VERTEX PHARMACEUTICALS INC / MA Q3 FY2024 earnings call

CF Commercial Execution: Continued strong commercial execution in CF, with $2.77 billion revenue in Q3 and poised for potential approval of the vanzacaftor triple in CF globally. ### CASGEVY Launch: Enthusiasm from patients, physicians, and policymakers for CASGEVY, with 45 authorized treatment centers (up from over 35 in Q2) and around 40 patients having cell collections. First commercial patients received infusions. ### Pipeline Progress: Rapidly progressed programs in clinical pipeline with three new programs advancing into Phase 3 clinical trials in the last quarter. Povetacicept's global Phase 3 RAINIER study in IgA nephropathy initiated. Suztrigine's FDA review for moderate to severe acute pain in the U.S. underway. Myotonic dystrophy type 1 program's MAD portion initiated. ### Other Highlights: CASGEVY approvals in Switzerland and Canada, and progress in VX-880, inaxaplin, and other programs.

In the third quarter, Vertex's CF segment delivered $2.77 billion in revenue, representing a 12% growth compared to Q3 2023. Year-to-date, based on strong growth and Q4 outlook, the full year product revenue guidance is increased to $10.8 billion to $10.9 billion. CASGEVY's early launch is progressing well with 45 authorized treatment centers and around 40 patients having cell collections, and the first commercial patients have received infusions of CASGEVY.

• Raising 2024 total product revenue guidance to $10.8 billion to $10.9 billion. - Operating expense guidance remains unchanged. - Non-GAAP tax rate for full year 2024 expected to be approximately 90% due to the non-deductible Alpine AIPR&D charge.

Q: Jessica Fye asked about how to get povetacicept approved for at-home delivery.

A: Reshma Kewalramani said plans are in place for at-home monthly subcutaneous administration and all regulatory interactions are planning for that.

Q: Salveen Richter asked about suzetrigine's Phase 2 LSR data and patient population confidence.

A: Reshma Kewalramani discussed mechanism of action, clinical evidence, and careful patient selection for the LSR study.

Q: Chris Garcia asked about suzetrigine's pricing inputs.

A: Stuart Arbuckle said pricing considers clinical benefit and unmet need.

Q: Mohit Bansal asked about suzetrigine vs Lyrica and competitor's NaV1.8 molecule.

A: Reshma Kewalramani discussed mechanism differences and competitor's molecule discontinuation.

Q: Tazeen Ahmad asked about LSR read-through to DPN and competitor's molecule.

A: Reshma Kewalramani discussed 993's reasons and inaxaplin's regulatory progress.

Q: Michael Yee asked about 993's benefits and inaxaplin's filing.

A: Reshma Kewalramani discussed 993's innovation and inaxaplin's regulatory plans.

Q: Evan Seigerman asked about CASGEVY commercial learnings.

A: Stuart Arbuckle discussed learnings on patient, physician, payer discussions.

Q: Phil Nadeau asked about suzetrigine's contracting and strategic initiatives.

A: Stuart Arbuckle discussed positive payer conversations and initiatives like co-pay assistance and retail distribution.

Q: Debjit Chattopadhyay asked about inaxaplin's enrollment and commercial potential.

A: Reshma Kewalramani and Stuart Arbuckle discussed enrollment and commercial opportunity.

Q: Olivia Brayer asked about LSR's internal bar for registrational program.

A: Reshma Kewalramani discussed looking at NPRS score magnitude and subgroup analysis.

Q: Liisa Bayko asked about suzetrigine's prior authorization confidence.

A: Stuart Arbuckle discussed policy and commercial efforts to influence prior authorization