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Alnylam Initiates Phase III ILLUMINATE-B Study on Lumasiran

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Alnylam Pharmaceuticals, Inc. (ALNY - Free Report)  announced that it has initiated ILLUMINATE-B, a global phase III pediatric study on its investigational, subcutaneously administered RNAi therapeutic, lumasiran. The drug is being evaluated for the treatment of primary hyperoxaluria type 1 (PH1).

The company also announced new positive efficacy results from the ongoing phase II open-label extension (OLE) study on lumasiran. All patients (N=20) from the phase I/II study on lumasiran transitioned to the phase II OLE study. As of Feb 8, the data cut-off date, 18 patients were dosed in the OLE study. Patients were on a range of lumasiran doses and regimens (1.0 mg/kg monthly, 3.0 mg/kg monthl, and 3.0 mg/kg quarterly).

In the study, patients dosed with lumasiran showed a 72% mean maximal reduction in urinary oxalate excretion relative to phase I/II baseline values across all dose cohorts. The drug also showed a mean maximal reduction in urinary 24-hour oxalate:creatinine ratio of 77% (range:57-91%) relative to phase I/II baseline values across all dose cohorts.  

There were no discontinuations from study treatment.  

Shares of Alnylam have declined 10% in the past year compared with the industry’s decline of 11.5%.

 

In February 2019, Alnylam initiated ILLUMINATE-A, a global phase III pivotal study on lumasiran, in children and adult PH1 patients with preserved renal function. The company expects to report top-line results from the study late 2019. If data are positive, the company plans to submit filings for global regulatory approvals 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. The drug is the first and only FDA-approved treatment for this indication. 

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

Alnylam’s expertise in RNAi therapeutics and broad intellectual property estate have allowed it to enter collaborations with leading pharmaceutical and life sciences companies, including Ionis Pharmaceuticals, Novartis (NVS - Free Report) , Roche, Takeda, Merck, The Medicines Company and Sanofi’s specialty care global business unit, Genzyme, among others.

Recently, Alnylam and Regeneron Pharmaceuticals (REGN - Free Report) extended their collaboration agreement. Both the 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 central nervous system (CNS), in addition to a select number of targets expressed in the liver.

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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