- Updated positive data from iadademstat in 1L AML unfit patients to be presented at EHA, with 100% ORR, 93% CRc and 79% strict CR at abstract submission cut-off
- Positive updated data from iadademstat in relapsed/refractory FLT3-mut AML also accepted at EHA, showing 67% CRc
- Ongoing broad expansion of iadademstat clinical development across hematological malignancies, solid tumors and non-malignant hematology
- Active preparations underway for FDA protocol resubmission of the PORTICO-2 Phase III trial with vafidemstat in aggression in BPD
- Continued advancement of vafidemstat programs in schizophrenia and autism spectrum disorder
- Continued strengthening of IP protection for iadademstat and vafidemstat
- Strong cash position at the quarter ended March 2026: $25.4 million (€22.1 million)
MADRID and CAMBRIDGE, Mass., May 14, 2026 (GLOBE NEWSWIRE) — Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today reported financial results for the three months ended March 31, 2026, and provided a corporate update on recent developments.
“During the first months of 2026, Oryzon has continued to execute strongly across both its oncology/hematology and CNS franchises, while maintaining a solid financial position,” said Dr. Carlos Buesa, Oryzon’s Chief Executive Officer. “Most importantly, iadademstat continues to deliver highly encouraging clinical data in acute myeloid leukemia, further reinforcing our conviction in the compound’s differentiated therapeutic potential and commercial opportunity.”
“We are particularly excited about the upcoming presentations at EHA 2026, where updated safety and efficacy results from both the ALICE-2 and FRIDA studies will be presented,” Dr. Buesa added. “In first-line AML, the triplet combination of iadademstat with azacitidine and venetoclax continues to demonstrate a highly competitive efficacy profile, which we believe compares very favorably with other emerging triplet regimens in the field. At EHA, we will present updated data from 18 patients, including encouraging activity in patients with adverse genetic backgrounds. Based on the progress achieved to date, we believe iadademstat-based combinations may offer a differentiated and competitive opportunity for accelerated clinical development in first-line AML and could support advancement into a potentially pivotal Phase II/III program with the objective of pursuing accelerated regulatory approval.”
“The iadademstat development platform continues to expand, reinforcing its potential as a high-value hematology and oncology asset,” Dr. Buesa continued. “Enrollment is advancing across multiple investigator-sponsored studies at leading U.S. cancer centers and National Cancer Institute-sponsored trials in hematologic malignancies and solid tumors. At the same time, emerging opportunities in non-malignant hematology, including sickle cell disease and essential thrombocythemia, could materially enlarge the commercial and strategic scope of the franchise. Taken together, we believe iadademstat will deliver increasingly diversified clinical catalysts and generate sustained data, supporting a strong cadence of newsflow over the coming quarters.”
“In CNS, we remain fully committed to advancing vafidemstat toward late-stage development,” continued Dr. Buesa. “We continue to work closely on the activities required to support the Phase III PORTICO-2 protocol resubmission following FDA feedback, while also advancing the EVOLUTION schizophrenia study and preparations for the new HOPE-2 study in autism spectrum disorder. We believe vafidemstat continues to represent an important long-term value driver for Oryzon.”
First Quarter and Recent Highlights
Iadademstat:
- Oryzon announced that updated positive data from the ongoing ALICE-2 Phase Ib clinical trial of iadademstat in combination with venetoclax and azacitidine in patients with newly diagnosed acute myeloid leukemia (AML) have been accepted for presentation at the European Hematology Association (EHA) 2026 Congress. As of the February 2026 data cutoff reported in the published abstract, the triplet combination continued to demonstrate favorable safety and high response rates. Among evaluable patients (n=14/15), the overall response rate (ORR) was 100% (14/14), with a complete response (CR) rate of 79% (11/14) and a composite complete remission rate (CRc: CR+CRh+CRi) of 93% (13/14). After a median follow-up of 6 months, the estimated 12-month overall survival rate was 74%. Updated data with additional patients and more mature responses will be presented at EHA in June 2026. This investigator-initiated study (IIS) is led by the Oregon Health & Science University (OHSU) Knight Cancer Institute and plans to enroll up to 24 patients to attain 21 evaluable patients. The trial continues to actively enroll patients.
- Updated positive data from the ongoing, fully enrolled Phase Ib FRIDA clinical trial of iadademstat in combination with gilteritinib in patients with relapsed or refractory (R/R) FLT3-mutated AML have also been accepted for presentation at EHA 2026. Updated data from the expansion cohort showed a favorable safety profile and a CRc rate of 67% (12/18 patients evaluable for response harboring canonical FLT3 mutations) in a heavily pre-treated patient population. These results compare favorably with gilteritinib monotherapy responses in contemporary real‑world cohorts enriched for heavily pre‑treated patients, which are reported to be 28% CR+CRi. Additional data from the study will be presented during the congress.
- A new Phase Ib trial of iadademstat in combination with an immune checkpoint inhibitor and radiotherapy in extensive-stage small cell lung cancer (ES-SCLC), sponsored by Yale University, has initiated patient enrollment. The study evaluates iadademstat in combination with atezolizumab and stereotactic body radiation therapy (SBRT), followed by maintenance therapy with atezolizumab and iadademstat, in patients with residual, progressive or recurrent ES-SCLC who previously received platinum-based chemotherapy with or without immune checkpoint inhibitor therapy.
- Enrollment has continued across additional ongoing iadademstat clinical studies, conducted under the Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI) in first line AML, myeloproliferative neoplasms and small cell lung cancer, as well as an investigator-initiated study in myelodysplastic syndrome.
- Oryzon continues to advance the RESTORE Phase Ib trial of iadademstat in adult patients with sickle cell disease (SCD). The study will evaluate the safety and tolerability of iadademstat, establish the Recommended Phase II dose (RP2D), and investigate iadademstat’s effect on inducing fetal hemoglobin (HbF) expression, a clinically meaningful endpoint in SCD. The trial is actively enrolling patients, and the Company expects initial clinical updates by year-end.
- Oryzon has received regulatory authorization from the European Medicines Agency (EMA) to initiate the IDEAL Phase II trial to evaluate iadademstat in adult patients with essential thrombocythemia (ET) who are resistant/intolerant to hydroxyurea. Site activation activities and study start-up preparations are ongoing.
- Oryzon has continued to strengthen the intellectual property position of iadademstat. The United States Patent and Trademark Office (USPTO) recently granted a patent covering methods for treating neoplastic diseases using combinations comprising iadademstat and other therapeutic agents, notably venetoclax. The patent is expected to remain in force until January 2039, including 681 days of patent term adjustment (PTA), excluding any potential patent term extension related to regulatory review. Patents covering combinations of iadademstat with venetoclax have also been granted in Australia, Brazil, Canada, Europe, India, Israel, Japan, Korea, Malaysia, Mexico, New Zealand, and Russia. In addition, Oryzon recently received a “Decision to grant” communication from the Mexican Patent Office covering combinations of iadademstat with PD-1 or PD-L1 inhibitors for cancer therapy. Once formally granted, such patent is expected to provide protection until at least 2040, excluding potential patent term extensions. Corresponding patents have already been granted or allowed in Australia, Europe, Japan, and Russia.
Vafidemstat:
- Oryzon continues active regulatory and development activities to support the advancement of the Phase III PORTICO-2 trial with vafidemstat in aggression in borderline personality disorder (BPD). Following receipt of written FDA feedback regarding study endpoints and certain non-clinical considerations, the Company is actively working on the generation of additional supporting information and protocol refinements required for resubmission. These activities include qualitative research and endpoint-validation work intended to further support the proposed clinical outcome measures.
- To further enhance its CNS clinical development capabilities, Oryzon appointed Rolando Gutierrez-Esteinou, M.D., as Chief Medical Officer for CNS programs. Dr. Gutierrez-Esteinou is a Harvard-trained psychiatrist and senior clinical development executive with extensive experience in late-stage neuroscience programs and regulatory interactions in psychiatry indications.
- Enrollment continues in the EVOLUTION Phase IIb clinical trial evaluating vafidemstat in schizophrenia, focused primarily on negative symptoms, with secondary endpoints assessing effects on cognitive impairment and positive symptoms. The study, initially conducted in Spain, continues its expansion into additional European countries (Bulgaria, Poland, Romania and Slovakia).
- Oryzon is completing preparations for the HOPE-2 Phase II trial to evaluate vafidemstat in aggression in autism spectrum disorder (ASD). The trial …