PQ-912 is primarily investigated for its potential therapeutic application in the treatment of Alzheimer’s disease. It functions as an inhibitor of the enzyme glutaminyl cyclase (QC), which is involved in the formation of neurotoxic pyroglutamate-modified amyloid-beta (pGlu-Aβ) peptides. These modified peptides are found in amyloid plaques in the brains of Alzheimer’s patients and are believed to contribute to disease progression due to their increased stability and neurotoxicity.

By inhibiting QC, PQ-912 reduces the production of pGlu-Aβ, thereby potentially slowing down amyloid plaque formation and associated neurodegeneration. In clinical development, PQ-912 has shown the ability to engage its target in the central nervous system and modulate biomarkers related to amyloid pathology. It is being evaluated for its impact on cognitive and functional outcomes in early-stage Alzheimer’s patients.

Due to its mechanism of action, PQ-912 represents a disease-modifying approach rather than symptomatic treatment, making it a candidate for early intervention strategies in Alzheimer’s disease. Research continues to assess its long-term efficacy, safety, and potential role in combination therapies.