Oryzon Genomics has presented its results for the fourth quarter and year-end 2025. The net result at the end of the fourth quarter of 2025 stands at -2.6 million euros compared to the fourth quarter of the previous year of -3.6 million, which represents a 1 million upgrade with respect to the closing of the fourth quarter of 2024.
Las investments in R&D increased by 50% as of December 31, 2025 to 12.6 million. Of this figure, 11.6 million correspond to development activities and 1 million to research activities.
At the end of December, the company had 28.4 million euros in cash and financial investments available, after having raised more than 60 million dollars in the first half of the year. He net worth stood at 117.8 million euross, 33.7% more year-on-year.
Oryzon breaks its trend and presents 2.6 million surplus (profit) in it statement of recognized income and expenses, by recognizing 5.2 million net subsidies of tax effectcompared to 3.1 million deficit (loss) in fiscal year 2024.
“After securing more than $60 million in the first half of 2025, marking a clear financial turning point for Oryzon, we closed the year with a solid cash position. 33.3 million dollars (28.4 million euros)«, commented the Dr. Carlos Buesa, CEO of Oryzon. “This solid financial position allows us to reinforce our focus on the main regulatory milestones of our two programs: oncology and CNS.”
Business evolution
Fourth quarter and other recent highlights
Iadademstat:
- Very encouraging preliminary data from the ongoing Phase Ib ALICE-2 clinical trial of iadademstat in combination with venetoclax and azacitidine in patients with newly diagnosed acute myeloid leukemia (AML) were presented at the American Society of Hematology (ASH) Annual Meeting in December 2025. The triple combination of iadademstat with venetoclax and azacitidine achieved an overall response rate (ORR) of 100% (n=10), with 90% of patients achieving strict complete remission (CR). 70% of patients underwent allogeneic hematopoietic stem cell transplantation (HSCT). Median overall survival (OS) was not reached and the 6-month OS rate was 66%. Treatment with iadademstat in combination with venetoclax and azacitidine was safe and well tolerated, with an adverse event profile similar to that of other combination therapies in the setting of newly diagnosed AML. The trial continues to enroll patients at dose level 2 (DL2). This investigator-initiated study (IIS) is led by the Knight Cancer Institute at Oregon Health & Science University (OHSU), and plans to recruit up to 24 patients to reach 21 evaluable patients. The company plans a new data update at the European Hematology Association (EHA) congress in June 2026.
- Positive preliminary results were also presented in ASH-2025 from the open-label, multicenter Phase Ib FRIDA clinical trial of iadademstat in combination with gilteritinib in patients with relapsed or refractory (R/R) AML with FLT3 mutation (FLT3mut+). The communication presented at ASH included data from 37 patients, with 4 dose level cohorts evaluated in the escalation phase. All doses evaluated in the escalation phase were safe according to dose-limiting toxicity (DLT) criteria. At the time of data cutoff, the study was in the expansion phase at a selected pharmacologically active dose, with a total of 17 patients enrolled at that dose level. Preliminary activity at the expanding dose showed a composite CR (CCR) rate of 67% (10/15 patients) and a CR+CRh rate of 47% (7/15) in 15 response-evaluable patients, which compares favorably with the results of the ADMIRAL trial (CR+CRh 34%), especially considering current clinical practice, in which many patients (47%) treated at this dose had previously failed venetoclax, a population with a markedly reduced response to gilteritinib monotherapy. Four patients underwent HSCT. The trial has completed recruitment and the company plans to present updated data at EHA-2026.
- Patient recruitment has begun in a new Phase Ib trial of iadademstat in combination with an immune checkpoint inhibitor and radiotherapy in extensive-stage small cell lung cancer (SCLC). The study, sponsored and conducted by Yale University, is a Phase Ib, open-label, non-randomized study that will evaluate the safety, tolerability, and efficacy of iadademstat in combination with atezolizumab and stereotactic body radiation therapy (SBRT), followed by maintenance treatment with atezolizumab and iadademstat in patients with residual, progressive, or recurrent EE-SCLC who have previously received platinum-based chemotherapy, with or without treatment with point inhibitors. immune control.
- Recruitment has also continued actively in additional ongoing trials with iadademstat, conducted under a Cooperative Research and Development Agreement (CRADA) with the US National Cancer Institute (NCI) in first-line AML, myeloproliferative neoplasms and SCLC, as well as an investigator-sponsored study in myelodysplastic syndrome.
- Beyond oncology, Oryzon has expanded clinical evaluation of iadademstat to non-malignant hematologic disorders, with a first trial in sickle cell anemia. This multicenter, open-label, Phase Ib trial, called RESTORE (REgulation of Sickling ThrOugh Reprogramming Epigenetics), will evaluate the safety and tolerability of iadademstat in adult patients with sickle cell anemia, and determine its recommended dose for Phase II (RP2D), in addition to evaluating the effect of iadademstat on the induction of fetal hemoglobin (HbF) expression. Increased HbF has already been recognized by the FDA as a clinically relevant endpoint for the treatment of sickle cell anemia. The trial is actively recruiting patients, with the first two cohorts already enrolled. The study is carried out in several centers in Spain and plans to include approximately 40 adult patients.
- Oryzon plans to initiate a clinical trial to evaluate iadademstat in essential thrombocythemia (ET) following the recent approval of the study by the European Medicines Agency (EMA). The study, called IDEAL (IaDademstat treatment for EssentiAL thrombocythemia), is a multicenter, open-label, single-arm Phase II trial that will be carried out in Spain in adult patients with ET who are resistant or intolerant to hydroxyurea. The primary objectives of the study are to evaluate the safety and tolerability of iadademstat, as well as to determine its effectiveness in reducing the percentage of adult patients with ET who have abnormal platelet counts. Secondary objectives include evaluation of the durable clinical hematologic response rate (DCHR), confirmation of the pharmacokinetic and pharmacodynamic profile of iadademstat in patients with ET, and evaluation of the duration of hematologic remissions (DHR).
- Oryzon has recently strengthened its patent portfolio for iadademstat, with a “Grant Decision” communication from the Japan Patent Office for its application titled “Combinations of iadademstat for cancer therapy,” regarding its use in combination with PD1 or PD-L1 inhibitors. Once formally granted, this patent will remain in force at least until 2040, not including possible extensions of the term of protection. Corresponding patents have already been granted or approved in Europe, Australia and Russia, and additional applications are pending in other countries.
Uncertainty:
- Oryzon continues to advance the Phase III PORTICO-2 trial with vafidemstat in aggression in Borderline Personality Disorder (BPD) after receiving written comments from the FDA, which included guidance on the study endpoints and certain non-clinical considerations. In preparation for submission of the revised protocol to the FDA and in line with the Agency’s recommendations, the Company is conducting various activities, including qualitative research to generate additional evidence on the conceptual validity and appropriateness of certain clinical scales proposed as endpoints.
- In order to continue strengthening its strategy and clinical execution, Oryzon has appointed Dr. Rolando Gutierrez-Esteinou as Medical Director for CNS programs. Dr. Gutierrez-Esteinou, a Harvard-trained psychiatrist and executive with extensive clinical development experience, has more than 20 years of leadership in drug development in neuroscience and psychiatry, including oversight of late-stage clinical trials and overall development strategy. Most recently, he served as Chief Medical Officer at Atai Life Sciences. He brings strong experience in conducting pivotal studies for innovative CNS therapies, reinforcing the Company’s medical leadership as it progresses vafidemstat into Phase III clinical development.
- Oryzon is completing preparations for a new Phase II trial to evaluate vafidemstat in the treatment of aggression in patients with autism spectrum disorder (ASD). This trial, called HOPE-2, plans to include genetically defined ASD subpopulations, such as individuals with Phelan-McDermid syndrome (PMS), and will initially be carried out in Spain as part of activities supported by the European IPCEI Med4Cure initiative.
- The Phase IIb EVOLUTION clinical trial with vafidemstat in patients with schizophrenia has continued to enroll patients. This study aims to evaluate the effectiveness of vafidemstat, mainly focused on the improvement of negative symptoms. As secondary objectives the trial will explore the effectiveness of vafidemstat in improving cognitive impairment and positive symptoms of schizophrenia. Initially conducted only in Spain, the study is now expanding to additional countries in the European Union.
- Oryzon has continued to strengthen its patent portfolio for vafidemstat, with a new “Grant Decision” communication from the Japan Patent Office. The approved claims cover the use of vafidemstat for the treatment of aggression and social withdrawal. Once formally granted, this patent will remain in force until at least 2038, not including possible extensions of the term of protection, which could provide additional years of protection. Additional patents in this family have already been granted or approved in Europe, Australia, Canada, Hong Kong, Israel, Korea, Malaysia, the Philippines and Russia, and applications are pending in other countries.
Programs in earlier phases:
ORY-4001, Oryzon’s highly selective histone deacetylase 6 (HDAC-6) inhibitor nominated as a candidate for clinical development for the treatment of certain neurological diseases such as Charcot Marie-Tooth disease (CMT), amyotrophic lateral sclerosis (ALS) and others, continues to advance in pre-regulatory IND studies that will prepare the compound for clinical studies.