Orphan Drug Designation For Tinostamustine In Extremely Rare Blood Cancer

Aggressive T-cell Prolymphocytic Leukemia Has Very Limited Treatment Options

The FDA has granted orphan drug status to EDO-S101 (Tinostamustine, Mundipharma EDO GmbH, and Imbrium Therapeutics L.P., a subsidiary of Purdue Pharma L.P.), an alkylating deacetylase inhibiting molecule for the treatment of T-cell prolymphocytic leukemia (T-PLL).

Tinostamustine (EDO-S101) is an alkylating deacetylase-inhibiting molecule in early phase clinical development for a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

Data from preclinical studies have shown that EDO-S101 could improve access to cancer cells’ DNA strands, break them, and correct damage repair. The data also indicate that these complementary and simultaneous actions have the potential to overcome resistance toward some other cancer treatments.

T-PLL is so rare that physicians may come across only one case of T-PLL every five to 10 years. The disease affects older adults, mostly male, with a median age of 61 years, and its rarity means that T-PLL can be misdiagnosed, which results in poor patient outcomes. Patients have a median survival of approximately seven months to one year, and the disease is typically resistant to conventional chemotherapy.

Currently, EDO-S101 is being studied in multiple myeloma, Hodgkin lymphoma, peripheral T-cell lymphoma, cutaneous T-cell lymphoma, T-cell prolymphocytic leukemia, soft tissue sarcoma, small cell lung cancer, triple-negative breast cancer, ovarian cancer, endometrial cancer, and MGMT-unmethylated glioblastoma.

Source: Mundipharma EDO, March 28, 2019