ORYZON receives the INNOVATIVE SME seal from the Spanish Ministry of Science and Innovation

La compañía biofarmacéutica ya ha sido inscrita en el Registro de Público de PYMES INNOVADORAS. 

Access to innovation

Oryzon Genomics S.A., (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announces that it has obtained the INNOVATIVE SME seal from the Spanish Ministry of Science and Innovation, and has consequently been registered in the Public Registry of INNOVATIVE SMEs. 

By awarding this distinction, which aims to reward and highlight the R&D activities of SMEs, the Ministry formally acknowledges the innovative and pioneering nature of Oryzon's studies and clinical advances in the field of epigenetics. This is the third consecutive time that Oryzon receives the INNOVATIVE SME. 

Oryzon is pioneering the development of LSD1 inhibitors in the field of psychiatric and neurodegenerative diseases. Vafidemstat is a Phase II LSD1 inhibitor, which has shown a good safety profile in clinical trials, with over 400 subjects treated, and has been shown to be effective in reducing agitation and aggression in Phase IIa clinical studies in patients with borderline personality disorder (BPD), attention deficit hyperactivity disorder (ADHD), autism spectrum disorder (ASD) and Alzheimer's disease. Vafidemstat is currently in clinical development in BPD, having recently completed the Phase IIb PORTICO trial, which has enrolled 210 patients in the US and Europe, and in schizophrenia, with the Phase IIb EVOLUTION trial, actively recruiting patients in Spain. The company presented positive and promising data from PORTICO on secondary endpoints reflecting clinically relevant improvements in overall BPD severity and agitation/aggressivity in the Top Line results presented at the JPMorgan-week in San Francisco and is currently finalizing in a satisfactory manner the analysis of the full study data. Based on the promising efficacy and safety results obtained in PORTICO, Oryzon is planning to request an end-of-Phase II meeting with the FDA to discuss plans for a registrational Phase III trial in BPD. The company plans to present final PORTICO data at a psychiatric conference later this year. 

Oryzon is also pioneering the field of CNS precision medicine, where it plans to explore vafidemstat as a potential treatment for certain diseases harboring mutations in certain genes that have been shown to be amenable to treatment with LSD1 inhibitors, such as Kabuki syndrome. PRESS RELEASE 2024 Pioneering Personalized Medicine in Epigenetics.

Currently, Oryzon has an ongoing clinical trial (FRIDA study) of iadademstat in combination with gilteritinib in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) harboring an FMStype tyrosine kinase mutation (FLT3mut+). Iadademstat is an orally active small molecule that acts as a potent and selective inhibitor of the epigenetic enzyme LSD1 (Lysine Specific Demethylase-1), and is the most potent compound among the LSD1 inhibitors in clinical development. The first cohort of the FRIDA study (six patients) has been completed, and the combination was safe and showed strong anti-leukemic activity. Recruitment of the second cohort (six patients) has been completed and is ongoing, and the company plans to present preliminary results from FRIDA at the next European Hematology Association congress, EHA-2024, in June in Madrid. 

Beyond AML, Oryzon is also developing iadademstat for the treatment of small cell lung cancer (SCLC). Iadademstat will be evaluated in a new clinical trial in preparation in combination with an immune checkpoint inhibitor (ICI) in patients with first-line metastatic SCLC, which will be conducted under a Cooperative Research and Development Agreement (CRADA) signed with the US National Cancer Institute (NCI) and led by Memorial Sloan Kettering Cancer Center (MSKCC). Iadademstat is also being evaluated in a collaborative Phase II clinical trial with the Fox Chase Cancer Center (FCCC) in combination with paclitaxel in platinum R/R SCLC and high-grade extrapulmonary neuroendocrine tumors.