MGI forecasts $1B peak US sales for novel thrombocytopenia drug AKR-501

MGI Pharma says that AKR-501, the novel, orally-available thrombocytopenia treatment that it has licensed from fellow USA-based biopharmaceutical firm AkaRx, could achieve peak sales of $1.0 billion in its home market. The drug is a full agonist that targets the c-Mpl receptor, and is designed to increase the production of platelets.

Thrombocytopenia is characterized by an abnormally low level of circulating platelets, either as a result of their autoimmune-mediated destruction caused by idiopathic purpura, or due to a reduction in the rate at which they are produced by bone marrow cells after chemotherapy. The condition is also linked to chronic hepatitis-C virus infection and, more specifically, to antiviral treatment for HCV, which can cause bone marrow toxicity.

Trials of AKR-50, reported by AkaRx in December last year at the American Society of Hematology meeting held in Orlando, Florida, indicated that it boosted platelet counts in healthy volunteers by around 50% and maintained this effect for 14 days. Currently, the drug is being examined in a Phase II study as a treatment for idiopathic thrombocytopenic purpura, with a Phase III assessment for this indication scheduled to start in the next 18 months.