New phase III results for ALN TTR02 (patisiran) in hereditary TTR-mediated amyloidosis shows evidence for patient cardiac disease- Alnylam Pharma

Alnylam Pharma presented baseline demographics from the APOLLO Phase III study of ALN TTR02 (patisiran) in hereditary TTR-mediated amyloidosis. A total of 225 hATTR patients with polyneuropathy (hATTR-PN) were enrolled at 44 sites in 19 countries around the world between December 2013 and January 2016. Ninety-five patients (42 percent) have the Val30Met TTR mutation, the most prevalent genotype known to be associated with hATTR-PN; the remaining 130 patients (58 percent) span 57 other mutations. In terms of disease severity, 104 patients (46 percent) were FAP Stage 1 at baseline, and 119 (53 percent) patients were Stage 2; only 2 patients (1 percent) were Stage 3. The mean baseline mNIS+7 score was 78.8 (range 8.0 � 165.0).

Additionally, baseline mNIS+7 and quality of life (QOL) scores were found to correlate with FAP Stage and polyneuropathy disability (PND) score, underscoring the clinical relevance of specific endpoints in the APOLLO Phase III study. A significant number of patients (N=122, 54 percent) had cardiac involvement at baseline. The Company believes this patient subset should allow for a controlled evaluation of effects of patisiran on cardiac parameters, including cardiac biomarkers NT-proBNP and troponin, left ventricular wall thickness, ejection fraction, and 10-meter walk test, all included as secondary or exploratory endpoints in the Phase III study protocol. Data were presented at the XV International Symposium on Amyloidosis. Alnylam expects to report data from the APOLLO trial in mid-2017.