Aplidin (plitidepsin) is an investigative anticancer agent under development by PharmaMar, a pharmaceutical company that commercializes anticancer drugs of marine origin. Aplidin is isolated from the sea squirt (Aplidium albicans) and has shown anti-myeloma activity even in myelomas resistant to other agents.
The drug has received orphan drug designation in the U.S., the European Union, and Switzerland.
How Aplidin works
The gene eEF1A2 is thought to play an important role in the development of cancers. Aplidin binds specifically to the protein product of the eEF1A2 gene that exists on the surface of tumor cells, inhibiting their development and viability by changing their shape and function. These changes lead to tumor cell death through a natural process called apoptosis or programmed cell death.
Aplidin studies have shown additive or synergistic effects with other anti-myeloma treatments.
Results from a small Phase 2 study (NCT00229203) of Aplidin as a three-hour infusion every two weeks alone or in combination with dexamethasone in 47 pre-treated patients with relapsing or drug resistant multiple myeloma had an overall response rate of 13 percent with Aplidin alone, which increased to 22 percent when dexamethasone was added. The treatment was well tolerated. Anemia and low platelet counts were the most frequent severe events and an increase in liver enzymes and fatigue were the most common side effects.
A larger Phase 3 study (NCT01102426), called ADMYRE, is still ongoing. It is a randomized, open-label, international, multicenter study of Aplidin in combination with dexamethasone vs. dexamethasone alone in people with relapsed/drug resistant multiple myeloma. It will measure progression-free survival (PFS) and evaluate tumor response, duration of response, overall survival, and any effect of the treatment on heart rhythm.
The study’s primary objective was met: the effectiveness of Aplidin in combination with dexamethasone compared to dexamethasone alone, determined using the International Myeloma Working Group criteria. It showed a statistically significant 35 percent reduction in the risk of progression or death over dexamethasone alone.
PharmaMar intends to submit a Marketing Authorization Application to the European Medicines Agency (EMA) soon, after a full description of the final data of the study is submitted for presentation at a medical meeting.
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