Ascelia Pharma (ACE) investor relations material

FDA feedback and U.S. regulatory path

• FDA acknowledged significant unmet medical need, no identified major safety concerns, and clinical evidence of technical performance as a T1-weighted MRI contrast agent.

• FDA said additional clinical evidence is needed; potential paths include a new-patient study or evaluation of existing SPARKLE images using a different primary endpoint.

• No new clinical study is currently planned; the most efficient U.S. path is being assessed with a focus on alternatives using the existing SPARKLE dataset.

• Potential endpoints are under evaluation, with FDA alignment planned before image reading; a Type C meeting is planned, with advisors and KOLs informing the review.

• FDA noted concerns about re-reading justification, reader-training bias, added utility versus T1-WI-only scoring, and post-hoc analyses; the SPARKLE re-read was designed under protocol to address reader-related issues.

Product quality

• FDA and Ascelia aligned on a strategy to resolve CRL quality issues involving drug-product specifications and proposed analytical methods, not manufacturing; implementation has begun.

• The Type A meeting clarified quality requirements and the path forward, reducing uncertainty on product-quality matters.

Development priorities and other markets

• Partnering continues, with interested potential partners and possible deal structures spanning development and commercialization; the aim is to advance discussions toward a signed partnership.

• European regulatory feedback will assess whether Orviglance could be approved using the existing data package and application; timing will be shared as plans develop.

• Current financing extends into Q2 next year, with an ambition to obtain FDA clarity within that financing period.