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Introduction & Market Context
Vertex Pharmaceuticals (NASDAQ:VRTX) reported its first quarter 2025 financial results on May 5, showing continued revenue growth as the company expands beyond its core cystic fibrosis business. The biotech firm posted $2.77 billion in revenue for the quarter, up from $2.69 billion in the same period last year, while raising the lower end of its full-year guidance.
The company’s stock closed at $501.15 on the day of the announcement but fell 3.2% to $484.20 in after-hours trading, suggesting investors may have expected stronger results despite the company’s progress with new product launches and pipeline advancements.
Quarterly Performance Highlights
Vertex reported total revenue of $2.77 billion for Q1 2025, representing modest year-over-year growth compared to Q1 2024. The company’s flagship cystic fibrosis treatment TRIKAFTA/KAFTRIO generated $2.54 billion, while newly launched ALYFTREK contributed $54 million in its initial quarter on the market. Other products accounted for $171 million in revenue.
As shown in the following financial highlights:
The company maintained strong profitability with non-GAAP operating income of $1.18 billion, translating to a 43% operating margin. Non-GAAP net income reached $1.05 billion, with diluted earnings per share of $4.06. Vertex ended the quarter with a robust cash position of $11.4 billion, providing significant resources for continued R&D investment and potential business development.
Product Launch Updates
Vertex highlighted significant progress with three key product launches that are diversifying its revenue streams beyond its established cystic fibrosis franchise.
ALYFTREK, the company’s newest cystic fibrosis treatment, received approval for patients aged 6 and older in both the U.S. and U.K., with additional approvals expected in the EU and other geographies throughout 2025. The company positioned ALYFTREK as a highly efficacious, once-daily CFTR modulator delivering equivalent improvement in lung function and greater CFTR function compared to TRIKAFTA.
As illustrated in the following slide:
CASGEVY, Vertex’s gene-editing therapy for sickle cell disease and transfusion-dependent beta thalassemia, continues to gain momentum globally with approvals in multiple regions including the U.S., U.K., EU, Saudi Arabia, Bahrain, Canada, Switzerland, and UAE. The company reported that more than 65 authorized treatment centers have been activated, with approximately 90 patients having completed first cell collections and 8 CASGEVY infusions administered in Q1 2025.
The launch progress for CASGEVY is shown here:
JOURNAVX, Vertex’s non-opioid pain medication approved by the FDA on January 30, 2025, is showing strong early uptake with over 20,000 prescriptions filled through April 18. The drug is now broadly available at approximately 33,000 retail locations, and the company has secured coverage for about 94 million lives, with 42 million having unrestricted access.
Pipeline Progress
Vertex continues to advance a diverse pipeline spanning multiple therapeutic areas. The company’s comprehensive clinical portfolio demonstrates its ambition to meet its goal of five launches over five years (by 2028):
In type 1 diabetes, Zimislecel (formerly VX-880) continues to show promising results in its pivotal trial, with dosing expected to complete in Q2 2025 and global regulatory submissions planned for 2026. The company highlighted that in the EASD 2024 data from 12 patients who completed the Day 180 visit, all achieved a reduction in HbA1c to below 7%, and 9 out of 12 were no longer using exogenous insulin.
In kidney disease, Povetacicept has completed enrollment of the interim analysis cohort in the Phase 3 RAINIER trial for IgA nephropathy, with potential for U.S. accelerated approval in the first half of 2026 if the interim analysis is positive. The company also announced plans to advance VX-407 into Phase 2 for autosomal dominant polycystic kidney disease (ADPKD) in the second half of 2025.
Financial Analysis
Based on Q1 performance, Vertex raised the lower end of its full-year 2025 revenue guidance to $11.85-$12.0 billion, up from the previous range of $11.75-$12.0 billion. The company maintained its expense guidance, with combined non-GAAP R&D, Acquired IPR&D, and SG&A expenses expected to be $4.9-$5.0 billion.
The updated guidance reflects continued growth in the cystic fibrosis franchise, the launch of ALYFTREK, ongoing uptake of CASGEVY, and contributions from JOURNAVX:
Forward-Looking Statements
Looking ahead, Vertex outlined multiple catalysts expected throughout 2025, including continued commercial expansion for ALYFTREK, CASGEVY, and JOURNAVX, as well as key clinical milestones across its pipeline.
The company remains focused on its "5-in-5" goal of launching five new products over five years, with three already launched (ALYFTREK, CASGEVY, and JOURNAVX) and additional candidates progressing through late-stage development.
Vertex’s strong start to 2025 demonstrates the company’s successful execution of its strategy to diversify beyond cystic fibrosis while maintaining leadership in that core therapeutic area. With multiple revenue-generating products now on the market and a robust pipeline advancing across several disease areas, the company appears well-positioned for continued growth despite the modest stock reaction to the Q1 results.
Full presentation:
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