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Price · 1Y
$516.34
Thesis
Vertex runs a near-monopoly in cystic fibrosis with the Trikafta/Kaftrio franchise generating roughly $10B+ in annual revenue, funding one of biotech's most disciplined R&D engines. That cash flow is now backing a diversification beyond CF into gene editing and non-opioid pain, which transforms the story from a single-disease specialist into a multi-franchise platform.
Casgevy, partnered with CRISPR Therapeutics, is the first FDA-approved CRISPR/Cas9 therapy and addresses sickle cell disease and transfusion-dependent beta thalassemia. Journavx (suzetrigine), approved January 2025, is a first-in-class NaV1.8 inhibitor for moderate-to-severe acute pain, opening a non-opioid category the company can own commercially.
Catalysts
- Casgevy patient activations and authorized treatment center ramp
- Journavx adoption in post-surgical settings and label expansion into chronic pain
- Next-gen CF triple combo (vanzacaftor) cannibalization economics
- Pipeline readouts in APOL1-mediated kidney disease and type 1 diabetes cell therapy
Recent events
Material filings, federal awards, and regulatory activity. Impact is scored against market cap.
- Sep 21Trial initiationClinicalTrials.gov
Vertex Phase 2/3 adaptive study of VX-147 in APOL1-mediated proteinuric kidney disease (NCT05312879) status recruiting as of 2026-09-21.
- Sep 21Trial initiationClinicalTrials.gov
Vertex initiates Phase 1 dose-escalation study of VX-581 in healthy participants (NCT07283770).
- Sep 18Trial initiationClinicalTrials.gov
Vertex initiates Phase 2/3 trial of povetacicept in primary membranous nephropathy (NCT07204275, recruiting).
- Sep 16Trial initiationClinicalTrials.gov
Phase 2a study NCT07161037 of VX-407 in ADPKD patients with subset of PKD1 variants updated to ACTIVE_NOT_RECRUITING status.
- Sep 16Trial readoutClinicalTrials.gov
CTX001 Phase 2/3 trial (NCT03745287) in severe sickle cell disease completed per ClinicalTrials.gov update.
- Sep 11Trial initiationClinicalTrials.gov
Vertex initiates Phase 2 trial (NCT07501702) of povetacicept in generalized myasthenia gravis; status recruiting.
- Sep 9Trial initiationClinicalTrials.gov
ClinicalTrials.gov update: NCT05312879 Phase 2/3 adaptive study of VX-147 in APOL1-mediated proteinuric kidney disease status RECRUITING.
- Sep 8Trial initiationClinicalTrials.gov
Vertex Phase 3 open-label study of povetacicept in IgA nephropathy (NCT07807995) status not yet recruiting.