Alnylam Pharmaceuticals has released new post hoc results from its HELIOS-B Phase 3 study, reporting lower rates of gastrointestinal (GI) adverse events in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) treated with vutrisiran, compared to placebo.
According to the company, in the overall study population, treatment with vutrisiran was associated with a 42 % lower rate of GI events versus placebo. In subgroup analyses, the company said a 37 % reduction was seen in the vutrisiran monotherapy cohort, and a 49 % reduction was observed among patients who were on tafamidis at baseline.
When assessing specific symptoms such as diarrhea, nausea, and vomiting, the data showed rate ratios (RR) below 1 across populations: for diarrhea (0.46, 0.48, 0.44), for nausea (0.35, 0.17, 0.48) and for vomiting (0.16, 0.25, 0.00) in the overall, monotherapy, and baseline-tafamidis groups, respectively. The company claims that the lower incidence of GI events appeared as early as three months into treatment and was consistent across hereditary and wild-type disease subtypes.
In a separate censored monotherapy analysis (excluding patients who later initiated tafamidis), vutrisiran treatment was associated with a 32 % reduction in risk for the composite endpoint of all-cause mortality and recurrent cardiovascular events through 36 months (hazard ratio 0.68; 95 % CI: 0.49–0.95; p = 0.022). These findings align with the trial’s primary monotherapy results, the company said.
The new analyses reinforce the safety and efficacy profile of vutrisiran as monotherapy and support its differentiated potential as a first-line therapy across varying patient health statuses.
