Astellas Pharma (4503) investor relations material

Pipeline overview and program updates

• Achieved proof of concept for flagship assets ASP2138, ASP7317, zitidagresib (ASP3082), and setidegrasib/ASP3082, with phase III trials initiated or planned in PDAC, NSCLC, and gastric/GEJ adenocarcinoma.

• Strategic focus on immuno-oncology, targeted protein degradation, blindness/regeneration, and genetic regulation, with disciplined portfolio management and resource reallocation.

• Terminated 21 clinical-stage programs to prioritize higher-value, lower-risk assets and improve pipeline quality.

• Pipeline includes late- and early-stage programs in oncology, ophthalmology, neuromuscular, and women's health, supported by strategic brands and externally acquired assets.

• 12 Phase 1 FSD NMEs, 1 Phase 3 study initiated, and 4 PoCs achieved, validating assets and platforms.

Clinical trial data and development milestones

• Zitidagresib/setidegrasib (ASP3082) showed strong efficacy in PDAC (ORR 58.3%, DCR 83.3%) and NSCLC (ORR 37.5%, median PFS 11.2 months), with phase III trials underway or planned.

• ASP2138 achieved POC in gastric cancer; phase III trial targeting low to moderate Claudin 18.2 expression patients is planned.

• ASP7317 achieved POC in severe visual impairment; phase Ib data to be presented at ARVO 2026, with global development plans in progress.

• AT-845 and ASP2957 advancing in gene therapy for Pompe disease and XLMTM, with ongoing and planned clinical trials.

• PADCEV plus pembrolizumab improved event-free and overall survival in muscle-invasive bladder cancer (EV-304).

R&D strategy and innovation priorities

• Focus Area Approach integrates biology, modality/technology, and disease to generate multiple programs from a single scientific foundation.

• Strategic shift toward high-value, de-risked pipeline progression and enhanced productivity, targeting 10 new molecular entities to enter Phase 3 by 2034.

• End-to-end operational model and agile practices have increased R&D productivity, reduced handovers, and accelerated development cycles.

• Data-driven portfolio review and strict discipline guide prioritization, with clear criteria for discontinuation and resource reallocation.

• Investment in talent, digital transformation, AI, and robotics to enhance research, trial design, and operational efficiency.