Janssen Acquires Rights to Novel Gene Therapy, Pioneering Treatment Solutions for Late-Stage Age-Related Macular Degeneration

Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, announced the acquisition of rights to Hemera Biosciences, LLC’s investigational gene therapy HMR59, administered as a one-time, outpatient, intravitreal injection to help preserve vision in patients with geographic atrophy, a late-stage and severe form of age-related macular degeneration (AMD). Financial terms of the transaction with Hemera Biosciences, a privately-owned biotechnology company, are not being disclosed.

Patients with AMD often have low levels of CD59, a protein that protects the retina from damage caused by an essential part of the body’s natural immune response called “complement.” In geographic atrophy, an overactivity of complement destroys cells in the macula, the central part of the retina responsible for central vision and seeing fine details, and results in a relentless progression to blindness. HMR59 is designed to increase the ability of retina cells to make a soluble form of CD59, helping to prevent further damage to the retina and preserve vision.

Geographic atrophy affects five millioni people globally, and is a leading cause of blindness in people over 50 years of age. The prevalence of geographic atrophy increases as the global population ages with roughly one in 29 people over age 75 affected, and nearly one in four people over age 90. There are currently no available therapies other than vitamins and low vision aids.

“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,” said James F. List, M.D., Ph.D., Global Therapeutic Area Head, Cardiovascular & Metabolism, Janssen Research & Development, LLC. “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.”

Gene therapy is an important modality for both therapeutic delivery and protein replacement, and one of the ways Janssen aims to significantly improve health outcomes for patients. Beginning with the eye, Janssen is rapidly developing expertise in the manufacturing, development, and commercialization of gene therapies across a range of mechanisms of action, building the case for future applications to other parts of the body.

“At Janssen, we pursue the best science to uncover transformational treatments that meet patient needs, which often means pioneering the solution. This is especially the case with gene therapy and late-stage eye diseases where the paths are largely uncharted,” said Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. “Through this acquisition, we are blazing the trail to bring innovative solutions to patients who are losing their vision.”

The Phase 1 study of HMR59 for patients with geographic atrophy is complete. A second Phase 1 study exploring HMR59 in patients with wet-AMD is currently conducting follow-up visits to evaluate long-term safety.