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Vertex Pharmaceuticals Inc VRTX Stock Quote

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Vertex's Narrow Moat Based on Intangible Assets From CF Drugs; Diversified Pipeline Supports Growth

Rachel Elfman Equity Analyst

Business Strategy and Outlook

| Rachel Elfman |

Vertex was once known for discovering Incivek, a blockbuster hepatitis C drug now overshadowed by a robust cystic fibrosis franchise with megablockbuster potential. The company's approved cystic fibrosis drugs are Kalydeco, Orkambi, Symdeko, and Trikafta, which will make Vertex eligible to treat about 90% of the CF population, assuming international and pediatric approvals. We expect Vertex to maintain its dominant position in CF, given the strong efficacy of its therapies, lengthy patents, and lack of competition, while developing pipeline candidates in other rare indications to spur growth.

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Business Description

Vertex Pharmaceuticals is a global biotechnology company that discovers and develops small-molecule drugs for the treatment of serious diseases. Its key drugs are Kalydeco, Orkambi, Symdeko, and Trikafta/Kaftrio for cystic fibrosis, where Vertex therapies remain the standard of care globally. In addition to its focus on cystic fibrosis, Vertex is diversifying its pipeline through gene-editing therapies such as CTX001 for beta-thalassemia and sickle-cell disease, small-molecule inhibitors targeting acute and chronic pain using non-opioid treatments, and small-molecule inhibitors of APOL1-mediated kidney diseases. Vertex is also investigating cell therapies to deliver a potential functional cure for type 1 diabetes.

Contact
50 Northern Avenue
Boston, MA, 02210
T +1 617 341-6100
Industry Biotechnology
Most Recent Earnings Jun 30, 2022
Fiscal Year End Dec 31, 2022
Employees 3,900

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