GlaxoSmithKline plc yesterday announced results from a global Phase II study of its investigational small molecule oral platelet growth factor, eltrombopag. Data from this study demonstrated that treatment with eltrombopag resulted in a significant increase in platelet count compared to placebo across a variety of typically poorly responding ITP patients including those with prior splenectomy or those having a very low baseline platelet count (
“ITP is a frightening disease which does not follow a predictable course. Severity and symptoms vary enormously from one patient to another,” says Shirley Watson, Founder of the ITP Support Association. “It is exciting news that a new drug is in development for ITP sufferers whose condition requires treatment. As eltrombopag stimulates platelet production rather than preventing platelet destruction, it offers a different approach for those with chronic refractory ITP.”
Also during the EHA, Joseph A Erhardt of GlaxoSmithKline gave an oral presentation of data (abstract 1024) showing that in vitro stimulation of platelet production by eltrombopag induces the same level of platelet function as those produced in vivo.2
“As we move into Phase III studies, the development of eltrombopag treatment represents a significant milestone for GSK Oncology in supportive care and underscores our continued commitment to delivering novel treatments to potentially meet a significant unmet medical need,” says Paolo Paoletti, MD, Senior Vice President, Oncology Medicine Development Centre, GSK.
About Eltrombopag
Eltrombopag is an investigational small-molecule thrombopoietin receptor agonist that has been shown in pre-clinical research and clinical trials to stimulate the proliferation and differentiation of megakaryocytes, the bone marrow cells that give rise to blood platelets, and thus may be considered a platelet growth factor. Because it is a small molecule, eltrombopag is administered orally as a tablet and may have less potential than large protein molecules for causing an immune system reaction.
Eltrombopag was discovered as a result of a research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. It is being developed by GlaxoSmithKline.
About Thrombocytopenia and ITP
A reduction in platelet count is the defining characteristic of any type of thrombocytopenia and diagnosis can be confirmed following a routine blood test. Thrombocytopenia occurs in 5% to 10% of all patients hospitalized for any cause.3
Idiopathic thrombocytopenic purpura is an autoimmune disorder that is marked by platelet destruction and/or inadequate platelet production. Primary ITP is estimated to affect 50 to 100 new persons per million per year in the United States and Europe.4 Some patients with ITP are asymptomatic or have mild bruising while others develop mucosal bleeding that can become severe.5 ITP is associated with a 5% fatality rate largely due to intracranial hemorrage.6
Thrombocytopenia also can occur as a consequence of chemotherapy treatment, interferon treatment, or chronic liver disease. Thrombocytopenia can impede a variety of medical treatments. It can prevent cancer patients from receiving their full dose of chemotherapy, prevent patients with hepatitis C infection from receiving interferon therapy or lead to dose reductions or discontinuation, and it can prevent or complicate procedures (surgical, dental etc.) in patients with chronic liver disease and ITP.
About GlaxoSmithKline
GlaxoSmithKline is one of the world’s leading research-based pharmaceutical and healthcare companies. GlaxoSmithKline is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information visit gsk.
Information about trial enrolment can be obtained by visiting itpstudy or clinicaltrials.
Cautionary statement regarding forward-looking statements
Under the safe harbor provisions of the US Private Securities Litigation Reform Act of 1995, the company cautions investors that any forward-looking statements or projections made by the company, including those made in this Announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect the Group’s operations are described under ‘Risk Factors’ in the ‘Operating and Financial Review and Prospects’ in the company’s Annual Report 2005.