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AstraZeneca details clinical trial failure of experimental heart disease drug

A Phase 3 study showed no added benefit from an experimental silencer drug when patients were already taking stabilizer medication.

The short version

  • AstraZeneca and Ionis Pharmaceuticals reported that their Phase 3 trial for an experimental ATTR-CM silencer drug failed to show additional benefits for patients already on a stabilizer drug.
  • Most trial participants were already taking a stabilizer medication at the beginning of the study.
  • The results affect rival companies in the multi-billion dollar ATTR-CM market, including Alnylam Pharmaceuticals and BridgeBio.
  • Industry analysts suggested the outcome strengthens the case that oral stabilizers may be more effective than injectable silencers.

Key facts

  • AstraZeneca provided details on the failure of a Phase 3 trial testing an experimental silencer drug developed with Ionis Pharmaceuticals for ATTR-CM.[STAT]
  • The trial demonstrated no additional health improvements for ATTR-CM patients who were already receiving a stabilizer drug.[STAT]
  • A majority of trial participants were taking a stabilizer drug at the start of the study.[STAT]
  • The findings carry commercial implications for competitors in the ATTR-CM treatment market, such as Alnylam Pharmaceuticals and BridgeBio.[STAT]

What remains uncertain

  • The full long-term commercial impact on injectable silencer drugs versus oral stabilizers remains subject to ongoing market assessment.[STAT]

Sources