Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals ended the year on a strong note, with full-year global CF product revenue rising 18% from 2021. The company delivered an earnings beat and several life-altering therapies for cystic fibrosis were developed using their unique R&D strategy, which is also intended to produce transformative medicines for critical diseases with high success rates, regardless of modality. Their mid- and late-stage clinical pipeline holds the opportunity to provide patients across 8 disease areas with potentially transformative impact. Also, preclinical research for the next phase of innovation, which includes initiatives for Duchenne’s muscular dystrophy and Type 1 myotonic dystrophy, is moving forward. Their regulatory applications for exa-cel for beta-thalassemia and sickle cell disease in the EU and UK were completed in late Q4. On the other hand, the MAA applications have recently received validation from the EMA and the MHRA. In addition, Vertex presented a positive picture of the potential introduction of the first CRISPR-based gene editing treatment as it completes the FDA submission. The management forecast for product revenue for the entire 2023 fiscal year is $9.55 billion to $9.7 billion, which, after factoring in an estimated 1.5% FX headwind, represents 7% to 9% growth year over year. We give Vertex Pharmaceuticals Incorporated a ‘Hold’ rating with a revised target price.