A Guide to Research in Dry AMD

PHARMACEUTICAL

Acucela

On June 4, 2008, Acucela Inc. announced that it had dosed the first subject in a Phase I trial for its lead compound ACU-4429 (formerly ACU-02), an orally available small molecule being developed for the treatment of dry AMD. ACU-4429 modulates the visual cycle by significantly reducing the accumulation of the retinal related toxic by-product A2E, believed to damage retinal cells.

Dr. Ryo Kubota, Acucela's chairman and chief executive officer, says that, because the drug is a non-retinoid (not derived from vitamin A), it has the potential to be safe for a wide range of people, including young patients and women of child-bearing age. Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness, says ACU-4429 may also be a beneficial treatment for people with Stargardt disease.

In January 2010, following the success of the Phase I clinical trial, Acucela began recruiting participants with dry AMD for Phase II. Known as the ENVISION Clarity Trial, Acucela is planning to enroll at least 56 participants at multiple sites throughout the U.S. Participants will receive either the drug or a placebo.

For more information about the trial, contact Covance Clinical Research Unit in Dallas, Laura Lonsdale at (214) 920-9053, Covance Clinical Research Unit in Austin, David Carter at (512) 302-6519, or visit www.clinicaltrials.gov and search for "Acucela."

More about Acucela.

Aspirin

Aspirin appears to be beneficial as a preventer of AMD. More about aspirin and AMD.

Carbon dioxide

AMD patients are benefitting from a new drug therapy involving a combination of medicines which includes carbon dioxide. More about carbon dioxide therapy.

Fenretinide

Fenretinide (ST-602), a drug that has been used to treat certain cancers, rheumatoid arthritis, acne, and psoriasis, has been found to also slow the production and accumulation of a toxin that leads to vision loss in Stargardt's patients. The toxin, called A2E, is a byproduct of vitamin A, the formation of which encourages production of waste deposits called lipofuscin. These deposits accumulate in the retinal pigment epithelium (RPE), interfering with the RPE's ability to nourish the photoreceptors. More about fenretinide.

Iluvian (fluocinolone acetonide)

Iluvian is a corticosteroid that has shown signs of preventing macular degeneration in animals. PSivida Ltd. has begun an early-stage clinical trial for testing a tiny implanted insert called Medidur for delivery of the drug to the retinas of patients with bilateral geographic atrophy (dry AMD).

OT-551

On February 26, 2007, Othera Pharmaceuticals presented new preclinical data demonstrating the safety and effectiveness of OT-551, an experimental drug in eye drop form for treatment of geographic atrophy (end stage dry AMD). Results from the Phase I trials demonstrated that when the compound is added to either Lucentis or Avastin treatment there is a synergistic effect versus either treatment alone. According to Dr. Len Parver, Othera's Medical Director, "OT-551 could potentially improve the outcome of patients already on Lucentis by treating the underlying macular degeneration and decreasing the need for frequent Lucentis injections."

On April 8, 2009, Othera announced positive interim data results from its Phase 2 trial of OT-551. The 12-month findings from the 2-year trial suggest an emerging trend for reducing moderate vision loss (i.e. 15 letters or more on the ETDRS chart) in treated patients compared to the placebo group.