Oryzon Genomics Announces First Patient Dosed in Phase I Extension Cohort of ORY-1001

Oryzon Genomics Announces First Patient Dosed in Phase I Extension Cohort of ORY-1001

Extension Cohort (Part 2) of the Current Phase I Clinical Trial to Assess the Efficacy of ORY-1001 in Patients With Acute Myeloid Leukemia in 10 Centers in Spain, UK and France

November 10, 2015 08:00 ET | Source: Oryzon
BARCELONA, Spain and CAMBRIDGE Mass., Nov. 10, 2015 (GLOBE NEWSWIRE) -- Oryzon Genomics, a clinical stage biopharmaceutical company leveraging epigenetics to develop therapies in oncology and neurodegenerative diseases, announced today the dosing of the first patient in the extension cohort (Part 2) of its ORY-1001 Phase I clinical trial.

Roche and Oryzon entered into a worldwide collaboration in April 2014 to research, develop and commercialize inhibitors of Lysine Specific Demethylase-1 (LSD1; KDM1A), an epigenetic modulator that regulates gene expression, including Oryzon's lead clinical candidate ORY-1001. Based on the multiple ascending dose (MAD) stage (Part 1) of Oryzon's Phase I clinical trial to evaluate the safety, tolerability and pharmacokinetics of ORY-1001 in patients with relapsed or refractory acute leukemia (AL), a recommended dose (RD) of ORY-1001 has been established. The preliminary results obtained during the MAD stage of the trial demonstrate the safety and tolerability of ORY-1001 in patients suffering from AL.

The objective of this extended cohort of the Phase I clinical trial is to evaluate the preliminary efficacy of ORY-1001. This extension cohort is performed to explore the ORY-1001 treatment efficacy at the recommended dose in various genetically selected subpopulations of patients suffering from acute myeloid leukemia (AML). This includes mixed lineage leukemia (MLL), a rare subset of AL in which leukemia stem cells are specially sensitive to LSD1 inhibition. To recruit a sufficient number of patients suffering from this rare form of leukemia, Oryzon has increased the number of active centers to 10. For a complete list of active centers visit www.oryzon.com.

The first patient of this extension cohort has been recruited and ORY-1001 treatment has been initiated.

"Patient recruitment in rare diseases always poses a challenge. We have included a sufficient number of reference centers to support patient enrollment over the next 6 months. We are eager to explore the efficacy profile of ORY-1001 as we continue to investigate the role of LSD1 inhibitors for the treatment of AML subgroups," said Dr. Cesar Molinero, Chief Medical Officer of Oryzon Genomics.


ORY-1001 is a highly selective and potent LSD1 inhibitor which is orally administered to patients. ORY-1001 affects AML stem cells, a sub-population of cancer cells that has been proposed to be responsible for frequent relapses of the disease. ORY-1001 significantly reduces tumor cell load and increases survival time in mouse models of Acute Lymphoblastic Leukemia. LSD1 is also associated with other malignancies such as solid tumors and other haematological diseases. ORY-1001 is part of the worldwide collaboration signed between Oryzon and Roche on April 2014. The agreement includes the licensing of two patent families that Oryzon has created and includes options for other Oryzon programs to be incorporated in future.


Founded in 2000 in Barcelona, Spain, Oryzon is a privately held, clinical stage biopharmaceutical company considered as the European champion in Epigenetics. The company has one of the strongest portfolios in the field and a clinical asset already partnered with ROCHE. Oryzon's LSD1 program is currently covered by 19 patent families and has rendered one compound in clinical trials and another one is anticipated to enter clinical trials in early 2016. In addition, Oryzon has ongoing programs for developing inhibitors against other epigenetic targets. The company has also a strong technological platform for biomarker identification and performs biomarker and target validation for a variety of malignant and neurodegenerative diseases. Oryzon's strategy is to develop first in class compounds against novel epigenetic targets through Phase II clinical trials, at which point it is decided on a case-by-case basis to either keep the development in-house or to partner or out-license the compound for late stage development and commercialization. The company has offices in Barcelona and Cambridge, Massachusetts. For more information, visit www.oryzon.com.

US Contact:
The Ruth Group
Lee Roth/Tram Bui
(646) 536-7012/7035

Ana Melgar/Patricia Cobo
+34 91 564 07 25

The Company:
Ms. Anna K Baran
IR Director
+44 (0) 752 1083 006
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