ORYZON announces FDA Orphan Drug Designation granted to iadademstat for treatment of Acute Myeloid Leukemia

  • The compound is currently in Phase II
  • Now has orphan designation in both U.S. and EU
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MADRID, SPAIN and CAMBRIDGE, MA, UNITED STATES, February 11th, 2021 – Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a public clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the company’s first-in-class LSD1 inhibitor iadademstat for the treatment of patients with acute myeloid leukemia (AML). Iadademstat is an investigational, oral, small molecule covalent inhibitor of the epigenetic enzyme LSD1, a chromatin remodeler that interacts with a variety of transcription factors involved in leukemia and other cancers.

“Receiving Orphan Drug Designation for iadademstat in AML is an important recognition of the role that new drugs with new mechanisms of action may bring to this patient community, where we do not yet have any potentially curative medicines besides stem cell transplant. Iadademstat is showing a high overall response rate of 85% in our ongoing clinical Phase II study ALICE, with a rapid onset of action and with durable responses. In addition, we have seen a good safety and tolerability profile in the combination treatment with azacitidine. Future Phase II clinical trials in further combination treatments of AML are planned for the second half of this year,” said Oryzon’s Global Head of R&D and CSO, Dr. Torsten Hoffmann.

The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.

Iadademstat was previously granted orphan drug designation by the European Medicines Agency for the treatment of AML.

Contact information

IR & Media, US & Europe:

Mary-Ann Chang - mchang@lifesciadvisors.com

+44 7483 284 853



Patricia Cobo/Carlos C. Ungría
pcobo@atrevia.com - cungria@atrevia.com
+34 91 564 07 25


Emili Torrel - BD Director - etorrell@oryzon.com
+34 93 515 13 13

Attached files
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More information

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 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 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), neuroendocrine tumors, medulloblastoma and others. Iadademstat has been tested in four clinical trials (two in monotherapy in SCLC and AML, and two in combination, in SCLC and AML) in more than 100 patients. In the combination studies, ALICE (ongoing), a Phase IIa trial in combination with azacitidine in elderly or unfit AML patients, and CLEPSIDRA (finalized), a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients, preliminary efficacy results have been reported.