Ascelia Pharma (ACE) investor relations material

Pipeline overview and strategic focus

• Two main products: Orviglance (diagnostic drug) and Oncoral (therapeutic drug), with Orviglance as the lead asset nearing FDA approval.

• Orviglance targets a well-defined unmet need in rare cancer patients with severe renal disease, representing an $800 million global market, half in the U.S.

• Manufacturing capacity is in place, and nine clinical studies have shown consistent positive results, leading to FDA submission.

• The company is not planning to commercialize alone but is actively seeking a partner for Orviglance commercialization.

• Oncoral, based on irinotecan, has completed phase I and is being developed for gastric and other solid cancers.

Orviglance clinical and regulatory progress

• Orviglance is a manganese-based, liver-specific MRI agent, avoiding gadolinium-related safety issues and offering minimal systemic exposure.

• Orphan Drug Designation from the FDA provides seven years of U.S. market exclusivity upon approval.

• Submission to the FDA occurred in September, with a PDUFA action date set for early July; the review process is on track with positive interactions.

• Phase III data show significant improvement in imaging quality for the target population, reducing the risk of missed liver cancer diagnoses.

• Additional patents filed for next-generation formulations and manufacturing processes to extend exclusivity.

Market opportunity and commercialization strategy

• U.S. market research indicates about 100,000 annual imaging procedures in the target population, concentrated in 400 large accounts.

• Pricing research and payer feedback support a strong value proposition for Orviglance.

• The company is in advanced discussions with potential partners, focusing on those with established hospital sales forces and distribution networks.

• Partnership structures under consideration include both global and U.S.-only rights, with flexibility to find additional partners for other regions.

• The company will provide product expertise and support to partners post-approval to maximize long-term value.