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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.”
News For ALNY From The Last 14 Days
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October 1, 2015
13:00 EDTALNYAlnylam recent weakness a buying opportunity, says Piper Jaffray
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07:01 EDTALNYGenzyme opts into ALN-AT3 Hemophilia Program
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September 30, 2015
07:11 EDTALNYLeerink to hold a roundtable
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September 28, 2015
14:49 EDTALNYAlnylam granted summary judgment by U.S. district court in patent dispute
Alnylam Pharmaceuticals announced that Chief Judge Patti Saris of the U.S. District Court for the District of Massachusetts granted Alnylam's summary judgment motion in an inventorship dispute, holding that Professor Brenda Bass, Distinguished Professor at the University of Utah, is not a co-inventor of certain patents, termed the Tuschl II patents, jointly owned by co-defendants the Max Planck Society, UMass, Massachusetts Institute of Technology, and the Whitehead Institute for Biomedical Research, and exclusively licensed to Alnylam. In 2011 Utah sued Alnylam, Max Planck, UMass, MIT, and Whitehead alleging that Dr. Bass was either the sole or a co-inventor of the Tuschl II patents, seeking correction of inventorship as well as unspecified monetary damages. In 2015 Utah dropped their sole inventorship claim. The Court found that there was no collaboration between Dr. Bass and Dr. Tuschl, which is a pre-requisite for co-inventorship, and also granted the defendants' motion for summary judgment on Utah's state law claims. While Utah can appeal this ruling, Alnylam remains firm in its belief that the inventorship of the Tuschl II patents as stated in the issued patents is correct, the company said.
11:14 EDTALNYAlnylam OLE data has positive read through to Phase 3 trial, says Piper Jaffray
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08:05 EDTALNYAlnylam reports new results from ongoing Phase 2 OLE study of patisiran
Alnylam Pharmaceuticals and collaborators announced new results from the company's ongoing Phase 2 open-label extension study of patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of TTR-mediated amyloidosis patients with familial amyloidotic polyneuropathy. In collaboration with investigators at The Scripps Research Institute and Misfolding Diagnostics, Inc., patisiran administration was shown to reduce pathogenic, misfolded TTR monomers and oligomers in FAP patients in the Phase 2 OLE study. Specifically, patisiran administration resulted in a rapid and sustained reduction of approximately 90% in serum non-native conformations of TTR. Since NNTTR is pathogenic in ATTR amyloidosis, these results provide direct mechanistic evidence supporting the therapeutic hypothesis that TTR knockdown has the potential to result in clinical benefit. In addition, Alnylam presented complete 12-month data from all 27 patients initially enrolled in the patisiran OLE study, showing a mean 3.1-point decrease in the modified Neuropathy Impairment Score at 12 months as well as sustained mean maximum reductions in total serum TTR of 91% for over 18 months. Moreover, patisiran administration was found to be generally well tolerated out to 21 months of treatment. Alnylam's ongoing Phase 2 OLE study is an open-label, multi-center trial designed to evaluate the long-term safety and tolerability of patisiran administration in FAP patients that were previously enrolled in a Phase 2 study. Patisiran is being administered once every 3 weeks at a dose of 0.3 mg/kg by intravenous infusion. The study is measuring a number of clinical endpoints every six months, including mNIS+7 which is an evaluation of muscle weakness, sensory and autonomic function, and nerve conductance, where neuropathy progression leads to an increased score over time. The change in the mNIS+7 measurement from baseline to 18 months is the primary endpoint in the company's ongoing Phase 3 APOLLO trial of patisiran in FAP patients.

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