Oryzon shares soar on safety clinical trial approval

BarcelonaOryzon Genomics shares rose this Monday after learning that the European Medicines Agency (EMA) has authorized the initiation of a Phase Ib clinical trial of the molecule yadademstate for sickle cell anemia (SCA). This drug is used against highly aggressive cancers such as leukemia and works by blocking the action of a protein called LSD1, which has been shown to promote tumor cell multiplication.

The company, whose shares are up more than 3%, highlights that this will be the first clinical trial investigating yadademstate in a non-malignant hematological indication. The Phase Ib study, called Restore, will be conducted at several centers in Spain. The goal is to recruit 40 adult patients with SCD to evaluate the safety and tolerability of yadademstate and establish its recommended dose for Phase 2. Shares closed the day up 2.36% at €2.820.

SCD is a chronic, inherited blood disorder caused by a mutation that leads to the production of hemoglobin S (HbS) instead of the normal hemoglobin A. Under low-oxygen conditions, HbS tends to polymerize, causing red blood cells to become sickle-shaped, rigid, and fragile. This leads to microvascular occlusion, hemolysis, and chronic inflammation.

"Highly promising" approach

"We are very pleased to be the only LSD1 inhibitor currently in clinical development for SCD. Inhibiting LSD1 represents a highly promising therapeutic approach for this disease, which affects approximately 7.7 million people worldwide," said Ana Limón, M.D., Senior Vice President of Clinical Development.

According to multiple market research reports, the market for SCD treatments is expected to grow substantially—from approximately $3 billion in 2025 to approximately $8 billion in 2032.

While approval by the U.S. Food and Drug Administration (FDA) is lacking, widespread use restricts access to much of the global patient population. Oxbryta, a once-approved oral therapy for SCD, reached annual sales of $328 million in a relatively short period of time before being withdrawn from the market. This highlights both the strong commercial potential and the urgent unmet need for effective, scalable, and affordable polar treatments for SCD.

Iadademstat has produced significant increases in HbF levels in baboons, the only animal model with strong translational relevance to humans, after a single dose. The increase in HbF levels mitigates—and potentially reverses—the pathological phenotype of the disease. Therefore, the FDA has already recognized increased HbF as a clinically relevant criterion for the treatment of SCD.