Biopharmaceutical company Oryzon Genomics, S.A., is collaborating with researchers from Columbia University in New York on a precision medicine therapy project focused on schizophrenia. The research will focus on a gene, SETD1A, linked to susceptibility for schizophrenia. The goal is to characterize the neuropsychiatric symptoms of people who have the SETD1A gene mutations.
The project seeks to build a foundation for a subsequent precision psychiatry clinical trial with vafidemstat, an LSD1 inhibitor developed by Oryzon for SETD1A-associated psychiatric disorders. The first stage will further characterize—at the molecular level—the therapeutic actionability of the SETD1A regulator protein encoded by this gene. During the second stage, the researchers will conduct an exhaustive functional psychometric characterization of people carrying mutations in the SETD1A gene.
Vafidemstat is currently in Phase II clinical development, and is being used in two Phase IIb clinical trials. It has previously been found effective for reducing agitation and aggression in clinical studies in participants with Alzheimer’s disease, borderline personality disorder (BPD), attention-deficit/hyperactivity disorder (ADHD) and autism. The current clinical trials are focused on BPD and on people with psychosis following infection with coronavirus disease 2019 (COVID-19). Vafidemstat is also being explored in studies of people with Phelan-McDermid syndrome – a variety of autism – who harbor a mutation in SHANK3.
In studies with mice carrying the SETD1A mutation, researchers with Columbia University found that the mutation created deficits in working memory and brain structure similar to the cognitive and behavioral deficits experienced by humans with schizophrenia. Administration of Oryzon’s LSD1 inhibitor vafidemstat produced a full rescue of both the behavioral and neuronal disease phenotypes. Because the treatment effects occurred in adult animals, the researchers believe that deficiency of SETD1A function during early developmental stages did not irreversibly compromise the function of disease-related neural circuits. They believe the finding opens the door for investigation of intervention with vafidemstat during a therapeutic window in adolescent and adult subjects.
Oryzon is based in Barcelona, Spain, with offices in the United States. It is a clinical stage biopharmaceutical company. Its LSD1 program has rendered two compounds, vafidemstat and iadademstat, in clinical trials. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets.
This was reported by Oryzon Genomics, S.A on December 17, 2020.
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