Oryzon Genomics, S.A. reported new preliminary blinded aggregate safety data from its ongoing Phase IIb PORTICO trial, investigating vafidemstat in Borderline personality Disorder (BPD). On June 26, 2023, blinded aggregate safety data were reviewed during the recent independent DMC meeting, corresponding to the initial 167 randomized patients (data cut-off, 23 May 2023). From the blinded data, there were no treatment-related serious adverse events or deaths.

An aggregated number of 306 adverse events, affecting 98 patients treated either with vafidemstat or placebo were reported, most of them were mild (216) or moderate (78), with only 12 reported as severe, in 9 patients, leading to 6 treatment discontinuations or patient withdrawals. The reviewed blinded PORTICO safety data is aligned with aggregated safety data collected from 7 completed vafidemstat clinical trials, in which almost 400 subjects have been treated with the drug. Following the blinded portion of the DMC, the independent DMC members reviewed the unblinded safety data, then provided the recommendation to continue the trial without modifications until full enrollment, which is planned for early Third Quarter 2023.

Current data of PORTICO continue to support that vafidemstat 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 (EudraCT No.: 2020-003469-20, ClinicalTrials.gov Identifier NCT04932291) is a multicenter, double-blind, randomized, placebo-controlled, Phase IIb trial to evaluate the efficacy and safety of vafidemstat in adult BPD patients. The trial has two primary independent objectives: to reduce agitation and aggression and to produce an overall improvement in BPD severity. The trial is currently actively recruiting patients in Europe and in the US and aims to include 188 patients distributed between two arms. PORTICO has an adaptive design with a pre-defined interim analysis to adjust the sample size in case of excessive variability around the endpoints.