Alnylam Pharmaceuticals announced today updated positive interim results of a Phase I study of pipeline drug ALN-APP for the treatment of Alzheimer’s disease (AD) and cerebral amyloid angiopathy (CAA).

The Phase I study is a multicentre, randomised, 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 (EOAD). ALN-APP is being developed in collaboration with Regeneron Pharmaceuticals.

Interim results

Updated results of Part A of the Phase I study, which involves 20 patients with EOAD, were presented during the Alzheimer’s Association International Conference (AAIC) ongoing from 16 to 20 July 2023 in Amsterdam. Previous results were announced in April 2023.

To date, the group who have received single doses of ALN-APP, administered by intrathecal injection, well-tolerated the drug with either mild or moderate adverse events. The results also indicate no remarkable elevations in cerebrospinal fluid CAA white blood cell count and total protein levels.

Haematology, serum chemistry, liver function, urinalysis, coagulation, and preliminary data for the exploratory biomarker neurofilament light chain showed no significant abnormalities. Patients treated with a single dose of 75mg ALN-APP showed a rapid and sustained reduction in cerebrospinal fluid of both soluble APPα (sAPPα) and soluble APPβ (sAPPβ).

How ALN-APP works

ALN-APP is an investigational drug that acts by inhibiting amyloid precursor protein to exhibit therapeutic intervention. The extracellular accumulation of misfolded immunoglobulin light chains as fibrillar amyloid deposits leads to the development of amyloidosis.

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Toronto Memory Program’s medical director and neurologist Dr Sharon Cohen said: “We’ve known for decades that mutations that increase APP production or alter its proteolysis cause EOAD, early-onset CAA or both. These Phase I results show that a single dose of ALN-APP can rapidly reduce APP production and that this effect is sustained for six months. Given the critical need for new and better treatments for AD and CAA, these results are promising, and the approach warrants further study.”

As a result of part A, the safety review committee has recommended the initiation of part B, which will be a multi-dose study. Patients from part A will be enrolled. The multiple-dose part of the study has received regulatory approval to proceed in Canada. However, it remains on partial clinical hold in the US due to findings in prior non-clinical chronic toxicology studies.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

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