Ankündigung • 22h
Oryzon Genomics S.A., Annual General Meeting, Jun 25, 2026 Oryzon Genomics S.A., Annual General Meeting, Jun 25, 2026. Ankündigung • May 15
Oryzon Genomics, S.A. Announces to Present Updated Positive Clinical Data for Iadademstat in Acute Myeloid Leukemia at EHA 2026 Oryzon Genomics, S.A. announced that updated positive clinical data from two Phase Ib clinical trials evaluating its selective LSD1 inhibitor iadademstat in acute myeloid leukemia (AML) will be presented in two poster sessions at the European Hematology Association (EHA) Annual Congress, taking place June 11-14, 2026 in Stockholm, Sweden. Presentations details: Title: Updated Safety and Efficacy Results of a Phase Ib Investigation of the LSD1 Inhibitor Iadademstat (ORY-1001) in Combination with Azacitidine and Venetoclax in Newly Diagnosed AML Abstract ID: EHA-4924 Presenter: Curtis Lachowiez, MD, Oregon Health & Science University, Portland, United States of America Presentation Date and Time: Saturday, June 13, 2026, 6:45-7:45 pm CEST As of the February 2026 data cutoff, the triplet combination of iadademstat, azacitidine and venetoclax evaluated in the ALICE-2 trial (NCT06357182) continues to demonstrate favorable safety and high response rates. Among evaluable patients (n=14/15) the overall response rate (ORR) was 100% with a complete response (CR) rate of 79% (n=11/14) and a composite complete remission (CRc: CR+CRh+CRi) rate of 93% (n=13/14). After a median follow-up of 6 months, the estimated 12-month OS rate was 74%. Updated data with additional patients and more mature responses will be presented at the meeting. Title: Safety and Efficacy of Iadademstat Plus Gilteritinib in the FRIDA Expansion Cohort of FLT3-Mutated Relapsed/Refractory Acute Myeloid Leukemia Abstract ID: EHA-5879 Presenter: Amir Fathi, MD, Massachusetts General Hospital, Boston, United States of America Presentation Date and Time: Friday, June 12, 2026, 6:45-7:45 pm CEST Updated data from a heavily pre-treated relapsed or refractory FLT3mut AML population in the FRIDA trial (NCT05546580) evaluating iadademstat plus standard of care treatment gilteritinib demonstrated a favorable safety profile and a CRc rate of 67% (n=12/18). Additional data will be presented at the conference. Iadademstat (ORY-1001) is an oral, highly selective inhibitor of the epigenetic enzyme LSD1, with a 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 hematological malignancies include an IIS in myelodysplastic syndrome (MDS) and U.S. 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 the 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 trials in sickle cell disease (approved by EMA, enrolling) and essential thrombocythemia (approved by EMA). Iadademstat has orphan drug designation for AML in the US and EU and for SCLC in the US. Ankündigung • Feb 24
Oryzon Genomics, S.A. Receives European Medicines Agency Approval to Initiate A Phase Ii Study of Iadademstat in Essential Thrombocythemia Oryzon Genomics, S.A. announced that the European Medicines Agency (EMA) has authorized its Clinical Trial Application (CTA) to initiate a Phase II study of iadademstat, Oryzon's potent and selective LSD1 inhibitor currently in clinical development in oncology and hematology, for the treatment of essential thrombocythemia (ET). The study, named IDEAL (IaDademstat treatment for EssentiAL thrombocythemia), is a multicenter, single-arm Phase II study to be conducted in Spain in adult patients with ET who are resistant/intolerant to hydroxyurea. Iadademstat is by far the most potent LSD1 inhibitor in clinical development, with more than 100-fold greater potency than any other LSD1 inhibitor currently in development. The IDEAL study will explore this potential of iadademstat, in addition to assessing its dose-dependent effects on platelet counts and thrombotic events in ET patients who are resistant or intolerant to standard-of-care treatment with hydroxyurea". Iadademstat is also being actively investigated in multiple oncology clinical trials, including the Phase Ib ALICE-2 study in combination with venetoclax and azacitidine in first-line AML. Highly encouraging preliminary data were presented at the American Society of Hematology (ASH) 2025 annual meeting, showing a 100% overall response rate (ORR) and 90% strict complete remission (CR) rate. Additional studies include the company-sponsored FRIDA trial evaluating iadademstat in combination with gilteritinib in relapsed/refractory FLT3-mutated AML, for which preliminary positive data were also presented at ASH-2025, as well as several trials conducted under a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute in first line AML, myeloproliferative neoplasms and small cell lung cancer, and investigator-initiated studies in myelodysplastic syndrome and small cell lung cancer. In addition, the company is conducting a clinical trial evaluating iadademstat In sickle cell disease. Ankündigung • Feb 12
Oryzon Genomics, S.A. Announces Chief Medical Officer For CNS Programs Changes Oryzon Genomics, S.A. announced the appointment of Rolando Gutierrez-Esteinou, M.D., as Chief Medical Officer, CNS Programs. Dr. Gutierrez-Esteinou is a Harvard-trained psychiatrist and an accomplished global clinical development executive with more than 20 years of leadership experience advancing neuroscience and psychiatry-focused drug development programs. He brings deep expertise in late-stage clinical execution, regulatory strategy, and pathways to market approval. Most recently, Dr. Gutierrez-Esteinou served as Chief Medical Officer at Atai Life Sciences, where he oversaw a portfolio of clinical-stage CNS programs and built a multidisciplinary development organization supporting multiple trials across psychiatric indications. Throughout his career, he has held senior leadership roles across global pharmaceutical companies—including Johnson & Johnson, Bristol Myers Squibb, and Novartis—as well as in biotech and CRO environments. His experience spans clinical development from Phase I through Phase IV across major neuropsychiatric disorders, including schizophrenia, mood disorders, addiction, and cognitive impairment. Dr. Gutierrez-Esteinou has contributed to multiple late-stage development programs and regulatory submissions, including supplemental New Drug Applications (sNDAs) and label expansions for established CNS therapies such as Risperdal (risperidone) and Abilify (aripiprazole). He has led regulatory interactions with major global health authorities, including the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). He completed his psychiatry residency at Harvard Medical School and held research fellowships at Harvard/McLean Hospital and the U.S. National Institutes of Health (NIH). Oryzon Genomics, S.A. also recognized Dr. Michael Ropacki, who has led the vafidemstat program as CMO–CNS over the past six years with significant success. Ankündigung • Feb 10
Oryzon Genomics, S.A. Announces First Patient Dosed in an Investigator-Initiated Phase Ib Study of Iadademstat in Extensive Stage Small Cell Lung Cancer Oryzon Genomics, S.A. announced that the first patient has been dosed in an investigator-initiated Phase Ib dose-finding trial evaluating iadademstat, Oryzon's potent and selective LSD1 inhibitor, in combination with radiotherapy and an immune checkpoint inhibitor in patients with residual, progressive or recurrent extensive stage small cell lung cancer (ES-SCLC). The study is sponsored and conducted by Yale University. The trial (NCT07113691), titled "Iadademstat and Radiation Therapy With Atezolizumab in Extensive Stage Small-cell Lung Cancer (ES-SCLC) Patients With Persistent, Recurrent or Progressive Disease After First Line Systemic Therapy", is an open-label, non-randomized Phase Ib study that will evaluate the safety, tolerability, and efficacy of iadademstat combined with atezolizumab and stereotactic body radiation therapy (SBRT) followed by maintenance therapy with atezolizumib and iadademstat. The study will enroll patients with residual, progressive or recurring ES-SCLC who previously received platinum-based chemotherapy with or without immune checkpoint inhibitor therapy. Dr. Anne Chiang at Yale University is serving as Principal Investigator. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors,afidemstat in CNS (Phase III-ready) and iadademstat in oncology (Phase II). The company has other pipeline assets directed against other epigenetic targets like HDAC-6 where a clinical candidate ORY-4001, has been nominated for its possible development in CMT and ALS. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. Iadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers. A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi. Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with azacitine and venetoclax in 1L AML in an investigator-initi 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 recently reported at ASH-2025 for both trials: 100% ORR and 90% strict CR in 1L AML, and 67% CCR (at the dose under expansion) in R/R AML. Additional studies in hemato-oncology include an IIS in MDS, and trials in myeloproliferative neoplasms and 1L AML both sponsored and conducted by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI. Beyond hematological cancers, the company-sponsored Phase IbFRIDA trial in relapsed/refractory FLT3-Mutant AML, with highly promising preliminary safety and efficacy data recently announced at ASH-2025. Ankündigung • Dec 09
Oryzon Genomics, S.A. Presents Data for Iadademstat Combinations in AML at the American Society of Hematology (ASH) 67th Annual Meeting Oryzon Genomics, S.A. reported updated data from clinical studies investigating iadademstat, the Company’s selective LSD1 inhibitor for onco-hematology indications. The results, recently presented at the 67th American Society of Hematology (ASH) Annual Meeting, highlight encouraging efficacy and safety findings from two ongoing studies evaluating iadademstat in combination with standard-of-care regimens in patients with acute myeloid leukemia (AML). ALICE-2 (NCT06357182), a Phase Ib investigator-initiated study sponsored by Oregon Health & Science University (OHSU) in newly diagnosed AML, evaluates treatment with iadademstat in combination with azacitidine and venetoclax, the standard of care in this setting for older or unfit patients. The study’s primary endpoint is the incidence of dose-limiting toxicities (DLTs). Secondary endpoints include efficacy measurements such as composite complete remission (CCR: complete remission [CR] + CR with partial hematologic recovery [CRh] + CR with incomplete recovery [CRi]), and overall response rate (ORR: CCR + morphologic leukemia free state [MLFS] + partial remission [PR]). Data for 10 patients are reported in the ASH publication. Treatment with iadademstat in combination with azacitidine and venetoclax was safe and well tolerated, with an AE profile similar to other combination treatments in newly diagnosed AML setting. Dose-finding for maximum tolerated dose (MTD) determination is ongoing. The combination treatment resulted in a highly encouraging ORR of 100% and a CCR rate of 90%, with 80% of patients attaining a strict CR. 70% of patients transitioned to allogeneic hematopoietic stem cell transplantation (HSCT). Median overall survival (OS) was not reached, and 6-month OS was 66%. The trial continues to enroll patients at dose level 2 (DL2), with a planned accrual of N=21 MTD-evaluable patients. FRIDA (NCT05546580), a Phase Ib clinical study sponsored by Oryzon, was designed to investigate iadademstat in combination with gilteritinib for the treatment of FLT3-mutant relapsed/refractory AML. The primary endpoints are incidence of treatment emergent adverse events (TEAEs) and determination of the recommended Phase II dose (RP2D). Secondary endpoints include response rates (CR, CRh, CRi, MLFS, CCR), event-free survival (EFS), and overall survival (OS). The ASH communication reports data for 37 patients, with 4 dose level cohorts evaluated in the escalation phase. All doses tested in the escalation phase were safe per DLT criteria. The study is in the expansion phase at one selected pharmacologically active dose, with a total of 17 patients enrolled in the study at this dose level per the ASH poster data cut-off. This dose continues to be well tolerated based on continuous safety monitoring, and has achieved the deepest responses which correlate with the target PK and PD values. Preliminary activity at the dose under expansion shows a 67% CCR (10/15 patients) and a 47% CR+CRh (7/15) in 15 patients evaluable for response, which favorably compares with the results of the ADMIRAL trial (CR+CRh 34%), particularly in light of contemporary practice with many patients (47%) treated at this dose after failing venetoclax, a population with markedly decreased response to gilteritinib monotherapy. Four patients have undergone HSCT. 
