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Alnylam Gets Second Product Approval Amid Stiff Competition

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We issued an updated report on Alnylam Pharmaceuticals Inc. (ALNY - Free Report) on Nov 28.

It is a development-stage biopharmaceutical company focused on the development of novel therapeutics based on RNA interference (RNAi). 

 In August 2018, the FDA approved Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and only FDA-approved treatment for this indication. The uptake of the drug has been strong, with more than 600 patients worldwide on commercial Onpattro treatment since launch as of Sep 30, 2019. The drug should drive revenues for the company as it will be an important treatment option for people suffering from this often fatal disease.

In November, the FDA approved givosiran injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). The approval came three months before the PDUFA date of Feb 20, 2020. Gvosiran injection will be marketed by the trade name of Givlaari. This is the second RNAi therapeutic from Alnylam approved by the FDA in the last sixteen months. Givlaari is expected to be available in the United States by the end of 2019. This approval should further boost sales for the company. The company plans to achieve regulatory approval from the European Medicines Agency (EMA) for Givlaari (givosiran) in early 2020 for the treatment of AHP. It also plans to execute on U.S. and EU commercial launches throughout 2020.

Shares of the company have soared 61.2% year to date compared with the industry’s growth of 5.2%.

Alnylam has an impressive pipeline.

One of the candidates is vutrisiran (ALN-AAT02), which is the first investigational RNA interference (RNAi) therapeutic targeting AAT with Alnylam’s enhanced stabilization chemistry plus (ESC+) GalNAc-conjugate technology.  During the third quarter of 2019, the company continued enrollment in the HELIOS-A phase III study on vutrisiran (ALN-TTRsc02) for the treatment of hATTR amyloidosis with polyneuropathy.  It expects to complete enrollment in the study in early 2020.

Earlier in the month, Alnylam initiated the HELIOS-B phase III study of vutrisiran. The company plans to continue enrollment in the study throughout 2020.

Alnylam advanced lumasiran, an investigational RNAi therapeutic in development for the treatment of primary hyperoxaluria type I (PH1). The company expects to file a new drug application (NDA) and a marketing authorization application (MAA) with the FDA and the EMA, respectively, in early 2020, assuming positive results from the phase III ILLUMINATE-A study. 

Alnylam is also evaluating inclisiran (formerly known as PCSK9si or ALN-PCSsc), an investigational RNAi therapeutic, in phase II ORION studies for hypercholesterolemia. The company’s partner, The Medicines Company MDCO, reported new results for the drug while developing the treatment for hypercholesterolemia. The company intends to file an NDA shortly.

The successful development and subsequent approval of these candidates will be a huge boost for the company.

Alnylam has entered several collaboration deals for the development and commercialization of its broad pipeline of RNAi therapeutic candidates.

In April, the company and Regeneron Pharmaceuticals REGN extended their collaboration agreement. Both companies will work together to discover, develop and commercialize new RNA interference (RNAi) therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and the central nervous system, in addition to a select number of targets expressed in the liver. The companies plan to advance programs directed to 30 targets. Other candidates also might be introduced into clinical development during the initial five-year discovery period, which may extend.

However, Alnylam derives a substantial amount of revenues from partnerships with companies like Sanofi SNY, Takeda and Monsanto, among others. The company is heavily dependent on partnerships for its operations and pipeline development activities. Moreover, Alnylam is not the only company working on RNAi-based therapeutics. Companies like Ionis, Sarepta Therapeutics and Roche Innovation Center are also involved in the development of RNA-based drugs.

Alnylam currently has 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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