- Business
- Oryzon Genomics S.A. (ORY.MC) is a clinical-stage biopharmaceutical company and the European leader in epigenetics-based therapeutics, focusing on personalized medicine approaches for central nervous system (CNS) disorders and oncology; the company develops proprietary drug candidates through Phase II clinical trials before deciding on in-house advancement, partnering, or out-licensing for late-stage development and commercialization. Founded in 2000 and headquartered in Cornellà de Llobregat, Spain, with additional offices in Boston, Massachusetts, and San Diego, California, United States, Oryzon operates primarily in Europe and the United States, targeting patients with cancer, neurodegenerative diseases, psychiatric disorders, and rare neurodevelopmental conditions through its advanced pipeline of lysine-specific demethylase 1 (LSD1) inhibitors and other epigenetic targets. Its core products include vafidemstat (ORY-2001), a CNS-optimized LSD1 inhibitor in Phase III-ready development for agitation/aggression in borderline personality disorder (BPD; PORTICO-2 trial protocol submitted to FDA in 1H2025), negative symptoms in schizophrenia (EVOLUTION Phase IIb trial ongoing), and genetically defined subpopulations in autism spectrum disorder (ASD), Alzheimer's disease, and other neurodevelopmental syndromes; iadademstat (ORY-1001), a highly selective LSD1 inhibitor in Phase II for acute myeloid leukemia (AML; including combinations with azacitidine, gilteritinib, and venetoclax), small cell lung cancer (SCLC; combinations with platinum/etoposide and PD-1/PD-L1 inhibitors), neuroendocrine tumors, and expanding into non-oncological hematology such as sickle cell disease and essential thrombocythemia; ORY-3001, a selective LSD1 inhibitor in preclinical development for non-oncological diseases; and ORY-4001, a histone deacetylase 6 (HDAC-6) inhibitor clinical candidate nominated for Charcot-Marie-Tooth disease (CMT) and amyotrophic lateral sclerosis (ALS); the company also maintains a proprietary platform for biomarker identification and target validation in malignant and neurological diseases.
Among recent developments, Oryzon secures a 13.26 million euro non-refundable grant (approximately 15 million USD) in May 2025 through the European IPCEI Med4Cure project for its VANDAM initiative, funding personalized medicine validation of vafidemstat in aggression subtypes of ASD (including Phelan-McDermid and Fragile-X syndromes) and iadademstat in rare neuroendocrine cancers and genetically driven hematological diseases through August 2026; the company raises over 60 million USD in non-dilutive and equity financing during the first half of 2025, including a 30 million euro capital increase via accelerated bookbuild in April 2025 to bolster its balance sheet, prepare a potential Nasdaq IPO, and expand neuroscience and oncology programs, while terminating a prior convertible bonds financing; Oryzon strengthens its intellectual property portfolio in September 2025 with Decisions to Grant from the Australian and European Patent Offices for iadademstat combinations with PD-1/PD-L1 inhibitors in cancer (valid to at least 2040) and vafidemstat in behavioral alterations like BPD, ASD, and schizophrenia-related social withdrawal (valid to at least 2038); it advances strategic collaborations, including a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI) for iadademstat in hematologic and solid tumors (with Phase I/II trials in SCLC and AML ongoing at sites like Memorial Sloan Kettering), and shifts toward a CNS-focused strategy by exploring partnerships for iadademstat while enhancing its value through investigator-initiated studies; additionally, Oryzon bolsters its clinical, regulatory, and strategic teams with late-stage development experts following FDA feedback on vafidemstat's PORTICO trial.