CStone Pharmaceuticals (2616) Investor Update summary

Business overview and strategy

• Achieved first profitability in H1 2024, driven by license fee income and cost control.

• Transitioned commercial model and divested ivosedinib, generating $50mn in total deal value.

• Focused on maximizing commercial value of marketed products and advancing innovative pipeline assets.

• Strong cash position of RMB 813.9mn as of June 2024, with reduced operating cash burn.

• Exploring global business development partnerships for key pipeline assets.

Key clinical program updates: CS5001 (ROR1 ADC)

• Phase I trial completed dose escalation with no dose-limiting toxicity up to 195 μg/kg/DL10.

• Well tolerated in heavily pre-treated B-cell lymphoma patients; most common TEAEs were manageable.

• Achieved 48% ORR in B-cell lymphoma, 60% in Hodgkin lymphoma, and 56% in non-Hodgkin lymphoma; 77% ORR at 125 μg/kg/DL8.

• Tumor shrinkage and durable responses observed, with ongoing treatment in several patients.

• Proprietary prodrug technology and tumor-cleavable linker minimize systemic toxicity and enable tumor-selective activation.

Key clinical program updates: CS2009 (PD-1/VEGFA/CTLA-4 trispecific antibody)

• First-in-class/best-in-class potential as a next-generation immune backbone for large indications (NSCLC, HCC, GC, etc.).

• Demonstrated superior in vivo efficacy and promising PK/tox profile in preclinical studies.

• Preferentially activates tumor-infiltrating lymphocytes and attenuates peripheral immunotoxicity.

• IND filing expected in Q4 2024; FIH trial to follow.

• Patent filed in Q3 2024; cell line development achieves high yield.