2.8. Alnylam’s big bet in amyloidosis

By late June or early July we’ll know whether Alnylam’s decision to alter the design of its phase 3 ‘Healios B’ study of Amvuttra (vutrisiran) in transthyretin amyloidosis with cardiomyopathy (ATTR-CM) was a good one. The short-interfering RNA (siRNA) drug is already approved in ATTR with polyneuropathy (ATTR-PN), a debilitating, fatal condition in which misfolded transthyretin proteins clump together and create damaging deposits in the peripheral nerves. 

But about ten times as many patients develop ATTR-CM, in which protein deposition occurs in cardiac tissue and gives rise to a form of heart failure. Alnylam is, therefore, attempting to establish Amvuttra in this category as well. 

An earlier drug from the same stable, Onpattro (patisiran), failed to deliver in ATTR-CM, but the company argues that it needed a longer, larger trial to detect the survival signals it is now seeking with Amvuttra in the Healios B study. Pfizer is the current category leader in this space with its Vyndaqel (tafadamis) product family. 

Alnylam maintains that extending the duration of the follow-up on Healios B will improve the study’s statistical power and strengthen its hand when commercializing the product. But as Alnylam’s leadership knows, changing the goalposts mid-trial can be a risky move. It’s a big play from CEO Yvonne Greenstreet and chief medical officer Pushkal Garg. 

Companies mentioned in this episode: 
Alexion Pharmaceuticals, Alnylam Pharmaceuticals, Athena Neurosciences, AstraZeneca, Eidos Therapeutics, FoldRx, Ionis Pharmaceuticals, Pfizer, Neurimmune, Novo Nordisk, Prothena Biosciences

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