ORYZON to attend the 2025 Phelan-McDermid Syndrome (PMS) Congress in Barcelona on June 26-29

Oryzon Genomics S.A., Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a European leader in epigenetics, announced today that its Chief Scientific Officer, Dr. Jordi Xaus will be attending the 2025 Phelan-McDermid Syndrome Congress in Barcelona on June 26-29, which comprises the CureSHANK 2nd Annual Phelan-McDermid Syndrome Drug Development Symposium (PMSDDS2025) and the III Scientific Conference of the Spanish Phelan-McDermid Syndrome Association. Dr. Xaus will participate in a panel discussion within PMSDDS2025 entitled “Industry Perspectives Discussion”, as part of the morning session “Noncommercial & Commercial Clinical Trials and Business Considerations”, on June 27.

Oryzon participates as Sponsor of both the CureSHANK’s 2nd Annual Phelan-McDermid Syndrome Drug Development Symposium, aimed at the scientific community, which will be held June 26-27; and the III Scientific Conference of the Spanish Phelan-McDermid Syndrome Association, aimed at families, doctors, researchers and other healthcare professionals, which will be held June 28-29.

Oryzon is committed to exploring the potential of vafidemstat, its LSD1 inhibitor for CNS disorders, in rare monogenic psychiatric disorders such as PMS or other ASD-related indications. In the Phase IIb PORTICO trial in Borderline Personality Disorder (BPD), vafidemstat demonstrated nominal statistical significance in reducing agitation and aggression. Agitation and aggression are key components of PMS. In addition, vafidemstat has also demonstrated efficacy in reducing agitation and aggression in a proof-of-concept basket clinical trial involving patients with Autism Spectrum Disorder (ASD), Attention Deficit Hyperactivity Disorder (ADHD), and BPD. The final results of this trial were recently published in Psychiatry and Clinical Neurosciences (see here).

Moreover, in collaboration with the Medical and Molecular Genetics Institute (INGEMM) and the Research Institute La Paz Hospital (IdiPaz) in Madrid, Oryzon recently published in the journal Frontiers in Psychiatry the results of an observational clinical study conducted in PMS to gather relevant data related to phenotypical and clinical characterization of PMS subjects, to serve as a foundation for this future precision psychiatry clinical trial with vafidemstat in this patient population (see here).

Dr. Jordi Xaus, Oryzon’s CSO, stated, “Monogenic rare disorders linked to alterations in the epigenetic machinery and other key neuronal genes have been identified as potential targets for precision medicine. The use of LSD1 inhibitors has been shown to partially or fully rescue the complex phenotypes caused by these genetic mutations. Vafidemstat is currently the only LSD1 inhibitor in clinical development in CNS and represents a promising opportunity for these patients, a possibility that will be explored thanks to the funds that will be received from the EU IPCEI Med4Cure project”.

Oryzon has been awarded 13.5 million € through the Important Project of Common European Interest (IPCEI) Med4Cure project, a macro-project that deploys 14 scientific projects to be developed by 13 companies as Direct Partners and 11 as Associated Partners. The project to be developed by Oryzon is called VANDAM and aims to validate epigenetic drugs like vafidemstat by applying a personalized medicine approach for rare and orphan diseases.