PharmaMar announced the start of a multicenter and prospective Phase 2 clinical trial to evaluate the efficacy of plitidepsin, a potential treatment option for adult patients with relapsed and refractory angioimmunoblastic T-cell lymphoma (AITL).
Plitidepsin is an investigational drug of marine origin, obtained from the ascidian Aplidium albicans. It binds to eEF1A2 targeting the non-canonical role of the protein and causing tumor cell death by apoptosis (programmed death).
In consultation with the U.S. Food and Drug Administration (FDA), and because AITL is a rare disease, the trial only has one study arm. Its primary endpoint is the evaluation of plitidepsin’s efficacy in terms of overall response rate (ORR), which will be assessed by an independent committee using the Lugano classification response criteria. The trial’s secondary endpoint is the evaluation of other efficacy parameters, including overall survival, progression free survival, duration of response, plitidepsin’s safety profile, and the identification of biomarkers that help to evaluate the drug’s predictive activity.
“This pivotal clinical trial will include 60 patients from approximately 25 investigative sites,” Dr. Arturo Soto, director of Clinical Development at PharmaMar’s Oncology Unit, said in a news release. “After the recent announcement of the positive results obtained with plitidepsin in patients with multiple myeloma, we are continuing with the development of this molecule in other hematological tumors, such as angioimmunoblastic T-cell lymphoma.”
The trial is registered with the European Union (2015-001909-14).
Plitidepsin is also being evaluated in a Phase 1b clincial trial in relapsed or refractory multiple myeloma patients, in combination with bortezomib and dexamethasone. Both the FDA and the European Union have designated plitidepsin an orphan drug to speed its development and testing.
Angioimmunoblastic T-cell lymphoma is a rare, aggressive (fast-growing) form of peripheral T-cell lymphoma (PTCL). While AITL only accounts for 1 percent to 2 percent of all NHL cases in the United States, it is one of the most common subtypes of mature T-cell lymphomas. Symptoms of AITL include high fever, night sweats, skin rash, and autoimmune disorders such as autoimmune hemolytic anemia (AIHA) and immune thrombocytopenia (ITP).