ALNYLAM Study of the ALN-APP Drug

ALN-APP is an investigational, intrathecally administered RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease (AD) and cerebral amyloid angiopathy (CAA). 

To learn more about the drug, please visit the site below.

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Study Timeline

24-month double-blind treatment period and optional 18-month open label extension period.

Inclusion Criteria

• Age 50 or above
• Cerebral Amyloid Angiopathy diagnosis based upon Boston Criteria version 2
• At least 4 microbleeds or superficial siderosis
• Mini-Mental State Examination (MMSE) of greater than or equal to 22
• Able to have MRI and tolerate LPs
• AChEIs (side effects from medication that include abdominal pain, chest pain, headaches, eye problems, and severe leg pain), memantine, antidepressants, antipsychotics, anxiolytics, benzodiazepines, anticonvulsants and mood stabilizers must be stable for at least 12 weeks prior to screening and dosing regiment must not be anticipated to change during the study.

Exclusion Criteria

• Moderate or severe state Alzheimer’s Disease (Clinical Dementia Rating of 2 or 3) or significant confidence interval (MMSE of less than or equal to 22) at screening
• Clinically significant abnormal laboratory values
• Antiplatelet or anticoagulant therapy within the 10 prior to screening or anticipated use during the study (Aspirin is allowed)
• History of seizures withing the last 6 months prior to screening