The biopharmaceutical company Oryzon Genomics, a member of Catalonia.health, has announced that the European Medicines Agency (EMA) has approved its Clinical Trial Application (CTA), the European equivalent to an IND, to initiate a Phase Ib trial of iadademstat in sickle cell disease (SCD). This will be the first clinical trial investigating iadademstat in a non-malignant haematological indication.
The Phase Ib study, named RESTORE (REgulation of Sickling ThrOugh Reprogramming Epigenetics), will be conducted at multiple sites in Spain and aims to enrol 40 adult patients with SCD. The trial’s primary objectives will be to evaluate the safety and tolerability of iadademstat and to establish its Recommended Phase 2 dose (RP2D). Secondary objectives include assessing iadademstat’s activity to induce fetal hemoglobin (HbF), among others.
Ana Limón, Senior Vice-president of Clinical Development and Medical Affairs at Oryzon said, “We are thrilled to be the only LSD1 inhibitor currently into clinical development for SCD. Targeting LSD1 presents a highly promising therapeutic approach for this disease, which affects approximately 7.7 million people worldwide as per 2025 estimates. Iadademstat has produced a significant increase in HbF levels in baboons, the only animal model with strong translational relevance to humans, after just a single dose. Increased HbF levels mitigate — and potentially reverse — the pathological phenotype of the disease and increases in HbF have already been recognized by the FDA as a clinically meaningful endpoint for the treatment of SCD. The trial has been carefully designed to deliver a rapid and clear signal of biological activity.”
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