- Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson, said Wednesday that it has acquired rights to an experimental gene therapy meant to treat a severe type of age-related vision loss.
- The therapy, known as HMR59, comes as a one-time injection into the eye, and is supposed to help retinal cells make a protein that protects them against damage from an overactive immune response. HMR59 has completed a small study which tested it in patients with an advanced form of a disease called age-related macular degeneration, or AMD, and is currently heading into a mid-sized clinical trial of about 130 patients.
- Janssen didn't disclose financial terms of its deal with Hemera Biosciences, the privately-owned biotech that's been developing HMR59. Even so, the company has already spent more than $100 million on a collaboration with New York-based biotech MeiraGTx to establish a position in the field of eye gene therapy.
Compared to fellow pharma giants like Novartis, Roche and Pfizer, J&J has taken a more conservative approach to gene therapy. The company hasn't inked any multibillion-dollar acquisitions or launched any first-of-their-kind genetic medicines.
But as J&J wades further into the field, it's making its aims clear. The company's initial focus is ocular diseases, which have been attractive targets for gene therapy developers since the eyes can be treated with much smaller doses than other parts fo the body, thereby lessening some of the challenges associated with administration and manufacturing.
Janssen's first key move came early last year with the MeiraGTx collaboration, which gave it exclusive rights to commercialize a few of the biotech's gene therapies for inherited retinal diseases, or IRDs. This July, the partners announced results from a very small study indicating treatment with one of those therapies may help to slow or stabilize vision loss in patients with a condition called X-linked retinitis pigmentosa. The companies said they plan to advance that therapy into late-stage testing.
Now, Janssen has access to another clinical-stage gene therapy through the Hemera deal. A Phase 1 trial of HMR59 wrapped up last December, having tested the therapy in 17 patients with the much more common, "dry" form of AMD. The companies said in a Dec. 2 statement that a second Phase 1 study investigating HMR59 in "wet" AMD is conducting follow-up visits with patients to evaluate long-term safety. Hemera's treatment is one of several gene therapies in development for AMD, among them candidates from Regenxbio and Adverum Biotechnologies.
Janssen sees particular value in the therapy's potential to treat an advanced kind of AMD known as geographic atrophy. The company estimates geographic atrophy, which causes severe vision loss and is more common in older populations, affects five million people around the world.
Geographic atrophy has also caught the attention of Novartis, which earlier this year agreed to buy the small gene therapy developer Vedere Bio for $150 million up front. Novartis claimed Vedere's technology has the potential to treat geographic atrophy as well as all IRDs.