Ascelia Pharma (ACE) investor relations material

Strategic focus and pipeline overview

• Focused on developing and commercializing novel drugs for rare cancer conditions, with two main assets: Orviglance (diagnostic) and Oncoral (therapeutic).

• Orviglance targets unmet needs in liver MRI imaging for patients with severe renal impairment, offering a gadolinium-free alternative.

• Orviglance has orphan drug designation and is under FDA review, with a PDUFA decision expected by July 3rd.

• Oncoral is an oral formulation of irinotecan, aiming to improve safety and efficacy in cancer treatment, with initial focus on gastric cancer.

• Commercialization strategy centers on partnering due to limited internal infrastructure.

Orviglance market opportunity and differentiation

• Addresses an $800 million global market, with nearly half in the U.S.; initial commercialization focus is the U.S.

• Orviglance is positioned as the only liver MRI agent for patients with severe renal impairment, avoiding gadolinium-related risks.

• Extensive clinical data from nine studies show strong efficacy and safety, with mostly mild gastrointestinal side effects.

• Market research indicates over 90% of healthcare professionals are concerned about gadolinium safety.

• Environmental and sustainability concerns over gadolinium are increasing, supporting the need for alternatives.

Regulatory and commercialization progress

• NDA for Orviglance submitted September 3rd; FDA Day 74 letter confirmed no major deficiencies and set July 3rd PDUFA date.

• Manufacturing is at commercial scale with ample capacity; orphan drug status supports regulatory and market positioning.

• Commercialization will target large hospitals, covering 75% of the U.S. market with 400 accounts.

• Ongoing discussions with multiple potential partners, aiming for attractive deal terms and efficient market entry.

• Active engagement with medical community and key opinion leaders to build awareness and prepare for launch.