Alnylam Submits CTA Application For ALN-APP, An Investigational RNAi Therapeutic For The Treatment Of Alzheimer’s Disease And Cerebral Amyloid Angiopathy

– Company Expects to Initiate a Phase 1 Study in Early-Onset Alzheimer’s Disease in Early 2022, with Initial Human Data at or Around Year-End 2022 –

– ALN-APP Marks the Industry’s First-Ever RNAi Therapeutic Program for CNS Disease and Represents the Start of Alnylam’s Expansion of RNAi Therapeutic Opportunities in Extra-Hepatic Tissues –

Alnylam Pharmaceuticals, Inc. the leading RNAi therapeutics company, announced that the Company has submitted a Clinical Trial Authorization (CTA) application to The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom to initiate a Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of Alzheimer’s disease (AD) and cerebral amyloid angiopathy (CAA). The Company plans to initiate the Phase 1 study in patients with early-onset AD in early 2022, upon obtaining MHRA approval, and expects to report initial human data at or around year-end 2022.

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“Neurodegenerative diseases like early-onset Alzheimer’s disease are devastating to patients and their families and remain an area of profound unmet medical need, requiring a paradigm shift in how we approach treatment of diseases impacting the central nervous system,” said Tim Mooney, Program Leader for the ALN-APP program at Alnylam. “To that end, we are excited to advance the first ever RNAi therapeutic targeting the CNS towards the clinic, with the support of our partners at Regeneron. ALN-APP represents Alnylam’s continued commitment to innovation both in pursuing new genetically validated disease targets and in advancing our platform to target extra-hepatic tissues.”

ALN-APP represents the first-ever RNAi therapeutic targeting CNS diseases. The clinical candidate was designed using Alnylam’s proprietary C16 conjugate technology for the delivery of short interfering RNA (siRNA) to the CNS. As such, ALN-APP represents the start of Alnylam’s expansion of RNAi therapeutic opportunities in extra-hepatic tissues. ALN-APP is being advanced in partnership with Regeneron as part of the companies’ 2019 agreement. Alnylam will lead global development and commercialization of ALN-APP, and Regeneron has exercised its 50-50 co-development and co-commercialization option under the collaboration.

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