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Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of VutrisiranAlnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with PolyneuropathyJan 07, 2021 – Vutrisiran Met Primary and All Secondary Endpoints at 9 Months, with Statistically Significant Improvements in Progression of Neuropathy, Quality of Life (QOL), and Gait Speed, Relative to Placebo – – Majority of Patients Showed Reversal of Disease Manifestations with Improvements in Neuropathy Impairment and QOL, Relative to Baseline – – Vutrisiran Showed Improvements in the 9-Month Exploratory Cardiac Endpoint of NT-proBNP, Relative to Placebo – – In Addition, Vutrisiran Demonstrated Encouraging Safety and Tolerability Profile – – Alnylam Intends to Present Full 9-Month Results and File New Drug Application (NDA) in Early 2021 – – Based on these positive results, the Company plans to submit a New Drug Application (NDA) for vutrisiran with the “We are excited to report positive topline results from the HELIOS-A study, which show that vutrisiran reduces neurologic impairment and improves quality of life in patients with hATTR amyloidosis with polyneuropathy as soon as 9 months, with an encouraging safety and tolerability profile. In addition, we’re very pleased to see evidence for reversal of polyneuropathy manifestations of disease and also favorable effects on the exploratory cardiac endpoint, NT-proBNP. We believe that vutrisiran, as a low-dose, once-quarterly, subcutaneously administered therapy, has the potential to be a highly attractive therapeutic option for patients living with this progressive, life-threatening, multi-system disease. We look forward to presenting the full 9-month results from HELIOS-A at a medical meeting in early 2021 and to announcing additional 18-month results, including additional exploratory cardiac endpoint data, in late 2021,” said HELIOS-A (NCT03759379) is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran. The study enrolled 164 patients with hATTR amyloidosis with polyneuropathy at 57 sites in 22 countries. Patients were randomized 3:1 to receive either 25mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (as a reference comparator) for 18 months. The primary endpoint is the change from baseline in mNIS+7 score at 9 months1, relative to historical placebo. Secondary endpoints at 9 months are the change from baseline in the Norfolk QoL-DN score and the timed 10-MWT, relative to historical placebo. Changes from baseline in NT-proBNP were evaluated as an exploratory endpoint at 9 months. The efficacy results of vutrisiran in HELIOS-A are compared to historical placebo control data from the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in a patient population similar to that studied in HELIOS-A. Additional secondary endpoints at 18 months will be evaluated in the HELIOS-A study, including change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified body mass index (mBMI), Rasch-built Overall Disability Scale (R-ODS), and serum transthyretin (TTR) levels. Additional exploratory cardiac endpoint data at the 18-month time point will be evaluated, including NT-proBNP, echocardiographic measures and cardiac amyloid assessments with technetium scintigraphy imaging. Following the 18-month study period, all patients are eligible to receive vutrisiran for an additional 18 months as part of an open-label extension study. Full 9-month results will be presented at a medical conference in early 2021 and topline 18-month results, including further exploratory cardiac endpoint data, are expected to be announced in late 2021. Vutrisiran demonstrated an encouraging safety profile. There were two study discontinuations (1.6 percent) due to adverse events in the vutrisiran arm by Month 9, both due to deaths, neither of which was considered related to study drug. There were two serious adverse events (SAEs) deemed related to vutrisiran by the study investigator, consisting of dyslipidemia and urinary tract infection. Treatment emergent adverse events (AEs) occurring in 10 percent or more patients included diarrhea, pain in extremity, fall and urinary tract infections, with each of these events occurring at a similar or lower rate as compared with historical placebo. Injection site reactions (ISRs) were reported in five patients (4.1 percent) and were all mild and transient. There were no clinically significant changes in liver function tests (LFTs). “The HELIOS-A results reinforce our commitment to building an industry-leading franchise of medicines for the treatment of ATTR amyloidosis which began with the development and approval of ONPATTRO as a treatment for patients with hATTR amyloidosis with polyneuropathy. Indeed, the vutrisiran results from HELIOS-A now serve as a second example of the potential for RNAi therapeutics to have a meaningful impact for patients, showing the ability to halt and potentially even reverse polyneuropathy manifestations of the disease. Furthermore, our robust development program, including the APOLLO-B and HELIOS-B studies, investigates the potential of patisiran and vutrisiran, respectively, to treat the cardiac manifestations of disease across a broad spectrum of patients with ATTR amyloidosis,” said Vutrisiran has been granted Orphan Drug Designation in Conference Call Information |
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Msg # | Subject | Author | Recs | Date Posted |
34950 | Re: Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran - Fierce Biotech | Steve_382 | 0 | 1/7/2021 10:55:11 AM |