Agios Pharmaceuticals, Inc.

Lucrezia De Pascalis

Brian Christopher Shook

Zenon D Konteatis

Heather Jane Finlay

Sandra King

The disclosure relates to compounds of Formula I or a pharmaceutically acceptable salt thereof, wherein, m, R-R, R, and L are defined herein. These compounds are useful in methods for stabilizing a mutant PAH protein or reducing blood phenylalanine concentration in a subject suffering from phenylketonuria. In some embodiments, the mutant PAH protein contains at least one R408W, R261Q, R243Q, Y414C, L48S, A403V, I65T, R241C, L348V, R408Q, or V388M mutation. In other embodiments, the mutant PAH protein contains at least one R408W, Y414C, I65T, F39L, R408Q, L348V, R261Q, A300S, or L48S mutation.

Growing community of inventors

Cambridge, MA, United States of America

Lucrezia De Pascalis