Alnylam Pharmaceuticals has shared detailed data further backing its rare disease drug Amvuttra (vutrisiran) from the Phase III trial Helios-B (NCT04153149) .
The company presented new data at the European Society of Cardiology (ESC) Congress on 30 August, which was simultaneously published in the New England Journal of Medicine. The Helios-B study focused on patients with transthyretin (ATTR) amyloidosis with cardiomyopathy, demonstrating significant reductions in all-cause mortality and recurrent cardiovascular events.
A subgroup analysis showed that the drug gave consistent benefits across all key patient segments, including participants already taking the only US Food and Drug Administration (FDA)-approved treatment for the disorder, Pfizer’s Vindaqel (tafamadis meglumine).
Alnylam had earlier reported positive topline results in June, showing that the therapy achieved statistically significant improvements across all secondary endpoints in both the combination therapy and monotherapy patient groups.
At the ESC Congress, Dr. Marianna Fontana, the trial’s principal investigator, pointed out that the study highlighted the changing treatment landscape for the disease with a high proportion of patients taking other treatments such as Vindaqel and SGLT2 inhibitors. “At present, there is no indication that we should not be able to treat older patients,” answered Fontana when asked about age restrictions related to the therapy.
In Helios-B, Amvuttra decreased the risk of all-cause mortality and recurrent cardiovascular events by 28%, specifically reducing the mortality in the Amvuttra patient group by 36% in the 42-month time-span. Furthermore, the Amvuttra treatment regimen had a similar tolerability profile to the placebo group with almost 62% of Amvuttra-treated patients experiencing serious adverse events compared to 67.1% of patients in the placebo group. Some adverse events in the trial were attributable to the disease pathology, Fontana explained.
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“It reflects the advancements we’ve made in this disease, that we are no longer using mortality, heart failure or hospitalisations; that we get to look at outpatient metrics to monitor disease advancement,” said Dr. Sarah Cuddy, the chair of a panel discussing the trial results, and a cardiovascular medicine specialist at Brigham and Women’s Hospital, Massachusetts.
The FDA approved Amvuttra for the treatment of the polyneuropathy of hATTR in adults. Amvuttra is an antisense RNAi oligonucleotide that treats ATTR amyloidosis by blocking the hepatic production of TTR messenger RNA before it is made.
ATTR amyloidosis with cardiomyopathy is a rare disease that affects approximately 2,500 to 5,000 US individuals per year, as per ESC data.
