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 (SCLC) patients. Oryzon has completed a Phase IIa trial (ALICE study) which evaluated 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; positive final data from this trial were presented at the American Society of Hematology (ASH) 2022 conference. Currently, Oryzon is performing a Phase Ib trial (FRIDA study) to evaluate iadademstat in combination with gilteritinib in patients with relapsed/refractory AML with FLT3 mutations. Iadademstat is also being evaluated in a collaborative Phase II trial (NET study) with the Fox Chase Cancer Center in combination with paclitaxel in patients with relapsed/refractory SCLC or extrapulmonary high grade neuroendocrine carcinomas.

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 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; the pilot, small scale SATEEN study in relapse-remitting and secondary progressive multiple sclerosis; and a Phase II trial in severely ill COVID-19 patients (ESCAPE study), which assessed the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection. Currently, a Phase IIb trial (PORTICO study) with vafidemstat in Borderline Personality Disorder patients and a Phase IIb trial (EVOLUTION study) in schizophrenia patients are ongoing.

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.