Astellas Pharma (4503) investor relations material

Pipeline overview and program updates

• Achieved proof of concept for ASP2138, ASP7317, and zitidagresib (ASP3082), with accelerated pipeline progress and 12 phase II FSDs over five years.

• Advancing four flagship assets: ASP2138 (immuno-oncology), ASP7317 (blindness/regeneration), AT845 (genetic regulation), and setidegrasib/ASP3082 (targeted protein degradation).

• Terminated 21 clinical stage programs to reallocate resources to higher-value, lower-risk assets, improving pipeline quality.

• Built a balanced portfolio with strategic brands, follow-on programs, externally acquired assets, and early-stage research supporting multiple franchises.

• Focused pipeline includes late-stage and early-stage programs in oncology, ophthalmology, neuromuscular, and women's health.

Clinical trial data and development milestones

• zitidagresib showed 58.3% ORR and 83.3% DCR in PDAC phase I, and 37.5% ORR with 11.2 months PFS in NSCLC; phase III trials initiated for both indications.

• ASP2138 achieved proof of concept in gastric/GEJ adenocarcinoma; phase III trial planned for patients with low to moderate claudin 18.2 expression.

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

• AT-845 gene therapy for Pompe disease is under review for final POC; ASP7317 achieved POC in severe visual impairment, with phase III planning underway.

• Decision to discontinue late-stage development of zitidagresib in colorectal cancer due to insufficient efficacy.

R&D strategy and innovation priorities

• Adopted a Focus Area Approach integrating 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.

• Implemented an end-to-end patient-centric R&D model, emphasizing productivity, portfolio discipline, and rapid decision-making.

• Actively investing in talent, AI, and digital transformation to accelerate research, trial design, and data analysis.

• Targeting 10 new molecular entities to enter Phase 3 by 2034.