Express | Subcutaneous RNAi Therapy Reached All Phase 3 Clinical Endpoints, New Drug Application Has Been Submitted

▎The content team editor of WuXi AppTec today, Alnylam Pharmaceuticals announced that the subcutaneous RNAi therapy vutrisiran developed by the company has reached the primary end point and all the secondary clinical trials in a phase 3 clinical trial for the treatment of patients with hereditary transthyretin (hATTR) amyloidosis.
To the end.

The company has submitted a New Drug Application (NDA) to the US FDA.

If approved, it will be Alnylam's fourth RNAi therapy approved by the FDA.

Hereditary transthyretin amyloidosis is a hereditary and progressive disease caused by mutations in the TTR gene.

Mutations in the TTR gene cause abnormal amyloid accumulation and damage body organs and tissues, such as peripheral nerves and the heart, leading to refractory peripheral sensory-motor neuropathy, autonomic neuropathy and/or cardiomyopathy, and other disease manifestations.

hATTR amyloidosis represents a major unmet medical need, with significant morbidity and mortality, and approximately 50,000 people are affected worldwide.

The median survival time after diagnosis is 4.
7 years, and the survival time of cardiomyopathy patients is only 3.
4 years.

Vutrisiran is a subcutaneously administered RNAi therapy that can target and silence specific mRNAs, thereby blocking the production of wild-type and mutant transthyretin, and delaying disease progression.

Alnylam's first FDA-approved RNAi therapy Onpattro in 2018 is an RNAi therapy for the treatment of hATTR amyloidosis.
However, that therapy needs to be administered intravenously every 3 weeks.

Vutrisiran utilizes Alnylam's Enhanced Stable Chemistry (ESC)-GalNAc-coupled delivery platform to improve its efficacy and metabolic stability, allowing patients to receive only one subcutaneous injection every 3 months.

▲Introduction to Onpattro and vutrisiran (picture source: Alnylam official website) In the phase 3 clinical study named HELIOS-A, a total of 164 patients with hATTR amyloidosis received vutrisiran or Onpattro treatment.

The results of the trial showed that at 9 months of treatment, the mNIS+7 score of patients in the vutrisiran group (a method of assessing neuropathic damage) decreased by an average of 2.
24 points from baseline, while the historical placebo group increased by an average of 14.
76 points (a rise in score means Symptoms worsen, and decline means symptom relief).

In terms of Norfolk QoL-DN score, which assesses the quality of life of patients, patients with vutrisiran have improved 16.
2 points compared with the historical placebo group.

At the same time, the evaluation of patients' walking speed and other physiological indicators has also been improved.

"Vutrisiran's encouraging efficacy and safety results in phase 3 clinical trials have further strengthened our confidence in the potential of RNAi therapy.

" Alnylam R&D President Dr.
Akshay Vaishnaw said, "We have completed the submission of the NDA application and look forward to it soon.
Bring this new treatment option of subcutaneous injection every quarter to hATTR amyloidosis patients.

Reference: [1] Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran.
Retrieved April 19, 2021, from https ://www.
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com/news/home/20210419005552/en Note: This article aims to introduce the progress of medical and health research, not to recommend treatment plans.

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