By acquiring the rights to Hemera Biosciences’ gene therapy candidate to treat geographic atrophy – the debilitating end-stage form of dry age-related macular degeneration – Johnson & Johnson’s Janssen Pharmaceuticals unit has made another strategic move to build its retina portfolio with novel therapies, as James F. List, MD, PhD, head of the Janssen unit overseeing the ophthalmology franchise, explained to OIS Weekly.
“We are entering ophthalmology from a pharmaceutical point of view driven by the high unmet needs of the space and the fact that we see very exciting science that potentially could meet that unmet need,” said Dr. List, global therapeutic area head of cardiovascular and metabolism of Janssen Research and Development, which oversees the company’s ophthalmology drug portfolio. “Like any space that we go into, we go in with the intention of being leaders, and retinal disease is no exception.”
He said the acquisition last week of rights to Hemera’s HMR59, a potential one-time intravitreal treatment for geographic atrophy (GA), fits that strategy.
Janssen’s busy year
The move caps a busy year in retina for the J&J unit. In January, Janssen entered into a strategic collaboration agreement with Exonate, an early-stage biotech that’s been developing an eye drop that uses messenger RNA (mRNA) to target age-related macular degeneration (AMD) and diabetic macular edema (DME). Its collaboration with MeiraGTx, which has been developing treatments for inherited retinal disease, resulted in a positive 12-month trial readout of the X-linked retinitis pigmentosa program.
Last month, J&J agreed to acquire Momenta Pharmaceuticals for $6.5 billion. Momenta is developing novel therapies for immune-mediated diseases, and its portfolio also includes M710, a proposed aflibercept biosimilar. This acquisition isn’t within Dr. List’s purview.
The recent moves in retina build on what Dr. List called J&J’s “heritage” in eye care, which harkens back 30 years to when the company completely disrupted the contact lens industry with disposable contact lenses. “Our entry into retina brings us all the way from the very front of the eye to the very back of the eye now and brings us into all three sectors – consumer, devices, and pharmaceutical – and as the world’s largest healthcare company, that makes sense,” he said.
Seeking transformational science
Hemera is a prime example of what Janssen aims to accomplish in retina, Dr. List added. A treatment for GA – for which no approved therapies exist – has been considered the holy grail in retina. “The only therapies that we have right now are vitamins, and of course, low-vision aides, so the opportunity to make a difference for these patients, to slow down the growth of the lesions in these patients, is tremendous,” he said. “It plays into one of the spaces where there’s a huge unmet need.”
It also plays into the sort of transformational science that interests Janssen, Dr. List noted. Much research in AMD has focused on disrupting the complement cascade – the biological chain reaction that leads to inflammation and, eventually, geographic atrophy. But the monthly or quarterly injections of anti-VEGF (vascular endothelial growth factor) drugs for retinal disease pose a burden for many patients.
The Hemera and MeiraGTx platforms utilize gene therapy for one-time treatments, what Dr. List called “one of the very transformational aspects.” He added, “This allows us to further grow our gene therapy platform.”
Entrée to gene therapy
The retina space also gives Janssen its entrée into gene therapy, Dr. List said. The MeiraGTx collaboration “gives us an opportunity to expand our gene therapy into a more common disease,” he said. “In this case, it’s not that a gene isn’t working, but it’s that we can express a therapeutic protein in the eye and leverage our growing expertise in gene therapy to be able to do that.”
The collaboration with Exonate to develop a topical drop to deliver drugs to the back of the eye extended Janssen’s retina portfolio beyond inherited retinal disease and AMD to target diabetic eye disease. “The idea there is a different approach to reduce the burden of injections into the eye for VEGF-driven diseases,” Dr. List said.
Janssen also has interest in developing treatments for glaucoma, Dr. List added. “People think of glaucoma as a pressure disease, but essentially intraocular pressure is the risk factor,” he noted. “Glaucoma is a retinal disease with loss of the ganglion cell neurons, so that’s another area that we’re quite interested in.”
Off and running
The collaborations in retina mean “we’ve started off hitting the ground running,” said Dr. List. Janssen is also building a discovery group in South San Francisco, CA (which, incidentally, is home to Genentech). Former National Eye Institute senior investigator Wai T. Wong, MD, PhD, has been brought in to lead the group, Dr. List added.
J&J, Hemera, and Exonate haven’t disclosed financial terms of the deals. BioSpace valued the MeiraGTx collaboration at $440 million.