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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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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.

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