Analyst Outlook
Verubecestat’s (Merck & Co) Phase Ib studies revealed highly impressive results in its capability to
almost abolish cerebrospinal fluid (CSF) amyloid-beta fragments. The clinical implications of
verubecestat’s biological efficacy will be brought to light in two large Phase III studies evaluating its
efficacy and safety in early and mild to moderate Alzheimer’s disease patients. Until the release of
Phase III results, relatively little is known about verubecestat apart from its impressive biological
effect and short-studied safety profile. While currently in a leading position, verubecestat is to face
fierce competition from other beta-site amyloid precursor protein cleaving enzyme (BACE) inhibitors
as they transition to Phase III development. This will likely prompt Merck to hasten verubecestat’s
clinical development as the race to obtain first-to-market status intensifies.
Drug Overview
Verubecestat is an inhibitor of the BACE1. BACE1 is one of two enzymes that cleaves amyloid-beta
precursor protein, giving rise to various amyloid-beta fragments. By inhibiting the activity of BACE1,
verubecestat reduces amyloid production and thus reduces amyloid plaque deposition in the brain, a
key feature of Alzheimer’s disease. Verubecestat is currently in Phase III development for the
treatment of prodromal and mild to moderate Alzheimer’s disease (Medtrack, 2015; Merck & Co,
2015).
TABLE OF CONTENTS
4 PRODUCT PROFILES
4 verubecestat : Alzheimer’s disease
LIST OF FIGURES
8 Figure 1: Verubecestat for Alzheimer’s disease – SWOT analysis
9 Figure 2: Datamonitor Healthcare’s drug assessment summary of verubecestat in Alzheimer’s
disease
10 Figure 3: Datamonitor Healthcare’s drug assessment summary of verubecestat in Alzheimer’s
disease
LIST OF TABLES
4 Table 1: Verubecestat drug profile
5 Table 2: Verubecestat Phase Ib data in Alzheimer’s disease
6 Table 3: Verubecestat Phase II/III trials in Alzheimer’s disease