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News Breaks
May 6, 2014
08:12 EDTALNYAlnylam advances development candidate ALN-AAT
Alnylam Pharmaceuticals is advancing its Development Candidate for ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin in development for the treatment of AAT deficiency-associated liver disease. New pre-clinical data were presented in a Late-Breaking Abstract Session at Digestive Disease Week, held May 3 – 6, 2014 in Chicago, Illinois. ALN-AAT is one of Alnylam’s genetic medicine programs, which are RNAi therapeutics directed toward genetically defined targets for the treatment of diseases with high unmet medical need. AAT deficiency-associated liver disease is caused by accumulation of mutant AAT protein in liver tissue with subsequent liver injury, fibrosis, cirrhosis, and, in some cases, hepatocellular carcinoma. It is estimated that approximately 12,000 people with AAT deficiency in the U.S. and E.U. have associated liver pathology. The company now plans to initiate IND-enabling studies with the goal of filing an IND or IND equivalent for ALN-AAT in mid-2015.
News For ALNY From The Last 14 Days
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November 19, 2014
07:52 EDTALNYInforma Business Information to hold a conference
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November 17, 2014
17:03 EDTALNYAlnylam presents new pre-clinical data on RNAi therapeutic programs
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07:09 EDTALNYAmerican Heart Association to hold a conference
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November 14, 2014
06:31 EDTALNYAlnylam to host conference call
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06:04 EDTALNYAlnylam announces positive initial phase 2 data with Revusiran
Alnylam announced positive initial Phase 2 data with revusiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of TTR cardiac amyloidosis. In the pilot Phase 2 study, revusiran was found to be generally well tolerated in TTR cardiac amyloidosis patients. Revusiran demonstrated clinical activity with an up to 98.2% knockdown of serum TTR – the disease causing protein. This included similar knockdown effects toward wild type and mutant TTR protein within V122I patients, who represent the most common genotype associated with inherited forms of TTR cardiac amyloidosis. In the five week course of treatment, there were no significant changes observed in a number of exploratory clinical measurements. Revusiran utilizes Alnylam’s proprietary GalNAc-conjugate delivery platform that enables subcutaneous delivery of RNAi therapeutics with a wide therapeutic index. The primary objective of the study was to evaluate the safety and tolerability of revusiran. The secondary objectives were to assess the clinical activity of revusiran toward serum levels of TTR and characterize the drug’s pharmacokinetic profile. In addition, a number of exploratory clinical measurements were performed at baseline and days 42 and 90 after start of dosing.
November 11, 2014
08:03 EDTALNYAlnylam adds ALN-HDV, ALN-PDL to hepatic infectious disease pipeline
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November 9, 2014
14:09 EDTALNYAlnylam management to meet with Needham
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