3 months a shot!

On January 21, 2022, Alnylam Pharmaceuticals announced positive results from a Phase 3 trial of its investigational RNAi therapy vutrisiran

hATTR amyloidosis is an inherited, progressive, debilitating and fatal disease caused by mutations in the TTR gene

Vutrisiran is an investigational subcutaneous RNAi therapeutic being developed for the treatment of ATTR amyloidosis, including hATTR and wild-type ATTR amyloidosis

▲ Introduction of Alnylam's ATTR amyloidosis product pipeline (Image source: Alnylam official website)

The phase 3 clinical trial enrolled 164 patients with hATTR-PN, randomized 3:1 to receive 25 mg vutrisiran (n=122) subcutaneously every three months, or 0.

The results of the trial at month 18 are as follows:

The Vutrisiran group (n=122) had a mean decrease from baseline in the Modified Neuropathic Impairment Score (mNIS+7) of 0.

Compared with a mean increase of 19.

Image source: Alnylam official website

Patients in the Vutrisiran group showed statistically significant improvements in measures of gait speed (10-MWT), nutritional status (mBMI), and disability (R-ODS) compared to placebo

In addition to meeting the trial's secondary endpoints, patients in the vutrisiran arm also demonstrated improvements in exploratory cardiac endpoints compared with placebo, continuing to support vutrisiran's potential to reduce cardiac amyloid burden and improve cardiac disease in patients

References:

[1] Alnylam Presents Positive 18-Month Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy.