Alnylam - Regeneron Partnered Alzheimer's Candidate's Initial Data Sounds Promising, Analyst Says
Alnylam Pharmaceuticals Inc and its partner, Greenburgh's Regeneron Pharmaceuticals, announced preliminary data from their Phase I study of their experimental drug dubbed ALN-APP.
ALN-APP is an investigational RNAi therapeutic targeting amyloid precursor protein (APP, a brain protein) in development for Alzheimer's disease and cerebral amyloid angiopathy (CAA). Patients treated with single-dose ALN-APP experienced dose-dependent, rapid and sustained reduction in cerebrospinal fluid of both soluble APPα (sAPPα) and APPβ (sAPPβ), biomarkers of target engagement, with a maximum reduction of 84% and 90%, respectively.
Further exploration of single-doses of ALN-APP is ongoing in Part A of the Phase 1 study (Canada, Netherlands, U.K., and U.S.) to evaluate longer-term safety and duration of action and to identify the multi-dose regimen for Part B
Alnylam and Regeneron are already moving into the second half of the Phase I study in Canada.
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