Vertex Pharmaceuticals Incorporated

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Vertex Pharmaceuticals Incorporated stock analysis VRTX overview
Bullish +75

2 Under-the-Radar Biotech Stocks Worth Buying for the Long Term

📈 Vertex Pharmaceuticals reported record annual revenue of $12 billion in 2025, marking a 9% increase led by cystic fibrosis therapies.

💰 Vertex generated annual EPS of $15.32 in 2025, recovering from a $2.08 loss in 2024 to show strong financial improvement.

🔬 Vertex received FDA approval on April 1 to expand its label extensions for CF therapies Trikafta and Alyftrek to cover 95% of U.S. patients.

⚠️ Vertex shares declined despite strong earnings growth due to underwhelming revenue from non-CF therapies, particularly Journavx and Casgevy.

🚀 Mirum Pharmaceuticals showed explosive revenue growth in 2025 with total sales of $521.3 million, up 54% from the prior year.

💊 Mirum's Livmarli drug generated $360 million in 2025 revenue, representing a 69% increase and driving significant market uptake.

🔮 Mirum forecasts product sales of $630 million to $650 million for 2026, projecting growth of 22.7% at the midpoint.

⚖️ Mirum improved its profitability trajectory with an annual EPS loss of $0.45 in 2025, down significantly from a $1.85 loss in 2024.

📅 Mirum has four potentially registrational readouts expected over the next 18 months focused on new drug indications.

💉 The company recently purchased Bluejay Therapeutics for $820 million to add brelovitug, a Breakthrough Therapy status treatment for HDV.

🩺 HDV is an aggressive disease with no currently approved U.S. therapies, potentially generating more than $750 million in annual revenue if addressed.

🤝 Mirum leverages its existing sales team that visits hepatologists treating rare liver diseases to market the new HDV treatment efficiently.

💴 Mirum maintains a strong balance sheet with $391 million in cash and investments, reducing pressure to sell stock for funding.

🌟 Vertex is expanding beyond cystic fibrosis with CRISPR-gene editing therapy Casgevy for sickle cell anemia and beta thalassemia.

🛡️ Vertex's new pipeline includes povetacicept for IgA nephropathy, which showed positive Phase 3 interim data in March.

Bullish Signals
  • Vertex Pharmaceuticals reported record annual revenue of $12 billion in 2025, representing a 9% increase led primarily by its cystic fibrosis therapies.
  • The company expects sales of $12.95 billion to $13.1 billion in 2026, with non-CF therapies accounting for $500 million or more.
  • Vertex improved its annual earnings per share significantly, moving from a loss of $2.08 in 2024 to record earnings of $15.32 in 2025.
  • Vertex received FDA approval on April 1 to expand label extensions for CF therapies Trikafta and Alyftrek to cover 95% of all U.S. patients with cystic fibrosis, solidifying its market dominance.
  • Mirum Pharmaceuticals achieved explosive revenue growth with $521.3 million reported in 2025, up 54%, where key drug Livmarli drove $360 million or a 69% increase over 2024.
  • Mirum forecasts product sales of $630 million to $650 million in 2026, representing a 22.7% growth at the midpoint.
  • Mirum is edging toward profitability with an annual EPS loss of only $0.45 compared to a loss of $1.85 in 2024.
  • The company has four potentially registrational readouts expected over the next 18 months, including studies for brelovitug to treat chronic hepatitis delta virus with no current approved therapies.
  • Mirum completed its $820 million purchase of Bluejay Therapeutics to add brelovitug to its pipeline, which holds Breakthrough Therapy status from the FDA and PRIME designation from the European Medicients Agency.
  • Vertex's gene editing therapy Casgevy continues to grow, used to treat severe sickle cell anemia and beta thalassemia with a potential blockbuster drug povetacicept showing positive Phase 3 interim data in March for IgA nephropathy.
  • Mirum maintains strong liquidity with $391 million in cash and investments after its Bluebird purchase, allowing it to fund research and development expansion without pressure to sell stock.
Risk Factors
  • Vertex Pharmaceuticals' shares have declined more than 2% this year, indicating recent market weakness despite steady financial growth.
  • Mirum Pharmaceuticals reported an annual EPS loss of $0.45 in the most recent period, compared to a larger loss of $1.85 in 2024, signaling it has not yet reached profitability.
  • The article notes that Vertex's stock has declined because the growth of its non-CF therapies revenue has been underwhelming, suggesting potential limitations outside its core cystic fibrosis franchise.
  • Mirum is forecasting product sales of $630 million to $650 million for 2026, which represents a slower 22.7% midpoint increase compared to the 54% revenue growth seen in 2025.
  • Vertex reported record revenue of $12 billion in 2025, but there is an explicit acknowledgment that growth outside its dominant CF therapies has been lacking.
  • The company relies heavily on a near monopoly on cystic fibrosis therapies, which could make it vulnerable to future regulatory challenges or patent expirations limiting its long-term upside.
  • Mirum still requires two years to reach profitability despite positive revenue trends, introducing execution risk and pressure to generate returns from its recent acquisitions.
Full Analysis
Vertex Pharmaceuticals (NASDAQ: VRTX) and Mirum Pharmaceuticals (NASDAQ: MIRM) are both biotech companies focusing on rare diseases with significant unmet medical needs, though they have adopted different strategic approaches to expand their platforms. Vertex has established a strong financial foundation based on its dominance in cystic fibrosis (CF) therapies, which affects approximately 112,000 people worldwide. The company recently received FDA approval on April 1 to expand label extensions for its CF therapies Trikafta and Alyftrek, making them available to 95% of U.S. patients with CF regardless of their specific CFTR protein variant, effectively solidifying its near-monopoly in this sector. Financially, Vertex reported record annual revenue of $12 billion in 2025 and expects sales to reach between $12.95 billion and $13.1 billion in 2026, with an annual earnings per share (EPS) of $15.32 for the year, up from a loss of $2.08 in 2024. Despite its robust CF portfolio, Vertex is also looking to grow through non-CF therapies, including CRISPR-gene editing therapy Casgevy and potential blockbuster povetacicept for IgA nephropathy, which showed positive Phase 3 interim data in March with a Biologics License Application filing expected in the first half of the year. Mirum Pharmaceuticals is demonstrating explosive revenue growth driven by its commercialization of Livmarli for rare liver diseases such as Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC). In 2025, Mirum reported total revenue of $521.3 million, a 54% increase from the prior year, with Livmarli accounting for $360 million. The company is forecast to generate between $630 million and $650 million in product sales for 2026, representing a midpoint increase of 22.7%. Mirum has already demonstrated a transition toward profitability, posting an EPS loss of $0.45 compared with a $1.85 loss in 2024, and is expected to become consistently profitable within the next year or two following its strategic acquisition of Bluebird Bio. A key driver for this growth is the purchase of Bluejay Therapeutics in January for $820 million, which added brelovitug to Mirum's pipeline. This drug holds Breakthrough Therapy status from the FDA and PRIME designation from the European Medicines Agency and targets chronic hepatitis delta virus (HDV), a condition affecting an estimated 15 to 20 million people worldwide for which no approved therapies currently exist in the United States. Both companies are entering high-reward periods supported by upcoming clinical data readouts that could expand their addressable markets significantly. Mirum anticipates registrational results over the next 18 months, including key data from the volixibat Vistas study expected in the second quarter and the Azure-1 and Azure-4 studies for brelovitug expected in the second half of the year to treat chronic hepatitis delta virus. Positive outcomes from these studies could allow Mirum to generate more than $750 million in annual revenue by addressing HDV, leveraging its existing sales infrastructure that already reaches hepatologists treating Alagille syndrome and PFIC. Vertex continues to strengthen its moat with Journavx, a non-opioid pain reliever showing promise alongside its core CF therapies. While Vertex shares have declined over 2% year-to-date due to slower-than-expected growth in non-CF revenue, it maintains strong cash flow, whereas Mirum's shares are up more than 20%, with sufficient cash and investments of $391 million post-acquisition to fund research and development without diluting shareholders.