ORYZON Initiates Patient Enrollment in IDEAL Phase II Trial of Iadademstat in Essential Thrombocythemia

MADRID and CAMBRIDGE, Mass., June 02, 2026 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, Ticker: ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the first patient has been enrolled in IDEAL, its Phase II study evaluating iadademstat in patients with essential thrombocythemia (ET).

IDEAL (IaDademstat treatment for EssentiAL thrombocythemia) is a multicenter, single-arm Phase II study being conducted in Spain in adult patients with ET who are resistant or intolerant to hydroxyurea. The study is designed to evaluate the safety, tolerability and clinical activity of iadademstat, including its efficacy in reducing the percentage of adult ET patients with abnormal platelet counts. Iadademstat will be administered for up to 24 weeks, with an additional 24-week extension period available for those benefiting from treatment.

Dr. Carlos Buesa, Oryzon’s Chief Executive Officer, said, “The enrollment of the first patient in IDEAL represents another important milestone in the continued expansion of the iadademstat franchise. Over the last couple of years, we have significantly broadened the clinical development of iadademstat across hematologic malignancies, solid tumors and non-malignant hematology, generating a growing number of clinical catalysts across multiple disease settings. We believe essential thrombocythemia represents an attractive opportunity to explore the therapeutic potential of LSD1 inhibition in a chronic myeloproliferative disease where additional treatment options are needed.”

“This milestone also comes at a particularly exciting time for the iadademstat program, as updated results from the ALICE-2 study in first line acute myeloid leukemia (AML) will be presented at EHA next week,” Dr. Buesa added. “The data reported to date have shown very encouraging efficacy, with 100% of responses and high complete remission rates, and we look forward to next week’s presentation with a larger patient cohort. We believe ALICE-2 continues to support the potential of iadademstat as a differentiated component of frontline AML treatment and highlights the progress being made across our hematology development portfolio.”

Dr. Ana Limón, Oryzon’s Senior Vice-President of Clinical Development and Global Medical Affairs, added: “IDEAL will allow us to assess not only the clinical effects of iadademstat on platelet counts and hematologic responses, but also to further characterize the biological impact of LSD1 inhibition in this disease. We believe the study may provide important insights into the potential role of iadademstat across the broader spectrum of myeloproliferative disorders.”

Essential thrombocythemia is the most common myeloproliferative neoplasm and is characterized by persistently elevated platelet counts and an increased risk of thrombotic complications. Despite available therapies, a significant proportion of patients become resistant or intolerant to first-line treatments such as hydroxyurea, highlighting the need for novel treatment options.

Preclinical and clinical data support the role of LSD1 inhibition in regulating megakaryocyte maturation and platelet production, providing a scientific rationale for the evaluation of iadademstat in ET.

Beyond ET, iadademstat continues to advance across a broad clinical development program in hematologic malignancies, solid tumors and non-malignant hematology. Updated data from the ongoing ALICE-2 study accepted for presentation at EHA 2026 showed a 100% overall response rate (ORR), 93% composite complete remission (CRc) rate, and 79% true complete remission (CR) rate at the abstract submission cut-off in first-line AML patients treated with the triplet combination of iadademstat, azacitidine and venetoclax. In addition, updated data from the FRIDA study evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutated AML demonstrated a favorable safety profile and a CRc rate of 67% in a heavily pre-treated patient population. Updated clinical results from both studies will be presented at the EHA 2026 Congress next week.

Oryzon is also advancing the RESTORE trial evaluating iadademstat in sickle cell disease, and additional trials are in progress conducted under the Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI) and as investigator-sponsored studies in myeloproliferative neoplasms, myelodysplastic syndrome and small cell lung cancer.

About Oryzon
Founded in 2000 and headquartered in Barcelona, Spain, Oryzon (ISIN: ES0167733015) is a clinical-stage biopharmaceutical company and a European leader in epigenetics, with a strong focus on personalized medicine for central nervous system (CNS) disorders and oncology. Oryzon’s team comprises highly experienced pharmaceutical professionals based in Barcelona, Boston, and New Jersey. The Company has an advanced clinical portfolio built around two LSD1 inhibitors: iadademstat, its oncology/hematology program, with several ongoing Phase I and II studies and which has demonstrated strong preliminary clinical activity in acute myeloid leukemia, including a 100% overall response rate (ORR) in first-line AML; and vafidemstat, its lead CNS program, which is Phase III–ready in Borderline Personality Disorder (BPD). In addition, Oryzon is advancing a broader epigenetics pipeline targeting other mechanisms, including HDAC6, for which the Company has nominated ORY-4001 as a clinical candidate for potential development in Charcot–Marie–Tooth disease (CMT), amyotrophic lateral sclerosis (ALS), and other neurological disorders. The Company also operates a robust platform for biomarker identification and target validation across malignant and neurological diseases. For more information, visit www.oryzon.com

About Iadademstat
Iadademstat (ORY-1001) is an oral, highly selective inhibitor of the epigenetic enzyme LSD1, with potent differentiating effect in hematologic cancers. Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L acute myeloid leukemia (AML) patients (ALICE trial). Iadademstat is currently being evaluated in combination with azacitidine and venetoclax in 1L AML in the ALICE-2 trial, an investigator-initiated study (IIS) led by OHSU, and in combination with gilteritinib in the company-sponsored Phase Ib FRIDA trial in relapsed/refractory FLT3-mutant AML, with highly encouraging preliminary safety and efficacy data in both trials: 100% overall response rate (ORR) and 93% composite complete remission rate (CRc), with 79% strict CR in 1L AML, and 67% CRc in R/R Flt3-mut AML. Additional studies in hematologic malignancies include an IIS in myelodysplastic syndrome (MDS) and National Cancer Institute (NCI)-sponsored trials in myeloproliferative neoplasms and 1L AML conducted under the Cooperative Research and Development Agreement (CRADA) between Oryzon and the NCI. Beyond hematological cancers, iadademstat is being evaluated in extensive stage small cell lung cancer (ED-SCLC) in a Phase I/II randomized trial in 1L in combination with immune checkpoint inhibition (ICI) sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center, and an IIS trial in combination with ICI and radiotherapy. Oryzon has also expanded iadademstat into non-oncological hematology indications, with ongoing trials in sickle cell disease and essential thrombocythemia. Iadademstat has orphan drug designation for AML in the US and EU and for SCLC in the US.

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