Ionis Pharmaceuticals: Lp(a)ye or Lp(nay)? See Focus Returning to sHTG Post Pela Update

研报英文原文证据摘录

J P M O R G A N

North America Equity Research

10 August 2026

Ionis Pharmaceuticals

Lp(aye) or Lp(nay)? See Focus Returning to sHTG Post

Pela Update

Based on our investor inbounds heading into pelacarsen’s phase III

Lp(a)HORIZON topline, we see this CVOT readout as a key hurdle to clear before

focus can return to Tryngolza’s launch in sHTG, which we see reading into the

outlook for IONS stock more broadly. Along these lines, and in conjunction with

our EU Biopharma colleague Richard Vosser’s deep dive on the trial, we wanted

to share our perspectives on what various outcomes could mean for IONS,

acknowledging that, if successful, we would not expect to have full insight into the

effect size with the topline release. Based on our buyside conversations and two

prior interims passing without the trial being stopped for efficacy, we think the

market views this trial as risky with a mix of investors thinking that the trial may

1) miss stat sig outright, or 2) deliver efficacy that is not sufficiently compelling

if it does hit, particularly in the context of multiple competitors coming up behind

pelacarsen, with 3) some of the view that the higher Lp(a) subset (≥90mg/dL)

represents the trial’s best shot.

As it relates to the stock, while our $76 IONS price target includes ~$6 of value for

pelacarsen, we think the market is pricing in less than this based on our buyside

conversations, perhaps LSD dollars/share, maybe up to $5 on the high end. On the

flip side, while the trial succeeding could add ~$4 to our valuation, we think the

stock would be up more than this on a positive topline given the difference we

perceive between our model and street expectations. Even acknowledging the

possibility of multiple competitors down the line, a hit in Lp(a)HORIZON would

put meaningful pelacarsen regulatory milestones in play for IONS, potentially

helping support the 2028 cash breakeven guidance. In either scenario, moving

beyond the topline would enable focus to shift more cleanly towards 1) Tryngolza’s

launch in SHTG, 2) ongoing launch momentum for Dawnzera, and 3) eventually,

the phase III Angelman readout 2H27. Net-net, with low Street expectations

…