Oryzon Genomics has announced new aggregated positive human efficacy data on its central nervous system (CNS) epigenetic drug vafidemstat from three different cohorts of psychiatric patients in a Phase II REIMAGINE trial.
Vafidemstat (ORY-2001) is an oral, brain penetrant drug that inhibits LSD1 and MAOB by reducing cognitive impairment, including memory loss and neuroinflammation. It also has neuroprotective effects.
The findings revealed that vafidemstat reduced aggression and improved function across the three psychiatric disorders of attention deficit hyperactivity disorder (ADHD), autistic spectrum disorder (ASD) and borderline personality disorder (BPD).
The Spanish biotechnology company had enrolled 30 patients across the three cohorts and the findings support vafidemstat as an emerging therapeutic option in the treatment of aggression.
After two months of vafidemstat treatment, Oryzon Genomics found a significant reduction of aggression measured by the Neuropsychiatric Inventory (NPI) four-item agitation/aggression subscale for all subjects (p<0.0001) and in each of the three individual cohorts.
There has also been a significant reduction of aggression measured by the Clinical Global Impression (CGI) of Severity (CGI-S) and Improvement (CGI-I) scales in the aggregated data for all subjects (p<0.0001 for both) and in each cohort.
Furthermore, the company found significant global improvement on the NPI total score in the aggregated cohorts (p<0.0001) and in each cohort.
The findings also revealed a notable reduction in suicidal thoughts in the BPD cohort, as per C-SSRS scale (p=0.0033). However, efficacy was not examined for the C-SSRS in the ADHD and ASD cohorts.
The company said that vafidemstat was safe and well-tolerated without significant adverse events across all three cohorts, demonstrating statistically significant improvements across clinical outcome assessments to measure levels of aggression and agitation.
Oryzon Genomics said that it is now preparing for a Phase IIb in BPD (PORTICO) and evaluating additional Phase IIb studies in ADHD and/or ASD.
REIMAGINE is a Phase IIa trial that evaluates the safety, tolerability and efficacy of vafidemstat in aggression in adult patients with BPD, ADHD and ASD.
The single-arm, open-label, eight-week treatment study has been conducted at the Vall d’Hebrón hospital in Barcelona.
The results have been presented at the 2019 International College of Neuropsychopharmacology (CINP) meeting in Athens, Greece.
The company is conducting a parallel REIMAGINE study to evaluate the safety, tolerability and efficacy of vafidemstat in aggression in subjects with moderate-to-severe Alzheimer’s disease.