Core Insights - Oryzon Genomics is hosting a virtual KOL event to discuss the unmet medical needs in Borderline Personality Disorder (BPD) and the design of vafidemstat's Phase III PORTICO-2 trial [1] - The event will address the lack of approved pharmacological treatments for BPD and the limitations of off-label medications, emphasizing the importance of addressing agitation and aggression in BPD and other psychiatric conditions [1] - Vafidemstat is highlighted as a promising treatment option due to its novel epigenetic mechanism of action, with ongoing discussions about its potential applications in BPD, schizophrenia, and autism spectrum disorder (ASD) [1] Company Overview - Oryzon Genomics, founded in 2000 in Barcelona, Spain, is a clinical-stage biopharmaceutical company specializing in epigenetics and personalized medicine for CNS disorders and oncology [9] - The company has a clinical portfolio that includes two LSD1 inhibitors: vafidemstat (Phase III-ready) and iadademstat (Phase II) [9] - Oryzon is also focused on biomarker identification and target validation for various malignant and neurological diseases [9] Vafidemstat Details - Vafidemstat (ORY-2001) is an oral, CNS-optimized LSD1 inhibitor that has shown efficacy in reducing cognitive impairment and neuroinflammation, as well as having neuroprotective effects [10] - The drug has demonstrated positive results in multiple Phase IIa clinical trials, including studies on aggression in psychiatric disorders and Alzheimer's disease [10] - Vafidemstat is advancing towards a Phase III trial in BPD following the completion of the Phase IIb PORTICO trial, with plans to investigate its effects on agitation and aggression [10]

Core Insights - Oryzon Genomics is participating in the 2025 Phelan-McDermid Syndrome Congress, highlighting its commitment to addressing rare genetic disorders through its research and development efforts [1][2] Company Participation and Events - Oryzon is sponsoring the CureSHANK's 2nd Annual Phelan-McDermid Syndrome Drug Development Symposium and the III Scientific Conference of the Spanish Phelan-McDermid Syndrome Association, which will take place from June 26 to June 29, 2025 [2] - Dr. Jordi Xaus, Oryzon's Chief Scientific Officer, will engage in a panel discussion on industry perspectives related to clinical trials and business considerations [1][7] Research and Development Focus - Oryzon is exploring the potential of vafidemstat, an LSD1 inhibitor, for treating rare monogenic psychiatric disorders, including Phelan-McDermid Syndrome (PMS) and Autism Spectrum Disorder (ASD) [3][5] - In the Phase IIb PORTICO trial for Borderline Personality Disorder (BPD), vafidemstat showed nominal statistical significance in reducing agitation and aggression, which are also key symptoms in PMS [3][7] Collaborations and Publications - Oryzon has collaborated with the Medical and Molecular Genetics Institute and the Research Institute La Paz Hospital to publish findings on the clinical characterization of PMS, which will support future clinical trials with vafidemstat [4] - The company has received €13.5 million from the EU IPCEI Med4Cure project to validate epigenetic drugs like vafidemstat through a personalized medicine approach for rare diseases [5] Product Pipeline and Clinical Trials - Vafidemstat is currently the only LSD1 inhibitor in clinical development for central nervous system (CNS) disorders, with ongoing trials in various psychiatric conditions, including BPD and schizophrenia [6][8] - The drug has demonstrated efficacy in reducing cognitive impairment and neuroinflammation in preclinical models, and it is advancing towards Phase III trials following positive results from earlier studies [8]

Core Insights - Oryzon Genomics, a clinical-stage biopharmaceutical company, is focused on epigenetics and personalized medicine, particularly in CNS disorders and oncology [3][4][5] Company Overview - Founded in 2000 in Barcelona, Spain, Oryzon is recognized as a European leader in epigenetics with a strong clinical portfolio [3] - The company has two LSD1 inhibitors: vafidemstat (Phase III-ready for CNS) and iadademstat (Phase II for oncology) [3] - Oryzon is also developing ORY-4001, targeting HDAC-6 for conditions like CMT and ALS, and has a robust platform for biomarker identification [3] Clinical Development - Iadademstat (ORY-1001) is a selective LSD1 inhibitor showing promising results in hematologic cancers, with ongoing trials in combination therapies for AML [4] - Vafidemstat (ORY-2001) is a CNS-optimized LSD1 inhibitor demonstrating efficacy in cognitive impairment and neuroinflammation, with positive results in various Phase IIa trials [5][6] Upcoming Events - Oryzon will participate in several international events, including Foro Medcap 2025 in Madrid, BIO International Convention 2025 in Boston, and Discovery and Development Europe 2025 in Basel, where they will engage with investors and present their research [2]

Core Insights - Oryzon Genomics has received a non-refundable grant of approximately €13.26 million (around $15 million) for its VANDAM project, which is part of the Med4Cure initiative aimed at supporting pharmaceutical research and innovation in Europe [1][3][4] - The grant covers 64% of the total budget for the VANDAM project, which is €20.68 million [1][4] - The VANDAM project focuses on developing therapies for rare neurodevelopmental disorders and neuroendocrine tumors, with a personalized medicine approach [2][3] Company Overview - Oryzon Genomics is a clinical-stage biopharmaceutical company based in Barcelona, Spain, specializing in epigenetics and personalized medicine for CNS disorders and oncology [5] - The company has two main drug candidates: vafidemstat, which is in Phase III readiness for CNS disorders, and iadademstat, currently in Phase II for oncology [5][6] - Oryzon's team consists of experienced professionals from the pharmaceutical industry, with operations in Barcelona, Boston, and San Diego [5] Project Details - The VANDAM project, which stands for "Validation of epigenetic Agents for Neuro-related rare Diseases Applying a personalized Medicine approach," is a 44-month initiative that began in January 2023 and will conclude in August 2026 [2][3] - The project aims to improve the quality of life for patients with rare diseases by developing targeted therapies based on a deeper understanding of the molecular causes of these conditions [2][3] Strategic Importance - The grant is considered a key component of Oryzon's clinical strategy, enabling the acceleration of clinical development for vafidemstat and further exploration of its therapeutic potential in managing aggression in rare diseases, including specific subtypes of Autism Spectrum Disorder (ASD) [2][4] - The funding will also support the investigation of iadademstat in rare tumors and genetically driven hematological diseases, areas with significant unmet medical needs [2][4] Collaborative Framework - Med4Cure is a pan-European project involving 14 scientific initiatives developed by 13 companies across six EU Member States, coordinated in Spain by the Centro para el Desarrollo Tecnológico y la Innovación (CDTI) [3] - Oryzon participates as an Associated Partner within this consortium, which is integrated into Spain's national Recovery and Resilience Plan [3]