Top-Line Results Released from Huntington’s Disease Study

Vaccinex announced topline results from the early manifest treatment arm (Cohort B1, N=179) of the Phase 2 double-blind, placebo-controlled SIGNAL trial of its lead clinical candidate, pepinemab, in patients with early manifest and prodromal Huntington’s disease (HD).

“HD affects multiple regions of the brain and disease progression impacts many critical functions including cognition and motor activity. The results reported strongly support a cognitive benefit to treatment with pepinemab and indicate that treatment with pepinemab antibody potentially targets cortical centers, including those that govern cognition. The first sign of HD is often chorea and cognitive disturbances generally follow later in disease progression. We believe the data, therefore, suggest that patients at a somewhat more advanced stage of HD may derive the greatest benefit from pepinemab,” Maurice Zauderer, Ph.D., president and chief executive officer of Vaccinex said. “The insights gained from this study also suggest that pepinemab might be an important treatment option for Alzheimer’s and other neurodegenerative diseases known to primarily affect frontal cortex and to impact cognition. As previously reported, imaging data indicate that these are the brain regions most affected by pepinemab treatment. The company has, accordingly, initiated screening and expects to begin enrolling patients this month in a new Alzheimer’s disease study of pepinemab at 15 clinical sites in the United States. In line with the Company’s ongoing efforts in Alzheimer’s, as well as in head and neck cancer, the Company also intends to examine and, as appropriate, prioritize and balance its budget and expenditures.”

The study had two co-primary endpoints, a family of two cognitive assessments from the Huntington’s Disease Cognitive Assessment Battery and Clinical Global Impression of Change (CGIC). Although the study did not meet pre-specified co-primary endpoints, the results of each of the two cognitive assessments demonstrated a strong trend for beneficial change (OTS, p=0.028 and PTAP, p=0.06). These cognitive assessments reflect changes in planning ability and memory associated with disease progression. Similarly, a trend of benefit in CGIC did not show a statistically significant difference between the placebo and pepinemab-treated groups, possibly due to small group size in this phase 2 study.

Pepinemab was well-tolerated with remarkably low treatment discontinuation and study drop-out rates over the extended 18 month treatment period.