Pipeline

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.

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.

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.

ADP027-ABC incorporates our proprietary ABC technology to improve brain penetration and modulates glycoprotein GPNMB, mimicking a protective genetic variant to treat both familial and sporadic Parkinson’s disease. By lowering GPNMB levels, ADP027-ABC seeks to normalize lysosomal function and enhance the microglial immune response. Restored lysosomal function enables the removal of alpha-synuclein, LRRK2, and GBA1 proteins, reducing inflammation and mitigating the progression of Parkinson’s disease.

ADP050-ABC incorporates our proprietary ABC technology to enhance brain penetrance and introduces GCase enzyme into the brain to replace the deficient or dysfunctional enzyme resulting from mutations in the GBA gene. This enzyme replacement therapy aims to restore the normal function of GCase, which is crucial for the breakdown of specific cellular waste products, particularly glucocerebroside. By replenishing GCase levels, ADP050-ABC seeks to alleviate the accumulation of glucocerebroside and subsequent cellular dysfunction associated with Parkinson’s disease and Lewy body dementia.