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Does Eplontersen’s Phase 3 Miss and Dravet Progress Change The Bull Case For Ionis (IONS)?

Simply Wall St·07/22/2026 01:29:40
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  • Ionis Pharmaceuticals and AstraZeneca recently reported that the large Phase 3 CARDIO-TTRansform trial of eplontersen in ATTR cardiomyopathy did not meet its primary cardiovascular endpoint, while Ionis separately dosed the first child in its Phase 1-2 ASCEND trial of ION337 for Dravet syndrome, a rare and severe epileptic encephalopathy.
  • This mix of a high-profile late-stage setback and early-stage progress in an underserved rare disease highlights both the uncertainty and breadth of Ionis’ RNA-targeted pipeline.
  • We’ll now examine how eplontersen’s Phase 3 outcome and ION337’s Dravet syndrome trial initiation could reshape Ionis Pharmaceuticals’ investment narrative.

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Ionis Pharmaceuticals Investment Narrative Recap

To own Ionis today, you need to believe its antisense platform can translate into multiple commercial drugs despite setbacks in individual programs. The eplontersen Phase 3 miss in ATTR cardiomyopathy is a real blow for one potential growth driver, but Ionis’ near term story still leans most heavily on execution around Olezarsen and Donidalorsen, with the biggest risk being that key upcoming approvals or pricing outcomes for these drugs fall short of expectations.

The ASCEND trial start for ION337 in Dravet syndrome is the most relevant offsetting announcement, showing Ionis continuing to push antisense into severe neurological diseases with limited options. While this early study does not replace lost eplontersen cardiomyopathy potential, it adds to a broader rare disease portfolio that could matter for long term optionality if later stage data are supportive and complements nearer term catalysts such as Olezarsen’s broader sHTG indication.

But while the platform breadth looks encouraging, investors should also be aware that...

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

Ionis Pharmaceuticals' narrative projects $2.4 billion revenue and $329.8 million earnings by 2029. This requires 31.0% yearly revenue growth and an earnings increase of about $656.8 million from -$327.0 million today.

Uncover how Ionis Pharmaceuticals' forecasts yield a $104.86 fair value, a 96% upside to its current price.

Exploring Other Perspectives

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

Before this news, the most optimistic analysts were banking on revenue reaching about US$2.8 billion and earnings of roughly US$456 million by 2029, assuming key late stage programs avoided major clinical or regulatory setbacks. You should recognize that this is a much more optimistic view than consensus, and events like the eplontersen result or future outcomes for ultra rare programs such as Alexander disease could cause both bullish and baseline narratives to shift meaningfully.

Explore 3 other fair value estimates on Ionis Pharmaceuticals - why the stock might be worth just $79.71!

The Verdict Is Yours

Don't just follow the ticker - dig into the data and build a conviction that's truly your own.

  • 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.

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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.