Vertex Pharmaceuticals (VRTX) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Cystic fibrosis and hematology franchise updates

• Expanded patient reach in cystic fibrosis through new geographies and younger age groups, with vanzacaftor NDA under review and a PDUFA date in January next year.

• VX-522 mRNA program targets the last 10% of CF patients who do not produce protein, progressing through phase I/II.

• CASGEVY, a new hematology franchise for sickle cell disease and beta thalassemia, shows strong early launch momentum and real-world patient benefit.

Pipeline and R&D strategy

• Multiple late-stage programs: suzetrigine (VX-548) for acute pain (NDA submitted), diabetic peripheral neuropathy (phase III), LSR (phase II, accelerated timeline), and Povetacicept in IgA nephropathy (phase III).

• Early-stage pipeline includes NaV1.7 (late preclinical), improved conditioning for CASGEVY, and regenerative medicine for type 1 diabetes.

• Disease-first approach, not limited by platform or therapeutic area, focusing on diseases with high potential for success.

Product launch and commercialization plans

• Vanzacaftor expected to launch in 2025, targeting patients who discontinued prior therapies and aiming for broad patient transition over time.

• Once-daily dosing and lower royalty burden are key advantages for vanzacaftor.

• Suzetrigine launch preparations include a trained field force, focus on institutional prescribing, and payer engagement for rapid uptake.

• No direct analogs for suzetrigine launch due to its unique profile as an oral small molecule for moderate to severe acute pain.

• Guidance for the pain franchise will be provided later in the year.