GLP Safety Studies

Iconic Therapeutics, Inc. initiated discussions with the Division of Anti-infective and Ophthalmologic Drugs to seek guidance on the proposed safety testing for hI-con1. The FDA recommended that Iconic should conduct intravitreal safety studies in the species where evidence of efficacy was demonstrated, i.e. the pig. The FDA specifically stated that monkey testing was not necessary. The need for systemic toxicology studies was also communicated by the FDA because of the extreme difficulty in demonstrating zero absorption from the eye. The FDA also indicated that Iconic would need to conduct a Tissue Cross Reactivity study.

Iconic submitted the study design summaries for the proposed GLP IVT and IV studies in mini-pigs. Comments were provided by an FDA pharmacology/toxicology reviewer. In response to those comments, both sexes (instead of one, as planned) were used in the IVT study and the high dose was increased by giving 2 injections over 3 days for each of the 3 monthly courses; the high IV dose was increased to a dose equivalent to complete absorption of the high IVT dose; and a study was conducted in a second species, the rabbit. The Tissue Cross Reactivity study was designed to measure the activity of hI-con1 against more than 20 normal tissue types taken from human cadavers.

Iconic engaged Charles River Laboratories to conduct the GLP IVT and IV safety studies in pigs and rabbits. Iconic engaged Comparative Biologics to conduct the Tissue Cross Reactivity study. Iconic has now successfully completed all its GLP IVT, IV and Tissue Cross Reactivity studies. Based on the results of these studies, Iconic has submitted an Investigational New Drug application (IND) with the FDA in 4Q10. It hopes to initiate phase 1/2a clinical studies in 4Q10.