Date: 2015-11-15
Type of information: Treatment of the first patient
phase: 1
Announcement: treatment of the first patient
Company: Oryzon Genomics (Spain)
Product: ORY-1001
Action
mechanism: enzyme inhibitor/LSD1 inhibitor. ORY-1001 is a selective inhibitor of Lysine Specific Demethylase-1 (LSD1, aka KDM1), an epigenetic modulator that regulates gene expression by demethylating specifically some lysines in the histones (lysine K4 and K9 in histone H3). LSD1 forms part of protein complexes involved in transcriptional regulation, and misregulation of these transcriptional complexes may result in disease. This orally-active subnanomolar specific LSD1 inhibitor reduces leukemic stem cell potential, potently inhibits colony formation, overcomes the differentiation block in AML cell lines, and induces apoptosis / inhibits proliferation at sub-nanomolar concentrations in selected AML cell lines.
Disease: relapsed or refractory acute leukemia
Therapeutic area: Cancer - Oncology
Country: France, Spain, UK
Trial details:
Latest
news: * On November 10, 2015, Oryzon Genomics announced the dosing of the first patient in the extension cohort (Part 2) of its ORY-1001 Phase I clinical trial. 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. The first patient of this extension cohort has been recruited and ORY-1001 treatment has been initiated. * On September 7, 2015, Oryzon Genomics announced that it has finalized the multiple ascending dose (MAD) stage of its Phase I clinical trial to evaluate the safety, tolerability and pharmacokinetics of ORY-1001 in patients with relapsed or refractory acute leukemia (AL). Through the establishment of a Recommended Dose (RD) of ORY-1001, Oryzon has achieved this development milestone included in the License Agreement with Roche signed in April 2014 and will receive a payment of $ 4 million. The preliminary results obtained so far demonstrate the safety and tolerability of ORY-1001 in patients suffering from AL. An extension arm will now commence to evaluate the preliminary efficacy of ORY-1001 in various subpopulations of acute myeloid leukemia (AML), including mixed lineage leukemia (MLL). * On January 16, 2014. Oryzon Genomics, a Spanish biopharmaceutical company with a strong focus on epigenetics, has announced that has received the approval from the British Drug Agency (MHRA) to initiate a Phase I/IIA clinical trial of ORY-1001, anovel and highly selective LSD1 inhibitor, in patients with relapsed or refractory acute leukemia in the United Kingdom.
The company got the approval from the Spanish Drug Agency (AEMPS) a few weeks ago. This first-in-human trial is currently open at the Hospital Vall d’Hebron in Barcelona, Spain and additional study sites in Spain and in the United Kingdom will be incorporated shortly. With this second approval, the company plan will be able to develop the clinical plan in the planned timeframe,
In August 2013, Oryzon received orphan designation for ORY-1001 for the treatment of Acute Myeloid Leukemia from the EMA. The company plans to apply for the same status to the FDA.
