Analyst Outlook
Aplidin (plitidepsin) is a cyclic depsipeptide antitumor agent developed by the Spanish company
PharmaMar. In 2004, Aplidin gained orphan drug designation in the US and EU for the treatment of
multiple myeloma (MM), and it is currently in Phase III development for the disease. PharmaMar is
positioning it as a fourth-line therapy for heavily pretreated MM patients in combination with
dexamethasone. Targeting fourth-line therapy will significantly limit the potential share for Aplidin in
the MM market. Datamonitor Healthcare also believes that Aplidin will face strong competition from
existing therapies with proven efficacy in heavily pretreated patient groups.
Drug Overview
Aplidin is an intravenous, marine-originated cyclic depsipeptide antitumor agent. The drug, which is
currently synthesized chemically, was originally isolated from the marine tunicate Aplidium albicans
(Cuadrado et al., 2003; PharmaMar, 2014). Aplidin’s exact mechanism of action is not fully
understood, but it is believed to induce cytotoxicity through several pathways. It has been shown to
inhibit the cancer-associated Eukaryotic elongation factor 12 (eEF1A2) (PharmaMar press release,
2015; Lee and Surh, 2009), as well as disrupting the glutathione homeostasis and activating mitogen-
activated protein kinases, leading to caspase-dependent apoptosis (García-Fernández et al., 2002;
González-Santiago et al., 2006). Aplidin has also been shown to block cell division in the G1/G2 phase,
and inhibit the secretion of vascular endothelial growth factor (Biscardi et al., 2005; Erba et al., 2002).
TABLE OF CONTENTS
4 PRODUCT PROFILES
4 Aplidin : Multiple myeloma
LIST OF FIGURES
7 Figure 1: Aplidin for multiple myeloma – SWOT analysis
8 Figure 2: Datamonitor Healthcare’s drug assessment summary of Aplidin in multiple myeloma
9 Figure 3: Datamonitor Healthcare’s drug assessment summary of Aplidin in multiple myeloma
LIST OF TABLES
4 Table 1: Aplidin drug profile
6 Table 2: Aplidin Phase III trials in multiple myeloma
6 Table 3: Aplidin Phase II data in multiple myeloma