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Vutrisiran UpdateAlnylam Reports Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with PolyneuropathyBUSINESS WIRE 4:00 PM ET 10/27/2021 – Vutrisiran Met All 18-Month Secondary Endpoints, Including Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), Gait Speed, Nutritional Status and Overall Disability, Relative to External Placebo – – At Month 18, Vutrisiran Also Showed Improvements in Exploratory Endpoints, Including Technetium Uptake Relative to Baseline in a Planned Cohort, Providing Potential Evidence for Reduced Cardiac Amyloid Burden – – In Addition, Vutrisiran Continued to Demonstrate an Encouraging Safety and Tolerability Profile – – Alnylam Intends to Present Full HELIOS-A 18-Month Results at a – Alnylam to Discuss Data as Part of “These results build on the positive vutrisiran data shared earlier this year and suggest that the reduction of neurologic impairment and improvement in quality of life in patients with hATTR amyloidosis with polyneuropathy observed as early as nine months are maintained at 18 months. We are also encouraged by the hypothesis-supporting, exploratory endpoints and cardiac amyloid imaging results,” said At 18 months, patients treated with vutrisiran showed quantitative improvement across a number of exploratory endpoints. Compared to placebo, patients in the vutrisiran arm demonstrated improvement in the cardiac biomarker endpoint, NT-proBNP, a measure of cardiac stress. In addition, patients treated with vutrisiran also demonstrated improvement in certain echocardiographic parameters, relative to placebo. Finally, in a planned cohort of 48 patients, treatment with vutrisiran was associated with an improvement in technetium uptake in the heart in a majority of patients, providing potential evidence for reduced cardiac amyloid burden. Vutrisiran demonstrated an encouraging safety profile. There were three study discontinuations (2.5 percent) due to adverse events in the vutrisiran arm during the 18 Month treatment period; the single new discontinuation since Month 9 was an event of cardiac failure considered unrelated to study drug by the investigator. During the 18 Month treatment period, there were two deaths (neither of which was considered related to study drug) and two serious adverse events (SAEs) deemed related to vutrisiran by the study investigator; these deaths and related SAEs all occurred by Month 9 and were previously reported. Treatment emergent adverse events (AEs) occurring in 10 percent or more patients included fall, pain in extremity, diarrhea, peripheral edema, urinary tract infection, arthralgia and dizziness; with the exception of pain in extremity and arthralgia, each of these events occurred at a similar or lower rate as compared with external placebo. Injection site reactions (ISRs) were reported in 5 patients (4.1 percent) and were all mild and transient. There were no hepatic safety concerns. Vutrisiran is under review by the Conference Call Alnylam Management will discuss the HELIOS-A 18-month topline during the third quarter 2021 results conference call on A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Alnylam website approximately two hours after the event. |
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