ORYZON Enters Cooperative Research and Development Agreement (CRADA) with U.S. National Cancer Institute to Develop Iadademstat in Different Cancers

• Oryzon and NCI will collaborate to assess the safety and efficacy of iadademstat in oncology patients with different hematological and solid tumors

MADRID, Spain and BOSTON, July 19, 2022 (GLOBE NEWSWIRE) -- 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, announced today that it has entered into a Cooperative Research and Development Agreement (CRADA) with the U.S. National Cancer Institute (NCI), part of the National Institutes of Health. Under the terms of the agreement, Oryzon and the NCI will collaborate on potential further clinical development of Oryzon’s clinical stage LSD1 inhibitor, iadademstat, in different types of solid and hematological cancers.

Dr. Carlos Buesa, President and CEO of Oryzon, said: “This research and development agreement with the NCI is, first, a strong validation of iadademstat, probably the most potent and selective LSD1 inhibitor currently in clinical development, and will also allow us to significantly expand our clinical development program for this compound.”

Dr. Douglas V. Faller, Oryzon’s Global Chief Medical Officer said: “This collaboration with the NCI on the development of iadademstat will allow us to work with some of the world’s leading oncology researchers to further expand its therapeutic potential. This collaboration also dramatically expands our ability to conduct clinical trials in a wide number of indications, and in combination with other novel or established therapies such as immuno-oncology and molecularly-targeted agents.”

Iadademstat is an orally active, highly potent and selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers. More than 100 cancer patients have been treated with iadademstat in several trials. In a still ongoing Phase IIa trial (ALICE trial) in elderly 1L acute myeloid leukemia (AML) patients (ALICE trial), iadademstat has shown encouraging safety and efficacy data in combination with azacitidine. The company is now starting a Phase Ib trial of iadademstat in combination with gilteritinib in FLT3 mutant relapsed/refractory AML patients, which recently received IND approval by the FDA. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors, medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial, finalized), preliminary efficacy results have been reported. Iadademstat has recently received orphan drug designation from the FDA for SCLC and has now orphan drug status in the US for SCLC and AML.

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

About Iadademstat
Iadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). In an ongoing Phase IIa trial in elder 1L-AML patients (ALICE trial), iadademstat has shown encouraging safety and efficacy data in combination with azacitidine (see Salamero et al., ASH 2021 poster). The company has recently obtained approval from the U.S. FDA for its IND for FRIDA, a Phase Ib trial of iadademstat plus gilteritinib in patients with relapsed/refractory AML with FLT3 mutations. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). New trials in combination in SCLC and NET are under preparation. In total iadademstat has been dosed so far to more than 100 cancer patients in four clinical trials.

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.