GREENWOOD VILLAGE, Colo., Oct. 7, 2013 /PRNewswire/ -- Ampio Pharmaceuticals, Inc. (NYSE MKT: AMPE) today announced interim results from the ongoing 450 patient, dose finding, 505 (b) 2 study of OptinaTM 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 thirty percent (30%) of patients had reached the first 4 week time point and their clinical results were considered representative data for the trial.
Dr. Vaughan Clift, Ampio's Chief Regulatory Officer explained: "As is common with interim analyses of this sort, the analysis parameters were defined a priori and could have resulted in three possible scenarios as follows: 1) Termination of the study due to lack of efficacy or safety concerns, 2) delayed analysis if no conclusion could be drawn or 3) 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 and stated:
"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".
Dr. Clift further explained "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 OptinaTM for all patients who have completed the trial and wish to continue treatment for an additional 12 weeks. Patients who received either dose of OptinaTM in the masked trial as well as those who received placebo will be eligible for inclusion in this Open Label Extension of the Trial.
"We are very pleased with both the IDRC recommendation and the rapid patient enrollment, which now exceeds 250 patients. We expect the trial to be completed in the first quarter of 2014, and anticipate releasing top-line results as soon as they become available to Ampio. 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."
About the Optina Trial
Patient enrollment for the 450 patient US trial began in February 2013 and over 250 patients have been enrolled to date. The multicenter, dose-finding trial is designed to evaluate the safety and efficacy of oral OptinaTM compared with placebo over 12 weeks in adult patients with DME. Patients are randomized (1:1:1) to receive one of two oral doses of OptinaTM (0.5mg per BMI and 1.0mg per BMI per day) or placebo and are treated for 12 weeks. The primary endpoint of the Phase IIb trial is improvement in best-corrected visual acuity (BCVA) in treated patients compared to placebo at 12 weeks. Secondary endpoints are (i) measurement of changes in central macular thickness in treated patients compared to a placebo and (ii) safety and tolerability of the two Optina doses.
About Open Label Extension of Trial
Following the active treatment period, all patients will be followed for four weeks without treatment in order to study any regression and then immediately allowed the option to enter into a 12 week open label extension of the trial in which they will receive the optimal dose of OptinaTM as determined by this interim analysis after which time their improvement in BCVA will be evaluated.
OptinaTM is a low-dose formulation of danazol that we are developing to treat diabetic macular edema. Danazol is a synthetic derivative of modified testosterone ethisterone, and we believe it affects vascular endothelial cell linkage in a biphasic manner. At low doses, danazol decreases vascular permeability by increasing the barrier function of endothelial cells. The lipophilic low-molecular-weight weak androgen has the potential to treat multiple angiopathies.
About Diabetic Macular Edema
Type 1 and type 2 diabetes mellitus affects 26 million people in the United States. One of the many symptoms of diabetes is the local and systemic inflammation of the microvascular system. Diabetic retinopathy is a complication of diabetes and is characterized by damage to the blood vessels of the retina and can either be proliferative or non-proliferative. Proliferative damage occurs when a reduction in oxygen levels in the retina due to impaired glucose metabolism causes fragile blood vessels to grow in the vitreous humor. Non-proliferative damage occurs when existing vessels experience poor endothelial cell linkage due to increased blood glucose levels and hypertension. Macular edema is the most common form of non-proliferative diabetic retinopathy. In diabetic macular edema, prolonged hyperglycemia compromises endothelial cell linkage leading to vascular permeability. The leakage of fluid, solutes, proteins and immune cells cause the macula to swell and thicken. This leads to damage of the central retinal tissue and can significantly impair sharp central vision. The prevalence of diabetes is 11.3% of the population above the age of 20, with an annual incidence of 1.9 million cases in the United States alone. In this population, the prevalence of diabetic macular edema is estimated at 30% of patients inflicted by the disease for 20 years or more.
About Ampio Pharmaceuticals
Ampio Pharmaceuticals, Inc. is a development stage biopharmaceutical company primarily focused on the development of therapies to treat prevalent inflammatory conditions for which there are limited treatment options. We are developing compounds that decrease inflammation by (i) inhibiting specific pro-inflammatory compounds by affecting specific pathways at the protein expression and at the transcription level; (ii) activating specific phosphatase or depletion of the available phosphate needed for the inflammation process; and (iii) decreasing vascular permeability.
Forward Looking Statements
Ampio's statements in this press release that are not historical fact and that relate to future plans or events are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by use of words such as "believe," "expect," "plan," "anticipate," and similar expressions. These forward-looking statements include statements regarding Ampio's expectations with respect to the completion, timing and size of the registered direct offering, as well as risks associated with clinical trials, expected results, regulatory approvals, and changes in business conditions and similar events. The risks and uncertainties involved include those detailed from time to time in Ampio's filings with the Securities and Exchange Commission, including without limitation, under Ampio's Annual Report on Form 10-K and Quarterly Reports on Form 10-Q. Ampio undertakes no obligation to revise or update these forward-looking statements, whether as a result of new information, future events or otherwise.
Investor Contact: Rick Giles, Director of Investor Relations, Ampio Pharmaceuticals, Inc., Direct: (720) 437-6530 Email: [email protected]