Oryzon Genomics announced the publication of final Phase IIa REIMAGINE study results, demonstrating the safety and efficacy of vafidemstat in reducing agitation and aggression in patients with borderline personality disorder (BPD), attention-deficit/hyperactivity disorder (ADHD), and autistic spectrum disorder (ASD). The study provided crucial data for advancing vafidemstat’s clinical development, specifically in BPD, leading to a Phase IIb trial and preparations for a Phase III trial. This research aimed to explore the potential of epigenetics, specifically LSD1 inhibition, in addressing unmet needs in psychiatry and neurodevelopmental disorders.
This development holds significant promise for patients struggling with agitation and aggression associated with these disorders. Current treatment options are often limited and may come with undesirable side effects. Vafidemstat offers a novel mechanism of action targeting LSD1, potentially providing a more effective and tolerable treatment alternative for these challenging symptoms. The positive findings in BPD particularly highlight the drug’s potential to significantly improve patients’ quality of life by addressing a core symptom that often disrupts interpersonal relationships and daily functioning.
The REIMAGINE study, a single-center, open-label basket trial, involved administering 1.2 mg/day of vafidemstat to adult patients for eight weeks. Results showed significant and consistent reductions in agitation and aggression across all three patient groups. Further, vafidemstat demonstrated improvements in non-aggressive symptoms and overall disease indicators. Building on these positive outcomes, Oryzon conducted the Phase IIb PORTICO trial in BPD, which further supported vafidemstat’s efficacy. Following a positive meeting with the FDA, Oryzon is now preparing for the Phase III PORTICO-2 trial. In parallel, vafidemstat is also being evaluated in a Phase IIb trial for negative symptoms of schizophrenia.
The positive results from the REIMAGINE and PORTICO studies coupled with FDA feedback suggest a promising future for vafidemstat. If the Phase III trial confirms its efficacy and safety profile, vafidemstat could become a valuable treatment option for managing agitation and aggression in BPD. Furthermore, its potential application in other CNS disorders like ADHD, ASD, and schizophrenia warrants continued investigation and could significantly broaden its impact on mental health care. This research also underscores the increasing importance of epigenetics in understanding and treating complex psychiatric conditions.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
