CSL (CSL) Investor Update summary

R&D strategy and portfolio focus

• R&D efforts are concentrated on core platforms: immunoglobulins (IG), plasma, and vaccines, with significant investment in innovation and process improvements such as Horizon 1 and Horizon 2 for IG yield maximization, SID, Nebulised Ig, and sa-mRNA.

• Strategic allocation: 65% of R&D spend on new product development, 35% on life cycle management and regulatory requirements.

• Portfolio management includes active prioritization, strategic partnerships, and productivity gains to maximize resource efficiency and accelerate promising programs.

• Setbacks in late-stage programs (e.g., Kcentra in trauma, Hizentra in dermatomyositis, clazakizumab in ABMR) have led to pivots toward new indications or alternative strategies.

• The pipeline is balanced across plasma, recombinant, and genetic medicines, with 21 products in clinical development and a focus on rare and serious diseases.

Key product and clinical program updates

• Hemgenix gene therapy is approved in nine countries, with long-term data confirming sustained efficacy and safety; further approvals expected in Japan and Europe.

• Kostaive, a self-amplifying mRNA COVID vaccine, is approved in Japan and pending EMA and US filings, with plans to update the formulation for new variants.

• RiaSTAP has a modernized process approved in Europe; US filing for acquired fibrinogen deficiency is planned for 2025, with a Phase 3 trial ongoing in Europe.

• Garadacimab showed ≥94% reduction in HAE attacks, with expected approvals in EU, US, and JP in 1H 2025; US approval delayed due to manufacturing questions, but no new clinical trials are required.

• Nebulized IG for non-cystic fibrosis bronchiectasis is advancing to Phase 2, targeting a large and growing patient population.

Product and platform innovation

• Immunoglobulin yield maximization through Horizon 1 (process improvement) and Horizon 2 (proprietary process) initiatives, with regulatory engagement to ensure product comparability and robust data generation.

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

• RiaSTAP® process improvements approved in EU; Hemopexin (CSL889) progressing to Phase II for SCD VOC.

• Plasma innovation platform focuses on digitization, donor experience, and yield growth.

• Automation and AI-powered labs are enhancing data collection, process optimization, and throughput, accelerating clinical development timelines.