Imbrium Orphan Status for T-cell Prolymphocytic Leukemia

FDA has granted Imbrium Therapeutics an orphan drug designation for its investigational drug EDO-S101 (tinostamustine), a potentially first-in-class alkylating deacetylase inhibiting molecule being studied in early phase clinical trials for treating T-cell prolymphocytic leukemia (T-PLL). The extremely rare and aggressive T-cell leukemia is characterized by out-of-control growth of mature T-cells, the company says. In addition to T-PLL, Imbrium has begun early phase clinical development of tinostamustine in a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

Imbrium says that preclinical studies have shown that tinostamustine has the potential to improve access to the DNA strands within cancer cells, break them, and counteract damage repair. “The preclinical data also suggest that these complementary and simultaneous modes of action have the potential to overcome resistance toward some other cancer treatments,” it says.