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ORYZON Publishes Paper in Journal of Clinical Oncology Demonstrating Relevance of Iadademstat in Leukemia
[October 15, 2020]

ORYZON Publishes Paper in Journal of Clinical Oncology Demonstrating Relevance of Iadademstat in Leukemia

  • Data support safety of the drug in elderly patients with acute leukemia

  • Paper describes mechanism of differentiation in patients and first antileukemic responses

  • Iadademstat showing robust results in ALICE Phase II trial

MADRID, Spain and CAMBRIDGE, Mass., Oct. 15, 2020 (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, announces the publication of a scientific paper in the peer-reviewed international medical journal, Journal of Clinical Oncology. The article describes the first-in-human clinical trial in acute myeloid leukemia (AML) patients with iadademstat (ORY-1001), the most potent and selective KDM1A inhibitor to date. KDM1A (also known as LSD1) is a specific lysine demethylase, whose activity is essential for the function and maintenance of the leukemic stem cells, a subset of malignant cells that is believed to be the ultimate reason for relapse in AML patients.

The manuscript, entitled "First-in-Human Phase I Study of Iadademstat (ORY-1001): A First-in-Class Lysine-Specific Histone Demethylase 1A Inhibitor, in Relapsed or Refractory Acute Myeloid Leukemia”, has been published in collaboration with a large group of highly respected scientists from leading hospitals around Europe. The article describes the Phase I/IIa clinical trial, which recruited 42 patients with refractory or relapsed AML, and reports that iadademstat has a good safety profile together with signs of clinical and biological activity as a single agent. This is notable because current treatment options in AML do not cure most patients, particularly those who are not suitable for intensive chemotherapy, and novel therapies are required.

Carlos Buesa, Oryzon’s CEO, commented: “Iadademstat is the first drug of its kind in the field of leukemia. This trial was pioneering and provided crucial data that have allowed us to advance in iadademstat’s clinical development. In the ALICE Phase II trial now underway in AML, iadademstat is demonstrating robust data on therapeutic activity in patients and a very promising level of duration of responses. The company expects to offer additional data from this trial at the ASH-2020 conference in December.”

The paper reports that patients with an aggressive AML variant, the mixed-lineage leukemia rearrangement, treated with iadademstat undergo a potent cell differentiation process, the first step for the leukemic cell to stop being cancerous. Complete remission with uncomplete hematological recovery (CRi) in one patient was also reported.

This first exploratory Phase I/IIa study provided the necessary knowledge to inform the ongoing Phase II ALICE study that is investigating iadademstat in combination with azacitidine to further delineate its activity in AML. Oryzon recently reported robust signs of clinical efficacy in the ALICE trial, with 77% objective responses, of which 60% were complete remissions (CR/CRi).

The Journal of Clinical Oncology is the official journal of the American Society of Clinical Oncology with a specialized audience of medical professionals in the oncology field. With an impact index of 32.9 and a manuscript acceptance rate of 13%, it is one of the most prestigious journals in the field worldwide.

The paper canbe accessed here:

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 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. For more information, visit

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 first Phase I/IIa clinical trial with iadademstat in refractory and relapsed acute leukemia patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi. 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), medulloblastoma and others. Oryzon is conducting two Phase IIa clinical trials of iadademstat in combination; the first one in combination with azacitidine in elderly AML patients (ALICE study) and the second one in combination with platinum/etoposide in second line SCLC patients (CLEPSIDRA study). In both studies, preliminary clinical results have been reported.

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.

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