CSL (CSL) Investor Update summary

R&D strategy and portfolio focus

• Emphasis on core immunoglobulins, plasma, and vaccine platforms, with investment in new modalities like self-amplifying mRNA and digital transformation initiatives.

• Active portfolio management and strategic partnerships drive efficient resource allocation, with 65% of R&D spend on new product development and 35% on life cycle management and regulatory requirements.

• Recent portfolio review prioritized IG, consolidated immunology and transplant, and created a combined cardiovascular/renal therapeutic area.

• Robust pipeline with 21 clinical programs balanced across plasma, recombinant, genetic medicines, immunoglobulins, haematology, cardiovascular/renal, vaccines, and transplant/immunology.

• Commitment to patient-centric innovation, digital transformation, sustainability, and AI-powered labs.

Key product and clinical program updates

• HEMGENIX®, KOSTAIVE®, RiaSTAP®, and garadacimab are advancing toward registration and approval in key regions, with major regulatory milestones expected through 2025.

• Hemgenix gene therapy for hemophilia B shows durable efficacy and safety, with global approvals and ongoing post-marketing commitments.

• KOSTAIVE® (sa-mRNA COVID-19 vaccine) approved in Japan, with EMA and FDA filings planned for 2025 and strong efficacy and durability data.

• RiaSTAP® process modernization approved in Europe; U.S. filing for acquired fibrinogen deficiency planned for 2025, with a Phase 3 trial ongoing in Europe.

• Garadacimab for hereditary angioedema is under review in multiple regions, with ≥94% reduction in HAE attacks and FDA requesting additional manufacturing data but no new clinical trials.

Innovation in plasma and IG platforms

• Horizon 1 and 2 initiatives maximize IG yield and modernize manufacturing, with regulatory engagement to ensure product comparability and process robustness.

• Prefilled syringes and nebulized IG (for bronchiectasis) enhance patient convenience and open new market opportunities, with Phase 2 trials planned.

• New plasma therapies like hemopexin (CSL889) for sickle cell disease and process improvements for RiaSTAP® highlight ongoing innovation.

• Plasma Innovation Platform Strategy Group established to drive collection-side innovation, digitization, donor experience, and cost reduction.

• HIZENTRA® PFS launched in multiple markets, offering greater convenience and flexibility for patients.