Alnylam under-skin injection RNAi therapy Phase 3 clinical trial results were positive

Hereditary thyroxine protein (hATTR) amyloid degeneration is a hereditary, regenerative disease caused by mutations in the TTR gene, which is usually fatal.
TTR gene mutations cause abnormal amyloid protein build-up and damage to body organs and tissues, such as exoskeric nerves and the heart, leading to stubborn exoskeric sensations - motor neuropathy, autonotic neuropathy and/or cardiomyopathy, and other disease manifestations.
hATTR amyloid represents a significant unsealed medical need, with significant morbidity and mortality, affecting approximately 50,000 people worldwide.
was 4.7 years after diagnosis and 3.4 years in patients with cardiomyopathy.
Vutrisiran is an RNAi therapy that is drugged under the skin and targets and silences specific mRNAs, blocking the production of wild and mutant transthyroxine proteins and slowing disease progression.
Alnylam's first FDA-approved RNAi therapy, Onpattro, in 2018, is an RNAi treatment for hATTR amyloid degeneration, which requires intravenous administration every three weeks.
Vutrisiran leverages Alnylam's Enhanced Stability Chemistry (ESC)-GalNAc-Even Delivery Platform to improve its potency and metabolic stability, allowing patients to receive only subsescape injections every three months.
in a randomized, open-label, global Phase 3 clinic called HELIOS-A, 164 patients with highly pathogenic neuropathy (polyneuropathy) were treated with vutrisiran or Onpattro, respectively.
9 months after treatment, the change in the mNIS-7 score (a way to assess neuropathic injuries) in patients in the vutrisiran group was significantly improved compared to the historical placebo group data.
the quality of life and the indicators of walking speed were also significantly improved.
"We are pleased to report the positive results of the HELIOS-A study, which showed that vutrisiran could reduce nerve function damage in patients with high ATTR amyloid with multiple neuropathy for up to nine months, improve quality of life, and have encouraging safety and tolerance characteristics.
we believe that vutrisiran, as a low-dose, quarterly subsection treatment, has the potential to be an attractive treatment option for patients.
," said Dr. Akshay Vaishnaw, President of Research and Development at Alnylam.
: This article is intended to introduce medical and health research, not treatment options recommended.
if you need guidance on treatment options, visit a regular hospital.
: s1. Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy. Retrieved January 7, 2021, from