Playing catch-up, Roche outlines plans for a PhIII hemophilia A program — next year
For now, the big focus in the hemophilia A gene therapy race has been centered on frontrunner BioMarin, with an expected OK just weeks away, and the crew from Sangamo/Pfizer, which has been looking to play catch-up with a pivotal study in the works.
But now Roche’s new gene therapy division at Spark has updated its own schedule, saying they’re looking at a Phase III start sometime next year as they optimize the dose and immunomodulatory regimen in order to avoid any future mishaps that could derail the program.
We already knew that 2 of 9 participants in the 2×1012 dose cohort “lost FVIII expression likely due to a capsid-based immune response,” says the company. “The seven other participants in the 2×1012 dose cohort and the five total participants in the 5×1011 vg/kg and 1×1012 vg/kg dose cohorts continue to show stable and durable factor FVIII expression. These data represent the longest stable expression of FVIII following investigational gene therapy and reinforce the ability of AAV gene therapy targeting hepatocytes to achieve stable and durable FVIII expression.”
SPK-8011 was linked to a 91% reduction in annualized bleed rate and a 96% drop in factor VIII infusions. And they have data stretching out past 3 years.
With such small numbers, though, any setbacks can afflict these programs with a lingering sting. And with several gene therapies in the mix now, analysts wonder which will be the dominant player.