AstraZeneca, Ionis data spark debate about RNA drugs’ impact on deadly heart disease
Results published in NEJM raised questions about the benefits “silencer” medications provide on top of standard therapies for TTR cardiomyopathy and amplified investor pressure on Alnylam.
On Friday, AstraZeneca disclosed the unexpected failure of a high-profile heart disease clinical trial, revealing that its experimental drug, a type of silencer, provided no additional benefits for patients who were already on a stabilizer, a different class of medication. The study found that patients with ATTR-CM, a progressive condition, who received AstraZeneca’s drug, a silencer, experienced no improvement if they were already taking a stabilizer, an effect so significant that it led to the entire Phase 3 study’s failure.
At the start of the trial, most participants were already taking a stabilizer. The trial’s outcome has far-reaching implications not just for AstraZeneca and its partnership with Ionis Pharmaceuticals, but for other companies that sell ATTR-CM drugs, including Alnylam Pharmaceuticals, which offers another silencer, and BridgeBio, the manufacturer of a new stabilizer.
Analysts have suggested that the study’s results support the view that stabilizers, oral medications, may be superior to injectable silencers.
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