Spanish biotech Oryzon Genomics is focused on epigenetics. Iadademstat is being explored for acute leukaemias and SCLC. Vafidemstat, its CNS asset, has completed several Phase IIa trials and a Phase IIb trial in BPD is now the lead study, but Oryzon is rapidly expanding its CNS R&D pipeline.
Oryzon develops small molecule inhibitors for epigenetic targets. The two lead drugs are iadademstat for oncology and vafidemstat for central nervous system (CNS) indications (both are LSD1 inhibitors, an epigenetic target). In oncology, the ALICE trial continues to show positive results in acute myeloid leukaemia (AML) while new trials FRIDA in AML and STELLAR in small cell lung cancer (SCLC) could potentially be pivotal, with the FDA having now granted orphan drug designation for iadademstat in AML and SCLC. In CNS, vafidemstat is being evaluated in a number of trials, PORTICO in borderline personality disorder and EVOLUTION in schizophrenia, both Phase IIb trials are currently enrolling patients. Oryzon is also hoping to register vafidemstat in its Phase I/II HOPE study targeting Kabuki syndrome, a rare disorder that affects multiple CNS systems, with an objective to target further orphan drug designation in this indication.
Oryzon is among the leading clinical stage drug developers with a second generation of epigenetic therapeutics, which have greater selectivity and potentially a favourable safety/efficacy profile than the first generation HDAC inhibitors.