ENGLEWOOD, Colo., Oct. 14, 2015 /PRNewswire/ — Ampio Pharmaceuticals, Inc. (NYSE MKT: AMPE) today announced that Ampio met with the Division of the Transplant and Ophthalmology Products of the FDA to discuss the results of the OptimEyes clinical trial of Optina™ and to seek guidance on the next steps to approval.
The guidance from the FDA was:
- Ampio perform a confirmatory study on patients with DME who are refractory to the currently available drugs, which if successful, would qualify Optina™ as a rescue medication for patients who have no treatment options (failed available therapies).
- The study will have significantly less patients than in our previous OptimEyes study, based on power calculations and guidance received from the FDA, and will include approximately 80 patients randomized 1:1 between placebo and Optina™.
- Optina™ will be compared to placebo, not to other anti-vascular endothelial growth factor (VEGF) drugs, since we are addressing a population that failed these alternative treatments.
- The FDA will consider improved vision as measured by BCVA (Best Corrected Visual Acuity), which is statistically and clinically meaningful, as determined by experts in the field.
- The duration of the study will be a maximum of 12 months.
A large number of patients who suffer from DME and who are either non-responsive to available therapies or who have contra-indications to intra-ocular therapies (such as glaucoma, post vitrectomy etc.), have very limited treatment options and are progressing toward blindness.
The FDA acknowledged that Optina™ qualifies as a drug in the 505(b)2 regulatory pathway, thus the safety profile for Danazol is already established at doses more than ten times higher than the Optina™ dose.
About Diabetic Macular Edema (DME)
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.
Optina™ (ultra-low dose danazol) is an oral therapy with few side effects that may delay the relentless progression to blindness in patients with Diabetic Macula Edema (DME). DME is a component of Diabetic Retinopathy that damages the eye. Palliative laser therapy and anti- VGEF injections in the eye are the only approved therapies. For some fraction of the DME patients, Optina™ could be an invaluable addition to existing therapies particularly in those patients no longer responding to the approved drugs.
About Ampio Pharmaceuticals
Ampio Pharmaceuticals, Inc. is a clinical trial 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.