MeiraGTx, J&J See Phase 3 Test Ahead for Vision Loss Gene Therapy
Summary
The condition, called X-linked retinitis pigmentosa, is part of a group of vision disorders that collectively represent the most common cause of inherited blindness. Secondary goals included retinal sensitivity, which after six months had improved or stabilized in five of the seven in the low and intermediate dose groups, according to the interim data. Most of the adverse events recorded were associated with the surgical procedure used to deliver the treatment, according to MeiraGTx; two of three patients in the high-dose cohort experienced inflammation, which was controlled with steroids. The interim results support advancing the treatment into a Phase 3 trial—a first for MeriaGTx, which was founded about five years ago, president and CEO Zandy Forbes said on a conference call Friday. J&J made a bet on the gene therapy pipeline being developed by MeiraGTx in January 2019, when the companies inked a $100 million deal giving the pharma giant rights to the biotech’s investigational XLRP treatment, plus others in its pipeline, as part of a pact to jointly develop and commercialize gene therapies to treat inherited retinal diseases.