Foundation research

ORYZON collaborates with the CMT Research Foundation in the United States


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  • Explore Oryzon’s HDAC6 Inhibitors on Charcot-Marie-Tooth disease
  • CMTRF to finance live efficacy testing in the CMT preclinical model

MADRID and BOSTON, July 26. 12, 2022 (GLOBE NEWSWIRE) — Oryzon Genomics, SA (ISIN: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with high unmet medical need, announced today today launched a preclinical collaboration with the CMT Research Foundation (CMTRF), an American patient-led non-profit organization focused on providing treatments and cures for Charcot-Marie-Tooth disease (CMT ), to explore Oryzon’s histone deacetylase 6 (HDAC6) inhibitors.

CMT is a progressive degenerative disease involving the peripheral nerves. It affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people worldwide. It is one of the most common conditions among rare diseases and currently lacks effective treatments or cures. CMT is caused by a variety of genetic mutations. CMT1A is the most common of all, accounting for about half of all people with CMT.

HDAC6 inhibitors have previously been described as potentially effective treatments for CMT. Oryzon recently completed an HDAC6 discovery program leading to the selection of two potential preclinical candidates with promising efficacy, selectivity and safety. As part of this collaboration, the CMTRF will fund a series of live tests with Oryzon’s HDAC6 candidates in a mouse model of CMT1A, which reliably recapitulates many symptoms of this disease in humans.

Dr. Jordi Xaus, CSO of Oryzon, said: “CMT is the most promising indication for our HDAC6 precandidates. CMTRF is the perfect ally to explore our compounds in this indication, as it has a close connection to the CMT patient community, a highly experienced scientific advisory body and a strong commitment to finding treatments for CMT. We are delighted to start this collaboration. If the results are positive, our HDAC6 program would be closer to clinical development and offer hope for patients with CMT. This program would become our second program in epigenetics in nervous diseases.

“We are delighted to collaborate with Oryzon on this important project,” said Cleary Simpson, CEO of the CMT Research Foundation. “HDAC6 inhibitors show promise as potential therapeutics for several forms of CMT, including the most common form, CMT1A. With funding from the CMT Research Foundation, Oryzon will be able to rapidly test its newly discovered HDAC6 inhibitors in CMT1A model mice, paving the way. People with CMT currently have no effective treatment or cure, and we believe this project has the potential to change that for millions of families.

The CMT Research Foundation (CMTRF) is solely focused on providing treatments and cures for CMT. Founded by two patients committed to accelerating drug delivery to people with CMT around the world, the organization funds research for drug development. The federal 501(c)(3) tax-exempt organization is supported by personal and corporate financial donations.

About OryzonFounded 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

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 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 detailed information about Oryzon and its management, as well as financial information statements.

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Source: Oryzon Genomics, SA