Geographic atrophy treatment : Janssen Pharmaceuticals has signed a licensing deal to acquire rights to HMR59, an investigational gene therapy for geographic atrophy, from clinical stage ocular gene therapy company Hemera Biosciences for an undisclosed price.

HMR59 is intended to be given as a one-time, outpatient, intravitreal injection for helping in preserving vision in patients having geographic atrophy. The intravitreal AAV gene therapy is being studied for the treatment of dry age-related macular degeneration (dry AMD).

Geographic atrophy is a late-stage and severe type of age-related macular degeneration.

In patients with age-related macular degeneration, the levels of the CD59 protein are low. The protein protects the retina from damage caused by an essential part of the natural immune response of the body called “complement.”

In geographic atrophy, an overactivity of complement damages cells in the macula, the core part of the retina, which is responsible for central vision and seeing fine details. Geographic atrophy if left untreated, could lead to relentless progression to blindness.

Geographic atrophy treatments

Presently, there are no available therapies for geographic atrophy, other than vitamins and low vision aids.

Dr. Adam Rogers – CEO and Founder of Hemera said: “Dry AMD is a debilitating visual disease that affects millions of people with no currently available treatment options. We are very excited that Janssen recognizes the value of our HMR59 program and sees this transaction as an important moment in furthering the goal of Hemera to provide a single injection treatment for patients with dry AMD.

“Janssen is a recognized leader in the pharmaceutical industry, with extensive manufacturing, clinical, regulatory and commercial expertise. As part of Janssen’s organization, HMR59 will be best positioned to achieve its maximum potential and benefit patients in need.”

HMR59 mechanism of action for geographic atrophy treatment

HMR59 gene therapy candidate has been designed to boost the ability of retina cells to make a soluble form of CD59, thereby helping to prevent more damage to the retina and preserve vision.

A phase 1 study of the intravitreal AAV gene therapy for patients with geographic atrophy has been completed. A second phase 1 study for HMR59 in patients having wet-AMD is currently undertaking follow-up visits for assessing its long-term safety.

James F. List – Global Therapeutic Area Head, Cardiovascular & Metabolism, Janssen Research & Development said: “Geographic atrophy is a devastating form of AMD that impacts the ability to accomplish everyday tasks, such as reading, driving, cooking, or even seeing faces.

“Our aim with this novel, single-administration gene therapy is to use our development expertise and deep heritage in vision care to help improve patient outcomes by intervening early, halting the progression to blindness, and preserving more years of sight.”

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