Alnylam records home run in key heart study of RNAi therapy vutrisiran
24 Jun 2024
Drug ApprovalClinical ResultPhase 3AHAPriority Review
Alnylam Pharmaceuticals is speeding ahead with new marketing applications for vutrisiran after the RNAi therapeutic met the primary and all secondary endpoints of a Phase III study for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM). The news, announced Monday, sent the company’s shares up 39% in premarket trading.
Top-line results showed that in the overall study population, vutrisiran reduced the composite of all-cause mortality and recurrent cardiovascular (CV) events by 28% compared to placebo, with this rising to a 33% drop in patients not on Pfizer's ATTR-CM drug Vyndamax (tafamidis) at baseline.
“We really feel that this data has set the bar for a new standard of care,” CEO Yvonne Greenstreet remarked. The FDA approved vutrisiran in 2022 under the name Amvuttra to treat nerve damage in adults with hereditary ATTR amyloidosis, with the product bringing in sales of $558 million last year.
Priority review voucher in play
“We believe we have in our hands a multibillion-dollar opportunity,” Greenstreet said, adding that expanding use of vutrisiran to ATTR-CM “really unlocks the potential for significant revenue growth” with the latest results representing “a really important moment for the company.” Alnylam indicated that it will use a priority review voucher in conjunction with its filing to the FDA.
The HELIOS-B study randomised 655 adults with ATTR-CM to receive vutrisiran or placebo subcutaneously once every three months. The trial evaluated the RNAi therapy’s effect on the reduction of all-cause mortality and recurrent CV events, both in the overall population and the 60% of patients not on Vyndamax at baseline.
Along with the impact on the primary endpoint, vutrisiran also reduced all-cause mortality by 36% in the overall population and by 35% in those not on Pfizer’s drug at baseline. Alnylam added that in addition, vutrisiran demonstrated clinically significant benefits on the 6-minute walk test (6-MWT), Kansas City Cardiomyopathy Questionnaire (KCCQ) and New York Heart Association (NYHA) Class at month 30.
In terms of safety, the company said that vutrisiran’s profile was “encouraging” in HELIOS-B, with rates of adverse events (AEs), serious AEs and AEs leading to study drug discontinuation similar between the two arms. Detailed results have been submitted as a late-breaking abstract to the European Society of Cardiology (ESC) for presentation.
Jitters put aside
Investors suffered some jitters earlier this year when Alnylam detailed changes to the statistical plan of HELIOS-B, including a longer minimum follow-up period and tweaks to the primary endpoint to include assessments in both the overall population and those not on Vyndamax at baseline. While BMO Capital Markets analyst Kostas Biliouris suggested at the time that the changes may be perceived as a "lack of management's confidence” in the study, the company’s chief medical officer Pushkal Garg said they were made "to optimise the study for success, and a strong and competitive label."
For related analysis, see Vital Signs: Alnylam loses precious time with HELIOS-B update.
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