• E-mail
• Print
• Comment
• Font Size
• Digg
• del.icio.us
• Discuss article

Icagen Reports Results of Phase II Open Label Extension Study of ICA-17043 for the Treatment of Sickle Cell Anemia

Posted on: Wednesday, 19 October 2005, 09:01 CDT

Icagen, Inc. (NASDAQ: ICGN) today reported the results of the Company's open label extension study to its Phase II study of ICA-17043 for the treatment of sickle cell anemia. The Phase II trial, the results of which have previously been reported, was a randomized, double-blind, placebo-controlled dose-range-finding study in 90 patients with sickle cell anemia conducted at 19 academic medical centers across the U.S. Following completion of the 12-week treatment period, and, for some patients, an eight week washout period, patients were offered the opportunity to enroll in the open label extension study, provided the study site had previously obtained institutional review board approval for such extension study. Of the 56 patients eligible to enroll in the open label extension study, 44, or 79%, elected to do so.

The open label extension study provided for a daily dose of 10 mg of ICA-17043 for a period of up to 48 weeks beyond completion of the Phase II study. All patients in the open label extension study received active study medication. In addition, some patients remained on concomitant hydroxyurea therapy, provided they had been on hydroxyurea therapy prior to entering the Phase II study. Unlike the Phase II trial, the open label extension study did not provide for an initial loading dose. The study was conducted primarily to evaluate further the long-term safety, and secondarily to evaluate descriptive evidence of efficacy, of this novel potential therapy. Since the study had no concurrent placebo control group, no formal statistical tests were performed.

ICA-17043 was generally well tolerated during this open label extension study. There were no serious adverse events that were attributed to ICA-17043. Of the 44 patients who enrolled in this study, 32 completed the 48-week treatment period, with only two patients discontinuing participation in the study as a result of adverse events that were considered possibly or probably related to study medication, one following a reversible increase in the level of gamma glutamyl transferase (GGT), an enzyme associated with the liver and biliary tract, and another following a diagnosis of interstitial nephritis. The only adverse events that occurred in two or more patients considered possibly related to study medication were GGT elevation, rash and headache.

During the Phase II study, ICA-17043 was demonstrated to improve the hematologic profile in patients with sickle cell anemia. In the open label extension study, hematologic data was collected during treatment for 42 of the 44 patients. Since no placebo group was available during this study for comparison, the last hematologic values collected while patients were taking ICA-17043 were compared to the beginning of Phase II study baseline values. Analysis of these hematologic parameters suggested that the pattern of beneficial effects of ICA-17043 observed during the Phase II study were maintained during the open label study. These effects included an increase in hemoglobin, hematocrit, and red blood cell count, as well as a decrease in dense cells, reticulocytes, bilirubin and lactate dehydrogenase (LDH).

Because there was no concurrent placebo control arm, there was no comparison group available in the open label extension study to appropriately assess the effect of ICA-17043 on vaso-occlusive crisis rates or average monthly pain intensity scores. Moreover, more than half of the patients in the open label extension study had a history of no crises or only one crisis in the year prior to the Phase II study, making it difficult to detect improvements in crisis rate in these patients. Accordingly, a descriptive analysis was performed for the 44 enrolled patients comparing the number of crises observed during the treatment phase of the open label extension study, as defined by the study protocol, to the number of crises during the year prior to enrollment in the Phase II study, as reported by patient recall. In this analysis, the historical number of crises in the one year period prior to the Phase II study was adjusted to account for the missing time on treatment in the open label extension study for those patients who dropped out of the open label extension study before its completion, as well as for the difference in the length of the measurement period between the 48-week treatment period of the open label extension study and the one year period of the historical data. Under this analysis, there were fewer crises during the open label extension study in comparison to the historical number of crises, especially in those patients with a history of two or more crises in the year prior to the Phase II study. Given that crises in this study were not adjudicated by an independent committee as was done in the Phase II study, and that there were a small number of self-selected patients, these descriptive results must be interpreted with caution. Average monthly pain intensity scores were relatively stable throughout the duration of the study.

"We are pleased to have successfully completed the Phase II open label extension study," stated P. Kay Wagoner, President and CEO of Icagen. "This study provides important long-term safety data, which further supports the favorable safety and tolerability profile of ICA-17043 that we have observed to date. Although not designed as an efficacy study, we were also pleased to note that the consistent pattern of improvements in hematologic parameters observed during the Phase II study were also seen in this open label extension study. Supporting the findings of our Phase II study, these results suggest that block of the Gardos channel with ICA-17043 results in a sustained reduction in hemolysis and improvement in anemia in patients with sickle cell anemia. We were also pleased to note a reduction in the number of crises, particularly in those patients with a history of two or more crises in the year prior to enrollment in the Phase II study. However, a placebo controlled trial such as our pivotal Phase III ASSERT study, which is currently underway in conjunction with McNeil Consumer and Specialty Pharmaceuticals, remains essential in determining the potential benefit of ICA-17043 on crisis rate."

About Sickle Cell Disease

Sickle cell disease is a chronic and debilitating genetic blood disorder, primarily affecting individuals of African descent, resulting in a variety of disease complications and a significantly shortened lifespan in the majority of patients. Sickle cell disease is the most common genetic disease among individuals of African descent and is prevalent worldwide. According to the American Medical Association, there are approximately 100,000 patients with sickle cell disease in the United States.

About Icagen

Icagen, Inc. is a biopharmaceutical company based in Research Triangle Park, North Carolina, focused on the discovery, development and commercialization of novel orally-administered small molecule drugs that modulate ion channel targets. Utilizing its proprietary know-how and integrated scientific and drug development capabilities, Icagen has identified multiple drug candidates that modulate ion channels. The Company's four most advanced programs are:

-- ICA-17043 for sickle cell disease, for which the Company is conducting a pivotal Phase III clinical trial;

-- a compound for atrial fibrillation, which is being developed by the Company's collaborator Bristol-Myers Squibb Company and is in Phase I clinical trials;

-- lead compounds for epilepsy and neuropathic pain, for which the Company is conducting preclinical studies; and

-- a compound for dementia, including Alzheimer's disease, which is being developed by the Company's collaborator Astellas Pharma, Inc., formerly Yamanouchi Pharmaceutical Co., Ltd., and is undergoing advanced preclinical testing.

Icagen is also conducting ongoing drug discovery programs focused on new therapeutics for pain disorders, inflammatory disorders and glaucoma.

Forward Looking Statements

This press release contains forward-looking statements that involve a number of risks and uncertainties. For this purpose, any statements contained herein that are not statements of historical fact may be deemed to be forward-looking statements. Without limiting the foregoing, the words "believes,""anticipates,""plans,""expects,""intends," and similar expressions are intended to identify forward-looking statements. Important factors that could cause actual results to differ materially from the expectations described in these forward-looking statements are set forth under the caption "Certain Factors That May Affect Future Results" in the Company's most recent Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission on August 15, 2005. These risk factors include risks as to whether the Company's products will be successful in the clinical trials process, the timing of such clinical trials, whether the results obtained in preliminary studies will be indicative of results obtained in clinical trials, whether the clinical trial results will warrant continued product development, whether and when, if at all, the Company's products, including ICA-17043, will receive approval from the U.S. Food and Drug Administration or equivalent regulatory agencies, and for which indications, and if such products receive approval, whether they will be successfully marketed; the Company's history of net losses and how long the Company will be able to operate on its existing capital resources; and the Company's dependence on third parties, including manufacturers, suppliers and collaborators. We disclaim any intention or obligation to update any forward-looking statements as a result of developments occurring after the date of this press release.

Source: Business Wire

More News in this Category