Table 1.
Demographic and Baseline Clinical Characteristics ofthe Patients.*
| Characteristic | Verubecestat 12-mg Group (N = 485) |
Verubecestat 40-mg Group (N = 484) |
Placebo Group (N = 485) |
|---|---|---|---|
| Demographic and clinical characteristics† | |||
| Age — yr | 71.7±7.1 | 71.0±7.4 | 71.6±7.1 |
| Female sex — no. (%) | 229 (47.4) | 244 (50.4) | 213 (44.0) |
| Race — no. (%)‡ | |||
| White | 396 (82.0) | 392 (81.0) | 390 (80.6) |
| Asian | 79 (16.4) | 85 (17.6) | 84 (17.4) |
| Other | 8 (1.7) | 7 (1.4) | 10 (2.1) |
| Geographic region — no. (%) | |||
| United States or Canada | 226 (46.8) | 224 (46.3) | 226 (46.7) |
| Europe, Australia, or New Zealand | 163 (33.7) | 163 (33.7) | 161 (33.3) |
| Japan | 57 (11.8) | 60 (12.4) | 59 (12.2) |
| Other | 37 (7.7) | 37 (7.6) | 38 (7.9) |
| APOE4 carrier — no. (%) | 328 (67.9) | 337 (69.6) | 335 (69.2) |
| MMSE score of ≥27 — no. (%)§ | 212 (43.9) | 211 (43.6) | 214 (44.2) |
| Treatment with acetylcholinesterase inhibitor or memantine for Alzheimer’s disease — no. (%) | 222 (46.0) | 225 (46.5) | 222 (45.9) |
| Education level below undergraduate degree — no. (%) | 243 (50.3) | 240 (49.6) | 250 (51.7) |
| Baseline scores on clinical outcome measures¶ | |||
| CDR-SB | |||
| No. of patients assessed | 465 | 458 | 469 |
| Score | 2.74±1.31 | 2.67±1.26 | 2.58±1.19 |
| CCS-3D | |||
| No. of patients assessed | 441 | 424 | 440 |
| Score | 0.03±1.04 | 0.02±0.99 | −0.06±0.98 |
| ADCS-ADLMCI | |||
| No. of patients assessed | 469 | 462 | 472 |
| Score | 42.2±5.9 | 43.1±5.4 | 42.8±5.9 |
| ADAS-cog13 | |||
| No. of patients assessed | 468 | 465 | 475 |
| Score | 22.1±6.6 | 22.6±6.5 | 21.9±6.4 |
| ADAS-cog11 | |||
| No. of patients assessed | 468 | 465 | 475 |
| Score | 13.4±4.9 | 13.8±4.8 | 13.4±4.7 |
| MMSE | |||
| No. of patients assessed | 466 | 463 | 475 |
| Score | 26.3±1.8 | 26.4±1.7 | 26.3±1.8 |
| NPI | |||
| No. of patients assessed | 463 | 459 | 472 |
| Score | 5.9±8.6 | 5.5±8.2 | 5.5±8.3 |
| Biomarker values¶ | |||
| Hippocampal volume on MRI — microliters | |||
| No. of patients assessed | 168 | 181 | 191 |
| Value | 6448±1107 | 6469±1106 | 6435±987 |
| Cortical amyloid load on PET — SUVR | |||
| No. of patients assessed | 63 | 59 | 65 |
| Value | 0.86±0.07 | 0.87±0.07 | 0.85±0.06 |
Plus-minus values are means ±SD. Percentages may not total 100 because of rounding. MRI denotes magnetic resonance imaging, PET positron-emission tomography, and SUVR standardized uptake value ratio.
Shown are data from patients who received at least one dose of the trial regimen (483 patients in the verubecestat 12-mg group, 484 patients in the verubecestat 40-mg group, and 485 patients in the placebo group).
Race was reported by the patients.
Scores on the Mini-Mental State Examination (MMSE) range from 0 to 30, with lower scores indicating poorer cognitive performance.
Shown are data from patients who were included in the modified intention-to-treat population. This population included patients who received at least one dose of the trial regimen and had a baseline measure and at least one valid follow-up outcome measure after randomization and positive findings for Alzheimer’s disease on PET (10 patients who had negative findings on PET but who had a positive tau:Aβ42 ratio in cerebrospinal fluid were also enrolled to validate a companion diagnostic test, but they are not included in the analyses). The number of patients evaluated for each outcome differed depending on which outcome measure was obtained. MRI and PET measures were obtained from a subgroup of patients. Scores on the Clinical Dementia Rating Scale-Sum of Boxes (CDR-SB) range from 0 to 18, with higher scores indicating worse cognition and daily function. Scores on the Composite Cognition Score-3 Domain (CCS-3D) are the mean z scores from multiple individual cognitive tests. Scores on the Alzheimer’s Disease Cooperative Study Activities of Daily Living for Mild Cognitive Impairment Inventory scale (ADCS-ADLMCI) range from 0 to 53, with lower scores indicating worse function. Scores on the 13-item cognitive subscale of the Alzheimer’s Disease Assessment Scale (ADAS-cog13) range from 0 to 85, with higher scores indicating worse cognition. Scores on the 11-item cognitive subscale of the Alzheimer’s Disease Assessment Scale (ADAS-cog11) range from 0 to 70, with higher scores indicating worse cognition. Neuropsychiatric Inventory (NPI) scores range from 1 to 144, with higher scores indicating more severe symptoms.