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Alnylam Files Clinical Trial Application for ALN-AAT02 in UK

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Alnylam Files Clinical Trial Application for ALN-AAT02 in UK

Alnylam Pharmaceuticals (ALNY) submits a Clinical Trial Authorization application in the U.K. to start a phase I/II study on its early stage candidate, ALN-AAT02, for treating alpha-1 liver disease.

Alnylam Pharmaceuticals, Inc. ALNY announced that it has filed a Clinical Trial Authorization (CTA) application to Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom to initiate phase I/II study on its early stage candidate ALN-AAT02. The candidate is being evaluated for the treatment of alpha-1 antitrypsin (AAT) deficiency-associated liver disease (alpha-1 liver disease).

ALN-AAT02 is the first investigational RNA interference (RNAi) therapeutic targeting AAT with Alnylam’s enhanced stabilization chemistry plus (ESC+) GalNAc-conjugate technology. The company expects to initiate the study by the end of 2018. However, the safety and efficacy of ALN-AAT02 are yet to be evaluated by any health authority including the FDA and EMA.

Per the press release, treatment of alpha-1 liver disease highlights an area of significant unmet medical need covering patients with liver transplantation. The treatment comes as the only available option for those suffering the indication. Hence, a positive feedback from MHRA will help the company address a new patient population.

Shares of Alnylam have plummeted 30.6% so far this year versus the industry’s decrease of 3.1%.


Notably, Alnylam is focused on developing novel therapeutics based on RNA interference (RNAi). The company’s pipeline of experimental RNAi therapeutics is focused on three strategic therapeutic areas (STArs), namely genetic medicines, cardio-metabolic disease and hepatic infectious disease.

In August 2018, the FDA approved Onpattro (patisiran) lipid complex injection, a first-of-its-kind RNA interference (RNAi) therapeutic, for treating polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Onpattro is the first and the only FDA-approved treatment for this indication. In the same month, the European Commission (EC) also granted a marketing authorization to Onpattro.

Zacks Rank & Stocks to Consider

Alnylam currently carries a Zacks Rank #3 (Hold).

Better-ranked stocks in the health care sector are Ligand Pharmaceuticals Inc. LGND, Caladrius Biosciences, Inc. CLBS and Athersys, Inc. ATHX, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Ligand’s earnings estimates have been revised 28.4% upward for 2018 and 6.5% for 2019 over the past 60 days. The stock has skyrocketed 89.3% so far this year.

Caladrius’ loss per share estimates has narrowed 5.7% for 2018 and 1.6% for 2019 over the past 60 days. The stock has soared 64.4% year to date.

Athersys’ loss per share estimates has narrowed 12.5% for 2018 and 4.3% for 2019 over the past 60 days. The stock has rallied 17.6% year to date.

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