Yung Chyung, Scholar Rock CMO (Business Wire)

A dark horse en­trant in­to the spinal mus­cu­lar at­ro­phy field dou­bles its val­ue on some PhII da­ta

The last four years have seen a sud­den ex­plo­sion in treat­ments for spinal mus­cu­lar at­ro­phy, a neu­rode­gen­er­a­tive con­di­tion that once led pa­tients — of­ten young ones — with a grim prog­no­sis and no op­tions. The prog­no­sis still isn’t rosy, but now there are three FDA-ap­proved op­tions, enough to make the choice of one dif­fi­cult.

Now a fourth po­ten­tial op­tion has en­tered the mix. To­day, Schol­ar Rock an­nounced the re­sults from a proof-of-con­cept test­ing their SMA drug by it­self and in com­bi­na­tion with Io­n­is’ Spin­raza, show­ing that all pa­tient co­horts im­proved on stan­dard scales used for mea­sur­ing mo­tor func­tion in peo­ple with SMA.

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