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Alnylam Pharma Announces Pipeline Goals For 2019 - Quick Facts

Alnylam Pharmaceuticals, Inc. (ALNY) announced, at its R&D Day, the company plans to discuss its pipeline goals for 2019, focusing on five late-stage investigational programs advancing toward potential commercialization. These programs include the wholly owned programs: givosiran for the treatment of acute hepatic porphyria, lumasiran for the treatment of primary hyperoxaluria type 1, and vutrisiran for the treatment of ATTR amyloidosis.

Alnylam announced initiation of a rolling submission of an NDA to the FDA for givosiran and initiation of the HELIOS-A pivotal Phase 3 study of vutrisiran in patients with hATTR amyloidosis. Regarding Lumasiran, Alnylam plans to report topline results from the ILLUMINATE-A Phase 3 study in late 2019.

The company announced that its first central nervous system (CNS)-targeted development candidate will be ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein for the treatment of cerebral amyloid angiopathy associated with intracerebral hemorrhage.

The company will also discuss commercial execution with ONPATTRO. In early January 2019, the company expects to pre-announce global product revenues for ONPATTRO and number of patients on commercial drug for the fourth quarter of 2018. The company announced that it has filed regulatory submissions for ONPATTRO in Canada and Switzerland, and expects regulatory decisions in 2019.

"In the coming year, we plan to continue our commercial execution for ONPATTRO, bringing this much needed medicine to eligible patients around the world, including an upcoming launch expected in Japan, assuming regulatory approval, and potential launches in other global markets. In addition, our continued execution, together with our partners, on our clinical pipeline of five late-stage investigational programs is poised to bring potential approvals of new RNAi therapeutics essentially on an annual basis in each of the coming years, with three expected Phase 3 program data readouts in 2019 alone and two potential NDA filings, one in mid-2019 and one at or around year-end 2019, assuming positive results," said John Maraganore, CEO of Alnylam.

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