ORYZON Starts Preclinical Collaboration on Autism with the Seaver Autism Center at Mount Sinai
MADRID, Spain and CAMBRIDGE, Mass., April 06, 2021 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with a strong unmet medical need, announced today the start of a preclinical collaboration on autism with researchers from the Seaver Autism Center for Research and Treatment at the Icahn School of Medicine at Mount Sinai led by Dr. Joseph Buxbaum.
Deletions or mutations at the end of chromosome 22 lead to a defect of the SHANK3 gene and produce in humans a variety of autism known as Phelan-McDermid Syndrome (PMS). Eighty percent of people with PMS have an autism spectrum disorder (ASD). It is thought that the inability of the single functioning copy of SHANK3 to produce enough Shank3 protein for normal functioning (haploinsufficiency) may be responsible for most of the neurologic symptoms (developmental delay, autism, and absent speech) associated with this disorder. Recent work published by US researchers has shown that the LSD1-HDAC2 complex is involved in PMS and that, in animal models defective for Shank3 which recapitulate many symptoms of the human syndrome, the inhibition of LSD1 restores neuronal electrophysiology and rescues learning deficits. This collaboration will explore the effects of vafidemstat in animal models developed and characterized at the Seaver Autism Center by the team of Dr. Buxbaum.
Vafidemstat is a selective, orally active LSD1 inhibitor in Phase II clinical development that has shown a very good safety profile and has been shown to be effective at reducing agitation and aggression in clinical studies in patients with Alzheimer's disease, Borderline Personality Disorder (BPD), Attention-Deficit/Hyperactivity Disorder (ADHD) and ASD. Vafidemstat is currently being explored in a Phase IIb clinical trial in BPD (PORTICO study) and the company is also preparing a Phase IIb trial in schizophrenia (EVOLUTION study). The company is also exploring the use of vafidemstat in the field of precision psychiatry.
Professor Buxbaum, Director of the Seaver Autism Center at the Icahn School of Medicine at Mount Sinai and principal investigator of the study, said: “We have worked on PMS for more than a decade and are very excited to advance this potential treatment for this disorder.”
Dr. Carlos Buesa, Oryzon’s CEO, said: "We are excited to initiate this collaboration with researchers at the internationally renowned Seaver Autism Center at Mount Sinai. Epigenetic dysregulation in the histone H3K4 methylation pathway has been proposed to be an important mechanism in the pathogenesis of autism. Oryzon’s vafidemstat with a good safety profile could bring a therapeutic option to these patients in the future.”