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Alnylam Stock Down Despite Positive Phase III Givosiran Data

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Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) announced that the phase III study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), met its primary efficacy endpoint and majority of secondary endpoints. In the ENVISION study, givosiran met the main goal of reducing the yearly number of attacks in patients with acute intermittent porphyria (AIP). Givosiran also achieved statistically significant results for five of nine secondary endpoints.

Despite the results being positive, shares of the company dipped about 4% following the news as investors were concerned about the high percentage of serious adverse events (SAEs) reported by patients on givosiran compared with those on placebo. Serious adverse events were reported by 20.8% of patients on givosiran compared to 8.7% on placebo. Adverse events (AEs)  such as renal impairment and elevated liver enzymes were reported in 89.6% of patients on the drug compared with 80.4% on placebo. One patient in the givosiran arm (2.1 percent) discontinued treatment due to an AE.

Shares of Alnylam have slumped 31.7% in the past year compared with the industry’s decline of 17.9%.

 

We remind investors that in October 2018, Alnylam announced positive early results from the interim analysis of the ENVISION phase III study. The results showed that treatment with givosiran led to a statistically significant reduction in urinary aminolevulinic acid (ALA) levels in AIP patients compared to placebo. The study also showed SAEs in 22% of givosiran patients and 10% of placebo patients in the interim analysis cohort of 43 patients, with one patient (4%) on givosiran discontinuing the treatment due to an increase in liver transaminase that resolved.

Alnylam plans to complete regulatory submissions for the drug to the U.S. and European Union regulators by mid-2019, and expects to launch the drug early 2020. 

We remind investors that the company’s only approved drug Onpattro is a first-of-its-kind 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 company also has other candidates in its pipeline. Alnylam along with partner The Medicine Company is evaluating inclisiran in phase III ORION studies for hypercholesterolemia.

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, including Ionis Pharmaceuticals, Novartis (NVS - Free Report) , Roche (RHHBY - Free Report) , Takeda, Merck, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others.

Zacks Rank

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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