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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, 2014
08:06 EDTALNYAlnylam initiates EXPLORE trial
Alnylam Pharmaceuticals and collaborators from the American Porphyria Consortium and The European Porphyria Network have initiated the EXPLORE trial, a prospective observational study of patients with hepatic porphyrias, including Acute Intermittent Porphyria, Variegate Porphyria, and Hereditary Coproporphyria, suffering from recurrent attacks. With this study, Alnylam and clinical investigators aim to learn more about the clinical course, management, and disease burden of patients with hepatic porphyrias that suffer from recurrent attacks. Alnylam is currently advancing ALN-AS1, a subcutaneously administered investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of hepatic porphyrias, and expects to file an IND or IND equivalent in late 2014 or early 2015. EXPLORE is a prospective, multi-center observational study designed to enroll up to 100 patients with hepatic porphyrias and a history of recurrent attacks. The study’s primary objective is to characterize the natural history, clinical management, and disease burden of patients with hepatic porphyrias. The study’s secondary objectives are to further characterize a number of disease features of hepatic porphyrias, including: (i) signs and symptoms of porphyria during acute attacks; (ii) levels of plasma and urinary aminolevulinic acid and porphobilinogen, the toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of the acute hepatic porphyrias; (iii) expression levels of aminolevulinic acid synthase-1 during acute attacks; and (iv) medical and family history of hepatic porphyria patients. Data obtained from EXPLORE are expected to further the understanding of hepatic porphyrias and to assist the design of clinical trials with ALN-AS1.
07:28 EDTALNYLeerink to hold a roundtable
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September 26, 2014
07:32 EDTALNYBioCentury to hold a conference
21st Annual NewsMakers in the Biotech Industry Conference to be held in New York on September 26.
September 24, 2014
08:08 EDTALNYAlnylam initiates DISCOVERY trial
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September 18, 2014
07:49 EDTALNYAlnylam resumed with an Outperform at Leerink
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