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 Issues 2023 Corporate Responsibility Report • Business Wire • 05/07/2024 12:00:00 PM
- Alnylam Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Period Activity • Business Wire • 05/02/2024 12:00:00 PM
- Medison Pharma and Alnylam Pharmaceuticals Announce Expansion of their Multi-Regional Partnership in Europe and Israel to Commercialize RNAi Therapeutics in additional LATAM and APAC markets including Australia • PR Newswire (US) • 04/30/2024 11:00:00 AM
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- Alnylam to Webcast Conference Call Discussing First Quarter 2024 Financial Results • Business Wire • 04/18/2024 12:00:00 PM
- Alnylam Presents Positive Results from the KARDIA-2 Phase 2 Study of Zilebesiran Added to Standard of Care Antihypertensives in Patients with Inadequately Controlled Hypertension • Business Wire • 04/07/2024 12:00:00 PM
- Alnylam to Webcast Investor Event to Discuss Results from KARDIA-2 Phase 2 Study of Zilebesiran at American College of Cardiology Scientific Session • Business Wire • 03/20/2024 12:00:00 PM
- Alnylam Launches Hereditary ATTR (hATTR) Amyloidosis Campaign to Help Shorten Time to Diagnosis for Inherited and Rapidly Progressive Disease • Business Wire • 03/13/2024 11:30:00 AM
- Alnylam meldet positive KARDIA-2 Topline-Studienergebnisse, die eine klinisch signifikante Blutdrucksenkung bei Zugabe von Zilebesiran zu Standard-Blutdrucksenkern belegen • Business Wire • 03/06/2024 11:50:00 AM
- Alnylam annonce des résultats positifs de l’étude KARDIA-2 démontrant des réductions cliniquement significatives de la pression artérielle lorsque le zilebesiran est ajouté à des antihypertenseurs standards • Business Wire • 03/05/2024 10:39:00 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 03/05/2024 09:22:04 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 03/05/2024 09:21:36 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 03/05/2024 09:21:19 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 03/05/2024 09:20:54 PM
- Alnylam Reports Positive KARDIA-2 Topline Study Results Demonstrating Clinically Significant Blood Pressure Reductions When Zilebesiran is Added to Standard of Care Antihypertensives • Business Wire • 03/05/2024 12:30:00 PM
- Form 8-K - Current report • Edgar (US Regulatory) • 03/04/2024 09:33:42 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/29/2024 09:18:42 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/29/2024 09:18:30 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/29/2024 09:18:02 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/29/2024 09:17:12 PM
- Form 144 - Report of proposed sale of securities • Edgar (US Regulatory) • 02/29/2024 09:00:48 PM
- Alnylam to Webcast Presentations at Upcoming March Investor Conferences • Business Wire • 02/27/2024 01:30:00 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/20/2024 09:08:59 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/20/2024 09:08:24 PM
- Form 4 - Statement of changes in beneficial ownership of securities • Edgar (US Regulatory) • 02/20/2024 09:07:31 PM
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