Home Madrid university ORYZON launches preclinical collaboration on Kabuki syndrome

ORYZON launches preclinical collaboration on Kabuki syndrome


MADRID, Spain and CAMBRIDGE, Mass., May 03, 2022 (GLOBE NEWSWIRE) — Oryzon Genomics, SA (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases high unmet medical need, today announced the start of a preclinical collaboration on Kabuki syndrome with researchers from the Kennedy Krieger Institute and Johns Hopkins University led by Dr. Jacqueline Harris, director of the clinic of Kennedy Krieger Institute Epigenetics and Assistant Professor of Pediatrics, Neurology and Genetics at Johns Hopkins University School of Medicine and Dr. Hans Bjornsson, Founder of the Epigenetics and Chromatin Clinic and Associate Professor of Pediatrics and Genetics at Johns Hopkins University School of Medicine.

Kabuki syndrome (KS) is an autosomal dominant/X-linked disorder that affects multiple organ systems including the neuro, immune, auditory, and cardiac systems. Patients present with characteristic distinctive facial features, growth retardation and mild to moderate intellectual disability and autoimmune disorders. The majority (>70%) of molecularly confirmed KS cases have loss-of-function variants in the KMT2D gene. This gene, aka MLL2, catalyzes the addition of methyl groups to histone 3 lysine 4, which are marks associated with open chromatin, thereby regulating the expression of critical target genes.

About Oryzon
Founded in 2000 in Barcelona, ​​Spain, Oryzon (ISIN Code: ES0167733015) is a clinical-stage biopharmaceutical company considered the European leader in epigenetics. Oryzon has one of the strongest pipelines in the field, with two LSD1 inhibitors, iadademstat and vafidemstat, in phase II clinical trials, and other assets in the pipeline directed against other epigenetic targets. Additionally, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignancies and neurological diseases. For more information visit www.oryzon.com

About Vafidemstat
Vafidemstat (ORY-2001) is an oral LSD1 inhibitor optimized for the CNS. 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 heightened aggression in SAMP8 mice, a model of accelerated aging and Alzheimer’s disease (AD), to normal levels, also reduces social avoidance and improves sociability in mouse models. Additionally, vafidemstat exhibits rapid, strong, and long-lasting efficacy in several preclinical models of multiple sclerosis (MS). Oryzon has completed two Phase IIa clinical trials on aggression 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 cases. Other phase IIa clinical trials completed with vafidemstat include the ETHERAL trial in patients with mild to moderate AD, where a significant reduction in the inflammatory biomarker YKL40 was observed after 6 and 12 months of treatment, and the trial small-scale SATEEN pilot in relapsing-remitting and secondary progressive MS, where anti-inflammatory activity was also observed. Vafidemstat has also been tested in a phase II in patients with severe Covid-19 (ESCAPE) assessing the drug’s ability to prevent ARDS, one of the most serious complications of viral infection, where it showed significant anti-inflammatory effects in severe Covid cases. -19 patients. Currently, vafidemstat is the subject of two phase IIb trials in borderline personality disorder (PORTICO) and in schizophrenic patients (EVOLUTION). The company is also deploying a CNS precision medicine approach with vafidemstat in genetically defined patient subpopulations with certain CNS disorders and preparing for a clinical trial in patients with Kabuki syndrome to begin in 1H 2022. company is also exploring the clinical development of vafidemstat in other neurodevelopmental syndromes.

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 regarding 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 these forward-looking statements are reasonable, investors and holders of Oryzon stock 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 Oryzon’s control which could cause actual results and developments to differ materially from those expressed, implied or projected by the forward-looking information and statements. These risks and uncertainties include those discussed or identified in documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are publicly available. Forward-looking statements are not guarantees of future performance and have not been reviewed by Oryzon’s auditors. You are cautioned not to place undue reliance on 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 anyone acting on its behalf are expressly qualified in their entirety by the above cautionary statement. All forward-looking statements included herein are based on information available to Oryzon as of the date hereof. Except as required by applicable law, Oryzon undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. This press release does not constitute an offer of securities for sale in the United States or any other jurisdiction. Oryzon securities may not be offered or sold in the United States absent registration or an exemption from registration. Any public offering of Oryzon securities to be made in the United States will be made by means of a prospectus obtainable from Oryzon or the selling securities holder, as the case may be, which will contain particulars of Oryzon and its management, as well as financial information statements.

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Ashley R. Robinson Sandya von der Weid Patricia Cobo Saikat Nandi
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