Earlier today Oryzon Genomics, a clinical stage biopharmaceutical company based in Barcelona (Spain) leveraging epigenetics to develop therapies in oncology and neurodegenerative diseases, announced the dosing of the first patient in the extension cohort (Part 2) of its ORY-1001 Phase I clinical trial. The trial will take place in 10 centers in Spain, UK and France.
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 or ALL. LSD1 is also associated with other malignancies such as solid tumors and other hematological diseases.
…recruitment in rare diseases always poses a challenge [but] we’re 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…
Worldwide Collaboration
In April 2014 Roche and Oryzon entered into a worldwide collaboration to research, develop and commercialize inhibitors of Lysine Specific Demethylase-1 (LSD1; KDM1A), an epigenetic modulator that regulates gene expression, including Oryzon’s lead LSD1 program which is currently covered by 19 patent families.
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.
Preliminary efficacy
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
“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,” noted Cesar Molinero, Chief Medical Officer of Oryzon Genomics.
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