Regenxbio has guided its Duchenne muscular dystrophy (DMD) gene therapy candidate through another test, reporting a clean safety profile and functional improvements as it heads toward pivotal data.
The biotech expects to publish pivotal top-line data on the gene therapy, RGX-202, early in the second quarter. Wednesday, Regenxbio shared a new cut of data from the phase 1/2 trial that underpinned its decision to take RGX-202 into the pivotal study, generating more evidence that its gene therapy may be free from the liver toxicity issues that have affected Sarepta Therapeutics’ Elevidys.
None of the 13 patients in the interim phase 1/2 safety dataset had serious adverse events or adverse events of special interest—including drug-induced liver injury—up to 24 months after treatment. Mean levels of two liver damage biomarkers among the 10 patients who received the pivotal dose were below the upper limit of normal up to 24 months.
Regenxbio, which shared an earlier cut of the data in January, posted its updated safety results alongside the latest efficacy readout. The readout included one-year disease trajectory data, as measured on the North Star Ambulatory Assessment (NSAA), on more patients who received the pivotal dose.
