Janssen acquires rights to novel AMD gene therapy
HMR59 is targeted at geographic atrophy: a late-stage, 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 seeing fine details, progressing 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 – administered as a one-time, outpatient, intravitreal injection – is designed to increase the ability of retina cells to make CD59: therefore helping preserve vision.
Therapy in Phase 1 studies
Massachusetts-based Hemera Biosciences – which was founded in 2010 to focus on using gene therapy for patients with AMD – has been investigating 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 exploring HMR59 in patients with wet-AMD is currently conducting follow-up visits to evaluate long-term safety.
Dr. Adam Rogers, Chief Executive Officer 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.
“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.”
It is estimated that geographic atrophy affects five million people globally; and is one of the leading causes of blindness in people over 50 years old. The prevalence of the disease increases as the global population ages: around one in four people over 90 are affected.
It affects the ability to accomplish everyday tasks such as reading, driving or cooking.
Gene therapy focus for Janssen
Johnson & Johnson’s Janssen is developing expertise in the manufacturing, development, and commercialization of gene therapies across a range of mechanisms of action for the eye. This expertise will then be used to address other parts of the body.
It established its eye diseases portfolio in 2018: and is working across both rare and common eye diseases: including achromatopsia and X-linked retinitis pigmentosa, age-related macular degeneration, diabetic retinopathy, and diabetic macular edema.
Gene therapy for late-stage eye diseases is ‘largely unchartered’, according to Mathai Mammen, M.D., Ph.D., global head of Janssen R&D. Its acquisition of HMR59 rights is part of its strategy to 'pursue the best science to uncover transformational treatments'.
"Through this acquisition, we are blazing the trail to bring innovative solutions to patients who are losing their vision," said Mammen.
In 2019, Janssen announced a worldwide collaboration and license agreement with MeiraGTx Holdings, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical stage inherited retinal diseases portfolio, including leading product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP).
It released 12 month data for the ongoing Phase 1/2 trail of XLRP last month, showing that low and intermediate doses of the investigational gene therapy were well-tolerated and continued to demonstrate statistically significantly sustained or increased vision improvement across multiple metrics and modalities.
The companies also have formed a research collaboration to explore new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology.
