Oryzon Genomics (ORY) Corporate Presentation summary

Strategic focus and platform

• Focuses on personalized medicine in epigenetics, targeting CNS and oncology indications with a growing pipeline and advanced clinical programs.

• Develops highly potent and selective LSD1 inhibitors, with two main programs: vafidemstat (CNS) and iadademstat (oncology).

• Raised €121M in equity since 2015, with €33.5M since 2022, and is listed in Madrid.

CNS pipeline: vafidemstat

• Vafidemstat is a Phase III-ready LSD1 inhibitor for CNS diseases, showing robust safety in over 430 subjects and no significant side effects.

• PORTICO Phase IIb in borderline personality disorder (BPD) showed all efficacy endpoints favoring vafidemstat over placebo, with a significant global treatment effect.

• FDA supports a Phase III trial in BPD, with agitation/aggression as a primary endpoint and a planned sample size of 350 patients.

• Vafidemstat is also in a Phase II trial for schizophrenia, targeting negative and cognitive symptoms, with strong preclinical and market rationale.

Oncology pipeline: iadademstat

• Iadademstat is a Phase II LSD1 inhibitor for oncology, with a focus on AML and SCLC, and multiple ongoing trials in collaboration with leading institutions.

• ALICE trial in AML showed rapid, deep, and durable responses, with 81% ORR and 91% MRD negativity in CR/CRi patients; results published in Lancet Haematology.

• FRIDA trial in FLT3mut+ R/R AML showed 69% ORR and fast time to response; recruitment ongoing to optimize dosing.

• SCLC program leverages strong preclinical synergy with ICIs; new pivotal trial in combination with ICI to start in 2025.