Healthcare Industry News: GSK
News Release - December 11, 2006
Trial Data Showed Promacta(TM) (Eltrombopag) Decreased Bleeding Episodes and Improved Platelet Counts in Patients With Chronic Idiopathic Thrombocytopenic PurpuraResults From Short-Term Phase II Study Demonstrated Potential for Promacta to Become First Oral Platelet Growth Factor
ORLANDO, Fla., Dec. 11 (HSMN NewsFeed) -- GlaxoSmithKline (GSK) today announced additional results from an international Phase II short-term, six- week study of its investigational non-peptide oral platelet growth factor, Promacta(TM) (eltrombopag). Analysis of data from this study showed that Promacta at 50mg or 75mg resulted in a positive trend in decrease in bleeding incidence (all grades) in adult patients with chronic idiopathic thrombocytopenic purpura (ITP), an autoimmune condition which results in low blood platelet counts and bleeding from the small blood vessels. These data were presented today at the 48th Annual American Society of Hematology (ASH) meeting in Orlando, Florida (abstract #475).(1)
The primary efficacy data from this study of (a statistically significant increase in platelet counts at 50mg and 75mg doses) were previously presented at the European Society of Hematology in June 2006.
"Current treatments for ITP, such as steroids or the removal of the spleen, have limitations and side effects," says James B. Bussel, MD, director of the Platelet Disorders Center, Children's Blood Foundation Division at the New York-Presbyterian Hospital/Weill Cornell Medical Center and principal investigator for this trial. "Bleeding events caused by ITP, whether categorized as moderate or severe, are of great concern to patients and physicians. These study results indicate there is a potential new oral treatment that can reduce bleeding episodes."
The Phase II trial was an international randomized, double-blind, placebo- controlled study that enrolled 117 adult patients with chronic ITP with baseline platelet counts of <30,000/microliter.(1) Patients were randomized to placebo, 30mg, 50mg or 75mg of Promacta once daily for six weeks. Bleeding events were assessed weekly during treatment and biweekly after treatment via the World Health Organization (WHO) bleeding scale and by adverse event reporting. The WHO bleeding scale measures bleeding severity from Grade 0 (no bleeding) through to Grade 4 (debilitating blood loss). The incidence of on therapy bleeding events regardless of grade and causality was 16%, 3% and 4% in the 30mg, 50mg and 75mg Promacta arms respectively, versus 10% in the placebo group. At doses of 30mg, 50mg and 75mg, the median platelet count on day 43 of treatment was 26,000/microliter, 128,000/microliter and 183,000/microliter respectively, in comparison to 16,000/microliter in the placebo group. Bleeding adverse events (AEs) included the following: hemorrhoids, hemorrhagic diarrhea and conjunctival hemorrhage in 3 patients receiving placebo; epistaxis, gingival bleeding and contusions in 5 patients receiving 30mg of Promacta; menorrhagia in 1 patient receiving 50mg of Promacta (who did not respond to Promacta); and contusion in 1 patient receiving 75mg of Promacta. The most common AE observed in this study was mild to moderate headache, reported in 21% of subjects on placebo, and 13%, 10% and 21% of subjects in the 30mg, 50mg and 75mg Promacta arms, respectively.(2) There was no apparent relationship between dose and incidence of AEs observed in this study.(3) Further studies will more fully characterize the safety profile.
"We are very excited by these results for Promacta, which show great promise for chronic ITP patients who failed different lines of treatment," said Paolo Paoletti, MD, Senior Vice President, Oncology Medicine Development Center, GSK. "Clinical trials for Promacta are ongoing, not only in ITP but also other conditions where thrombocytopenia is a clinical issue -- for example liver disease and patients receiving chemotherapy. Our robust clinical trial program reflects our continued commitment in delivering novel treatments to address unmet medical needs."
Promacta is an investigational non-peptide 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. Promacta is administered orally as a tablet and may have less potential than large protein molecules for causing an immune system reaction. The safety profile will be further examined in the ongoing clinical trials program. Promacta was discovered as a result of a research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. It is being developed by GlaxoSmithKline. Promacta is an investigational compound that has not received regulatory approval in any market for any indication at this time.
About Idiopathic Thrombocytopenia Purpura
ITP is an autoimmune bleeding disorder characterized by increased platelet clearance and/or inadequate platelet production, creating thrombocytopenia (low platelet count). Its cause is currently unknown. Primary ITP is estimated to affect 50 to 100 new persons per million per year in the United States and Europe.(3) People with ITP often bleed from small blood vessels underneath the skin causing bruises, nosebleeds, bleeding from the gums during dental work, or other bleeding that is difficult to stop. Bleeding in the brain or gastrointestinal (GI) tract is rare but can be life threatening if it occurs.(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% ten year fatality rate primarily from intracranial hemorrhage.(6)
A normal blood platelet count is greater than or equal to 150,000/microliter and less than or equal to 400,000/microliter.(7) A reduction in platelet count (to a level <150,000/microliter) 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.(8)
Thrombocytopenia can occur as a consequence of an autoimmune abnormality, chemotherapy treatment, interferon treatment, viral infection or chronic liver disease. Thrombocytopenia can impede a variety of medical treatments. It can prevent cancer patients from receiving their full dose of chemotherapy, preclude patients with hepatitis C infection from receiving interferon therapy or lead to dose reductions or discontinuation, and complicate surgical or dental procedures.
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 http://www.GSK.com.
For further information on Promacta trials please visit http://www.itpstudy.com or http://www.clinicaltrials.gov.
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.
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(1) Bussell, James, B., et al. Analysis of bleeding in patients with idiopathic thrombocytopenic purpura (ITP), a randomized, double-blind, placebo-controlled trial of eltrombopag, an oral platelet growth factor. Presented 11 December 2006, 48th Annual American Society for Hematology meeting, Orlando, USA.
(2) Newland, A, Cheng, G, et al. Eltrombopag increases platelets during 6-week treatment of ITP -- results of a randomized, double-blind, placebo- controlled phase II study. Presented 18 June 2006, 11th Congress of the European Hematology Association meeting, Amsterdam, the Netherlands.
(3) Cines DB, McMillan R. Management of adult idiopathic thrombocytopenic purpura. Annu Rev Med. 2005;56:425-442.
(4) National Heart, Lung, and Blood Institute. Signs and Symptoms Index. http://www.nhlbi.nih.gov/health/dci/Diseases/Itp/ITP_SignsAndSymptoms.html. Accessed Oct 19 2006.
(5) Stasi, R., Provan, D. Management of immune thrombocytopenic purpura in adults. Mayo Clin Proc. 2004;79:504-522.
(6) George, JN., Woolf, SH., Raskob, GE., et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood. 1996,88:3-40.
(7) http://www.nlm.nih.gov/medlineplus/ency/article/003647.htm. Accessed Oct 19 2006.
(8) Mocharnuk R. Growth factors in granulocytopenia and thrombocytopenia. Presented 44th Annual Meeting of the American Society of Hematology meeting, Philadelphia, USA.
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