The European Medicines Agency (EMA) has approved Oryzon Genomics’ clinical trial application (CTA) to begin the Phase II HOPE-2 study of vafidemstat in patients with Phelan-McDermid syndrome (PMS).
The single-arm, single-centre, open-label trial will evaluate the therapy in 12 adult patients in Spain.
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Primary endpoints focus on determining the safety and tolerability profile of vafidemstat over a 12-week treatment course, with participants eligible to extend therapy to 24 weeks based on investigator evaluation of clinical benefit.
Secondary endpoints will assess treatment effects on aggression and anger using the Aberrant Behaviour Checklist (ABC) irritability subscale, Clinical Global Impression of Severity (CGI-S), Repetitive Behaviour Scale-Revised (RBS-R), and the Phelan-McDermid Syndrome Assessment of Severity (PMSA-S).
PMS is described as a genetic neurodevelopmental disorder stemming from SHANK3 gene deletions or mutations, characterised by developmental delays, speech impairments, and autism spectrum disorder symptoms.
Oryzon is collaborating with the Spanish Phelan-McDermid Syndrome Association to assist with participant identification.
Oryzon CNS chief medical officer Rolando Gutierrez-Esteinou said: “The initiation of the HOPE-2 study bolsters our strategy to expand the applicability of vafidemstat in CNS indications.
“Vafidemstat is the only LSD1 inhibitor in clinical development for central nervous system disorders, with a potent and unique mechanism of action that modulates transcriptional programmes involved in neural plasticity, neuroinflammation and neuronal excitability.
“LSD1 inhibition has been shown to trigger a ‘reset’ of neuronal transcription and reverse social behaviour and aggression phenotypes in ASD [autism spectrum disorder] genetic models, including SHANK3-deficient mice. These preclinical findings and the clinical results observed in the REIMAGINE Phase IIa trial in ASD patients lead us to believe vafidemstat has unique potential to provide a promising treatment option for PMS.”
The HOPE-2 trial is being conducted under Oryzon’s VANDAM project, which forms part of the Med4Cure Important Project of Common European Interest on Health.
It is backed by NextGenerationEU funding through the Spanish Ministry of Science, Innovation and Universities and the Centre for the Development of Industrial Technology and Innovation.
