Vafidemstat’s Remarkable Safety Record in Phase IIb PORTICO Trial for Borderline Personality Disorder

ORYZON Genomics, S.A., a clinical-stage biopharmaceutical company using epigenetics to develop effective therapies for diseases with a major unmet medical need, today released preliminary aggregate safety data from its ongoing Phase IIb PORTICO trial. This trial is assessing the efficacy of vafidemstat as a potential treatment for Borderline Personality Disorder (BPD). These results are a crucial step towards potential approval and a potential life-changing therapy for the millions affected by this disorder.

At a recent independent Data Monitoring Committee (DMC) meeting, blinded aggregate safety data collected from 167 randomized patients was reviewed up to data cut-off of 23 May 2023. There were no treatment-related serious adverse events or deaths, and 306 adverse events in total were reported, with most being mild or moderate in severity.

Severe adverse events were only reported in 9 patients, leading to 6 treatment discontinuations or patient withdrawals. These findings provided additional support for vafidemstat’s safety and well-tolerance and the DMC recommended continuing the trial until full enrollment, expected in early Q3 2023. The total number of patients treated with vafidemstat across all seven completed clinical trials now stands at almost 400, suggesting that this treatment for is safe and well-tolerated.

A predefined independent interim analysis (IA) to assess the signal size and futility was done on March 23, 2023 with the data of the first 90 patients that had concluded at least two-thirds of the trial. The outcome of this IA was that PORTICO was not futile and should continue as it is without increasing the number of patients to be recruited.

PORTICO is an exciting clinical trial that promises to advance the understanding and treatment of bipolar disorder in adults! This multicenter, double-blind, randomized Phase IIb trial tests the efficacy and safety of the investigational drug vafidemstat for reducing agitation and aggression as well as overall improvement in BPD severity. Patients will be enrolled in the US and Europe, and the adaptive design of the trial comes with a pre-defined interim analysis to make sure sample sizes are adjusted according to the endpoints. This is a great opportunity for those looking to contribute to the betterment of bipolar treatment.

About Oryzon

Oryzon, established in 2000 in Barcelona, Spain, is a pioneering biopharmaceutical company in the field of epigenetics – leading the way in breakthroughs in personalized medicine, particularly in CNS disorders and oncology. With teams located in Europe and America, Oryzon’s advanced clinical portfolio includes two LSD1 inhibitors – vafidemstat and iadademstat – currently undergoing Phase II clinical trials. However, that’s only the beginning as the company is also engaged in a host of other therapeutic endeavors and biomarker validation projects in neurological and malignant diseases. Oryzon is creating life-changing treatments and transforming use of personalized medicine – join us in making a monumental contribution to society!

About Vafidemstat

Vafidemstat (ORY-2001) is a remarkable oral, CNS-optimized LSD1 inhibitor that demonstrates an impressive range of effects. In various animal studies, this powerful molecule quickly and effectively restores memory, reduces the exaggerated aggression of SAMP8 mice, aids in social avoidance, and enhances sociability. Its capacity goes even further with powerful and durable efficacy in multiple sclerosis models. Unforunately, the effects of cognitive impairment, including memory loss and neuroinflammation, can be devastating.

But don’t worry – clinical trials were conducted to test vafidemstat’s impact on psychiatric disorders, Alzheimer’s, and even severe Covid-19. As a result, promising clinical results- including reduced inflammatory biomarker YKL40- have been reported across the board. Vafidemstat is currently in Phase IIb trials in particular borderline personality disorder and schizophrenia, and Oryzon is discovering its potential in other neurodevelopmental disorders. Thanks to vafidemstat, major leaps in reversing cognitive decline may be right around the corner.


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