Alnylam Releases Positive Top-Line Rare Disease Lumasiran Data
Alnylam Pharmaceuticals Inc. ALNY, announced positive top-line results from the ILLUMINATE-B pediatric phase III study of its RNAi candidate, lumasiran, in development for the treatment of primary hyperoxaluria type 1 (PH1). The ultra-rare disease causes a progressive decline in kidney function and can lead to end-stage renal disease.
Lumasiran targets glycolate oxidase to counter the overproduction of oxalate that leads to kidney damage in patients with PH1.
Shares of Alnylam have gained 26.4% year to date compared with the industry’s growth of 3%.
The ILLUMINATE-B (NCT03905694) study enrolled 18 patients with PH1 belowthe age of six. Lumasiran was administered according to a weight-based dosing regimen. The primary efficacy endpoint of the study was the percent change from baseline to Month 6 in spot urinary oxalate to creatinine ratio averaged across Months 3 to 6. At six months, relative to baseline, lumasiran demonstrated a clinically meaningful reduction in spot urinary oxalate:creatinine ratio.
The safety and efficacy of lumasiran were consistent with that reported for the ILLUMINATE-A study in patients aged six years and older, demonstrating that lumasiran can significantly reduce the hepatic production of oxalate across all ages.
The company believes that a significant reduction in urinary oxalate levels has the potential to favorably impact disease progression and management in very young patients.
Lumasiran enjoys Orphan Drug, Breakthrough Therapy and Pediatric Rare Disease designations for treating PH1.Alnylam has filed a new drug application (NDA) for lumasiran with the FDA. The FDA has granted Priority Review tothe NDA and set an action date of Dec 3, 2020. Further, the marketing authorization application (MAA) for lumasiran has been submitted to and validated by the EMA, and received Accelerated Assessment designation.
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. The company and 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 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 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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