Expect top line results in Feb. 2021
Alnylam Completes Enrollment of HELIOS-A Phase 3 Study of Vutrisiran for the Treatment of hATTR Amyloidosis with Polyneuropathy
Feb 18, 2020
– HELIOS-A Topline Results Expected in Early 2021 –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 18, 2020-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that it has achieved full enrollment in its HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis. The study was designed to enroll 160 patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy across 68 sites in 22 countries. Alnylam is on track to report topline results from HELIOS-A in early 2021.
“We are pleased to have reached another important milestone for our TTR program by completing enrollment of the HELIOS-A study,” said Rena Denoncourt, Program Leader, Vutrisiran Program at Alnylam. “We are committed to developing multiple therapeutic options for this progressive, life-threatening and multisystem disease, and believe that vutrisiran, as a low-dose, once-quarterly, subcutaneously administered investigational therapeutic, can be an attractive option for patients. We look forward to sharing topline results in early 2021.”
HELIOS-A is a Phase 3 global, randomized, open-label study to evaluate the efficacy and safety of vutrisiran in patients with hATTR amyloidosis with polyneuropathy. The trial randomized patients 3:1 to receive either 25mg of vutrisiran subcutaneously once every 12 weeks or 0.3 mg/kg of patisiran intravenously once every three weeks. For most endpoints, results from the vutrisiran arm will be compared to results from the placebo arm of the landmark APOLLO Phase 3 study, which evaluated the efficacy and safety of patisiran in people with hATTR amyloidosis with polyneuropathy. The co-primary endpoints of HELIOS-A are the change from baseline in the modified Neurologic Impairment Score +7 (mNIS+7) and in the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) score, at 9 months. Secondary endpoints include the change from baseline in key clinical evaluations including the timed 10-meter walk test (10-MWT), modified body mass index (mBMI), and Rasch-built Overall Disability Scale (R-ODS). The percent reduction in serum transthyretin (TTR) levels in the vutrisiran arm will be compared to the within-study patisiran arm. Additional exploratory endpoints will be assessed to determine the effect of vutrisiran on other aspects of the multisystem nature of this disease, including manifestations of cardiac amyloid involvement.
Vutrisiran has been granted Orphan Drug Designation in the United States and the European Union for the treatment of ATTR amyloidosis. The safety and efficacy of vutrisiran are also being evaluated in the ongoing HELIOS-B Phase 3 clinical trial in patients with ATTR amyloidosis with cardiomyopathy. The HELIOS-B study was initiated in late 2019 and is currently enrolling at sites around the world. Together, the HELIOS-A and -B studies comprise a comprehensive clinical development program intended to demonstrate the broad impact of vutrisiran across the multisystem manifestations of disease and the full spectrum of patients with ATTR amyloidosis.
Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and mutant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s next-generation delivery platform known as the Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform. The safety and efficacy of vutrisiran have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency or any other health authority.