Pharma Stocks

A Look at Ionis Pharmaceuticals’s Valuation as Zilganersen Trial Delivers Landmark Results for Alexander Disease

Ionis Pharmaceuticals (IONS) reported positive topline results from its pivotal trial of zilganersen for Alexander disease, showing clear disease stabilization. This development has generated fresh investor optimism regarding the company’s rare disease pipeline.

See our latest analysis for Ionis Pharmaceuticals.

Ionis Pharmaceuticals’ upbeat Phase 3 results for zilganersen caught the market’s attention. Along with recent analyst upgrades and some notable insider selling, these developments have fueled renewed debate around the company’s upside. The share price has seen a gentle but steady climb this year, and while the 1-year total shareholder return is just under 1%, momentum may be building as the company’s late-stage pipeline milestones come into focus.

If new clinical advances have you rethinking your watchlist, it’s a great moment to explore more biotech and pharmaceutical innovators with our See the full list for free.

With Ionis Pharmaceuticals delivering landmark clinical results and the stock making steady gains, the key question now is whether these advances are fully reflected in the share price or if there is still a true buying opportunity for investors.

Most Popular Narrative: 1.6% Undervalued

With a fair value set at $69.48, just above the last close price of $68.40, the dominant narrative positions Ionis Pharmaceuticals as modestly undervalued. The real story, however, lies in the bold commercial moves and evolving pipeline underlying these projections.

The rapid revenue growth and positive launch trajectory for Tryngolza in familial chylomicronemia syndrome (FCS), along with the imminent launch of Donidalorsen for HAE and multiple late-stage pipeline assets reading out or launching by 2027, are set to drive sustained, stepwise increases in top-line revenue and operating leverage as Ionis transitions from R&D heavy to commercial stage. Expanding addressable patient populations, from rare diseases to larger segments such as severe hypertriglyceridemia (sHTG), combined with favorable physician feedback and significant unmet need, position Ionis to capture substantial market share and revenue growth from trends tied to the rise in chronic disease and an aging population.

Read the complete narrative.

How is this nearly on-par fair value justified? Analysts build their case on a whirlwind of revenue growth, ambitious pipeline launches, and a pricing model that could rival some tech giants. What assumptions make up this bullish thesis? Discover the surprising numbers and high-stakes projections driving this target.

Result: Fair Value of $69.48 (UNDERVALUED)

Have a read of the narrative in full and understand what’s behind the forecasts.

However, execution risk remains as Ionis expands pipeline indications, and any regulatory setbacks could quickly challenge current growth expectations.

Find out about the key risks to this Ionis Pharmaceuticals narrative.

Another View: Multiples Tell a Very Different Story

Looking at Ionis Pharmaceuticals through the lens of its price-to-sales ratio, the outlook shifts. The company trades at 11.5 times its sales, which is far above both the industry average of 9.7x and the peer average of 6.9x. Even more striking, this is well over the fair ratio of 3.7x that the market could eventually revert to. This gap highlights a potential overvaluation and suggests risk if market sentiment changes.

See what the numbers say about this price — find out in our valuation breakdown.

NasdaqGS:IONS PS Ratio as at Oct 2025

Build Your Own Ionis Pharmaceuticals Narrative

If you see the numbers differently or want to dig into the data firsthand, you can craft your own take in just a few minutes: Do it your way.

A great starting point for your Ionis Pharmaceuticals research is our analysis highlighting 2 key rewards and 2 important warning signs that could impact your investment decision.

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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.

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