• EN

    • -+ 0.00%
    -+ 0.00%
    -+ 0.00%

    Does Pelacarsen’s Phase 3 Miss And ZANVASTRO’s FDA Win Change The Bull Case For Ionis (IONS)?

    Simply Wall St·09/05/2026 14:19:12
    Listen to the news
    • In early September 2026, Ionis Pharmaceuticals announced that partner Novartis’ Phase 3 Lp(a)HORIZON cardiovascular outcomes trial of pelacarsen failed to meet its primary endpoint, even though the drug achieved substantially lower lipoprotein(a) levels in more than 8,000 patients with established cardiovascular disease.
    • Just days earlier, the FDA approved Ionis’ ZANVASTRO as the first disease-modifying therapy for ultra-rare Alexander disease, underscoring the company’s ability to convert its RNA-targeted pipeline into commercial products despite setbacks in partnered cardiovascular programs.
    • We’ll now examine how the FDA approval of ZANVASTRO, alongside pelacarsen’s outcomes miss, reshapes Ionis’ investment narrative and risk profile.

    Rare earth metals are an input to most high-tech devices, military and defence systems and electric vehicles. The global race is on to secure supply of these critical minerals. Beat the pack to uncover the 30 best rare earth metal stocks of the very few that mine this essential strategic resource.

    Ionis Pharmaceuticals Investment Narrative Recap

    To stay invested in Ionis today, you have to believe its antisense platform and growing rare disease franchise can offset volatility from partnered cardiovascular programs and an unprofitable base business. In that context, Novartis’ pelacarsen miss removes a potential long term cardiovascular royalty stream, but does not directly change the near term focus on Olezarsen and Donidalorsen approvals and launches, which still look like the key near term catalysts and the biggest execution risks.

    The most relevant new development is the FDA approval of ZANVASTRO, which adds a third wholly owned commercial product and validates Ionis’ ability to bring ultra rare neurology programs to market. While Alexander disease is an extremely small indication, ZANVASTRO slightly diversifies revenue, supports the case for Ionis’ RNA-targeted platform, and partially offsets the disappointment around pelacarsen as investors watch how quickly Tryngolza and DAWNZERA can scale.

    Yet behind the FDA win, investors should be aware of the risk that pricing pressure across broader indications could...

    Read the full narrative on Ionis Pharmaceuticals (it's free!)

    Ionis Pharmaceuticals’ narrative projects $2.3 billion revenue and $222.2 million earnings by 2029.

    Uncover how Ionis Pharmaceuticals' forecasts yield a $88.00 fair value, a 51% upside to its current price.

    Exploring Other Perspectives

    IONS 1-Year Stock Price Chart
    IONS 1-Year Stock Price Chart

    Some of the most optimistic analysts were assuming Ionis could lift revenue nearly 50% a year to about US$2.9 billion by 2029, but pelacarsen’s failure and the risk that ultra rare launches like ZANVASTRO fall short of those expectations show how widely views can differ and why it is worth comparing these bullish scenarios with more cautious ones.

    Explore 4 other fair value estimates on Ionis Pharmaceuticals - why the stock might be worth just $60.82!

    The Verdict Is Yours

    Disagree with existing narratives? Extraordinary investment returns rarely come from following the herd, so go with your instincts.

    • A great starting point for your Ionis Pharmaceuticals research is our analysis highlighting 3 key rewards that could impact your investment decision.
    • Our free Ionis Pharmaceuticals research report provides a comprehensive fundamental analysis summarized in a single visual - the Snowflake - making it easy to evaluate Ionis Pharmaceuticals' overall financial health at a glance.

    Contemplating Other Strategies?

    Markets shift fast. These stocks won't stay hidden for long. Get the list while it matters:

    This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned.