An eye drop for macular degeneration shows promise in animal studies

Drugs such as Roche’s Lucentis and Regeneron’s Eylea help slow down the progression of age-related macular degeneration (AMD), a leading cause of blindness in people over age 65. But those medicines have to be injected into the eye, making them inconvenient for patients.

Scientists at the University of Birmingham have been developing an alternative treatment—this one in the form of an easy-to-use eye drop. After proving the drop has a similar therapeutic effect as injections do in rodents last year, the team began investigating the drug in rabbits and pigs, which have eyes that are similar to those of humans.

The team discovered that the drop can deliver an effective amount of AMD-fighting drug to the larger mammals. They reported their results in the journal Investigative Ophthalmology & Visual Science.

The eye drop contains a peptide that can penetrate cells in the retina. It is now being developed by a startup in the U.S. called Macregen, according to a statement. The company is working with the University of Birmingham team to complete proof-of-concept studies.

"From the outset, we [realized] that delivering drugs through eye drops would mean that patients can administer their treatment themselves, and this would be less costly, save time for patients and healthcare providers, and reduce the potential complications that can arise from injections," said Felicity de Cogan, a research fellow at the University of Birmingham’s Institute of Microbiology and Infection, in the statement.

As successful as Lucentis and Eylea have been, improving on them has been challenging. The drugs work by inhibiting the protein VEGF, which subsequently plugs up the leaky blood vessels that are a hallmark of AMD. Regeneron tried combining its drug with the Bayer antibody nesvacumab, but disappointing trial results prompted the partners to scrap a planned phase 3 study last year.

Meanwhile, a race is on to develop alternate AMD therapies. Last year, New Jersey-based PanOptica raised $11 million in venture capital to move its AMD drop into phase 1/2 trials. And Gemini Therapeutics of Cambridge, Massachusetts, hauled in $42.5 million to develop AMD drug candidates that target genetic abnormalities behind the disease. More recently, a team led by the University of Southern California’s Roski Eye Institute reported promising results from a small clinical trial of a retinal implant they made from embryonic stem cells. They are testing the implant for the treatment of the "dry" form of AMD.

Macregen and the University of Birmingham team are aiming to start clinical trials of their eye drop next year. The trials could start as early as spring, the company predicted.