Ampio

What is Optina™
Optina™ is a low dose version of danazol. A completely unexpected beneficial effect was discovered by Dr. David Bar-Or and is being studied as a new medication to prevent leakage from small blood vessels caused by inflammation in many diseases including Diabetes Mellitus.

What does Optina™ treat?
Leakage from small blood vessels is often the early phase of inflammatory diseases. In Diabetes, for example, leakage from the small blood vessels at the back of the eye (macula) causes loss of vision which can become permanent.

Several diseases such as diabetic nephropathy (kidney disease), age related macular degeneration (a common cause for blindness), allergic rhinitis (hay fever) and many other conditions are all associated with vascular leakage and are potential targets for Optina™.

What is a Phase II clinical trial?
Once the initial safety of the study drug has been confirmed in Phase I trials, Phase II trials are performed on larger groups of patients and are designed to assess how well the drug works, as well as to continue safety assessments. Phase II studies involve both dose ranging (how much drug should be given) and efficacy (how well the drug works).

Optina™ is based on a drug with an extensive safety record (approved and used for other indications for decades) and is administered at a fraction of the previously approved dose.

Because danazol is an approved and marketed drug in many countries including the U.S., the approval process for Optina™ will fall under special legislation which shortens the path to approval compared to New Chemical Entities (NCEs) which require larger and more comprehensive studies. This approach takes advantage of the already known safety and side effects of the drug and bypasses the lengthy trials required to establish safety.

Drugs that fall into this category may be approved with only a few hundred patients, in trials lasting months and not years.

What clinical trials is Ampio conducting with Optina™?
Optina™ is being tested in patients with Diabetic Macular Edema (DME) in a Phase II clinical trial. DME is a common complication of Diabetes Mellitus. The high glucose levels encountered in diabetes trigger biochemical changes that lead to leaky blood vessels resulting in a swollen macula (the visual center of the eye). When this happens in the eye it eventually leads to permanent damage. Dr. Bar-Or discovered that, at very low doses, Optina™ has the effect of preventing the leakage.

The study’s main goal is to assess the efficacy and safety of two oral doses of Optina™ in patients with early signs of DME. The efficacy is being measured by standard tests including visual acuity improvement.

What is a New Chemical Entity (NCE)?
This is a general term used by many regulatory bodies in different countries to refer to a drug that has never been approved. Unlike Optina™, most drugs are created in the laboratory or extracted from plants and are entirely new compounds. Regulators need extensive evidence of safety in animals and humans often involving thousands of subjects to allow progression to Phase II.

Is Optina™ protected by patents?
Yes, Ampio owns an extensive patent portfolio surrounding Optina™ and its uses.

What side effects does Optina™ have?
Optina™ is anticipated to have fewer side effects than danazol (the active ingredient of Optina™) which has been given to tens of thousands of patients for nearly 35 years. When administered for the currently approved conditions, danazol is used at much higher doses than Optina™ and still, the side effects are uncommon and generally benign.

What are the competitors to Optina™?
Laser therapy and Lucentis^® are the major currently known potential competitors to Optina™. Laser therapy is the standard of care and slows the progress of the disease but does not stop the eventual blindness. It also causes damage to the retina.

Lucentis^® was recently approved in Europe for the treatment of DME. It is an effective therapy that requires injection directly into the eye several times a year, is relatively expensive and has been associated with the rare destruction of the eye due to complications related to the injection.

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