Drug Research
Drug Discovery & Development

Alnylam scraps RNAi drug development over safety concerns

PBR Staff Writer Published 06 October 2016

Alnylam Pharmaceuticals is stopping the development of revusiran, a therapeutic intended at treating hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), due to safety concerns.

ATTR amyloidosis disease occurs due to the deposition of transthyretin in peripheral tissues.

The company said study safety monitors recommended that the phase 3 trial be stopped after patients on a previous study developed neuropathy, or nerve pain.

The data-monitoring committee told Alnylam that the benefit-risk profile did not support continued dosing.

The company then reviewed unblinded data that showed an imbalance of mortality compared with placebo.

Alnylam CEO John Maraganore said: “We have stopped all dosing and are actively monitoring patients across revusiran studies to ensure their safety. We will also continue to evaluate ENDEAVOUR data to understand the potential cause of these findings.

"While this outcome is disappointing given the lack of available treatment options for patients suffering from this devastating disease, we remain committed to serving the needs of the ATTR amyloidosis community.”

The company said its discontinuance of revusiran will not affect patisiran, now in phase 3 trials to treat hATTR amyloidosis with polyneuropathy, or any of its other investigational RNAi therapeutic programs.

Alnylam is partnering with The Medicines Company on a potential treatment for hypercholesterolemia known as ALN-PCSsc or PCSK9si.

In 2014, Alnylam and a Sanofi unit formed an alliance to develop drugs intended at treating rare genetic diseases.