-- Positive clinical data presented at EHA for iadademstat in first line
acute myeloid leukemia (AML); iadademstat+azacitidine+venetoclax resulted
in 100% ORR, 89% CRc and 78% CR, including in patients with TP53 or RAS
mutations; the triplet continued to demonstrate a favorable safety
profile
-- In FLT3-mutated refractory-relapsed patients, iadademstat with
gilteritinib continued to show a favorable safety profile and a high CRc
rate of 67%
-- Enrollment initiated in Phase II trial of iadademstat in essential
thrombocythemia
-- U.S. patent granted for vafidemstat in borderline personality disorder
(BPD)
-- Cash position as of June 30, 2026 was $16.7 million (EUR14.7 million),
excluding the gross proceeds of EUR12.0 million ($13.7 million) raised in
the capital increase completed after quarter end
-- On July 1, 2026, the company entered into a financing agreement with
COFIDES with a commitment to invest EUR25 million in future capital
increases
MADRID and CAMBRIDGE, Mass., July 27, 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 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 EUR12 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 EUR25 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 <MU>g
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 30(th) , 2026, compared to $3.0 and $5.8 million for the quarter and six months ended June 30(th) , 2025.
General and administrative expenses were $1.3 and $2.8 million for the quarter and six months ended June 30(th) , 2026, compared to $1.4 and $2.7 million for the quarter and six months ended June 30(th) , 2025.
Net losses were $1.6 and $3.6 million for the quarter and six months ended June 30(th) , 2026, compared to $1.7 and $3.4 million for the quarter and six months ended June 30(th) , 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 30(th) , 2026, compared to a negative net result of $1.8 million (--$0.03 per share) for the six months ended June 30(th) , 2025.
Cash, cash equivalents, and marketable securities totaled $16.7 million (EUR14.7 million) as of June 30, 2026.
After quarter-end, Oryzon raised gross proceeds of EUR12.0 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 EUR25 million, subject to the satisfaction of certain corporate, financial, business, and social impact-related conditions.
ORYZON GENOMICS, S.A.
BALANCE SHEET DATA (AUDITED)(1)
(Amounts in thousands US $)
June 30th, June 30th,
2026 2025
---------- ----------
Cash and cash equivalents 16,729 36,465
Marketable securities 0 0
Total Assets 160,216 162,205
---------- ----------
Deferred revenue 0 0
---------- ----------
Total Stockholders' equity 136,389 133,927
---------- ----------
ORYZON GENOMICS, S.A.
STATEMENTS OF OPERATIONS (AUDITED)(1)
(US $, amounts in thousands except per share data)
Three Months Ended Six Months Ended
June 30th June 30th
----------------------- ----------------------
2026 2025 2026 2025
----------- ---------- ---------- ----------
Collaboration Revenue 0 0 0 0
Operating expenses:
Research and Development 6,319 2,962 11,443 5,760
General and
administrative 1,275 1,382 2,756 2,654
----------- ---------- ---------- ----------
Total operating expenses 7,594 4,344 14,199 8,414
----------- ---------- ---------- ----------
Loss from Operations -7,594 -4,344 -14,199 -8,414
----------- ---------- ---------- ----------
Other income, net 6,007 2,623 10,638 4,976
Net Loss -1,587 -1,721 -3,561 -3,438
=========== ========== ========== ==========
Net Financial & Tax 1,422 1,901 2,002 1,627
Net Result -165 180 -1,559 -1,811
=========== ========== ========== ==========
Loss per share allocable to common
stockholders:
Basic 0.00 0.00 -0.02 -0.03
----------- ---------- ---------- ----------
Weighted average Shares
outstanding
Basic 77,518,039 77,513,039 77,515,719 71,165,325
----------- ---------- ---------- ----------
(1) Spanish GAAP
* Exchange Euro/Dollar (1.1394 for
2026 and 1.1720 for 2025)
About Oryzon
Founded in 2000 and headquartered in Barcelona, Spain, Oryzon (ISIN: ES0167733015) is a clinical-stage biopharmaceutical company and a European leader in epigenetics, with a strong focus on personalized medicine for central nervous system $(CNS)$ disorders and oncology. Oryzon's team comprises highly experienced pharmaceutical professionals based in Barcelona, Boston, and New Jersey. The Company has an advanced clinical portfolio built around two LSD1 inhibitors: iadademstat, its oncology/hematology program, with several ongoing Phase I and II studies and which has demonstrated strong preliminary clinical activity in acute myeloid leukemia, including a 100% overall response rate (ORR) in first-line AML; and vafidemstat, its lead CNS program, which is Phase III--ready in borderline personality disorder (BPD). In addition, Oryzon is advancing a broader epigenetics pipeline targeting other mechanisms, including HDAC6, for which the Company has nominated ORY-4001 as a clinical candidate for potential development in Charcot--Marie--Tooth disease (CMT), amyotrophic lateral sclerosis (ALS), and other neurological disorders. The Company also operates a robust platform for biomarker identification and target validation across malignant and neurological diseases. For more information, visit www.oryzon.com
About Iadademstat
Iadademstat (ORY-1001) is an oral, highly selective inhibitor of the epigenetic enzyme LSD1, with 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 reported at EHA-2026: 100% overall response rate (ORR) and 89% composite complete remission rate (CRc), with 78% strict CR in 1L AML, and 67% CRc in R/R Flt3-mut AML. Additional studies in hematologic malignancies include an IIS in myelodysplastic syndrome (MDS) and 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 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 ongoing trials in sickle cell disease and essential thrombocythemia. Iadademstat has orphan drug designation for AML in the US and EU and for SCLC in the US.
About Vafidemstat
Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor with potential to address neuropsychiatric disorders through epigenetic modulation. In preclinical studies, vafidemstat has demonstrated effects on cognition, neuroinflammation, aggression, and social behavior, as well as neuroprotective and anti-inflammatory activity across multiple CNS disease models. Oryzon has completed several Phase II clinical trials with vafidemstat, including the REIMAGINE and REIMAGINE-AD trials in aggression in patients with different psychiatric disorders and in aggressive/agitated patients with moderate or severe AD, respectively, with positive clinical results reported in both trials. Following completion of the global randomized double-blind Phase IIb PORTICO trial in borderline personality disorder (BPD), vafidemstat is advancing as a Phase III-ready asset for aggression in BPD (PhIII in preparation). Vafidemstat is also being evaluated in the ongoing double-blind, randomized, placebo-controlled Phase IIb EVOLUTION trial in negative symptoms of schizophrenia. In addition, Oryzon is also deploying a CNS precision medicine approach with vafidemstat in genetically defined patient subpopulations of certain CNS disorders, as well as in neurodevelopmental syndromes,
including preparations for a new clinical trial in aggression in autistic conditions such as Phelan-McDermid syndrome.
FORWARD-LOOKING STATEMENTS
This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words "expects, " "anticipates," "believes," "intends," "estimates" and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any person acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward--looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation $(EU)$ 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration.
Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors. mcordera@atrevia.com com
(END) Dow Jones Newswires
July 27, 2026 14:23 ET