ORYZON presents safety and efficacy data of vafidemstat from the Phase II SATEEN trial in multiple sclerosis at ECTRIMS-2021
- Long-term vafidemstat treatment was safe and well tolerated with drug exposures up to 2 years
- Anti-inflammatory activity observed in vafidemstat-treated patients
- SATEEN was a pilot, small scale, trial not powered to get definitive efficacy data
MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, October 13th, 2021 - Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, presented today final data from the Phase II trial SATEEN on vafidemstat’s ability to reduce the inflammatory response in multiple sclerosis (MS) patients. The data were presented at the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis, ECTRIMS-2021, in an e-poster entitled “Safety And Efficacy Data From SATEEN Trial In Multiple Sclerosis”.
Eighteen MS patients were enrolled in this Phase II randomized, double-blind, placebo-controlled, parallel group, dose-finding trial that evaluated the safety and tolerability of two vafidemstat doses (0.6 mg or 1.2 mg, randomization ratio 2:3:3) in relapse-remitting MS (RRMS) or secondary progressive MS (SPMS) patients. Data analysis occurred after 9 and 15 months of treatment, with an optional additional open-label extension for SPMS patients.
The median age of the patients was 49 years; 72% were female and 67% were RRMS. The mean study permanence was 408 ± 156 days, with the longest exposure being 756 days. Treatment was well tolerated, and no serious adverse events were reported. Out of the 55 adverse events reported in the full population, only 13 in 7 patients were assessed as potentially related to treatment, 4 of which (including the only one considered severe during the trial) in 2 placebo treated patients.
This was a pilot, small scale trial not powered to get conclusive efficacy data. Accordingly, there were no statistically significant differences between groups in MRI, OCT or EDSS evaluations. Relapse or disease progression was recorded in 4 patients (22.2%).
Yet, selected patients treated with vafidemstat showed improvement in one or more clinical readouts. In addition, promising pharmacodynamic anti-inflammatory activity was reported in most of the vafidemstat-treated patients compared to placebo. Particularly, serum Th1/Th2 cytokine ratios were modulated with vafidemstat treatment, reaching statistical significance in 3 instances, including a clear dose-dependent decrease of the IFN γ/IL-4 ratio. Vafidemstat also promoted changes in the expression of several soluble markers important in MS pathogenesis, such as BDNF1 or EGF2, among others, and these differences were overall markedly larger when a positive clinical outcome was observed in the studied patient. Finally, in the same patients, plasma chemokine levels assessed by specific immunoassays (IP-10, MCP-1, RANTES) were also overall decreased upon vafidemstat treatment compared to placebo.
Dr. Jordi Xaus, Oryzon’s CSO, commented: “SATEEN has provided the longest-duration vafidemstat clinical data so far, with exposures ranging from 15 to 24 months, and has confirmed the excellent tolerability of long lasting vafidemstat treatments. Despite the power limitations of the study, the clinical activity observed is encouraging and supports that vafidemstat has the potential to reduce the neuroinflammation component in MS. Neuroinflammation is a core feature in MS but also in other CNS conditions. These anti-inflammatory findings come to confirm the ones recently reported in severe COVID-19 patients treated for one week with vafidemstat and emphasize the antiinflammatory activity of vafidemstat.”
A copy of the e-poster presented at ECTRIMS-2021 is available here
For more information about ECTRIMS-2021, please visit ECCMID’s website
About Oryzon
Founded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company considered as the European champion in Epigenetics. Oryzon has one of the strongest portfolios in the field. Oryzon’s LSD1 program has rendered two compounds, vafidemstat and iadademstat, in Phase II clinical trials. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets. Oryzon has a strong technological platform for biomarker identification and performs biomarker and target validation for a variety of malignant and neurological diseases. Oryzon has offices in Spain and the United States. Oryzon is one of the most liquid biotech stocks in Europe with +90 M shares negotiated in 2020 (ORY:SM / ORY.MC / ORYZF US OTC mkt). For more information, visit www.oryzon.com
About Vafidemstat
Vafidemstat (ORY-2001) is an oral, CNS optimized LSD1 inhibitor. 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 sociability 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. 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 showed significant anti-inflammatory effects in severe Covid-19 patients. Currently, vafidemstat is in two Phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenia patients (EVOLUTION). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically-defined patient subpopulations of certain CNS disorders.
FORWARD-LOOKING STATEMENTS
This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events or otherwise. This press release is not an offer of securities for sale in the United States or any other jurisdiction. Oryzon’s securities may not be offered or sold in the United States absent registration or an exemption from registration. Any public offering of Oryzon’s securities to be made in the United States will be made by means of a prospectus that may be obtained from Oryzon or the selling security holder, as applicable, that will contain detailed information about Oryzon and management, as well as financial statements.