Alnylam Pharma Reports Additional Positive Interim Phase 1 Results For Alzheimer's Disease Treatment

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) Wednesday announced additional positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer's disease and cerebral amyloid angiopathy (CAA).

ALN-APP is the first clinical-stage program using Alnylam's proprietary C16-siRNA conjugate platform for central nervous system (CNS) delivery and the first investigational RNAi therapeutic to demonstrate gene silencing in the human brain. ALN-APP is being developed in collaboration with Regeneron.

"Today's results showcase the exciting emerging profile of ALN-APP. This novel approach appears generally well-tolerated and is able to target the amyloid pathway successfully, robustly lowering target engagement biomarkers sAPPa and sAPPß and maintaining a significant effect up to 10 months after administration," said Dr. Cath Mummery, Consultant Neurologist and Head of Clinical Trials, Dementia Research Centre, University College London.

"For the first time, we also see that single doses of ALN-APP can reduce cerebral spinal fluid levels of Aß42 and Aß40, which are the amyloidogenic peptides that are the primary components of amyloid deposits in Alzheimer's disease and CAA. This approach warrants further study to evaluate whether it can potentially interrupt relentless progression of these two devastating diseases."

Single doses of ALN-APP achieved sustained pharmacodynamic activity up to 10 months after administration, the company said.