ALN-TTRsc clinical activity establishes new benchmark for consistent ttr knockdown of ~90%.
Multiple doses of aln-ttrsc found to be generally safe and well tolerated in phase i study.
Results Establish human translation of Alnylam's proprietary galnac-sirna conjugate platform for subcutaneous administration of RNAi Therapeutics with wide therapeutic index
Company to host conference call at 8:00 a.m. today to discuss clinical results.
Co announced positive interim results from its Phase I clinical trial of ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene for the treatment of TTR-mediated amyloidosis. The data are being presented today at the Heart Failure Society of America 17th Annual Scientific Meeting being held September 22 -- 25, 2013 in Orlando, Fla. Results show that ALN-TTRsc administration led to robust, consistent, and statistically significant knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR, the disease causing protein in ATTR, was found to be rapid, dose dependent, and durable. To date, ALN-TTRsc has been found to be generally safe and well tolerated in this study.
In addition, knockdown of TTR, the disease causing protein in ATTR, was found to be rapid, dose dependent, and durable. To date, ALN-TTRsc has been found to be generally safe and well tolerated in this study. These human data are the first to be presented for Alnylam's proprietary GalNAc-siRNA conjugate delivery platform, enabling subcutaneous dosing of RNAi therapeutics with a wide therapeutic index, and demonstrate human translation for this platform. Moreover, these results establish a new benchmark for consistent TTR knockdown of approximately 90% for RNA therapeutics in development for the treatment of ATTR.
ALN-TTRsc, which is being developed for the treatment of FAC, is a subcutaneously administered RNAi therapeutic that comprises an siRNA conjugated to a GalNAc ligand that enables receptor-mediated delivery to the liver. ALN-TTRsc is the first GalNAc-siRNA -- and the first subcutaneously delivered systemic RNAi therapeutic -- to enter clinical development stages. Alnylam is also developing ALN-TTR02, an intravenously administered RNAi therapeutic targeting TTR for the treatment of ATTR patients with FAP.
The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects. The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc.
Interim data from the 28 subjects enrolled and analyzed in this study to date showed that single- and multi-dose administration of ALN-TTRsc resulted in rapid, dose-dependent, consistent, and durable knockdown of serum TTR levels. In the multi-dose cohorts, there was a statistically significant knockdown of serum TTR at doses of 2.5 mg/kg, 5.0 mg/kg, and 10.0 mg/kg as compared to placebo.
In 2012, Alnylam entered into an exclusive alliance with Genzyme, a Sanofi company, to develop and commercialize RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, for the treatment of ATTR in Japan and the broader Asian-Pacific region. Alnylam plans to develop and commercialize the ALN-TTR program in North and South America, Europe, and rest of the world.
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
Element79 Gold Corp Appoints Warren Levy to Board of Directors • ELMGF • Aug 28, 2024 9:51 AM
Integrated Ventures Announces Strategic Entry Into Health & Wellness Industry with Focus on GLP-1 Products • INTV • Aug 28, 2024 8:30 AM
ELEMENT79 ANNOUNCES UPLISTING TO OTCQB VENTURE MARKET • ELMGF • Aug 26, 2024 10:03 AM
North Bay Resources Announces Gold Assays up to 2.2 Ounces per Ton, Fran Gold Project, British Columbia • NBRI • Aug 26, 2024 10:00 AM
PickleJar Unveils Latest Venue Managed Services Innovations in Upcoming Webinar • PKLE • Aug 23, 2024 1:11 PM
Element79 Gold Corp Provides Update on Nevada Portfolio • ELMGF • Aug 23, 2024 8:00 AM