VX15 blocks the activity of semaphorin 4D (SEMA4D), a molecule that is thought to promote chronic inflammatory responses in the brain.
“We are very pleased that the FDA has granted this important designation for VX15 for the treatment of Huntington’s disease,” Maurice Zauderer, Vaccinex’s chief executive officer, said in a press release. “Currently, there are few therapeutic options available for this patient population, and these are limited to palliative measures. The FDA Fast Track program holds the promise of giving patients more rapid access to potentially effective treatments.”
Vaccinex launched a Phase 2 multi-center, randomized, double-blind, placebo-controlled clinical trial in subjects with late prodromal (pre-manifest) and early manifest Huntington’s disease in June 2015.
The trial SIGNAL will enroll about 94 patients, ages 21 or older, by invitation only. Researchers expect an interim analysis to be ready for presentation by the third quarter of 2016. Top line data is expected in 2018.
The trial’s primary objective is to evaluate the safety and tolerability of monthly intravenous administration of a single dose of VX15 (or placebo). Secondary objectives include determining the effect of VX15 on brain volumes and clinical features of Huntington’s disease that include cognition, motor function, behavior, functional abilities and global function.
Further objectives include determining the pharmacokinetics (how the drug moves in the body) and pharmacodynamics (effects and mechanism) profile of the drug, its immunogenicity (ability to provoke response) and any exploratory biomarkers.
The study will be divided into cohort A and B: in cohort A, subjects will receive either VX15 or placebo at a 1:1 ratio for six months, followed by three months of follow-up; in cohort B, subjects will receive VX15 or placebo at 1:1 ratio for 18 months, followed by three months of follow-up.
VX15 is being studied for its potential to prevent or delay disease onset in people with a confirmed mutation. The Fast Track program will allow Vaccinex to move the development processes and review of VX15 faster than normal.