RNAi targeting TTR improves functional capacity in ATTR amyloidosis with cardiomyopathy

Results of the APOLLO-B phase 3 study with the RNAi patisiran showed that the primary endpoint was met. Treatment with patisiran resulted in a significant and clinically meaningful benefit on functional capacity – measured by the 6-mintue walk test (6-MWT) – at 12 months, compared with placebo in patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

These findings were presented at the 18th International Symposium on Amyloidosis (ISA).

A median difference of 14.7 meters (P=0.0152) was observed between the two treatment groups, with a benefit for the patisiran group. In addition, the first secondary endpoint – health status and quality of life, measured by the KCCQ-OS score, was also met (LS mean difference of 3.7 points, P=0.0397, with benefit for patisiran treatment). The composite endpoint of all-cause mortality, frequency of CV events and change from baseline in 6-MWT over 12 months was not different between the patisiran group and the placebo group. Safety and tolerability profile was encouraging in patients with ATTR amyloidosis and cardiomyopathy.

APOLLO-B is a phase 3, randomized, double-blind, placebo-controlled multicenter global study. The trial enrolled 360 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy at 69 sites in 21 counties. Patients were randomized 1:1 to patisiran (0.3 mg/kg) or placebo IV administered every three weeks over a 12-month period. After 12 months, all patients will receive patisiran in an open-label extension.

Additional results from the APOLLO-B study will be presented at the Heart Failure Society of America annual meeting which will be held in Washington DC, Sept 30 – Oct 3, 2022.

Source: press release Alnylam, Sept 8 2022