ORYZON reports financial results and corporate update for half year ended June 30th, 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 three months ended June 30, 2026, and provided a corporate update on recent developments.

“During the second quarter of 2026, Oryzon reported important clinical progress while strengthening our financial position,” said Dr. Carlos Buesa, Oryzon’s Chief Executive Officer. “At the European Hematology Association (EHA) conference, we presented updated results from the ALICE-2 and FRIDA trials that further support the potential of iadademstat in AML. In first-line AML, the triplet combination of iadademstat with azacitidine and venetoclax continues to demonstrate a highly competitive efficacy profile, including encouraging activity in patients with adverse genetic backgrounds, while maintaining a favorable and manageable safety profile. We are also encouraged by the proportion of patients who have been able to proceed to allogeneic hematopoietic cell transplantation, a potentially curative treatment option that may contribute to improved long-term outcomes. We believe the ALICE-2 results compare favorably with those reported for other emerging triplet regimens and further reinforce the differentiated positioning of iadademstat in this setting.”

“We expect to present final data from ALICE-2 and FRIDA by year-end,” Dr. Buesa continued. “Subject to confirmation of the current efficacy and safety findings in the final ALICE-2 dataset, we remain on track to engage with the FDA on the design of a potentially registrational study in first-line AML, with the objective of initiating the trial in 2027. We also see iadademstat as an increasingly diversified asset across oncology and hematology, having enrolled the first patient in the IDEAL Phase II trial in essential thrombocythemia.”

“We continue to advance vafidemstat toward a Phase III trial in aggression in borderline personality disorder (BPD), as well as a new Phase II trial in aggression in autism spectrum disorder (ASD), while enrollment progresses in the ongoing Phase IIb trial in schizophrenia”, Dr. Buesa added. “The recent grant of a U.S. patent significantly extends our IP protection for vafidemstat in BPD and strengthens our long-term development plans for the asset.”

“To support our pipeline through the clinical catalysts expected this year and beyond, we raised €12 million through a recent capital increase and entered into a financing agreement with the Social Impact Fund managed by COFIDES,” Dr. Buesa concluded. “Under the agreement, the Fund has committed to invest €25 million in future capital increases to support Oryzon's mental health programs, subject to certain conditions. These additional resources provide us with greater financial flexibility as we work to translate our innovative epigenetic therapies into clinical benefit for patients and long-term value for our stakeholders.”

Second Quarter and Recent Highlights

Iadademstat:

• Oryzon shared updated positive data from the ongoing ALICE-2 (NCT06357182) Phase Ib clinical trial of iadademstat in combination with azacitidine and venetoclax in patients with newly diagnosed AML at the EHA 2026 Congress. As reported, the iadademstat-azacitidine-venetoclax triplet combination demonstrated high levels of clinical activity and continued to exhibit a favorable safety profile. Among evaluable patients (n=18), a 100% (18/18) overall response rate (ORR), an 89% (16/18) composite complete remission (CRc) rate and a 78% (14/18) complete response (CR) rate were observed. CRs occurred early, most of them in cycle 1. Efficacy was observed across different genomic risk groups, including TP53 and RAS pathway mutations and patients with complex karyotypes, all considered adverse risk: patients with TP53-mutated disease (2/2) attained CR and showed a reduction in TP53 variant allele frequency (14% to undetected and 22% to 1%, respectively), and patients with RAS pathway mutations (3/3) achieved CR. After a median follow-up of 8 months, median overall survival (OS) and event-free survival (EFS) were not reached; estimated 12-month OS and EFS were 79% and 71%, respectively. Additionally, 9 patients successfully transitioned to allogeneic HCT, with an estimated 12-month OS of 88%. This investigator-initiated study (IIS) is led by the Oregon Health & Science University (OHSU) Knight Cancer Institute and continues to actively enroll patients. The trial aims to enroll 21 evaluable patients; the 18 evaluable patients reported at EHA represent around 85% of the target enrollment.
• Updated positive data from the fully enrolled Phase Ib FRIDA (NCT05546580) clinical trial of iadademstat in combination with gilteritinib in patients with relapsed or refractory (R/R) FLT3-mutated AML were also presented at EHA 2026. Updated data from the expansion cohort at the selected pharmacological active dose (PAD, 75 μg iadademstat) included 18 patients evaluable for response. The iadademstat+gilteritinib combination showed a favorable safety profile and a CRc rate of 67% (12/18) 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.
• 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 extensive-disease small cell lung cancer (ED-SCLC), as well as investigator-initiated studies in myelodysplastic syndrome and ED-SCLC.
• Oryzon has initiated and enrolled the first patient in the IDEAL Phase II trial to evaluate iadademstat in adult patients with essential thrombocythemia (ET) who are resistant/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.
• 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.

Vafidemstat:

• Oryzon continues active regulatory and development activities to support the advancement of the Phase III PORTICO-2 trial with vafidemstat in aggression in BPD. Following receipt of written FDA feedback regarding study endpoints and certain non-clinical considerations, the Company is actively generating 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.
• Patient enrollment is ongoing in the EVOLUTION Phase IIb trial of vafidemstat in schizophrenia. The study is primarily assessing its effects on negative symptoms, with cognitive impairment and positive symptoms included as secondary endpoints. Initially launched in Spain, EVOLUTION is being extended to additional European countries (Bulgaria, Poland, Romania and Slovakia).
• Oryzon is finalizing preparations for the HOPE-2 Phase II study of vafidemstat for the treatment of aggression in autism spectrum disorder (ASD). The trial will focus on genetically-defined ASD subpopulations, in particular individuals with Phelan-McDermid syndrome (PMS). The study will initially be conducted in Spain and forms part of the activities supported by the Med4Cure IPCEI EU initiative.
• Oryzon continues to reinforce its IP portfolio for vafidemstat, as the United States Patent and Trademark Office (USPTO) recently granted U.S. Patent No. 12,673,044, entitled “Methods of treating borderline personality disorder”. The granted claims cover methods of treating non-aggressive symptoms of borderline personality disorder (BPD) using LSD1 inhibitors, including vafidemstat, complementing Oryzon’s patent portfolio covering the treatment of aggression. The patent is expected to expire in March 2043, including 1,095 days of Patent Term Adjustment (PTA) awarded by the USPTO to compensate for delays during patent prosecution. This estimate does not include any potential Patent Term Extension (PTE) that may become available following regulatory review, if applicable. A corresponding patent application has also been allowed in Canada.

Earlier stage programs:

• ORY-4001, Oryzon’s highly selective histone deacetylase 6 (HDAC6) inhibitor for neurological disorders, continues IND-enabling studies to enable future clinical trials. The program remains focused on potential applications in Amyotrophic Lateral Sclerosis (ALS), Charcot-Marie-Tooth disease (CMT) and other neurological disorders.

Financial Update: First half 2026 Financial Results

Research and development (R&D) expenses were $6.3 million and 11.4 million for the quarter and six months ended June 30th, 2026, compared to $3.0 and $5.8 million for the quarter and six months ended June 30th, 2025.

General and administrative expenses were $1.3 and $2.8 million for the quarter and six months ended June 30th, 2026, compared to $1.4 and $2.7 million for the quarter and six months ended June 30th, 2025.

Net losses were $1.6 and $3.6 million for the quarter and six months ended June 30th, 2026, compared to $1.7 and $3.4 million for the quarter and six months ended June 30th, 2025. 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 $1.6 million (–$0.02 per share) for the six months ended June 30th, 2026, compared to a negative net result of $1.8 million (–$0.03 per share) for the six months ended June 30th, 2025.

Cash, cash equivalents, and marketable securities totaled $16.7 million (€14.7 million) as of June 30, 2026.

After quarter-end, Oryzon raised gross proceeds of €12.0 million ($13.7 million) through a capital increase, without the issuance of warrants, as announced on July 1, 2026.

On the same date, the Company announced that it had entered into a share subscription agreement with the Social Impact Fund, managed by Compañía Española de Financiación del Desarrollo (COFIDES), an entity attached to the Spanish Ministry of Inclusion, Social Security and Migration. Under the agreement, the Social Impact Fund has committed, as an anchor investor, to subscribe for newly issued Oryzon shares in a future financing for an aggregate investment of €25 million, subject to the satisfaction of certain corporate, financial, business, and social impact-related conditions.

For more information, please see the attached press release.