Sparks - Oryzon Genomics

Oryzon Genomics has announced that the first patient has been enrolled in the RESTORE trial, a Phase Ib, multi-centre, open-label study assessing iadademstat in adults with sickle cell disease. This news swiftly follows the update in August 2025 that the European Medicines Agency had provided regulatory clearance for RESTORE, an encouraging indicator that plans are progressing as anticipated. RESTORE aims to recruit c 40 participants and is designed to assess the safety and tolerability of iadademstat in this patient population. It will also determine a recommended dose for Phase II, and measure the effect of the candidate in inducing foetal haemoglobin expression (an FDA-recognised outcome measure for sickle cell disease).

Since iadademstat has already demonstrated safety and tolerability in c 200 participants in prior clinical trials, the RESTORE study represents a potentially expandable opportunity for Oryzon, exploring iadademstat beyond oncology indications.