J&J-partnered MeiraGTx aims at Biogen with new eye gene therapy data
Several months after Biogen touted the first proof-of-concept data from its $800 million eye gene therapy, MeiraGTx and J&J are out with their own early data for a rival treatment.
In a small Phase I/II trial for their gene therapy for the degenerative eye disease x-linked retinitis pigmentosa, MeiraGTx found that the 7 patients in the two lowest dose cohorts saw their vision improve on multiple measures for 6 months. The study will provide the basis for the New York biotech to move into a pivotal Phase III for the gene therapy that J&J paid $100 million to get in on last year.

“It’s really exciting data,” Jim List, Janssen’s global therapeutic head for cardiovascular and metabolic disease, told Endpoints News. “This shows the potential of the therapy to have an improvement in vision from this treatment, but also for this to slow or halt the progression of the disease, and that would be amazing.”
The retinitis program is MeiraGTx’s lead program and one of two major gene therapy plays for the disorder, which is generally diagnosed in adolescence and causes slow deterioration in vision for years after. Biogen spent $877 million last year to acquire Nightstar Therapeutics and their gene therapy program.
In January, Biogen published a similar set of Phase I/II data for Nightstar’s treatment in Nature Medicine, also showing that the drug was safe and that a subset of patients saw improvement that was sustained over 3 to 6 months.
Like Biogen, MeiraGTx also found though that a subset of patients experienced an inflammatory response. Although that response was quickly resolved with oral steroids, it appeared to interfere with the effectiveness of the drug. Patients on the highest dose of MeiraGTx’s trial did not see a strong improvement, and the biotech will conduct their Phase III with the low and intermediate dose.
The 4 patients on the intermediate dose arms saw a retinal sensitivity increase of 1.02 db, and an increase of central visual field progression rate at 1.26 db/year. Central visual field progression rate improved 1.10 db/year for the low doses. For the high dose, both of those numbers were negative.

Michel Michaelides, lead investigator and MeiraGTx’s scientific founder, said that Biogen did not look at some of the core measures of vision that they did. He also pointed to the difference in improvements between eyes. Only one of each patients’ eyes received therapy – but cautioned that cross-trial comparisons were inherently fraught.
“I’m not sure they have such a clear disparity between treated and untreated,” he said.
Michaelides also said patients reported improved vision, greater clarity, greater ease tracking signs and reading on a computer. There’s no placebo-control for those metrics yet and that will have to wait for Phase II. Still, Michaelides said the earlier trials have already started changing scientists’ goals.
“It wasn’t long ago where the ambition for degenerative diseases was to slow halt progression, and what we’re showing here is improvement,” he said. “That’s huge for patients.”