First patient recruited to Phase I Early Alzheimer’s Disease study

UCLH have recruited the first participant in the world to a new global phase I study for early Alzheimer’s Disease (AD).

The study (NCT07234942) is testing an experimental study drug (RG6627/RO7812653) designed to reduce the amount of a protein called ApoE in the brain. Studies have demonstrated that ApoE contributes to worsening of brain health in people with AD. For example, ApoE speeds up the accumulation of abnormal proteins called amyloid and tau in the brain. These proteins cause brain inflammation and damage and lead to memory loss in people with AD. By reducing the ApoE protein in the brain this drug may have an impact on AD. 

Professor Catherine Mummery, UCLH consultant neurologist at the Dementia Research Centre, at the National Hospital for Neurology and Neurosurgery (NHNN), clinical lead for the NHNN Cognitive Disorders Clinic and head of Clinical Trials at UCL Dementia Research Centre, is leading the trial at UCLH. The study is being delivered at the NIHR UCLH Clinical Research Facility at NHNN.

Currently treatments provide modest clinical benefit for symptoms and against the progression of AD, so there is need for alternative treatments. There is compelling evidence suggesting that once AD has started, ApoE contributes to several key manifestations of the disease1.

Professor Mummery says: “This tremendously exciting treatment approach is based on the fact that ApoE is a major risk factor for the development of Alzheimer’s disease. This is the first drug trialled that directly addresses ApoE-related disease progression using gene silencing methods to lower production of ApoE and therefore reduce levels in the brain. The phase I trial is the first step in assessment in patients, and we are very proud to be collaborating with Roche on this trial, with the first patient in the world recruited at UCLH.”

The major aim of this Phase 1 study is to investigate safety and tolerability of RG6627/ RO7812653. Six countries will be supporting the study, with five sites the UK.

The results from this study will inform future trials of RG6627/RO7812653 and guide further clinical development as a treatment for AD.

F. Hoffmann-La Roche Ltd (or “Roche”) is the study sponsor and is developing RG6627/RO7812653, which was discovered by and licensed from Ionis Pharmaceuticals.

Notes to editors

1. ApoE contributes to Alzheimer’s disease manifestations including amyloid plaque formation, neurodegeneration and neuroinflammation. It is anticipated therefore that lowering ApoE levels in the central nervous system may result in a beneficial effect on the clinical progression of the disease.