ORYZON announces First‑Patient‑In (FPI) in RESTORE Phase Ib trial of iadademstat in sickle cell disease
Recently approved by the European Medicines Agency, RESTORE will evaluate iadademstat’s ability to raise fetal hemoglobin (HbF) in adult patients with SCD
Oryzon Genomics S.A.,. (ISIN Code: ES0167733015, Ticker: ORY), a clinical‑stage biopharmaceutical company and global leader in epigenetics, today announced that the first patient has been enrolled in RESTORE, its multi‑center, open‑label Phase Ib clinical trial of iadademstat in adults with sickle cell disease (SCD). The study, to be conducted across several sites in Spain, aims to enroll approximately 40 adult patients. It is designed to evaluate the safety and tolerability of iadademstat in SCD and to establish the recommended Phase 2 dose (RP2D) as well as to evaluate iadademstat’s effect on inducing fetal hemoglobin (HbF) expression. Increases in HbF have already been recognized by the FDA as a clinically meaningful endpoint for the treatment of SCD.
Dr. Ana Limón, Senior Vice President of Clinical Development and Medical Affairs at Oryzon, said: “Iadademstat has demonstrated PoC in preclinical baboon models of SCD. Achieving FPI in RESTORE in a record time is an important milestone as we bring the first oral LSD1 inhibitor into clinical testing for SCD in Europe. Our goal is to deliver to SCD patients a clinical benefit comparable to the one achieved with certain gene therapies through inducing HbF, with the potential advantages of an oral, scalable approach”.
Dr. Carlos Buesa, Oryzon’s CEO, stated: “SCD represents a major unmet medical need, and we are excited to expand the scope of iadademstat’s clinical development to hematology. This is an important step toward offering new hope and potential treatment options for patients living with this condition. Given the open- label design of RESTORE, we expect to obtain initial insights within the next few months that will help us better understand iadademstat’s potential in this indication.”
Iadademstat is an oral, highly potent and selective LSD1 inhibitor in clinical development in onco- hematology designed to re‑induce HbF via epigenetic reprogramming of the hemoglobin switch, a mechanism that underlies certain FDA‑approved gene therapies for SCD. Iadademstat has shown good tolerability in first-in-man and combination studies across onco-hematology indications. Nearly 200 patients have been treated to date, supporting confidence in its safety profile entering the RESTORE trial.
SCD remains the most common inherited blood disorder in the United States and represents a significant unmet medical need, with limited effective and accessible treatment options. By increasing HbF levels,
iadademstat aims to reduce vaso-occlusion, hemolysis, and organ damage—key drivers of morbidity and decreased survival in SCD. Epidemiological studies have consistently shown a direct correlation between higher HbF levels and improved life expectancy in patients with SCD.