Boosting Fair Value on Alnylam After Drug Trial

We are raising

Patisiran’s gene-silencing mechanism showed impressive efficacy across a broad spectrum of patients with hereditary ATTR amyloidosis with polyneuropathy, including those with cardiac involvement, which could set the drug up for a broader label than expected. Hereditary ATTR amyloidosis is a rare disease with high unmet need that has been historically categorized as familial amyloid cardiomyopathy (or FAC, accumulation of amyloid in the heart) and familial amyloid polyneuropathy (or FAP, accumulation of amyloid in the nerves), but today the industry views the disease as more of a spectrum (FAP typically precedes FAC). Alnylam’s phase III Apollo trial met its primary and secondary endpoints and showed no serious side effect imbalances against the placebo arm. Despite patisiran’s less convenient intravenous dosing, which requires the use of steroid pre-treatment, we believe the drug’s clean safety profile makes it an easy winner against Ionis’ inotersen. Alnylam’s next-generation drug ALN-TTRsc02, with subcutaneous dosing every couple of months, decreases the chance that Ionis’ drug will take meaningful market share.

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