Alnylam Pharmaceuticals, Inc. ALNY announced positive phase III results from the ILLUMINATE-A study on its RNAi candidate, lumasiran in development for the treatment of primary hyperoxaluria type 1 (PH1).. Data from the study showed that PH1 patients treated with lumasiran met the primary efficacy endpoint of percent change from baseline, relative to placebo. The study also achieved statistically significant results for all six tested secondary endpoints, including the proportion of patients achieving a near-normalization or normalization of urinary oxalate.
Lumasiran achieved the ILLUMINATE-A primary endpoint with a 53.5% mean reduction in urinary oxalate relative to placebo and showed a 65.4% mean reduction in urinary oxalate relative to baseline. In the study, 84% of patients on lumasiran achieved normal or near-normal levels of urinary oxalate and more than half of patients reached normalization compared with zero percent in the placebo group.
The drug demonstrated an encouraging safety and tolerability profile with no serious or severe adverse events (AEs) and with mild injection site reactions as the most common drug-related AE.
The substantial and sustained reductions in urinary and plasma oxalate demonstrated that lumasiran addresses the underlying pathophysiology of PH1 by reducing the production of the toxic metabolite responsible for the clinical manifestations of this serious and progressive disease.
Based on these results, Alnylam has filed a new drug application (NDA) with the FDA. The FDA has granted a Priority Review to the NDA with an action date of Dec 3, 2020.
In addition, the marketing authorization application (MAA) for lumasiran has received an accelerated assessment from the European Medicines Agency (EMA).
Shares of Alnylam have gained 10.8% year to date compared with the industry’s growth of 6.9%.
Meanwhile, the company is also evaluating several other candidates. A few significant ones include vutrisiran, inclisiran and fitusiran. The HELIOS-A phase III study is evaluating vutrisiran for the treatment of hATTR amyloidosis with polyneuropathy. Another phase III study, HELIOS-B, is evaluating the candidate in hereditary and wild-type ATTR amyloidosis with cardiomyopathy. The company is developing fitusiran in partnership with Sanofi SNY in the ATLAS phase III program for the treatment of hemophilia A or B with and without inhibitors. Alnylamand partner Novartis’ NVS inclisiran is under review in the United States and Europe for treating heterozygous familial hypercholesterolemia and atherosclerotic cardiovascular disease.
Alnylam has a collaboration agreement with Regeneron Pharmaceuticals REGN to discover, develop and commercialize new RNAi therapeutics for a broad range of diseases by addressing disease targets expressed in the liver, eye and central nervous system.
Alnylam Pharmaceuticals, Inc. Price
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Alnylam is a Zacks Rank #3 (Hold) stock, currently. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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