Ampio Pharmaceuticals, Inc., a clinical trial stage biopharmaceutical company focused on the development of therapies to treat prevalent inflammatory conditions, announced that further data analysis from the 12-week multi-center, placebo-controlled, double-masked randomized trial identified a reversal of pathological changes and a synergistic effect with other medication.

As previously reported, oral treatment with Optina (low-dose danazol) has been shown to be safe and confer significant improvements in visual acuity (VA) and reductions in central retinal thickness (CRT) in patients with diabetic macular edema (DME) when given the optimal dose.

An independent specialized ophthalmology company reviewed a representative set of images of the eye from the trial and identified positive changes in Optina treated patients compared to placebo. These changes included the reversal of complications such as cystic lesions and subretinal fluid.

Additionally, analysis revealed that 69 per cent of the patients in the Optina study received a standard of care medication that manages kidney-induced high blood pressure. The drugs referred to as ACE (Angiotensin Converting Enzyme) inhibitors or ARBs (Angiotensin Receptor Blockers) are commonly prescribed to diabetic patients. When this group received the optimal Optina dose, they showed a six-letter improvement compared to placebo (p = 0.01), regardless of whether or not they previously had anti-VEGF eye injections. There was a significant 34 micron reduction in central retinal thickness (CRT) over placebo (p = 0.02). 60 per cent of these eyes showed a restoration of at least one line of vision compared to only 27 per cent of placebo (p = 0.05).

Pamela Rath, MD, a practicing ophthalmologist and lead investigator in the OptimEyes trial, stated “After reviewing the data, there appears to be a very real change in a short amount of time in patients treated with Optina for diabetic macular edema. It will be nice to see longer-term data as well. But with the results we are seeing thus far, Optina should prove to be a nice adjunct to anti-VEGF eye injections therapy for DME and may replace injections in some patients.”

Michael Macaluso, Ampio Pharmaceuticals chief executive officer, stated “Diabetes is not an eye disease, but a systemic disease. The fact that we demonstrate synergy with other medications that treat diabetic complications, opens the door for broader use.” Ampio plans to publish this data and present it at various conferences.

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 causes 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 per cent 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 per cent of patients inflicted by the disease for 20 years or more.

OptinaTM (ultra-low dose danazol) is an oral therapy with few side effects that may delay the relentless progression to blindness in patients with DME. DME is a component of diabetic retinopathy that damages the eye. Palliative laser therapy and anti-VEGF 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.