Share price of Alnylam Pharmaceuticals Inc. (ALNY - Free Report) increased 25.4% year to date compared with the industry’s growth of 15.9%. Alnylam is a development-stage biopharmaceutical company focused on the development of novel therapeutics based on RNA interference (RNAi).
In August 2018, the company’s lead drug, Onpattro (patisiran) received regulatory approvals in the United States and Europe for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and only FDA-approved treatment for this indication. Sales of the drug initiated on a strong note, with more than 200 patients in the United States and EU on commercial Onpattro treatment during the fourth quarter of 2018. The drug is expected to further boost sales of the company in 2019.
Alnylam is also planning to expand the label of Onpattro. In this regard, the company obtained regulatory alignment with the FDA on the design of its phase III study, which will evaluate Onpattro in hereditary and wild-type ATTR amyloidosis patients with cardiomyopathy. The study is expected to start mid-2019. The drug is also under review in Japan, Canada and Switzerland.
The company also has several other promising candidates in its pipeline.
Vutrisiran (ALN-AAT02) is the first investigational RNAi therapeutic, targeting AAT with Alnylam’s enhanced stabilization chemistry plus (ESC+) GalNAc-conjugate technology. However, the safety and efficacy of ALN-AAT02 are yet to be evaluated by any health authority like the FDA or the EMA. The company initiated HELIOS-A phase III study in hereditary ATTR amyloidosis patients with polyneuropathy. Late 2019, Alnylam plans to initiate an additional phase III study, HELIOS-B, on vutrisiran in hereditary and wild-type ATTR amyloidosis patients with cardiomyopathy.
The company is advancing givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria (AHP). It initiated rolling submission of a new drug application (NDA) in 2018 with plans to pursue full approval based on complete results from the ENVISION phase III study. Alnylam plans to complete the rolling submission of the NDA and file a Marketing Authorization Application (MAA) mid-2019.
Alnylam along with The Medicine Company (MDCO - Free Report) is evaluating inclisiran in phase III ORION studies for hypercholesterolemia. The company expects to file an NDA in the United States at or around 2019-end, assuming positive phase III results.
Alnylam’s expertise in RNAi therapeutics and broad intellectual property estate have allowed the company to enter into collaborations with leading pharmaceutical and life sciences companies like Ionis Pharmaceuticals, Novartis (NVS - Free Report) , Roche (RHHBY - Free Report) , Takeda, Merck, Monsanto, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others. The companies collaborated to optimize the development and commercialization of certain products for the treatment of rare genetic diseases. Alnylam generates revenues from research collaborations, grants and licensing of the RNAi technology outside its core focus area.
These factors should further drive the share price of the company in 2019.
Alnylam currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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