Q&A

Oryzon Genomics is an epigenetics-focused biotechnology company developing two clinical-stage drug candidates, iadademstat and vafidemstat, that inhibit lysine specific demethylase 1 (LSD1). As a clinical-stage company, Oryzon does not make money from commercial revenues. It funds development by raising capital, most recently a €12m equity placing completed in July 2026 that extended its cash runway. Near-term development is concentrated on iadademstat in haematology, with first-line acute myeloid leukaemia (AML) and sickle cell disease (SCD) as the central indications. Vafidemstat is a longer-term central nervous system (CNS) programme, with borderline personality disorder (BPD) as the lead indication. Our model assumes Oryzon self-sponsors the registrational trials in AML and SCD and licenses vafidemstat in 2027, and our FY27 estimates include a risk-adjusted €50m upfront payment from that assumed global licensing agreement. Successful business development is a key determinant of future shareholder dilution.

In our July 2026 update, we valued Oryzon Genomics at €1.0bn, or €12.4 per share, using a risk-adjusted net present value (rNPV) valuation. After positive interim ALICE-2 data at EHA 2026, we raised the probability of success for the acute myeloid leukaemia (AML) programme to 35%, from 30%, with other core assumptions unchanged. The per-share value stayed at €12.4 because the higher valuation was largely offset by the increase in shares to 84.3m from 79.9m after the €12m equity financing. Our model assumes Oryzon self-sponsors registrational trials in AML and sickle cell disease and licenses vafidemstat in 2027, so the valuation is sensitive to the assumed timing and terms. We exclude essential thrombocythaemia (ET) from the valuation and view it as a source of upside.

Our forecasts for Oryzon Genomics show an FY26 operating loss of €7.3m, with R&D expense of €21.0m and other income of €20.0m from the expected capitalisation of development expenditure. In July 2026, we raised our R&D estimate from €17.7m to reflect faster development activity in H126 and the start of the IDEAL Phase II trial. We also raised our other income estimate from €16.8m. For FY27, we forecast operating profit of €42.5m, which assumes a global licensing agreement for vafidemstat including a €50m upfront payment. Our model assumes a $1.5bn licensing deal including $150m upfront; the €50m included in our FY27 forecasts reflects risk adjustment across the respective indications. The timing of that partnering transaction is the key sensitivity in our financial model. If licensing is delayed beyond our assumptions, we estimate Oryzon would require c €20m of additional financing during FY27.

The investment case for Oryzon Genomics centres on iadademstat in first-line acute myeloid leukaemia (AML) and sickle cell disease. ALICE-2, a Phase Ib study of iadademstat with venetoclax and azacitidine in newly diagnosed AML, showed a 100% overall response rate and an 89% composite complete remission rate across 18 evaluable patients at EHA 2026. Final data are expected by end-2026, likely at ASH 2026 in December, a major potential catalyst in our view. If the findings are confirmed, Oryzon intends to engage with the FDA on ALICE-3, a potentially registrational Phase II/III study in first-line AML, targeted to start in 2027. For vafidemstat in borderline personality disorder, the Phase IIb PORTICO study established an efficacy signal but missed statistical significance on both primary endpoints, so FDA-acceptable endpoints are the key milestone before Phase III. The main risks are typical of drug development: clinical, regulatory, competitive, financing and commercial. Successful business development is a key determinant of future shareholder dilution.