Vafidemstat Phase IIb PORTICO Trial Results in BPD Unveiled

Oryzon Genomics, a biopharmaceutical firm specializing in epigenetics, has reported findings from the Phase IIb PORTICO trial on the drug vafidemstat for the treatment of Borderline Personality Disorder (BPD). The study, set to be discussed at the 7th Sachs Annual Neuroscience Innovation Forum, indicated that while the primary goals were not met, certain secondary measures showed positive results.

The PORTICO trial, which involved 210 patients, aimed to evaluate the impact of vafidemstat on BPD severity and agitation/aggression. Although the main objectives did not achieve statistical significance, the secondary endpoint of Borderline Evaluation of Severity (BEST) demonstrated a notable reduction in disease severity from weeks 8 to 12. Similarly, the State-Trait Anger Expression Inventory 2 (STAXI-2) Trait Anger, which assesses agitation and aggression, showed significant improvement over the placebo group during the same period.

Vafidemstat proved to be safe and well-tolerated, aligning with its established safety profile. The number of participants experiencing treatment-emergent adverse events was slightly lower in the vafidemstat group compared to the placebo group. The majority of these events were mild or moderate in severity, and most resolved by the end of the trial.

Dr. Michael Ropacki, Oryzon's Chief Medical Officer, highlighted the significance of the findings, suggesting that they provide a clear direction for the future development of vafidemstat as a potential treatment for BPD. The company plans to engage with the FDA to discuss the results and the design of a Phase III study.

Dr. Douglas V. Faller, Oryzon's Global CMO, emphasized the uniqueness of vafidemstat as an LSD1 inhibitor in clinical development for CNS indications. He noted its potential in improving sociability and memory while restoring NMDR functionality, which is implicated in BPD.

CEO Dr. Carlos Buesa expressed gratitude to all involved in the trial and considered the results to be very positive, despite not meeting the primary endpoints. He pointed out the importance of the study in defining future clinical trials and establishing the necessary effect size for a well-powered Phase III trial.

The comprehensive data from the PORTICO trial will be presented at a psychiatric conference and published in a peer-reviewed journal. Oryzon, founded in 2000, is a leader in European epigenetics with a focus on personalized medicine in CNS disorders and oncology. Vafidemstat, an LSD1 inhibitor, is currently in Phase IIb trials for BPD and schizophrenia, and Oryzon is exploring its potential in other neurodevelopmental syndromes.

BPD is a debilitating psychiatric disorder affecting a significant portion of the population, characterized by emotional instability and impulsivity. Currently, there are no FDA-approved treatments specifically for BPD, making the development of effective therapies a critical unmet need in healthcare.