Oryzon Genomics, S.A. announced that the company presented an update on vafidemstat's ongoing Phase IIb PORTICO trial in borderline personality disorder (BPD) at the 36th European College of Neuropsychopharmacology (ECNP) Congress, held on October 7-10 in Barcelona, Spain. Oryzon presented a poster communication entitled "PORTICO -- Double-blind, randomized placebo-controlled, adaptive phase IIb trial with vafidemstat in borderline personality disorder -- aggregated baseline characteristics, demographics and safety", which was presented by Dr. Michael Ropacki, Oryzon's CMO for CNS. PORTICO enrolled the last patient in July 2023.

The data presented at ECNP-2023 correspond to the analysis of aggregated blinded safety data cut as of August 23, 2023. As of September 2023, PORTICO randomized 210 participants, and 131 of the originally planned participants (N = 150) already completed the trial. Results obtained confirm that PORTICO enrolled a representative real-world BPD population allowing common comorbidities and concomitant medications that are typically exclusionary in other BPD trials, as well as allowed subjects to receive psychotherapy during the trial.

The screen failure and dropout rates were lower than in the most recent BPD clinical trial. Finally, the aggregated blinded safety data on the fully enrolled sample supports that vafidemstat has been extremely safe and well-tolerated, with a low number of discontinuations (2%) due to treatment-emergent adverse events (TEAEs) and 0% due to serious TEAEs (STEAEs). Only one serious TEAE deemed severe was reported, which fully recovered/resolved during the study.

Oryzon's team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS and iadademstat in oncology, in several Phase II clinical trials. The company has other pipeline assets directed against other epigenetic targets.

The molecule acts on several levels: it reduces cognitive impairment, including memory loss and neuroinflammation, and at the same time has neuroprotective effects. In animal studies vafidemstat not only restores memory but reduces the exacerbated aggressiveness of SAMP8 mice, a model for accelerated aging and Alzheimer's disease (AD), to normal levels and also reduces social avoidance and enhances social avoidance and enhances socialability in murine models. In addition, vafidemstat exhibits fast, strong, and durable efficacy in several preclinical models of multiple sclerosis (MS).

Oryzon has performed two Phase IIa clinical trials in aggressiveness in patients with different psychiatric disorders (REIMAGINE) and in aggressive/agitated patients with moderate or severe AD (REIMAGINE-AD), with positive clinical results reported in both. Additional finalized Phase IIa clinical trials with vafidemstat include the ETHERAL trial in patients with Mild to Moderate AD, where a significant reduction of the inflammatory biomarker YKL40 has been observed after 6 and 12 months of treatment, and the pilot, small-scale SATEEN trial in Relapse-Remitting and Secondary Progressive MS, where anti-inflammatory activity has also been observed. Vafidemstat has also been tested in a Phase II in severe Covid-19 patients (ESCAPE) assessing the capability of the drug to prevent ARDS, one of the most severe complications of the viral infection, where it has been observed.