 Aug 12;86(6):459-475. doi: 10.1016/j.jacc.2025.04.055.
- Witteles RM, Garcia-Pavia P, Damy T, Grogan M, Sheikh FH, Morbach C, Bender S, Exter J, Eraly SA, Fontana M. Vutrisiran Improves Survival and Reduces Cardiovascular Events in ATTR Amyloid Cardiomyopathy: HELIOS-B. J Am Coll Cardiol. 2025 Apr 30:S0735-1097(25)06170-4. doi: 10.1016/j.jacc.2025.04.008. Online ahead of print.
- Maurer MS, Witteles RM, Garcia-Pavia P, Sheikh FH, Morbach C, Rodriguez Duque D, Aldinc E, Eraly SA, Gillmore JD. Impact of Heart Failure Severity on Vutrisiran Efficacy in Transthyretin Amyloidosis With Cardiomyopathy. J Am Coll Cardiol. 2025 May 27;85(20):1927-1939. doi: 10.1016/j.jacc.2025.03.477. Epub 2025 Mar 17.
- Fontana M, Maurer MS, Gillmore JD, Bender S, Aldinc E, Eraly SA, Jay PY, Solomon SD. Outpatient Worsening Heart Failure in Patients With Transthyretin Amyloidosis With Cardiomyopathy in the HELIOS-B Trial. J Am Coll Cardiol. 2025 Feb 25;85(7):753-761. doi: 10.1016/j.jacc.2024.11.015. Epub 2024 Nov 18.
- Fontana M, Berk JL, Gillmore JD, Witteles RM, Grogan M, Drachman B, Damy T, Garcia-Pavia P, Taubel J, Solomon SD, Sheikh FH, Tahara N, Gonzalez-Costello J, Tsujita K, Morbach C, Pozsonyi Z, Petrie MC, Delgado D, Van der Meer P, Jabbour A, Bondue A, Kim D, Azevedo O, Hvitfeldt Poulsen S, Yilmaz A, Jankowska EA, Algalarrondo V, Slugg A, Garg PP, Boyle KL, Yureneva E, Silliman N, Yang L, Chen J, Eraly SA, Vest J, Maurer MS; HELIOS-B Trial Investigators. Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy. N Engl J Med. 2025 Jan 2;392(1):33-44. doi: 10.1056/NEJMoa2409134. Epub 2024 Aug 30.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Vutrisiran | Small interfering RNA (siRNA) | 25 mg | Subcutaneous |
Indications
No indication recorded.