Regeneron Pharmaceuticals (REGN) Investor update summary

Genetics and multiomics platform

• Nearly 4M multi-omics profiles are linked to longitudinal health records, including ~3.3M generated internally and ~700,000 in-licensed; sequencing access is projected to reach 10M–50M in 5–10 years.

• Human genetics has discovered or validated ~50 targets, with six programs in the clinic; rare protective-variant-backed targets showed ~6x higher development success or likelihood of approval than targets without genetic support.

• The dataset includes 1M participants of non-European ancestry; genetic insights supported Dupixent indication expansion, while genetics, proteomics, and AI inform indication selection, trial design, risk prediction, and potential healthcare tools.

• A proteomic MASH risk score tracked biopsy-based classification and outcomes, identified 90% of ≥F2 fibrosis cases at an 11% false-positive rate, and outperformed other non-invasive tests on sensitivity, specificity, and area under the curve.

MASH pipeline and clinical data

• CIDEB loss-of-function variants, carried by ~1 in 500 people, are associated with 43% lower liver-disease risk and 22% lower liver fat; the siRNA aims to mimic this protective biology.

• A single CIDEB siRNA dose reduced liver fat 58%–80% at week 36; 62% normalized liver fat to <5% and 93% achieved ≥30% reductions. Mean liver fat approached or fell below the upper limit of normal at week 12; ALT fell 32% versus a 5% placebo increase.

• CIDEB siRNA was generally well tolerated through week 36, with adverse-event rates similar to placebo and no nausea or vomiting; no triglyceride or LDL-C elevations were reported. One severe serious hyperglycemia event was not attributed to treatment.

• A 360-patient Phase 2b registration-intent study in F2/F3 MASH has started, with liver-fat change at week 52 as the primary endpoint; final data are expected in 2029. A Phase III non-invasive safety study in MASLD is also underway.

• PNPLA3 siRNA reduced liver fat by up to 50% for up to 24 weeks; Phase II work is underway, with an F2/F3 genetic MASH study anticipated next year. HSD17B13 siRNA development was deprioritized.

Market opportunity and positioning

• U.S. MASH affects ~13M adults; 5.0M–5.5M have moderate-to-advanced fibrosis, fewer than 10% of addressable patients are diagnosed, and 20%–25% of diagnosed patients receive advanced treatment.

• The U.S. MASH market was ~$1B in 2025 and is projected to exceed $15B by 2035; treated patients are projected to rise from ~100,000–130,000 to 750,000–1M.

• Initial focus is F2/F3 disease. Potential CIDEB differentiators include direct liver-fat reduction, broad MASH applicability, favorable early tolerability, and twice-yearly dosing; potential use alongside GLP-1 therapies and efficacy on histology and clinical outcomes remain subject to validation.