Alnylam reveals data supporting vutrisiran as heart drug standard

Alnylam Pharmaceuticals presented detailed Phase III study data on Friday for its RNAi therapeutic, vutrisiran, which it hopes will become the leading treatment for ATTR amyloidosis with cardiomyopathy (ATTR-CM). Despite the promising data, the company's shares dropped by up to 20% in premarket trading. The findings were shared at the European Society of Cardiology (ESC) congress and also published in The New England Journal of Medicine.

In June, Alnylam announced top-line results from its HELIOS-B trial. The trial demonstrated that vutrisiran reduced the composite of all-cause mortality and recurrent cardiovascular (CV) events by 28%, compared to a placebo, over a 36-month period. This reduction increased to 33% in patients who were not receiving Pfizer’s ATTR-CM drug, Vyndamax (tafamidis), at the beginning of the study. Furthermore, vutrisiran showed a 36% reduction in all-cause mortality in the overall population up to 42 months, and a 35% reduction in those not on Vyndamax at baseline.

The detailed findings revealed Friday showed that vutrisiran lowered the relative risk of death by 31% through month 36 in the overall study population, with this figure rising to 36% by month 42. Among those not on Vyndamax at baseline, the drug was associated with a non-significant 30% reduction in mortality over the initial 36 months.

Alnylam previously highlighted that the trial met all of its primary and secondary endpoints, demonstrating significant clinical benefits in several measures. These included the 6-minute walk test (6-MWT), the Kansas City Cardiomyopathy Questionnaire (KCCQ), and the New York Heart Association (NYHA) Class. At the ESC congress, it was reported that by month 30, patients on vutrisiran had a decline of 26.46 meters in the 6-MWT from baseline, compared to a 32.1-meter decline in the placebo group.

Alnylam's chief medical officer, Pushkal Garg, commented that while the results have yet to be reviewed by regulatory authorities, the data suggest that vutrisiran has the potential to become a new standard of care for treating ATTR-CM. The company plans to submit marketing applications for vutrisiran later this year and intends to use a priority review voucher for its filing in the United States.

The FDA approved vutrisiran in 2022 under the name Amvuttra for treating nerve damage in adults with hereditary ATTR amyloidosis. The drug generated $558 million in sales last year.

In conclusion, Alnylam Pharmaceuticals' detailed Phase III study data for vutrisiran show significant promise in treating ATTR amyloidosis with cardiomyopathy. With plans for marketing applications underway, the company aims to establish vutrisiran as a new standard of care for this condition.

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