Alnylam and Regeneron Report Positive Interim Phase 1 Clinical Data on ALN-APP, an Investigational RNAi Therapeutic for Alzheimer’s Disease and Cerebral Amyloid Angiopathy

– Single Doses of ALN-APP Demonstrated Dose-Dependent, Rapid and Sustained Reduction of sAPPα and sAPPβ in Cerebrospinal Fluid, with Up to 90% at Highest Dose to Date –

– Encouraging Clinical Safety and Tolerability Profile Observed with Single Dosing to Date –

– Results Provide First Demonstration of Gene Silencing by RNAi Therapeutics in the Human Brain Using Alnylam’s Proprietary C16 Platform –

Excerpt from the Press Release:

CAMBRIDGE, Mass. & TARRYTOWN, N.Y.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) and Regeneron Pharmaceuticals, Inc. (Nasdaq: REGN) announced today positive interim results from the ongoing single ascending dose part 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).

Twenty patients have been enrolled in three single-dose cohorts in Part A of the ongoing Phase 1 study in patients with early-onset Alzheimer’s disease. In this study to date, single doses of ALN-APP, which are administered by intrathecal injection, have been well tolerated. All adverse events were mild or moderate in severity, with available cerebrospinal fluid data for white blood cells and protein appearing similar to placebo. Early data for neurofilament light chain from a subset of cohorts (2 of 3 studied) looked comparable to placebo. Patients treated with 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 maximum reduction of 84% and 90%, respectively. Median decreases of both biomarkers of greater than 70% was sustained for at least three months at the highest dose tested. Detailed interim results from this study are planned to be reported at an upcoming scientific conference.

“ALN-APP, via its upstream targeting mechanism, has the potential to address the underlying cause of two devastating CNS diseases, Alzheimer’s disease and CAA, which affect many millions of people and their families around the world.

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