A team of researchers from Gothenburg University in Sweden presented data from a Phase 4 study on Ceplene® earlier this week at the American Association for Cancer Research (AACR) Annual Meeting in New Orleans, LA. These data could have a profound impact on future use of the drug in elderly patients with Acute Myeloid Leukemia (AML). Ceplene®, whose rights in the U.S. belong to Immune Pharmaceuticals (IMNP), is already approved in Europe and Israel. Immune plans to use these data to attract a development partner for Ceplene®, as the company prepares to advance the drug towards regulatory approval in the U.S.
Quick Background on Ceplene
Ceplene® is a drug Immune acquired in 2013 with the takeover of EpiCept. Until recently, Ceplene® sat on the back-burner at Immune. However, recent data has piqued the interest of management and now it seems as though there is a potential to monetize Ceplene® through a partnership that aims to fund clinical studies designed to gain regulatory approval in the U.S. Ceplene® was approved in the EU in October 2008. EU rights are controlled by Swedish-based Meda Pharmaceuticals. Meda recently refocused its business on respiratory, dermatology, and pain / inflammation products, so the company does not actively promote the product. As such, it is likely that any company interested in the U.S. rights to Ceplene® may also be interested in acquiring the EU rights as well. This presents an interesting in-licensing / out-licensing opportunity for Immune.
Ceplene® (histamine dihydrochloride), in combination with interleukin-2 (IL-2), is believed to work by activating T and Natural Killer (NK) cells into a tumor killing mode. Ceplene® binds to the histamine H2 receptors (H2R) that are predominantly expressed on FAB subtypes with monocytic differentiation. Binding to H2R results in down-regulation of NOX2, which results in decreased production of Reactive Oxygen Species (ROS), and subsequent protection of deleterious immune cells from deactivation and death.
Phase 3 data with Ceplene® used in combination with IL-2 demonstrated an improvement in leukemia-free survival (LFS) in patients with AML. Specifically, complete remission patients in the post-consolidation phase of treatment for AML receiving Ceplene® plus IL-2 achieved a 3-year LFS rate of 40% vs. only 26% for a no treatment control (Brune et al., 2006). Both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) have granted Ceplene® orphan drug status for the treatment of AML.