Spanish biotech Oryzon Genomics is focused on epigenetics. Iadademstat is being explored for haematology-oncology, where the strategic priority is in first-line acute myeloid leukaemia; there is potential for an accelerated approval pathway should clinical data continue to be supportive. It is also being evaluated in other indications, such as myelodysplastic syndrome, myeloproliferative neoplasms and sickle cell disease, as well as in small cell lung cancer. Central nervous system asset vafidemstat has completed a Phase IIb trial in borderline personality disorder and preparations are underway for Phase III. It is also currently involved in a Phase IIb trial for schizophrenia, and management is preparing for an additional Phase II trial in autism spectrum disorder.
There are four key reasons why Oryzon may be considered a compelling investment opportunity:
1. Iadademstat presents a differentiated opportunity in haematological diseases.
Iadademstat is an orally administered inhibitor of LSD1, a target involved in the regulation of gene expression in cancer cells and leukemic stem cell survival. Recent clinical data in first-line acute myeloid leukaemia (AML) have shown highly promising interim efficacy signals, including a 100% response rate in combination with venetoclax and azacitidine (Ven-Aza, the current standard of care), which compare favourably to Ven-Aza alone. As such, the programme is being positioned in first-line AML, which represents a sizeable market with an unmet medical need, since around a third of patients have primary resistance or have suboptimal responses to Ven-Aza. The potential for an accelerated approval pathway, if supported by upcoming data, also provides a clear route to value creation. Beyond AML, iadademstat is also being evaluated in other indications, such as myelodysplastic syndrome, myeloproliferative neoplasms and sickle cell disease as well as in small cell lung cancer.
2. A dense pipeline of near-term catalysts may drive investor attention.
Oryzon is entering a period of heightened news flow, with multiple clinical readouts expected across 2026–27. Key milestones include advanced interim results from the ALICE-2 trial in first-line AML at the European Hematology Association conference in June, followed by the final readout at the American Society of Hematology conference in December, alongside interim updates from the RESTORE study in sickle cell disease. These events are expected to inform registrational strategies and could represent meaningful inflection points for the investment case, particularly given the company’s increasing near-term strategic focus on iadademstat.
3. Vafidemstat offers longer-term upside in CNS indications.
Vafidemstat is being developed for neuropsychiatric disorders, including borderline personality disorder (BPD) where there are currently no approved pharmacological treatments. Key opinion leaders in the field believe the clinical data to date have been encouraging, and preparatory activities for a Phase III programme are underway. As such, vafidemstat represents a key value driver over the longer term, particularly given its potential applicability across multiple CNS indications.
4. A diversified pipeline and dual therapeutic focus helps mitigate binary risk.
Oryzon’s portfolio spans both oncology-haematology and CNS, with multiple indications under investigation for each lead asset. Iadademstat alone is being explored across several haematological and solid tumour settings, while vafidemstat is being assessed in BPD, schizophrenia and autism spectrum disorder. This breadth provides multiple shots at value creation and reduces reliance on any single clinical outcome. Combined with an established safety database across both assets, this diversification supports a more balanced risk profile relative to single-asset peers.
In summary, Oryzon presents a compelling investment case, underpinned by a differentiated clinical pipeline, multiple upcoming catalysts and exposure to high-value therapeutic areas. With near-term momentum in iadademstat and longer-term potential from vafidemstat, the company is positioned to deliver meaningful clinical and shareholder value.
Published 22 May 2026
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Carlos Buesa
CEO