Ampio Pharmaceuticals, a development stage biopharmaceutical company, has announced interim results from the ongoing 450 patient, dose finding, 505(b)(2) study of Optina as a treatment for Diabetic Macular Edema (DME). This interim analysis was conducted by an Independent Data Review Committee (IDRC) comprised of a statistician and an ophthalmologist/retinologist, who were permitted to view the unmasked data from the trial. At least 30% of patients had reached the first four-week time point and their clinical results were considered representative data for the trial.
Dr. Vaughan Clift, Ampio's chief regulatory officer, said, "As is common with interim analyses of this sort, the analysis parameters were defined a priori and could have resulted in three possible scenarios, including termination of the study due to lack of efficacy or safety concerns, delayed analysis if no conclusion could be drawn or indication of an optimum dose if sufficient differentiation from placebo was present to indicate clinical benefit and no serious adverse events. The IDRC elected the third option.
IDRC said, "After a thorough review of the interim data from the ongoing study, it was determined that there was a treatment dosage that was demonstrating a potentially beneficial anatomic effect. Given that there were no significant safety concerns identified in the study to date, a recommendation to continue the trial was made."
Clift said, "This IDRC decision will allow for the continuation and completion of the trial and, importantly, the immediate initiation of an open label extension study using this optimum dose of Optina for all patients who have completed the trial and wish to continue treatment for an additional 12 weeks. Patients who received either dose of Optina in the masked trial and those who received placebo will be eligible for inclusion in this open label extension of the trial.”
Patient enrollment now exceeds 250 patients. The trial is expected to be completed in the first quarter of 2014. FDA has agreed to the 505(b)(2) pathway for this drug which, given no significant safety concerns, could allow approval based on this single clinical trial.