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Gene Editing And New Therapies Will Transform Global Healthcare

Published
18 Jul 24
Updated
04 Sep 25
AnalystConsensusTarget's Fair Value
US$479.83
20.4% undervalued intrinsic discount
10 Sep
US$381.87
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1Y
-17.5%
7D
-1.4%

Author's Valuation

US$479.83

20.4% undervalued intrinsic discount

AnalystConsensusTarget Fair Value

Last Update04 Sep 25
Fair value Decreased 0.25%

Analysts maintain a cautious-but-constructive stance on Vertex Pharmaceuticals, citing strong CF performance and resilient topline results offset by pipeline setbacks and future revenue concentration risks, with the consensus price target unchanged at $481.04.


Analyst Commentary


  • Bullish analysts highlight Vertex’s continued strong execution track record, robust base cystic fibrosis (CF) business, solid Q2 results with topline beats, and a positive long-term growth outlook beyond near-term setbacks.
  • Bearish analysts cite pipeline setbacks, notably the non-opioid pain candidate VX-993 failing to show statistical significance in Phase 2 and the discontinued advancement of Journavx, raising concerns about future revenue diversification.
  • The cystic fibrosis franchise's dominance is viewed as both a strength and a risk, as any credible threat to Vertex’s technological monopoly—such as from competitors like Sionna—could disproportionately impact the stock.
  • Some analysts see the recent share price pullback as an attractive entry point, emphasizing that pipeline progress (outside of pain) remains on track and the near-term catalyst path appears “clean.”
  • Moderation in outlooks includes cautiousness around macroeconomic uncertainties, slightly lowered guidance, and expense management initiatives such as reduced headcount growth to address cost pressures for fiscal 2026 and beyond.

What's in the News


  • Vertex completed a share buyback program totaling 5.76 million shares (2.24% of shares outstanding) for $2.43 billion.
  • Vertex reiterated 2025 full-year earnings guidance with expected revenue of $11.85–12 billion.
  • The Phase 2 trial for VX-993 in acute pain showed no significant efficacy versus placebo; development as monotherapy will not proceed.
  • ALYFTREK, a once-daily CFTR modulator for cystic fibrosis, received Health Canada approval, broad reimbursement in NHS England, and European Commission approval; positive longer-term and pooled clinical data were also presented.
  • Vertex announced durable clinical benefits for CASGEVY (CRISPR/Cas9 gene therapy) in sickle cell disease and beta thalassemia, and entered an exclusive collaboration with Ono Pharmaceutical for povetacicept in Asia; the company was dropped from several Russell Value indices but added to the Russell 1000 Dynamic Index.

Valuation Changes


Summary of Valuation Changes for Vertex Pharmaceuticals

  • The Consensus Analyst Price Target remained effectively unchanged, at $481.04.
  • The Consensus Revenue Growth forecasts for Vertex Pharmaceuticals remained effectively unchanged, at 9.7% per annum.
  • The Future P/E for Vertex Pharmaceuticals remained effectively unchanged, at 26.54x.

Key Takeaways

  • Expanding access to innovative therapies and a diversified pipeline in genomics position the company for sustained revenue and margin growth in precision medicine markets.
  • Robust global launches, improving reimbursement, and disciplined reinvestment strengthen future earnings and broaden opportunities amid rising healthcare spending and AI-driven drug development trends.
  • Dependence on cystic fibrosis drugs, regulatory and pricing pressures, and uncertain pipeline outcomes threaten Vertex's profitability, margins, and long-term revenue growth.

Catalysts

About Vertex Pharmaceuticals
    A biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF).
What are the underlying business or industry changes driving this perspective?
  • Rapid progress in launching and expanding access for new therapies such as ALYFTREK (CF), CASGEVY (sickle cell disease/beta thalassemia), and JOURNAVX (acute pain) across additional geographies and patient segments, fueled by rising prevalence of chronic diseases and aging population, is likely to drive significant top-line revenue growth over the next several years.
  • Vertex's pipeline diversification, including programs in pain, kidney, and type 1 diabetes, leverages global advances in genomic and gene-editing technologies, positioning the company to capture long-term growth from accelerating personalized and precision medicine adoption, supporting both revenue and long-term margin expansion.
  • Growing global healthcare spending and improving reimbursement/access in emerging markets (with international launches and fast-track approvals for new products) should increase the number of patients reached and extend the revenue base for existing and next-generation therapies, driving sustainable growth.
  • Commercial success and broad payer coverage for recent launches, particularly JOURNAVX and CASGEVY, are setting the stage for larger market uptake and eventual margin improvement as early launch support programs unwind and operational leverage is realized, positively impacting net margins and earnings.
  • Strong cash flows and a robust balance sheet, bolstered by meaningful share repurchases and prudent reinvestment in R&D and commercial infrastructure, support future earnings power and the ability to capitalize on long-term industry trends such as faster drug development via AI and high-throughput screening, replenishing the pipeline and widening growth opportunities.

Vertex Pharmaceuticals Earnings and Revenue Growth

Vertex Pharmaceuticals Future Earnings and Revenue Growth

Assumptions

How have these above catalysts been quantified?
  • Analysts are assuming Vertex Pharmaceuticals's revenue will grow by 9.4% annually over the next 3 years.
  • Analysts assume that profit margins will increase from 31.9% today to 37.4% in 3 years time.
  • Analysts expect earnings to reach $5.6 billion (and earnings per share of $21.77) by about September 2028, up from $3.6 billion today. However, there is a considerable amount of disagreement amongst the analysts with the most bullish expecting $7.2 billion in earnings, and the most bearish expecting $4.5 billion.
  • In order for the above numbers to justify the analysts price target, the company would need to trade at a PE ratio of 26.5x on those 2028 earnings, down from 27.9x today. This future PE is greater than the current PE for the US Biotechs industry at 15.3x.
  • Analysts expect the number of shares outstanding to decline by 0.44% per year for the next 3 years.
  • To value all of this in today's terms, we will use a discount rate of 6.78%, as per the Simply Wall St company report.

Vertex Pharmaceuticals Future Earnings Per Share Growth

Vertex Pharmaceuticals Future Earnings Per Share Growth

Risks

What could happen that would invalidate this narrative?
  • Heavy reliance on the cystic fibrosis (CF) franchise persists, despite diversification efforts, exposing Vertex to ongoing patent expiry and the risk of increased competition from new entrants that could erode long-term revenue streams and profitability.
  • High R&D expenditures and the uncertain clinical and regulatory outcomes in pain, kidney, and gene-editing programs (e.g., setbacks in PNP indications and reliance on next-generation pipelines) may lead to prolonged periods of elevated costs without significant revenue offsets, putting downward pressure on net margins and earnings.
  • Growing pricing pressure from global payers (e.g., reimbursement negotiations, expansion of government payer models, and healthcare cost containment) could restrict the future pricing power of Vertex's new therapies, limiting sustainable revenue growth and compressing margins.
  • Increased competition is likely in crowded markets targeted by Vertex's pipeline (such as generalized myasthenia gravis), with the potential for next-generation genetic medicines or alternative rare disease treatments to limit Vertex's market share, top-line growth, and operating margins.
  • Regulatory uncertainty-including stricter requirements for demonstrating clinical and real-world effectiveness, as well as delays in broadening indications and obtaining approvals outside core areas-could delay product launches, increase post-approval costs, and create variability in revenue and earnings visibility.

Valuation

How have all the factors above been brought together to estimate a fair value?
  • The analysts have a consensus price target of $479.833 for Vertex Pharmaceuticals based on their expectations of its future earnings growth, profit margins and other risk factors. However, there is a degree of disagreement amongst analysts, with the most bullish reporting a price target of $624.0, and the most bearish reporting a price target of just $330.0.
  • In order for you to agree with the analyst's consensus, you'd need to believe that by 2028, revenues will be $14.9 billion, earnings will come to $5.6 billion, and it would be trading on a PE ratio of 26.5x, assuming you use a discount rate of 6.8%.
  • Given the current share price of $396.48, the analyst price target of $479.83 is 17.4% higher.
  • We always encourage you to reach your own conclusions though. So sense check these analyst numbers against your own assumptions and expectations based on your understanding of the business and what you believe is probable.

How well do narratives help inform your perspective?

Disclaimer

AnalystConsensusTarget is a tool utilizing a Large Language Model (LLM) that ingests data on consensus price targets, forecasted revenue and earnings figures, as well as the transcripts of earnings calls to produce qualitative analysis. The narratives produced by AnalystConsensusTarget are general in nature and are based solely on analyst data and publicly-available material published by the respective companies. These scenarios are not indicative of the company's future performance and are exploratory in nature. Simply Wall St has no position in the company(s) mentioned. Simply Wall St may provide the securities issuer or related entities with website advertising services for a fee, on an arm's length basis. These relationships have no impact on the way we conduct our business, the content we host, or how our content is served to users. The price targets and estimates used are consensus data, and do not constitute a recommendation to buy or sell any stock, and they do not take account of your objectives, or your financial situation. Note that AnalystConsensusTarget's analysis may not factor in the latest price-sensitive company announcements or qualitative material.

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