Ascelia Pharma (ACE) ABGSC Investor Days summary

Pipeline and Product Development

• Orviglance, a manganese-based MRI agent, completed phase III and targets FDA orphan drug designation, with NDA submission planned for mid-2025.

• Orviglance addresses unmet needs in rare oncology, focusing on patients with severe renal disease requiring liver imaging.

• Oncoral, an oral irinotecan chemotherapy, completed phase I and is phase 2–ready for gastric and other solid tumors, with a clinical collaboration in place.

• Orviglance demonstrated strong efficacy and safety in nine clinical studies, with commercial scale manufacturing ready in the U.S.

• Orviglance clinical data accepted for presentation at major scientific conferences in 2024–2025.

Clinical Results and Regulatory Progress

• Orviglance phase III met primary and key secondary endpoints, showing significant improvement in liver imaging.

• Safety profile consistent with earlier studies, mainly mild GI symptoms.

• FDA meeting provided positive feedback for NDA structure; submission on track for mid-2025.

• NDA includes all clinical, non-clinical, and manufacturing data; timeline for submission is July 1, plus or minus six weeks.

• Launch expected next year, with a 10-month review timeline post-NDA submission.

Market Opportunity and Commercialization Strategy

• Global addressable market for Orviglance is $800 million, with half in the US, focused on a fragile patient subset.

• Commercialization will be via a dedicated partner, aiming for significant investment and successful launch.

• 75% of U.S. target patients are concentrated in 400 medical accounts; about 2,000 radiologists cover this segment.

• Over 90% of healthcare professionals express concern about gadolinium-based agents, and 84% of US physicians are likely to use Orviglance for target patients.

• Rollout strategy is focused and data-driven, leveraging existing relationships with key opinion leaders.