logo
Plus   Neg
Share
Email

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.

For comments and feedback contact: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Shares of General Electric Co. are rising more than 10 percent on Thursday after the industrial conglomerate said it plans to establish a new, independent company focused on building a comprehensive Industrial Internet of Things or IIoT software portfolio. In addition, longtime bearish analyst Stephen Tusa of J.P. Morgan upgraded shares of GE to neutral. Apple Inc. said Thursday that it will invest $1 billion to build a new corporate campus in North Austin that will eventually create 15,000 jobs. Further, the tech giant announced plans to establish new offices and expand to over 1,000 employees each in three cities - Seattle, San Diego and Culver City, California. In January, Apple said it will create over 20,000 jobs over the next five years. German telecom giant Deutsche Telekom, Chinese conglomerate Alibaba's Alibaba Cloud, banking giant Citi and 13 other organizations have joined Hyperledger Blockchain project, an open source collaborative effort aimed to advance cross-industry blockchain technologies. Hyperledger, a project of The Linux Foundation that started less than three years ago, is a multi-project.
Follow RTT