• Recent U.K. MHRA Scientific Advice Meeting (SAM) that resulted in full alignment to initiate confirmatory Phase 3 trial in U.K. with existing Phase 2 drug product paved the way Conditional Marketing Authorisation Application (CMAA) submission in the coming weeks and Phase 3 trial initiation in mid Q4/'26 following Phase 2b drug product requalification
  • MHRA's SAM participants' recommendation to Project Orbis U.K. leadership to take the global regulatory leadership mantle with U.S. FDA and EMA, coupled with granting the Company access to U.K. national patient registry, the UK Clinical Practice Research Datalink (CPRD) to strengthen comparator data while Phase 3 is ongoing to further improve interpretability of data during early market access phase
  • Since 2021, 29 MHRA-led products have been approved by U.S. FDA via Project Orbis
  • Company continues to engage with U.S. FDA on the non-Orbis Accelerated Approval route after successful Type C Statistical Methods Meeting that resulted in alignment on amended Phase 3 study design as buildout of OST-400 natural history database expands to include U.K. national patient registry data and Company awaits FDA decisions on RMAT, Rolling Review and CNPV ahead of the pending Q4/'26 completion of its ongoing BLA filing initiated in Q1/'26
  • BLAs granted via Project Orbis are eligible to receive Priority Review Vouchers (PRVs) and may also be granted via the Accelerated Approval Program
  • Patient previously reported as lost to follow-up confirmed to be alive