Alnylam Pharmaceuticals Inc. (ALNY) announced Monday updated positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy or CAA.
ALN-APP, being developed in collaboration with Regeneron Pharmaceuticals, Inc., is an investigational, intrathecally administered RNAi therapeutic targeting amyloid precursor protein or APP. It is the first clinical-stage program using Alnylam’s proprietary C16-siRNA conjugate platform for central nervous system or CNS delivery and the first investigational RNAi therapeutic to demonstrate gene silencing in the human brain.
The updated results were presented at the 2023 Alzheimer’s Association International Conference being held July 16-20, 2023 in Amsterdam, The Netherlands.
The Phase 1 study is a multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of ALN-APP in patients with early-onset Alzheimer’s disease or EOAD, the most common neurodegenerative disease and the most common form of dementia.
The trial includes single ascending dose phase, i.e., Part A; and multiple dose phase, i.e., Part B in patients with EOAD.
In Part A of the ongoing Phase 1 study, twenty patients with EOAD have been enrolled in three single-dose cohorts. In this trial to date, single doses of ALN-APP, which are administered by intrathecal injection, have been well tolerated.
Further exploration of single doses of ALN-APP is ongoing in Part A. In addition, the safety review committee has recommended initiation of Part B, the multiple-dose part of the study, which will enroll patients from Part A.
The trial has already received regulatory approval to proceed in Canada, where the majority of the Part A clinical trial patients were enrolled.
In the U.S., the multiple dose part of the study remains on partial clinical hold due to findings observed in prior non-clinical chronic toxicology studies.
The safety and efficacy of ALN-APP have not been evaluated by the FDA, EMA, or any other health authority.
In addition to ALN-APP, Alnylam and Regeneron have named 10 targets in the CNS as part of their exclusive collaboration established in 2019 to discover RNAi therapeutics for eye and CNS diseases.
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