Vertex Pharmaceuticals (VRTX) Goldman Sachs 45th Annual Global Healthcare Conference summary

Business and strategy overview

• Strong growth in cystic fibrosis (CF) with expanding geographic reach and younger patient penetration; continued innovation in CF pipeline, including mRNA collaboration with Moderna, with updates expected late 2024 to early 2025.

• 2024 marks commercial diversification with the launch of CASGEVY for sickle cell disease and beta thalassemia, showing strong early uptake and global approvals.

• Acquisition of Alpine Immune Sciences adds a Phase III-ready asset (povetacicept) for IgA nephropathy and other renal indications, aligning with strategic focus on serious diseases with unmet need.

• Pipeline updates anticipated for type 1 diabetes, mRNA, and other programs by late 2024 or early 2025.

Acute pain program and commercial strategy

• VX-548 for acute pain is in rolling NDA submission, targeting a multi-billion dollar market with a specialty-focused commercial model.

• U.S. acute pain market includes over 80 million patients annually, with institutional settings (hospitals, surgical centers) as key prescribing hubs.

• Early commercial efforts will focus on key opinion leaders and high-volume institutions, using a targeted specialty sales force and digital enablement.

• Full commercial staffing for pain expected in the current quarter; payer and formulary discussions are underway, with strong interest in opioid alternatives.

• Legislative tailwinds include the NOPAIN Act (effective January 2025) and proposed Alternatives to PAIN Act, supporting opioid alternatives and reducing access barriers.

Scientific and clinical pipeline updates

• Nav1.8 inhibitor suzetrigine shows broad efficacy in acute and neuropathic pain; additional Nav1.8 and Nav1.7 inhibitors in development, with potential for combination therapy.

• No need anticipated for triple therapy; focus remains on advancing suzetrigine, 9 series, and Nav1.7 programs.

• Vanzacaftor triple for CF demonstrates superior efficacy over TRIKAFTA, with higher rates of patients reaching carrier-level CFTR function, especially in children.

• Strong physician and patient interest in vanzacaftor; expected significant switching from existing therapies and re-engagement of previously untreated patients.