July 23, 2018 10:42am

Indirect analysis shows Patisiran’s Impact on measures of Neuropathy and quality of life in APOLLO study relative to those measured in randomized study of Tafamidis

ALNY jumps +$1.35 or +1.27% to $107.28 because of widening clinical percentage ranges

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Alnylam Pharmaceuticals (ALNY presented new analyses from the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic for the treatment of hereditary ATTR (hATTR) amyloidosis, in six presentations at the 2018 Peripheral Nerve Society (PNS) Annual Meeting being held July 22-25, in Baltimore, MD.

 

The new data presented underscore the potential clinical benefit of patisiran for patients with hATTR amyloidosis. First, the results of the exploratory EQ-5D-5L and EQ-VAS assessments show that patisiran treatment, relative to placebo, may help patients maintain or improve mobility and independence, reduce anxiety and depression, and favorably impact overall health.

 

Overall health status was an exploratory endpoint assessed in APOLLO using EuroQOL-5-dimension 5-level (EQ-5D-5L), a standardized measure of health status based on five dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, and the EuroQOL visual analogue scale (EQ-VAS), a measure of a patient’s global impression of their overall health as evaluated on a scale of zero (worst possible health) to 100 (best possible health).

 

At 18 months, a larger proportion of patients on patisiran than placebo, respectively, showed preservation (defined as no change in score) or improvement relative to baseline in each EQ-5D-5L domain: mobility, 70 versus 22%; self-care, 66 versus 21%; usual activities, 72 versus 25%; pain/discomfort, 73 versus 31%; anxiety/depression, 81 versus 45%. Overall health, as measured by EQ-VAS, improved by an average of 2.4 points in patients on patisiran, while declining by an average of 7.1 points in placebo patients, indicating a 9.5 point difference (nominal p value less than 0.001).

 

The percentages (%) do speak … as do the initial open day's range.