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MOA / MOD


The mechanism of ATTR12,13

Amyloidogenic TTR cascade


TTR
Secreted as a tetramer

TTR
dissociation
The tetramer breaks into monomers

Amyloid fibril
formation
Misfolded monomers aggregate into amyloid fibrils

Deposition of
fibrils
Amyloid fibrils accumulate in different parts of the body, such as the nerves

Vyndaqel® is a selective stabilizer of TTR12,13

* As measured by a TTR stabilization assay.
† Study design: In the 1.5-year, randomized, double-blind, placebo-controlled pivotal study, Val30Met patients received Vyndaqel® 20mg once daily or placebo. Coprimary endpoints: Neuropathy Impairment score of The Lower Limb (NIS-LL) responder analysis (change of less than 2 points on NIS-LL) and treatment group difference in the mean change from baseline in Norfolk Quality of Life-Diabetic Neuropathy total score ((Norfolk QOL-DN) in ITT population (n = 125) and EE (n=87) populations. Secondary endpoints: changes in neurological function, small and large nerve fiber function, and nutritional status.27
ⱡ Study design: A 1-year, open-label, single-arm study. Non-Val30Met patients received Vyndaqel® 20mg once daily. Primary endpoint TTR stabilization at week 6 (n=19). Secondary endpoints: TTR stabilization at month 6 (n=18) and month 12 (n=17).28