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ABBV-916

A randomized phase 1b/2 trial of ABBV-916 in adults with early Alzheimer's disease

Year of Publication: 2026

Authors: Lynch SY et al.

Journal: Alzheimer's & Dementia

Citation: Alzheimers Dement 2026;22(8):e71715

Link: https://doi.org/10.1002/alz.71715

Bottom Line

ABBV-916 at ≥300 mg IV every 4 weeks produced dose-dependent amyloid-plaque clearance and blood-biomarker changes broadly comparable to approved anti-amyloid therapies, with substantial ARIA rates (ARIA-E 33.8%, ARIA-H 25.0%) that were strongly APOE ε4–dependent; the program was subsequently discontinued for business reasons.

Major Points

  • Dose-proportional PK; amyloid PET reduced significantly vs placebo at 30 mg (-25.1 CL), 300 mg (-61.9 CL), and 900 mg (-83.7 CL) at week 24
  • Week-24 amyloid negativity (<24.1 CL): 100% at 900 mg, 54.6% at 300 mg, 71.4% in 2000→900 mg down-titrated cohort
  • ARIA-E in 33.8% and ARIA-H in 25.0% of ABBV-916 patients; symptomatic ARIA-E in 10.0%; no macrohemorrhage; no deaths in the double-blind period
  • ARIA risk rose with APOE ε4 gene dose: 16.7% (non-carrier), 38.6% (heterozygote), 83.3% (homozygote) for ARIA-E
  • Selective targeting of pyroglutamate-modified Aβ (AβpE3) did NOT reduce ARIA versus approved therapies
  • Sponsor (AbbVie) discontinued the program before dose expansion due to lack of differentiation from approved anti-amyloid therapies

Design

Study Type: Randomized Controlled Trial (Phase 1b/2 multiple ascending dose)

Randomization: 1

Blinding: Double-blind, placebo-controlled

Allocation Ratio: 3:1 ABBV-916:placebo within each cohort

Enrollment Period: Aug 15, 2022 - May 9, 2024

Follow-up Duration: 24-week double-blind period + 16-week safety follow-up (or 2-year extension)

Centers: Multicenter, global

Countries: USA (multiple sites; central IRB Advarra)

Sample Size: 106

Analysis: Full analysis set (all randomized patients receiving ≥1 dose with baseline amyloid PET); MMRM for amyloid change; hypothetical strategy for intercurrent events

Registration: NCT05291234


Inclusion Criteria

  • Adults aged 50–90 years
  • Met NIA-AA 2018 Research Framework criteria for stage 3 or 4 AD
  • MMSE score 20–28
  • Plasma amyloid probability score (C2N Diagnostics) consistent with increased likelihood of positive amyloid PET
  • Amyloid PET scan ≥37 Centiloids by florbetapir
  • No clinically significant findings for laboratory parameters as assessed by the investigator

Exclusion Criteria

  • MRI findings of vasogenic edema
  • ≥4 cerebral microhemorrhages on MRI
  • Any area of superficial siderosis
  • Any macrohemorrhage
  • Severe white matter disease
  • Other clinically significant or unstable medical conditions
  • History that would make the patient unsuitable for the study per investigator

Baseline Characteristics

CharacteristicPlacebo (n=26)ABBV-916 all doses (n=80)ABBV-916 300 mg (n=17)ABBV-916 900 mg (n=28)
Age (yrs, mean ± SD)76.4 ± 7.476.2 ± 7.576.4 ± 7.776.8 ± 6.4
Female42.3%57.5%64.7%50.0%
Male57.7%42.5%
White96.2%88.8%94.1%82.1%
Black3.8%10.0%
Asian0%1.3%
Hispanic or Latino23.1%23.8%
BMI (mean ± SD)27.6 ± 3.926.8 ± 4.5
APOE ε4 carrier57.7%62.5%58.8%57.1%
APOE ε4/ε4 homozygous19.2%7.5%

Arms

FieldControlABBV-916 10 mgABBV-916 30 mgABBV-916 100 mgABBV-916 300 mgABBV-916 900 mgABBV-916 2000→900 mg
InterventionIV placebo every 4 weeks x6 doses over 24 weeks10 mg IV Q4W x630 mg IV Q4W x6100 mg IV Q4W x6300 mg IV Q4W x6900 mg IV Q4W x6 (cohorts 5 + 7 combined)2000 mg IV first dose(s), down-titrated to 900 mg from week 4 due to safety review
Duration24 weeks (double-blind)24 weeks24 weeks24 weeks24 weeks24 weeks24 weeks
N26688172813

Outcomes

OutcomeTypeControlInterventionHR / OR / RRP-value
Safety, PK, and change from baseline to week 24 in brain amyloid plaque deposition by florbetapir PET (Centiloids)PrimaryPlacebo (mean baseline): 97.9 CL · ABBV-916 30 mg vs placebo at week 24: -25.1 CL difference (p < 0.05) · ABBV-916 300 mg vs placebo at week 24: -61.9 CL difference (p < 0.0001) · ABBV-916 900 mg vs placebo at week 24: -83.7 CL difference (p < 0.0001) · ABBV-916 900 mg absolute change from baseline: -77.0 CL at week 24 (-50.4 CL at week 12)
Amyloid negativity (<24.1 CL) at week 24 - 300 mgSecondaryValue: 54.6%
Amyloid negativity (<24.1 CL) at week 24 - 900 mgSecondaryValue: 100%
Amyloid negativity (<24.1 CL) at week 24 - 2000→900 mgSecondaryValue: 71.4%
Plasma Aβ42/Aβ40 change at 24 wk (300 mg)SecondaryValue: +15.3% · Placebo: -2.3%
Plasma Aβ42/Aβ40 change at 24 wk (900 mg)SecondaryValue: +15.7% · Placebo: -2.3%
Plasma p-tau217 change at 24 wk (300 mg)SecondaryValue: -23.8% · Placebo: +15%
Plasma p-tau217 change at 24 wk (900 mg)SecondaryValue: -29.8% · Placebo: +15%
Plasma GFAP change at 24 wk (300 mg)SecondaryValue: -20.0% · Placebo: +11.9%
Plasma GFAP change at 24 wk (900 mg)SecondaryValue: -31.5% · Placebo: +11.9%
Antidrug antibodiesSecondaryValue: 3.8% (3/80) transient low-titer
Any TEAE (placebo)Adverse69.2%
Any TEAE (ABBV-916 all)Adverse78.8%
Serious AE (placebo)Adverse0%
Serious AE (ABBV-916)Adverse11.3%
Discontinuation due to AE (placebo)Adverse0%
Discontinuation due to AE (ABBV-916)Adverse20.0%
ARIA-E (ABBV-916 all)Adverse33.8%
ARIA-H (ABBV-916 all)Adverse25.0%
Symptomatic ARIA-EAdverse10.0% (8/80)
Symptomatic microhemorrhageAdverse5.0% (4/80)
Superficial siderosisAdverse13.8% (11/80)
MacrohemorrhageAdverse0%
Headache (ABBV-916)Adverse15.0% (vs 3.8% placebo)
Falls (ABBV-916)Adverse10.0% (vs 0% placebo)
UTI (ABBV-916)Adverse10.0% (vs 11.5% placebo)
Dizziness (ABBV-916)Adverse7.5%
Nausea (ABBV-916)Adverse7.5%
COVID-19 (ABBV-916)Adverse6.3%
Hypertension (ABBV-916)Adverse6.3%
Infusion-related reactions (900 mg)Adverse10.7%
Deaths (double-blind period)Adverse0

Subgroup Analysis

ARIA-E incidence by APOE ε4 status: non-carrier 16.7% (5/30), heterozygous carrier 38.6% (17/44), homozygous carrier 83.3% (5/6). ARIA-H incidence: non-carrier 13.3%, heterozygous 29.5%, homozygous 50.0%. Clear APOE ε4 gene-dose relationship for ARIA risk.


Criticisms

  • Very small sample size (N=106) and short 24-week double-blind duration
  • Predominantly White population (88.8%) limits generalizability across race/ethnicity
  • Program terminated before dose expansion (Stage B) due to sponsor's business/strategic reasons, so optimal dose and long-term efficacy/safety could not be established
  • Cohort 6 required mid-study down-titration from 2000 mg to 900 mg due to safety, confounding dose-response interpretation for the highest planned dose
  • No clinical/cognitive outcomes (CDR-SB, iADRS, etc.) - amyloid PET surrogate only
  • Interruptions and early discontinuations may have introduced confounding for the amyloid clearance analysis
  • Selective targeting of AβpE3 did not translate into the hypothesized ARIA reduction versus approved anti-amyloid antibodies

Funding

AbbVie Inc.

Based on: ABBV-916 (Alzheimer's & Dementia, 2026)

Authors: Lynch SY et al.

Citation: Alzheimers Dement 2026;22(8):e71715

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