Hybrigenics’ inecalcitol gets Orphan Drug designation for the treatment of Acute Myeloid Leukemia in the United States

18-Aug-2015 - France

Hybrigenics announces the Orphan Drug Designation by the American Food and Drug Administration of inecalcitol for the treatment of acute myeloid leukemia (AML) in the United States. This favourable decision is based on in vitro and in vivo preclinical evidence showing the synergy between inecalcitol and azacytidine or decitabine, two hypo-methylating anticancer drugs, to inhibit the growth of human AML cell lines in vitro and, in vivo, to prolong the survival of mice in two different experimental models of AML.

The molecular basis of their synergy with inecalcitol, a vitamin D receptor agonist, has been elucidated: azacytidine or decitabine “unmask” the gene coding for vitamin D receptors (by reducing the methylation of its promoter region). As a consequence, more vitamin D receptors are expressed and available to be activated by inecalcitol, resulting in an improved efficacy to limit leukemia progression over the hypo-methylating agents alone.

Azacytidine (Vidaza(R), Celgene) and decitabine (Dacogen (R), Janssen-Cilag) are two hypo-methylating agents already used for AML in older (>65 years old) or frail patients not eligible to standard induction chemotherapy. Inecalcitol alone has already been studied in a Phase II clinical trial in chronic lymphocytic leukemia.

https://www.bionity.com/en/news/154144/hybrigenics-inecalcitol-gets-orphan-drug-designation-for-the-treatment-of-acute-myeloid-leukemia-in-the-united-states.html