Novartis (NOVN) ASCO Investor Event 2024 summary

Novartis legacy and unmet needs in CML

• Novartis transformed CML from a deadly disease to a chronic condition with high survival rates over the past 20 years.

• 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 a nearly 20% higher MMR rate at week 48 versus investigator-selected TKIs (67.7% vs 49.0%) and a 30% higher MMR rate versus imatinib (69.3% vs 40.2%), with consistent benefits across all subgroups.

• Scemblix led to faster and deeper molecular responses, reducing median time to MMR by about a third compared to other TKIs (24.3 weeks vs 36.4 weeks) and doubling deep molecular response rates.

• 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.

• Scemblix, a first-in-class STAMP inhibitor, targets the ABL myristoyl pocket for enhanced efficacy and reduced off-target effects.

• Compared to 2G TKIs, Scemblix had numerically higher MMR rates, earlier achievement of MMR, and deeper responses.

Safety and tolerability

• Scemblix demonstrated a favorable safety profile, with fewer grade ≥3 adverse events and lower rates of dose adjustments, interruptions, and discontinuations than imatinib or 2G TKIs.

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

• Non-hematological adverse events impacting quality of life were less frequent and severe with Scemblix.

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

• Fewer patients discontinued Scemblix due to adverse events compared to imatinib (5% vs. 11%) and 2G TKIs (5% vs. 10%).