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May 21, 2014
08:03 EDTALNYAlnylam announces publication of pre-clinical results with ALN-AS1
Alnylam Pharmaceuticals announced the publication in the Proceedings of the National Academy of Sciences of pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 for the treatment of hepatic porphyrias, including acute intermittent porphyria. In the paper, titled “RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice,” Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City documented results from pre-clinical models of the human disease showing that RNAi therapeutics targeting ALAS-1 can completely block the abnormal production of toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of AIP. This new paper provides proof of concept for an RNAi therapeutic for the treatment of AIP. “Our data in a mouse model of AIP, now published in PNAS, demonstrate that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid, and durable suppression of the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP. As such, these findings provide key pre-clinical proof-of-concept data for our ALN-AS1 program. We believe ALN-AS1 has the potential to be a transformative therapy for patients with hepatic porphyrias including AIP, an ultra-rare genetic disease with enormous unmet medical need,” said Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development of Alnylam. “We are currently advancing our Development Candidate for ALN-AS1, which employs our Enhanced Stabilization Chemistry-GalNAc-conjugate technology. This technology enables subcutaneous dosing with improved potency and durability, and a wide therapeutic index, and is now clinically validated based on results from our hemophilia program. We are on track to file an Investigational New Drug application for ALN-AS1 in late 2014 or early 2015, and look forward to advancing this investigational medicine to patients.”
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June 23, 2015
16:18 EDTALNYAlnylam weakness a buying opportunity, says Deutsche Bank
Deutsche Bank said Alnylam's Phase 1 AT-3 conference presentation update was positive, as expected, supporting safety and efficacy. The firm's analyst said weakness could be due to fast money selling and recommends buying shares on weakness.
14:31 EDTALNYAlnylam reports new positive clinical data for ALN-AT3
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June 15, 2015
05:36 EDTALNYAlnylam price target raised to $180 from $155 at Piper Jaffray
Piper Jaffray analyst Edward Tenthoff raised his price target for shares of Alnylam Pharmaceuticals to $180 after the company reported preliminary Phase I ALN-CC5 data at the European Hematology Association meeting. Hemolysis was lowered by an average of 43% with a max knockdown of 61% after a single dose, making ALN-CC5 the company's fourth RNAi drug to achieve human proof-of-concept, Tenthoff pointed out. He views Alnylam as an emerging orphan drug play with multiple potential blockbusters and reiterates an Overweight rating on the name. The stock closed Friday down $2.46 to $130.28.

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