AstraZeneca and partner Ionis Pharmaceuticals announced Phase III CARDIO-TTRansform trial evaluating Wainua eplontersen adults transthyretin-mediated amyloid cardiomyopathy ATTR-CM did not meet primary efficacy endpoint significant setback investigational indication.
CARDIO-TTRansform – Key Update
- Drug: Wainua eplontersen | Indication Tested: ATTR-CM transthyretin-mediated amyloid cardiomyopathy | Trial: Phase III CARDIO-TTRansform global study 1400+ patients one of largest ATTR-CM trials
- Primary Endpoint: Reduce composite cardiovascular death and recurrent cardiovascular clinical events up to 140 weeks vs placebo – Failed to achieve statistically significant improvement
- Safety: Profile consistent previous studies generally well tolerated
- Next Steps: Continue analyzing full dataset secondary endpoints predefined subgroup analyses determine potential next steps regulatory authorities full findings to be presented future medical congress
- Existing Approval Unaffected: Wainua already approved several countries hereditary transthyretin-mediated amyloid polyneuropathy ATTRv-PN – Unsuccessful ATTR-CM trial limits expansion into larger cardiomyopathy population
Tags: AstraZeneca Ionis Wainua eplontersen Phase III CARDIO-TTRansform ATTR-CM transthyretin-mediated amyloid cardiomyopathy failed primary endpoint cardiovascular death, 1400 patients 140 weeks placebo safety tolerated ATTRv-PN approved indication unaffected regulatory next steps
