Strong cash position at year-end 2025: $33.3 million (€28.4 million) CNS (Vafidemstat)Appointed renowned Rolando Gutierrez-Esteinou, M.D. as new CMO for CNS Preparing protocol resubmission to incorporate FDA guidance on Phase III PORTICO-2 trial in aggression in BPD Ongoing expansion of Phase IIb schizophrenia trial into additional EU countries Finalizing preparations for new Phase II trial in aggression in autism spectrum disorder as a part of the IPCEI EU Grant in personalized medicine Oncology – Hematology (Iadademstat)Seven ongoing oncology trials, with six sponsored by the NCI or top-tier U.S. institutions Positive data in 1L AML unfit patients presented at ASH with 100% ORR (90% strict CR) Positive data in R/R FLT3+ AML presented at ASH; 67% CCR at the selected dose under expansion Initiated enrollment in new Phase Ib trial in small cell lung cancer in combination with ICI and radiotherapy Continued momentum in Phase Ib sickle cell disease trial with first two cohorts enrolled New Phase II trial in essential thrombocythemia approved by EMA
Appointed renowned Rolando Gutierrez-Esteinou, M.D. as new CMO for CNS Preparing protocol resubmission to incorporate FDA guidance on Phase III PORTICO-2 trial in aggression in BPD Ongoing expansion of Phase IIb schizophrenia trial into additional EU countries Finalizing preparations for new Phase II trial in aggression in autism spectrum disorder as a part of the IPCEI EU Grant in personalized medicine
Seven ongoing oncology trials, with six sponsored by the NCI or top-tier U.S. institutions Positive data in 1L AML unfit patients presented at ASH with 100% ORR (90% strict CR) Positive data in R/R FLT3+ AML presented at ASH; 67% CCR at the selected dose under expansion Initiated enrollment in new Phase Ib trial in small cell lung cancer in combination with ICI and radiotherapy Continued momentum in Phase Ib sickle cell disease trial with first two cohorts enrolled New Phase II trial in essential thrombocythemia approved by EMA
MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, February 27th, 2026 – 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 twelve months ended December 31, 2025 and provided a corporate update on recent developments.
“After securing over $60 million in the first half of 2025, marking a clear financial turnaround for Oryzon, we ended the year with a solid cash position of $33.3 million (€28.4 million),” said Dr. Carlos Buesa, Oryzon’s Chief Executive Officer. “This financial strength allows us to sharpen our focus on key regulatory catalysts across both of our programs: oncology and CNS.”
“In oncology-hematology, iadademstat continues to receive strong external validation, with seven ongoing trials, six of which are sponsored by the NCI or leading U.S. institutions, including the most recent study sponsored by Yale University,” Dr. Buesa added. “In first-line AML, the triple combination has shown a 100% overall response rate to date, with no dose-limiting toxicities, highlighting a highly competitive profile among emerging triplet regimens. The trial continues to enroll rapidly, and we plan to present data from 15-16 patients at the European Hematology Association Annual Congress (EHA) in June. This would represent approximately 75% of the planned enrollment, providing a very meaningful interim assessment of efficacy and safety. Encouraging results are also emerging in MDS and MPN.”
“In sickle cell disease, we are advancing iadademstat into what we believe represents a new paradigm for the pharmaceutical industry — medium-priced therapies addressing large patient populations. The first two cohorts of the RESTORE trial have been enrolled, and we expect to report meaningful data later this year.”
“In CNS, we have further strengthened our medical and regulatory capabilities with the appointment of renowned CMO Dr. Rolando Gutierrez, who brings extensive experience from the psychiatric field to lead vafidemstat through late-stage development and regulatory interactions, particularly with the U.S. Food and Drug Administration (FDA),” continued Dr. Buesa. “Discussions with the Agency are expected to continue in the coming months, and the Company anticipates a resubmission of the Phase III protocol before year-end, in line with standard regulatory timelines. At the same time, we continue to advance our programs in schizophrenia and ASD.”
Fourth Quarter and Recent Highlights
Iadademstat:
Vafidemstat:
Earlier stage programs:
Financial Update: Fourth quarter 2025 Financial Results
Research and development (R&D) expenses were $5.2 million and $14.8 million for the quarter and twelve months ended December 31, 2025, compared to $2.1 and $8.7 million for the quarter and twelve months ended December 31, 2024.
General and administrative expenses were $1.7 and $5.6 million for the quarter and twelve months ended December 31, 2025, compared to $0.9 and $3.7 million for the quarter and twelve months ended December 31, 2024.
Net losses were $2.1 and $6.7 million for the quarter and twelve months ended December 31, 2025, compared to net losses of $1.1 and $4.6 million for the quarter and twelve months ended December 31, 2024. The result is as expected, given the biotechnology business model where companies in the development phase typically have a long-term maturation period for products and do not have recurrent income.
Negative net result was $3.1 million (–$0.04 per share) for the twelve months ended December 31, 2025, compared to a negative net result of $3.7 million (–$0.06 per share) for the twelve months ended December 31, 2024.
Cash, cash equivalents, and marketable securities totaled $33.3 million as of December 31, 2025.