2 April 2019 - - The US Food and Drug Administration has granted orphan drug designation to US-based biopharmaceutical company Imbrium Therapeutics L.P.'s investigational drug tinostamustine, a potentially first-in-class alkylating deacetylase inhibiting molecule being studied in early phase clinical trials, for the treatment of T-cell prolymphocytic leukemia (T-PLL), the company said.

Imbrium Therapeutics L.P., an operating subsidiary of Purdue Pharma L.P., is working in conjunction with Mundipharma EDO GmbH.

T-PLL is an extremely rare and aggressive T-cell leukemia that is characterized by out of control growth of mature T-cells.

There are very limited effective treatment options for T-PLL. The disease typically progresses rapidly and does not respond well to standard multi-agent chemotherapy.

The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

Orphan drug designation is intended to facilitate drug development for rare diseases and may provide certain incentives to drug developers.

T-PLL is an extremely rare and typically aggressive blood cancer. It is so rare that healthcare professionals may only see one case of T-PLL every five to 10 years.

Due to its rarity, T-PLL can be misdiagnosed, resulting in poor patient outcomes with a median survival of around one year.

There is no guarantee that tinostamustine, an investigational agent, will successfully complete clinical development or gain FDA approval.

In addition to T-PLL, Imbrium Therapeutics has initiated the early phase clinical development of tinostamustine in a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

T-cell prolymphocytic leukemia (T-PLL) affects approximately 2% of all patients with mature lymphocytic leukemias.

It is characterized by the out of control growth of mature T-cells (T-lymphocytes). T-cells are a type of white blood cell that protects the body from infections.

The majority of patients present with hepatosplenomegaly and generalized lymphadenopathy, with skin infiltration, anemia and thrombocytopenia often seen.

T-PLL affects older adults with a median age at diagnosis of 61 years, and it is more common in men than in women.

T-PLL typically has rapid progression and does not respond well to standard multi-agent chemotherapy.

Tinostamustine (EDO-S101), is a novel multi-action therapy in Phase 2 clinical development for a range of rare and difficult-to-treat blood cancers and advanced solid tumors.

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.

Tinostamustine is currently 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.

Imbrium is a clinical-stage biopharmaceutical company dedicated to advancing medical science through the development of important new pharmacologic and biologic therapeutics.

We are pursuing oncology chemotherapeutics, treatments for disorders of the central nervous system, and non-opioid approaches to the management of pain.

As an operating subsidiary of Purdue Pharma L.P., Imbrium strives to develop and bring to market new medicines that serve the unmet needs of patients, physicians and health systems worldwide.

Mundipharma EDO is part of the Mundipharma global network of privately-owned independent associated companies, which operate in over 120 countries worldwide.