Novartis (NOVN) ASCO Investor Event 2024 summary

Novartis legacy and unmet needs in CML

• Over 20 years, Novartis transformed CML from a deadly disease to a chronic condition with high survival rates.

• Despite advances, 40-50% of first-line CML patients switch therapy within 5 years due to resistance or intolerance.

• Only 25% of treated CML patients achieve treatment-free remission, with persistent adverse events driving non-adherence.

• Current first-line treatment is split between imatinib and 2G TKIs, but many patients do not reach efficacy goals.

• Faster, deeper molecular responses with better tolerability remain key unmet needs for newly diagnosed patients.

Key clinical data and study results

• Phase III ASC4FIRST trial showed Scemblix achieved superior major molecular response rates at week 48 versus both investigator-selected TKIs and imatinib, with a nearly 30% improvement over imatinib and nearly 20% over other TKIs.

• Scemblix led to earlier and deeper molecular responses, reducing median time to major molecular response by about three months compared to other TKIs.

• The trial's median follow-up was 16.3 months for Scemblix and 15.7 months for SoC TKIs, with the next analysis scheduled at week 96.

• The trial randomized 405 newly diagnosed CML patients to Scemblix or investigator-selected TKIs, reflecting real-world clinical practice.

• Superior efficacy was consistent across all demographic and prognostic subgroups.

Safety, tolerability, and regulatory recognition

• Scemblix demonstrated fewer grade ≥3 adverse events and lower rates of treatment discontinuation and dose adjustments than imatinib or 2G TKIs.

• Hematologic and non-hematologic toxicities, including diarrhea, rash, nausea, muscle spasms, and edema, were lower for Scemblix-treated patients.

• No new safety concerns were observed in newly diagnosed patients.

• Scemblix received Breakthrough Therapy Designation from the FDA for newly diagnosed CML, with global submissions planned for H2 2024-2025.

• Data was published in the New England Journal of Medicine and highlighted at ASCO and EHA.