Clinical Trials

Oryzon has two drugs in clinical development, iadademstat  (also known as ORY-1001), a selective LSD1 inhibitor for oncology, and vafidemstat  (also known as ORY-2001), a CNS optimized LSD1 inhibitor for neurological disorders.

Oryzon has performed a Phase I clinical trial with iadademstat  in patients with relapsed/refractory acute leukemia, and especially in those with alteration in the MLL gene and other specific genomic alterations, and a Phase IIa trial (CLEPSIDRA study) with iadademstat in combination with platinum-etoposide in relapsed small cell lung cancer patients. Currently, Oryzon is performing a Phase IIa trial (ALICE study) to evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with standard treatment with azacitidine in newly diagnosed patients with Acute Myeloid Leukemia (AML) not eligible for intensive chemotherapy, with positive preliminary clinical results reported.

With vafidemstat , after completing a Phase I clinical trial in healthy volunteers, Oryzon has performed Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders: Autism Spectrum Disorder, Borderline Personality Disorder and adult Attention Deficit Hyperactivity Disorder (REIMAGINE study), and in aggressive/agitated patients with moderate or severe Alzheimer’s disease (REIMAGINE-AD study), with positive preliminary clinical results reported in both clinical trials. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL and ETHERAL-US studies in patients with mild to moderate Alzheimer’s disease, where a significant reduction of an inflammatory biomarker has been observed after 6 months of treatment, and the pilot, small scale SATEEN study in relapse-remitting and secondary progressive multiple sclerosis. A new Phase IIb trial (PORTICO study) with vafidemstat in Borderline Personality Disorder patients is ongoing, and a Phase IIb trial (EVOLUTION study) in schizophrenia has been recently authorized. Vafidemstat is also being evaluated in a Phase II trial in severely ill COVID-19 patients (ESCAPE study), assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection.

Both Oryzon’s experimental drugs target the Lysine-specific demethylase LSD1 , a critical enzyme involved in the regulation of histone and chromatin remodeling. By doing this, they regulate important genes involved in the control of cellular differentiation, survival and proliferation.

Our compounds are investigational medicines, meaning that they are being evaluated in clinical trials and are not yet available for use outside of a clinical trial setting. Approval by regulatory authorities such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) at the end of their clinical development is the way to make our medicines available to the patients in need of these medicines.

In the meantime, our priority is to advance in the clinical trials in order to obtain the data required for the review and approval of our medicaments by the regulatory authorities. We provide below a summary of the clinical trials sponsored by Oryzon.