Lp(a): 93.9 percentage-point reduction vs placebo · Time-averaged from day 60 through day 180
ALPACA · Phase 2 · Peer-reviewed trial · 400 mg at baseline; pooled 400 mg groups · Treatment-regimen estimand; placebo-adjusted time-averaged change from day 60 to 180.
Limitations: The value averages a 120-day interval; it is not a single day 180 measurement. Two 400 mg groups were pooled before their second dosing assignments.
Status in this atlas: Current approval status is not summarized in this therapy record. A trial phase or a brand name does not establish approval for this use.
Targets the messenger RNA for apolipoprotein(a), reducing its production in liver cells.
Uses, doses and availability depend on the formulation, indication and country. Study doses below describe research; they are not prescribing instructions.
The molecule
GalNAc-conjugated Dicer-substrate siRNA with a tetraloop architecture and three GalNAc conjugates.
Small interfering RNA
Lepodisiran
GalNAc-conjugated Dicer-substrate siRNA with a tetraloop architecture and three GalNAc conjugates.
The primary trial publishes a drug-specific structure schematic in Supplement 3, eFigure 4. No validated whole-drug 3D coordinates are included; this description must not be replaced by a generic DNA helix.
A next milestone is not summarized for this therapy. This does not mean development has ended.
Development history3 selected milestones
Lepodisiran · compound history across indications
Selected sourced milestones, not a complete development history. Dates distinguish study starts, publications, announcements and approvals; the first listed date is not necessarily when development began.
Publication
Phase 1 results published
A randomized dose-ascending study of 48 adults reported long-duration Lp(a) lowering after a single injection. This early human study established pharmacodynamic proof of concept, not prevention of cardiovascular events.
The registry records the actual start of phase 3 cardiovascular outcomes testing. Eligibility includes patients with established ASCVD and a separate group at risk for a first cardiovascular event.
The plotted ALPACA trial reported sustained Lp(a) lowering. The pooled 400-mg groups had a placebo-adjusted, time-averaged reduction of 93.9 percentage points from day 60 through day 180; this is not a single-visit estimate.