Spark Therapeutics

Spark Therapeutics IncONCE revealed Friday the publication in "The Lancet" of long-term data from a first stage study of voretigene neparvovec (SPK-RPE65), its most advanced product candidate. Incidentally, the drug candidate received breakthrough therapy and orphan product status for the treatment of inherited retinal disease (IRD) caused by mutations in the RPE65 gene.

Spark Therapeutics said the publication detailed safety and efficacy results in 11 subjects, who underwent administration of voretigene neparvovec to the contralateral, or second previously uninjected eye. The company added that every subject participated in an earlier first stage trial in which the worse of the two eyes were injected with voretigene neparvovec.

Related Link: Cantor Fitzgerald Sees 81% Upside In Spark; Initiates At Buy

The company said that the publication was the first to report complete cohort results of injection of the second eye with an AAV vector, and illustrated the overall safety of the approach. Aside from that, the data demonstrated long-term stable and persistent gain for a minimum of three years, with observation ongoing, in retinal and visual function and functional vision after voretigene neparvovec injection.

Spark's CEO, Jeffrey Marrazzo, commented, "We remain encouraged about the durability of benefit shown, underscoring the utility this treatment may have for patients suffering from this debilitating, blinding disorder."

The company indicated that it intends to present efficacy analyses of both the initial one-year post and two-year follow data from the intervention subjects in the pivotal final stage study in the third quarter. Moreover, the company plans to provide efficacy analyses on the four-year follow-up data from the relevant cohort of the second Phase 1 trial.

At time of writing, Spark was up 2.44 percent at $52.38.