2024-09-02 12:20:58
Alnylam Pharmaceuticals announced detailed results from the phase 3 HELIOS-B study of vutrisiran, an investigational RNAi therapy for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The data is presented in the Congress of the European Society of Cardiology (ESC) 2024 and has been published in ‘The New England Journal of Drug‘. As previously mentioned, the HELIOS-B study met all 10 primary and secondary endpoints in the combination and monotherapy populations, with statistical significance.
Patients included are mainly Class I or II patients New York Heart Association (NYHA) with wild-type disease and has been diagnosed by non-invasive methods, with special treatment sometimes with available standard treatments, such as tafamidis and SGLT2 inhibitors, representing the ATTR patient population -CM of today.
In the study, treatment with vutrisiran reduced the risk of death and cardiovascular events compared to placebo. In the general population, vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events by 28%, with similar reductions in death and cardiovascular events as the primary endpoint. Mortality in this population was significantly reduced by 31% during the double-blind period and by 36% up to 42 months.
In the monotherapy population, vUtrisan significantly reduces the risk of death for all causes and recurrent cardiovascular events by 33% and significantly reduced the risk of death by 35% up to month 42. As a component of the primary endpoint, a non-significant 30% reduction in death (p-value 0.1179) was observed in the monotherapy population during the double-blind period.
Treatment with vutrisiran was also associated with benefits over placebo on several well-established clinical measures of disease progression, including the six-minute long test, Kansas City Cardiovascular Questionnaire and NYHA class, as well as the heart biomarker NT-proBNP. Subgroup analyzes showed consistent benefits in all key patient groups, including patients receiving background tafamidis.
Trends of greater effect were observed in patients with earlier disease (ie, younger patients and those with lower NT-proBNP baseline levels). In the HELIOS-B study, the safety and tolerability profiles of vutrisiran were consistent with the established profile. The rate of impact of events (AE), serious AEs, serious AEs, and AEs leading to treatment discontinuation were similar in the placebo and placebo arms.
Cardiac AEs were similar or lower in the vutrisiran group compared with the placebo group. AEs that occurred in more than 15 percent of all patients were similar or less in the vutrisiran group compared to placebo (heart failure, Covid-19, atrial fibrillation, gout, dyspnea, and falls).
Reducing TTR toxicity improves survival
“We are proud to share HELIOS-B information with the community at ESC Congress 2024. With this research, it has been shown to accelerate the reduction of toxicity of TTR observed with vutrisiran improved survival and reduced cardiovascular hospitalizations and disease progression compared to placebo, with benefits consistently observed in all populations, and regardless of the use of underlying stabilizers,” Alnylam Medical Director tell, Pushkal Garg.
“Although the results have not yet been reviewed by the regulatory authority, the data shared today suggest that vutrisiran has the potential to become the new standard treatment for ATTR-CM, a progressive and ultimately fatal disease with limited treatment options. We would like to thank everyone who contributed to the success of this study, including patients, caregivers, researchers, study staff, and my colleagues at Alnylam. “In light of this data, we work quickly to present these data to regulatory agencies and bring this medicine to patients around the world,” he added.
In the HELIOS-B study, vutrisiran showed a satisfactory safety and tolerability profile, consistent with the established profile of the drug. Them impact of event rates (AEs), serious adverse events, serious adverse events, and adverse events leading to discontinuation of study drug were similar between the placebo and placebo arms.
Cardiovascular adverse events were similar or lower in the other arm compared with placebo. Adverse events that occurred in more than 15 percent of patients overall were similar or lower in the vutrisiran arm compared to placebo (heart failure, Covid-19, atrial fibrillation, gout, dyspnea, and falls).
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