AstraZeneca, Ionis Unveil Complete Phase 3 Data For ATTR Candidate, Says Drug Halts Disease Progression

Ionis Pharmaceuticals Inc IONS and AstraZeneca Plc AZN announced that the Phase 3 NEURO-TTRansform study for eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group.
The companies released topline data in the last week of March.
Ionis and AstraZeneca seek regulatory approval for eplontersen for ATTRv-PN in the US, with a PDUFA action date of December 22.
Eplontersen halted patients' disease progression after 66 weeks in ATTRv-PN, a fatal neurodegenerative disease.
In a composite score of polyneuropathy clinical measurements like muscle strength and blood pressure, patients taking eplontersen recorded a 0.28-point average increase from baseline compared to a 25.06-point average increase in the placebo group.
The difference of 24.8 points between the groups was good for a p-value of p<0.0001.
Two other co-primary endpoints showed eplontersen resulted in an 82% reduction of serum TTR concentration in active arm patients compared to 11% in the placebo group and a 19.7-point average increase in quality of life questionnaire.
Eplontersen is currently being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and fatal condition that typically leads to progressive heart failure and often death within three to five years from disease onset.
Price Action: IONS shares are down 3.55% at $35.62, and AZN shares are up 0.13% at $75.70 on the last check Monday.

Market News and Data brought to you by Benzinga APIs

-

-

-

-

-

-

-

-

-

-

-

-

-

-

Comments