Spanish biotech Oryzon Genomics is focused on epigenetics. Iadademstat (Phase IIa) is being explored for acute leukaemias and SCLC; its CNS product vafidemstat is in Phase IIb trials in BPD, schizophrenia and also being investigated for precision medicine. ORY-3001 is being developed for certain orphan indications.
Oryzon develops small molecule inhibitors for epigenetic targets. It has completed five Phase II and has three ongoing Phase II trials with two assets: iadademstat and vafidemstat. Oryzon reported data in March 2021 from its two Phase IIa trials focusing on AD (ETHERAL and REIMAGINE-AD) that confirm vafidemstat is safe and effective in controlling aggression/agitation, a key focus area. In July 2020, results from the Phase IIa REIMAGINE trial with vafidemstat for aggressiveness in psychiatric diseases showed improvement in all cohorts (BPD, ADHD and ASD). Oryzon is conducting the Phase IIb trial for vafidemstat in BPD (PORTICO) and a Phase IIb trial for schizophrenia (EVOLUTION) which received approval from Spanish regulators in July. Oryzon is also developing a precision medicine approach for vafidemstat in genetically defined patients with certain CNS disorders. In June 2021, Oryzon presented an update from the Phase II ALICE trial (iadademstat plus azacitidine) in AML, which continues to show a large response benefit.
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.