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Alnylam's Kidney Disease Drug Succeeds in Late Stage Study

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Alnylam Pharmaceuticals, Inc. ALNY announced positive top-line results from the phase III study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1) — a rare genetic kidney disorder. The ILLUMINATE-A study met its primary efficacy endpoint and all tested secondary endpoints.

Lumasiran met the primary efficacy endpoint of percent change from baseline compared to placebo in 24-hour urinary oxalate excretion averaged across months 3 to 6 (p less than 0.0001). The study also achieved statistically significant results for all six tested secondary endpoints. The candidate also demonstrated an encouraging safety and tolerability profile.

The company plans to submit a new drug application (NDA) and file a Marketing Authorization Application (MAA) for lumasiran in early 2020.

However, despite the positive results, the stock dropped about 3%.

Shares of the company have surged 59.8% year to date compared with the industry’s growth of 8.6%.

Alnylam now expects to have four marketed products by the end of the next year. It currently has two approved products in the United States — Givlaari and Onpattro. Onpattro is a first-of-its-kind RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The drug is the first and only FDA-approved treatment for this indication. Givlaari is the second RNAi therapeutic from Alnylam approved by the FDA. 

Alnylam also has other candidates in its pipeline. The company along with partner The Medicine Company is evaluating inclisiran in phase III ORION studies for hypercholesterolemia.

Earlier in the year, Alnylam and Regeneron Pharmaceuticals REGN extended their collaboration agreement. Both companies will work together to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and the central nervous system (CNS), in addition to a select number of targets expressed in the liver.

Zacks Rank & A Stock to Consider

Alnylam is a Zacks Rank #3 (Hold) stock, currently.

A better-ranked stock in the biotech sector is Alkermes Plc. ALKS, sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Alkermes’ earnings per share estimates have increased from 36 cents to 52 cents for 2019 in the past 60 days. The company delivered a positive earnings surprise in the trailing four quarters by 236.80%, on average.  

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