EUR 850.00 For Business Accounts Only

Morningstar | Alnylam's Patisiran Supports Positive Moat Trend; Maintaining $110 FVE

We are maintaining our fair value estimate of $110 per share after no-moat Alnylam reported second-quarter results that were in line with our expectations. On July 27, the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion of patisiran for hATTR amyloidosis with stage 1 or 2 polyneuropathy, which will be launched as Onpattro later this year. We're anticipating the U.S. Food and Drug Administration to follow suit next week, as the drug is under priority review as a breakthrough therapy with an approval deadline of Aug. 11. Ionis' inotersen continues to be a competitor with more convenient subcutaneous dosing, but we believe patisiran benefits from a more attractive safety profile. Pending approval, the firm is on track for several launches through 2021, including givosiran for acute hepatic porphyrias, lumasiran for ultra-rare disease primary hyperoxaluria, fitusiran for hemophilia, and inclisiran for hypercholesterolemia. For these pipeline candidates, we're maintaining probabilities of success of 65%, 50%, 70%, and 60%, respectively. The company reported a slight hiccup in slower-than-expected enrollment for phase 2 trials of cemdisiran in atypical hemolytic-uremic syndrome, delaying the data readout to 2019 from late 2018, but this does not affect our valuation, which rests on patisiran and late-stage candidates. Even with this minor setback, Alnylam has several near-term catalysts slotted for the second half of 2018, including the anticipated launch of Onpattro (patisiran) in the U.S. and Germany, additional Onpattro filings, and data readouts from givosiran and lumasiran trials. We believe patisiran's positioning for a global label and the firm's ample pipeline are indicative of the firm's positive moat trend and strengthening competitive advantage.