Evidence map›Paper›PMID 41673497›Full record

Trial reportNature aging2026

Exploratory analyses of clinical outcomes from the BIIB080 phase 1b study in mild Alzheimer's disease.

Melanie Shulman, Shuang Wu, Nick Ziogas, Amanda Edwards, Jessica Collins, Lin Lin, Irene Tien, Gioacchino Curiale, Yumeng Li, Catherine Mummery and 7 more

Registry-linked trialAbstract readRandomized Controlled TrialMulticenter StudyClinical Trial, Phase I
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Nature aging, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03186989 (A Randomized, Double-Blind, Placebo-Controlled Study, Followed by an Open-Label Extension, to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 814907 in Patients With Mild Alzheimer's Disease), which is not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03186989 phase1completednot on this map

A Randomized, Double-Blind, Placebo-Controlled Study, Followed by an Open-Label Extension, to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 814907 in Patients With Mild Alzheimer's Disease

TypeinterventionalSponsorIonis Pharmaceuticals, Inc.Ran2017 to 2022Enrolled46ConditionsMild Alzheimer's DiseaseArmsIONIS MAPTRx, Placebo
3 · Its place in the literature

Who cites it

8 citing papers in PubMed.

  1. Trial
  2. Review
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

17 authors.

Melanie ShulmanBiogen, Inc., Cambridge, MA, USA. melanie.shulman@biogen.com.ORCID http://orcid.org/0009-0002-5550-9077
Shuang WuBiogen, Inc., Cambridge, MA, USA.
Nick ZiogasBiogen, Inc., Cambridge, MA, USA.
Amanda EdwardsBiogen, Inc., Cambridge, MA, USA.
Jessica CollinsBiogen, Inc., Cambridge, MA, USA.
Lin LinBiogen, Inc., Cambridge, MA, USA.
Irene TienBiogen, Inc., Cambridge, MA, USA.
Gioacchino CurialeBiogen, Inc., Cambridge, MA, USA.
Yumeng LiBiogen, Inc., Cambridge, MA, USA.
Catherine MummeryDementia Research Centre, National Hospital for Neurology and Neurosurgery, University College London, London, UK.ORCID http://orcid.org/0000-0002-6739-0803
Roger LaneIonis Pharmaceuticals, Carlsbad, CA, USA.
Candice JungeIonis Pharmaceuticals, Carlsbad, CA, USA.
John BeaverBiogen, Inc., Cambridge, MA, USA.
Ying TianBiogen, Inc., Cambridge, MA, USA.
Jaren LandenBiogen, Inc., Cambridge, MA, USA.
Szofia BullainBiogen, Inc., Cambridge, MA, USA.
Diana GallagherBiogen, Inc., Cambridge, MA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

This study conducted exploratory analyses of the effects of BIIB080, a MAPT (microtubule-associated protein tau)-targeting antisense oligonucleotide, in participants with mild Alzheimer's disease. A multicenter, randomized, double-blind, phase 1b trial was conducted as a placebo-controlled, multiple-ascending dose (MAD) study followed by an open-label, long-term extension (LTE). During the MAD study, participants were randomized and received either intrathecal placebo or BIIB080 10 mg (n = 6), 30 mg (n = 6) or 60 mg (n = 9) every 4 weeks or 115 mg (n = 13) every 12 weeks (Q12W). During the LTE, participants received high-dose BIIB080 (60 mg (n = 7) or 115 mg (n = 9) Q12W). BIIB080 was generally well tolerated. Here we present findings from exploratory analyses, which showed a consistent trend of slowed decline on cognitive, functional and global measures favoring BIIB080 high-dose groups at the end of both study periods. This favorable trend is supported by reported reductions from baseline in brain neurofibrillary tangles measured with tau positron emission tomography. Trial registration: ClinicalTrials.gov identifier: NCT03186989 .

Indexed as

Alzheimer DiseaseOligonucleotidesOligonucleotides, Antisensetau ProteinsAgedAged, 80 and overCognitionDose-Response Relationship, DrugDouble-Blind MethodFemaleHumansIndolesMaleTreatment OutcomeIndoleslatrepirdineMAPT protein, humanOligonucleotidesOligonucleotides, Antisensetau Proteins

Identifiers

PMID41673497
PMCPMC12920120

What OpenQuestion holds

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.