Early Access

10-KPeriod: FY2021

VERTEX PHARMACEUTICALS INC / MA Annual Report, Year Ended Dec 31, 2021

Filed February 9, 2022For Securities:VRTX

Summary

Vertex Pharmaceuticals Inc. (VRTX) reported strong revenue growth in 2021, driven by the continued success of its cystic fibrosis (CF) treatments, particularly TRIKAFTA/KAFTRIO. The company achieved a 22% increase in net product revenues, reaching $7.57 billion, fueled by international expansion of KAFTRIO and the expanded indication for TRIKAFTA in younger children in the U.S. Vertex is actively expanding its CF portfolio with a new triple combination therapy in Phase 3 trials and pursuing gene-editing approaches for the remaining CF patient population. Beyond CF, Vertex is making significant progress across a diversified pipeline targeting serious diseases such as sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, type 1 diabetes, and pain. Key developments include advancing CTX001 (CRISPR gene-editing therapy) towards regulatory submissions for sickle cell disease and beta thalassemia, and positive early data for VX-880, a cell therapy for type 1 diabetes. The company's strong financial position, with $7.5 billion in cash, cash equivalents, and marketable securities, supports its ongoing investment in research and development and strategic collaborations.

Financial Statements
Beta

Key Highlights

  • 1Net product revenues increased by 22% to $7.57 billion in 2021, primarily driven by the strong performance and international expansion of TRIKAFTA/KAFTRIO for cystic fibrosis (CF).
  • 2The company advanced its CF pipeline, initiating Phase 3 trials for a new triple combination therapy (VX-121/tezacaftor/VX-561) and is progressing mRNA therapeutics for CF patients not served by small molecules.
  • 3Vertex made significant strides in its non-CF pipeline, with CTX001 (CRISPR gene-editing therapy for sickle cell disease and beta thalassemia) anticipated for regulatory submissions in late 2022.
  • 4Positive Phase 1/2 data was announced for VX-880, a stem cell-derived therapy for type 1 diabetes, with continued patient dosing planned for 2022.
  • 5VX-147 showed positive Phase 2 results for APOL1-mediated kidney disease, with pivotal development expected to begin in Q1 2022.
  • 6Research and development expenses increased significantly to $3.05 billion in 2021, largely due to a $900 million upfront payment to CRISPR Therapeutics for the CTX001 collaboration.
  • 7Vertex ended 2021 with a robust cash position of $7.5 billion, providing ample resources for ongoing research, development, and business development activities.

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