Two years ago, Alnylam Pharmaceuticals presented study data showing that people with a deadly heart condition would live longer, and better avoid hospital stays, if they received a drug combination involving nucleic acid-based medicine it had developed.
That finding suggested there are additive benefits to using Alnylam’s medication, known as Amvuttra, on top of standard medications. The results added billions of dollars to Alnylam’s market value, and led to the drug’s approval in that heart condition, called transthyretin-mediated amyloidosis cardiomyopathy.
New study results published in the New England Journal of Medicine and presented at a medical meeting on Friday, however, raise questions about the impact drugs like Amvuttra have. In that trial, partners AstraZeneca and Ionis Pharmaceuticals found that a similar kind of medication didn’t have the same benefits. Since their top-line release in July, the data sparked questions about why eplontersen might have fallen short and whether Alnylam’s drug is as impactful as testing indicated.
Called Waiuna and known scientifically as eplontersen, Ionis and AstraZeneca’s drug is already approved for a rarer form of transthyretin amyloidosis that affects the nerves. The companies had hoped to make the drug more widely available by initiating the “CARDIO-TTRansform” study, a large study testing the treatment in people with TTR cardiomyopathy.
