Results from the APOLLO phase III trial of ALN TTR02 in hereditary ATTR amyloidosis show benefits in cardiac system.- Alnylam Pharma.

Alnylam Pharmaceuticals has presented new results from the APOLLO Phase III study of ALN TTR02 (patisiran), for the treatment of hereditary ATTR (hATTR) amyloidosis. Specifically, there were improvements in cardiac structure (reduction in mean left ventricular [LV] wall thickness) and function (reduction in LV-end diastolic volume and global longitudinal strain) at 18 months. Patisiran was also associated with a favorable effect on gait speed in the cardiac subpopulation, relative to placebo. Furthermore, patisiran treatment led to a significant reduction in levels of a cardiac stress biomarker, NT-proBNP, relative to placebo at 9 and 18 months (p = 7.7 x 10-8). Higher levels of NT-proBNP are associated with increased mortality in cardiac amyloidosis.

The frequency of cardiac adverse events (AEs) and serious AEs (SAEs) were similar in patisiran- and placebo-treated patients in the overall APOLLO study population. Deaths occurred with similar frequency in the patisiran (4.7 percent) and placebo (7.8 percent) arms. Results were presented at the 16th International Symposium on Amyloidosis (ISA).

Comment: ALN TTR02 is currently filed in both the US and EU for hereditary ATTR (hATTR) amyloidosis, and in the EU it has been granted accelerated approval. Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world, including certain Central and Eastern European countries of the European Union.