Pipeline
We’re developing a pipeline of firsts.
Program
Target
Progranulin
Candidate
Phase 3
Indication
FTD-GRN
Partner
Target
Progranulin
Candidate
Phase 2
Indication
FTD-C9orf72
Partner
Target
Progranulin
Candidate
Phase 1b
Indication
Alzheimer’s disease
Partner
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AL001
Target
Progranulin
Indication
Frontotemporal dementia (FTD) in granulin mutation
Development Stage
Phase 3
AL001 modulates progranulin (PGRN), a key regulator of immune activity in the brain with genetic links to multiple neurodegenerative disorders. We are initially developing AL001 for frontotemporal dementia (FTD) with progranulin mutation (FTD-GRN). The global INFRONT-3 Phase 3 clinical trial is currently enrolling both at-risk and symptomatic participants with FTD-GRN.
AL001 is being developed in collaboration with GSK.
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AL001
Target
Progranulin
Indication
Frontotemporal dementia (FTD) in C9orf72 mutation carriers
Development Stage
Phase 2
AL001 is being studied in Phase 2 trial for the treatment of frontotemporal dementia patients with a C9orf72 mutation (FTD-C9orf72). FTD-C9orf72 is associated with abnormal accumulation of the protein TDP-43. The Phase 2 study, which also includes participants with progranulin mutations (FTD-GRN) is designed to assess the safety and tolerability of chronic dosing of AL001.
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AL101
Target
Progranulin
Indication
Neurodegenerative diseases
Development Stage
Phase 1b
AL101 is designed to increase progranulin levels for the treatment of more prevalent neurodegenerative diseases including Alzheimer’s disease and Parkinson’s disease. Mutations that moderately reduce the expression levels of PGRN have been shown to increase the risk of developing Alzheimer’s disease and Parkinson’s disease, and increased PGRN levels have been demonstrated to be protective for these diseases in animal models.
AL101 is currently being studied in a Phase 1 clinical trial of healthy volunteers designed to assess safety, tolerability and pharmacokinetics and pharmacodynamics. AL101 is being developed in collaboration with GSK.
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AL002
Target
TREM2
Indication
Alzheimer’s disease
Development Stage
Phase 2
Research suggests that reduction of Triggering Receptor Expressed on Myeloid cells 2 (TREM2) functionality may lead to Alzheimer’s disease and other forms of dementia. AL002 is intended to counteract this decreased functionality by improving TREM2 signaling to enhance microglia activity.
The randomized, controlled Phase 2 study, INVOKE-2, in patients with early Alzheimer’s disease, will enroll approximately 265 participants and assess
AL002’s impact on disease progression, as well as changes in multiple fluid and imaging biomarkers. AL002 is being developed in collaboration with AbbVie.