Icagen Inc. began a pivotal study of its lead product, ICA-17043, for sickle cell disease.
The company, which recently went public, will conduct the randomized, double-blinded, placebo-controlled trial in 300 patients. Called ASSERT, (A Stratified Sickle Event Randomized Trial), the study will be held at about 60 sites across the U.S. and in selected other countries.
Company officials could not be reached for comment.
Earlier this month, the company raised $40 million in its initial public offering, pricing 5 million shares at $8 apiece. (See BioWorld Today, Feb. 4, 2005.)
With that financial injection, the Research Triangle Park, N.C.-based company is moving forward with Phase III for ICA-17043. To be eligible, patients must range between 16 and 65 years old and have a sickle cell disease diagnosis, and their history must include at least two vaso-occlusive crises requiring a visit to a medical facility in the year prior to enrollment. Under the study protocol, they are to be treated for a year.
The study's primary endpoint is vaso-occlusive crisis rate.
A number of secondary endpoints also will be evaluated, including many that were analyzed in Phase II. Results from a 12-week study were reported late last year at the American Society of Hematology. The trial met its primary endpoint, the change in hemoglobin level from baseline, at a 10-mg dose (p<0.001). That increase was accompanied by a corresponding increase in hematocrit and red blood cell count, and ICA-17043 also demonstrated a dose-dependent and statistically significant improvement across several laboratory measures, such as dense red blood cells, reticulocyte count, LDH and indirect bilirubin. The company said that such measures, when taken together, provide evidence that ICA-17043 reduces red blood cell destruction and improves anemia in sickle cell patients.
Hemoglobin level is being measured as a secondary endpoint in the Phase III program.
The drug is partnered with McNeil Consumer & Specialty Pharmaceuticals, a division of McNeil-PPC Inc. and a unit of New Brunswick, N.J.-based Johnson & Johnson. The FDA has named the product an orphan drug and also has given it fast-track status.
In addition to ICA-17043, Icagen focuses on orally administered small molecules that modulate ion channel targets. It has two other products in clinical studies.
Phase I trials are under way for ICA-69673, a drug for epilepsy and neuropathic pain, as well as for a compound for atrial fibrillation. The latter is being developed in collaboration with Bristol-Myers Squibb Co., of New York.
Another partnered compound, for dementia such as Alzheimer's disease, is in advanced preclinical testing under the watch of Yamanouchi Pharmaceutical Co. Ltd., of Tokyo. Icagen's ongoing drug discovery programs are focused on pain disorders, inflammatory disorders and glaucoma.
On Tuesday, Icagen's shares (NASDAQ:ICGN) gained 29 cents to close at $7.10.