ESC 2024: Positive Cardiomyopathy Prospects for Alnylam's Amvuttra

Alnylam Pharmaceuticals has unveiled further promising data from its Phase III Helios-B trial regarding its rare disease drug, Amvuttra (vutrisiran). The findings, presented at the European Society of Cardiology (ESC) Congress on August 30, have also been published in the New England Journal of Medicine.

The Helios-B trial concentrated on patients suffering from transthyretin (ATTR) amyloidosis with cardiomyopathy. The results demonstrated notable reductions in both all-cause mortality and recurrent cardiovascular events among the patients treated with Amvuttra. An analysis of different patient subgroups revealed that the drug provided consistent benefits across all key segments, including those already using Pfizer’s Vindaqel (tafamadis meglumine), the only FDA-approved treatment for this condition.

Earlier, in June, Alnylam had reported positive topline results from the trial. The therapy showed statistically significant improvements across all secondary endpoints for both the combination therapy and monotherapy groups. During the ESC Congress, Dr. Marianna Fontana, the principal investigator for the trial, highlighted the evolving treatment landscape for this disease. She pointed out that a substantial proportion of patients were already on other treatments such as Vindaqel and SGLT2 inhibitors. Addressing concerns about potential age restrictions for the therapy, Dr. Fontana stated that there was no indication that older patients should be excluded from treatment.

In Helios-B, Amvuttra demonstrated a 28% reduction in the risk of all-cause mortality and recurrent cardiovascular events. Specifically, the mortality rate in the Amvuttra-treated group decreased by 36% over a 42-month period. Additionally, the treatment's tolerability was similar to that of the placebo group, with approximately 62% of patients on Amvuttra experiencing serious adverse events compared to 67.1% in the placebo group. Dr. Fontana explained that some of these adverse events were attributable to the underlying disease pathology.

Dr. Sarah Cuddy, a cardiovascular medicine specialist at Brigham and Women’s Hospital, Massachusetts, and chair of a panel discussing the trial results, remarked on the advancements in treating this disease. She noted that the focus has shifted from mortality, heart failure, and hospitalizations to outpatient metrics for monitoring disease progression.

The FDA has approved Amvuttra for treating the polyneuropathy of hATTR in adults. Amvuttra is an antisense RNAi oligonucleotide that combats ATTR amyloidosis by inhibiting the hepatic production of TTR messenger RNA before it is synthesized.

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