A Phase 2 Clinical Trial for a New Glaucoma Treatment

photograph of retina showing glaucomatous cupping of the optic disc

Aerie Pharmaceuticals, Inc. has announced positive results for a Phase 2a clinical trial of experimental/investigational drug AR-13324 as a potential eye drop treatment for primary open-angle glaucoma.

About Aerie Pharmaceuticals

Aerie Pharmaceuticals, Inc., located in Bedminster, New Jersey and Research Triangle Park, North Carolina, is a privately held biotechnology company that is focused on the discovery and development of novel treatments for glaucoma. Aerie’s Phase 2a study enrolled 80 patients at 11 locations, from March to July, 2012, in the following states: California, Connecticut, Georgia, Kansas, Missouri, New York, Oklahoma, Pennsylvania, Tennessee, and Texas.

Aerie’s primary product, still in development, is AR-12286, which has also demonstrated its safety and effectiveness in ongoing clinical trials.

About Open-Angle Glaucoma

The most common type of glaucoma is primary open-angle glaucoma. This type of glaucoma occurs when the entrances to the eye’s drainage canals are clear, but a blockage develops within the canal, trapping the aqueous humor [i.e., a fluid contained within the eye] and causing an increase in intraocular [i.e., within the eye] pressure. Vision loss is usually slow and gradual and often there are no early warning signs. There is a strong genetic predisposition for this type of glaucoma.

Aqueous humor is a clear, watery fluid contained in a chamber behind the cornea that helps bring nutrients to the ocular tissue. It is made behind the lens and flows to the front of the eye, where it is drained by a tissue called the trabecular meshwork. Problems with the flow of this fluid can lead to problems with the pressure inside the eye.

About the Clinical Trial

From a Science Business news release:

The patients enrolled in the double-blinded trial had [either] open angle glaucoma, the most common form of glaucoma, or higher than normal intraocular pressure known as ocular hypertension.

The study tested the safety and tolerability of AR-13324 as well as its [effectiveness]. AR-13324 simultaneously lowers intraocular pressure by enhancing fluid outflow, while decreasing fluid inflow to the eye.

In the study, patients received one of three dosage levels of AR-13324 — 0.01, 0.02, or 0.04 percent — or a placebo. Patients received the drug once a day in the morning for seven days and were evaluated at four different time points during the day. The findings show patients taking the drug had lower intraocular pressure than patients taking the placebo. In addition, the patients testing the drug maintained those reductions over 24 hours.

Double-Blind Studies

In order to receive approval from the U.S. Food and Drug Administration (FDA), a new drug or treatment must be proven to be both safe and effective by undergoing a rigorous series of controlled unbiased studies. To prevent bias, neither the patient nor the examiners can know which patients receive the actual treatment and which are the untreated (or “control”) subjects.

These are called “double-blind” or “double-masked” studies and usually produce the most reliable results. The medication is coded and patients are placed at random into either the treatment or control group. When the study is concluded, the code is revealed and it is then possible to determine who received the actual drug or treatment, and who received the inactive substance, or placebo.

More about Clinical Trials

Most clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer:

  • In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and effective dosage range, and identify possible side effects.
  • In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to determine if it is effective and to further evaluate its safety.
  • In Phase III studies, the study drug or treatment is given to even larger groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
  • In Phase IV studies, after the Food and Drug Administration (FDA) has approved the drug, continuing studies will determine additional information, such as the drug’s risks, side effects, benefits, and optimal use.

Additional Information

VisionAware will provide updates on this important research as they become available. You can learn more about glaucoma risk factors, treatments, and resources at the VisionAware website.