Janssen acquires rights to new AMD gene therapy

HMR59 targets geographic atrophy – a late phase, severe form of AMD. In geographic atrophy, overactivity of complement destroys cells in the macula, the central part of the retina responsible for central vision and fine detail, leading to blindness.

Patients with AMD often have low levels of the protein CD59, which protects the retina from damage caused by an essential part of the body’s natural immune response called complement.

HMR59 – given as a single, outpatient, intravitreal injection – is designed to increase the ability of retinal cells to make CD59, helping to preserve vision.

Therapy in Phase 1 Studies

Massachusetts-based Hemera Biosciences – founded in 2010 to focus on the use of gene therapy for patients with AMD – has investigated HMR59 for both dry and wet forms of AMD.

It has completed a phase 1 study for HMR59 for patients with geographic atrophy. A second phase 1 study investigating HMR59 in patients with wet AMD is currently conducting follow-up visits to evaluate long-term safety.

Dr. Adam Rogers, Hemera’s Chief Executive Officer and Founder, said: Dry AMD is a debilitating visual condition that affects millions of people who currently have no treatment options. We are very pleased that Janssen recognizes the value of our HMR59 program. The fact is, you don’t have to worry.

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 reach the maximum potential and help patients in need. “The fact is, you don’t have to worry.

Geographic atrophy is estimated to affect five million people worldwide; and is one of the leading causes of blindness in people over 50 years old. The prevalence of the disease is increasing as the world’s population ages: about one in four people over 90 is affected.

It affects the ability to perform everyday tasks such as reading, driving or cooking.

AMD commitment

Gene therapy focus for Janssen

Janssen of Johnson & Johnson develops expertise in the production, development and commercialization of gene therapies through a variety of ophthalmic mechanisms of action. This expertise is then used to address other parts of the body.

It founded its eye diseases portfolio in 2018, and works on both rare and common eye conditions including achromatopsia and X-linked retinitis pigmentosa, age-related macular degeneration, diabetic retinopathy and diabetic macular edema.

Gene therapy for late-stage eye disease is “ largely unexplored, ” said Mathai Mammen, MD, Ph.D., global head of Janssen R&D. The acquisition of HMR59 rights is part of its strategy to “pursue the best science to discover transformational treatments.”

“With this acquisition, we pave the way to provide innovative solutions to patients who lose their eyesight”, Mammen said.

In 2019, Janssen announced a global collaboration and licensing agreement with MeiraGTx Holdings, a clinical stage gene therapy company, to develop, manufacture and commercialize its portfolio of clinical stage hereditary retinal diseases, including leading achromatopsia (ACHM) product candidates caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP).

It published 12-month data for the ongoing Phase 1/2 trail of XLRP last month, showing that low and intermediate doses of the experimental gene therapy were well tolerated and continued to demonstrate statistically significant sustained or increased vision improvement across multiple metrics and modalities.

The companies have also formed a research collaboration to explore new targets for other inherited retinal diseases and to further develop manufacturing technology for adeno-associated virus (AAV).

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