Oryzon Genomics S.A.

Oryzon Genomics, S.A. announced that the European Medicines Agency has authorized its Clinical Trial Application to initiate a Phase II study of vafidemstat for the treatment of Phelan-McDermid Syndrome. The study, named HOPE-2, is a single-center, single-arm, open-label Phase IIa trial that will enroll 12 adult patients with PMS, a severely disabling genetic disorder related to autism with a U.S. prevalence estimated at approximately 1 in 7,300 people. The primary objective is to evaluate the safety and tolerability of vafidemstat, with secondary objectives assessing its effect on anger and aggression and overall disease efficacy; vafidemstat will be administered for 12 weeks, after which the investigator will assess whether participants may continue treatment through week 24 based on clinical benefit. The study will be conducted in Spain as part of Oryzon's VANDAM project, which is part of the Med4Cure Important Project of Common European Interest on Health and has received funding from the Spanish Ministry of Science, Innovation and Universities and the Centre for the Development of Industrial Technology and Innovation under the Recovery, Transformation and Resilience Plan funded by the European Union – NextGenerationEU. Oryzon will collaborate with the Spanish Phelan-McDermid Syndrome Association to support identification of potential participants.

Oryzon Genomics reported positive clinical data for iadademstat in acute myeloid leukemia and strengthened its financial position with a €25 million financing commitment. At the EHA 2026 Congress, the iadademstat-azacitidine-venetoclax triplet showed a 100% overall response rate, 89% composite complete remission rate, and 78% complete response rate in newly diagnosed AML patients, including those with TP53 or RAS mutations. In relapsed/refractory FLT3-mutated AML, iadademstat plus gilteritinib demonstrated a 67% composite complete remission rate. The company also initiated a Phase II trial of iadademstat in essential thrombocythemia and received a U.S. patent for vafidemstat in borderline personality disorder. Cash as of June 30, 2026 was $16.7 million, and after quarter-end Oryzon raised €12.0 million in a capital increase and entered a share subscription agreement with COFIDES for a €25 million future investment.