Eltrombopag, being developed by GSK for thrombocytopenia, demonstrates encouraging Phase I results

Eltrombopag is the first in a new class of oral, small-molecule, non-peptide agonist of the thrombopoietin receptor, being developed by GSK for the treatment of thrombocytopenia (low platelet count) due to various causes. Results published in the journal Blood suggest that suggest that the pharmacodynamic, pharmacokinetic, and safety profile of eltrombopag supports further investigation in Phase II trials.

In this Phase I placebo-controlled trial, eltrombopag was given to 73 healthy male volunteers as once-daily oral capsules for 10 days at doses ranging from 5 to 75 mg. Increases in platelet counts were seen at 30, 50 and 75 mg doses of eltrombopag, and started rising at Day 5 of treatment and peaking on Day 15. The pharmacokinetics of eltrombopag were dose dependent and linear, and platelet counts increased in a dose-dependent manner. There were no apparent differences in the incidence of adverse events between volunteers in the eltrombopag or placebo groups.

The authors conclude ‘These observations indicate that eltrombopag is a once-daily, oral Thrombopoietin receptor agonist with demonstrated thrombopoietic activity in human subjects, encouraging further studies in patients with thrombocytopenia.’

Source: http://bloodjournal.hematologylibrary.org/

NOTE: GSK seem to be investigating the use of Eltrombopag in various indications, including patients with idiopathic thrombocytopenic purpura and patients with low platelet counts who are receiving chemotherapy with Adriamycin and Ifosfamide.