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April 2, 2014
08:08 EDTALNYAlnylam receives positive opinion for orphan drug designation ALN-TTRsc
Alnylam Pharmaceuticals announces that the European Medicines Agency Committee for Orphan Medicinal Products has adopted a positive opinion recommending ALN-TTRsc for designation as an orphan medicinal product for the treatment of transthyretin-mediated amyloidosis. ALN-TTRsc is currently in a pilot Phase 2 clinical trial for the treatment of ATTR patients with TTR cardiac amyloidosis; this study is aimed at evaluating the tolerability of ALN-TTRsc in approximately 15 patients. In addition, the study will assess preliminary clinical activity as measured by knockdown of serum TTR levels and additional exploratory tests, such as cardiac imaging, circulating cardiac biomarkers, 6-minute walk test, New York Heart Association classification, and measures of heart failure symptoms and quality of life. The company expects to present data from the Phase 2 trial in late 2014. Patients completing the Phase 2 trial will be eligible to participate in an open-label extension study for further assessment of general tolerability and clinical activity with long-term dosing; the ALN-TTRsc Phase 2 OLE study is expected to be initiated in mid-2014. Assuming positive results, Alnylam expects to begin a Phase 3 trial in TTR cardiac amyloidosis patients by the end of 2014.
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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