The Phase II trial is an open-label, multi-dose study of ALN-TTRsc, designed to enroll approximately 15 TTR cardiac amyloidosis patients with FAC or SSA. The primary objective of the study is to evaluate the general tolerability of ALN-TTRsc. Patients will receive 5 daily doses followed by 5 weekly doses of 5 mg/kg, with follow-up through Day 90; in the Phase I ALN-TTRsc study, this dose resulted in an up to 93% TTR knockdown and a mean nadir knockdown of approximately 88%, and was found to be generally safe and well tolerated. Secondary objectives include assessment of clinical activity as measured by knockdown of serum TTR levels and additional tests, such as cardiac imaging (including echocardiography and cardiac MRI), circulating cardiac biomarkers (NT-proBNP and troponins T and I), 6-minute walk test, New York Heart Association (NYHA) classification, and measures of heart failure symptoms and quality of life (Kansas City Cardiomyopathy Questionnaire and EQ-5D QOL). Patients completing the Phase II trial will be eligible to participate in an open-label extension (OLE) study for further assessment of general tolerability and clinical activity with long-term dosing; the ALN-TTRsc Phase II OLE study is expected to be initiated in mid-2014.
Recent ALNY News
- Alnylam présente les résultats détaillés de l'étude de phase 3 HELIOS-B positive de Vutrisiran▼ chez les patients atteints d'amylose ATTR avec cardiomyopathie au congrès de la Société européenne de cardiologie • Business Wire • 08/30/2024 08:03:00 PM
- Alnylam präsentiert detaillierte Ergebnisse der positiven HELIOS-B-Phase-3-Studie mit Vutrisiran▼ bei Patienten mit ATTR-Amyloidose mit Kardiomyopathie auf dem Kongress der Europäischen Gesellschaft für Kardiologie • Business Wire • 08/30/2024 05:10:00 PM
- Alnylam Presents Detailed Results from the Positive HELIOS-B Phase 3 Study of Vutrisiran▼ in Patients with ATTR Amyloidosis with Cardiomyopathy at the European Society of Cardiology Congress • Business Wire • 08/30/2024 10:00:00 AM
- Alnylam to Webcast Presentations at Upcoming September Investor Conferences • Business Wire • 08/28/2024 08:00:00 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/23/2024 12:01:25 AM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 08/20/2024 09:10:42 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/14/2024 08:12:07 PM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 08/12/2024 08:07:40 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/08/2024 08:10:22 PM
- Europäischer Kardiologenkongress 2024: Alnylam legt detaillierte Ergebnisse der Phase-3-Studie HELIOS-B zu Vutrisiran▼ bei ATTR-Amyloidose mit Kardiomyopathie vor • Business Wire • 08/08/2024 02:08:00 AM
- Alnylam présentera des résultats détaillés de l'étude HELIOS-B de phase 3 pour le vutrisiran▼ chez les patients atteints d'amylose ATTR avec cardiomyopathie lors du congrès 2024 de la Société européenne de cardiologie • Business Wire • 08/07/2024 11:04:00 PM
- Alnylam to Present Detailed Results from the HELIOS-B Phase 3 Study of Vutrisiran▼ in Patients with ATTR Amyloidosis with Cardiomyopathy at the European Society of Cardiology Congress 2024 • Business Wire • 08/07/2024 12:00:00 PM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 08/06/2024 08:07:57 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 08/03/2024 12:01:26 AM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 08/01/2024 08:01:31 PM
- Form 10-Q - Quarterly report [Sections 13 or 15(d)] • Edgar (US Regulatory) • 08/01/2024 12:22:58 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 08/01/2024 12:11:34 PM
- Alnylam Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Period Activity • Business Wire • 08/01/2024 12:00:00 PM
- Alnylam to Webcast Conference Call Discussing Second Quarter 2024 Financial Results • Business Wire • 07/18/2024 12:00:00 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 07/13/2024 12:02:23 AM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 07/11/2024 08:40:50 PM
- Alnylam Reports Positive Topline Results from HELIOS-B Phase 3 Study of Vutrisiran, Achieving Statistical Significance on Primary and All Secondary Endpoints in Both Overall and Monotherapy Populations • Business Wire • 06/24/2024 11:00:00 AM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 05/31/2024 11:00:52 PM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 05/29/2024 08:01:35 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 05/20/2024 08:34:56 PM
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