Clinical and regulatory path forward for the Phase 2b SAH study

Strategic focus for the study design of phase 2b

• Study design based on the objective of meeting the requirements for Conditional Approval and/or Compassionate Use in Europe and the US respectively.
• Planned pre-IND meeting with the FDA in the US and a corresponding meeting with the EMA for Europe and/or national medicines agencies within Europe.
• Efficacy will be assessed using the GOSE scale after 3 months as the primary efficacy measure and NFL (brain injury marker) as the secondary efficacy measure.
• The study comprises approximately 45 active patients and is an open-label study in which patients receiving active treatment are compared with matched historical controls in the Nordic region and Europe, taking into account patient ethics considerations.
• Interim analysis at 30 treated patients (Q4 2027/Q1 2028) for statistical efficacy and effect size analysis ahead of regulatory dialogue regarding Compassionate Use authorisation in the US and Conditional Approval in Europe following completion of the Phase 2b study, i.e. after a total of 45 patients.