Data from Pivotal FIREFISH and SUNFISH Studies Demonstrate Clinical Benefit of Risdiplam in Type 1, 2, & 3 Spinal Muscular Atrophy Patients

SUNFISH

MFM32

Aged 2-11(n=24)

Aged 12-25(n=19)

Aged 2-25(n=43)*

Total MFM change from baseline, mean (SD)

3.47 (3.77)

1.64 (3.43)

2.66 (3.70)

Total MFM change from baseline, median (range)

4.17 (-6.3, -9.4)

2.08 (-7.3, -6.3)

3.13 (-7.3, -9.4)

Proportion of patients who achieveimprovement (i.e. a change frombaseline in MFM score ?3), % (n)

70.8 (17/24)

42.1 (8/19)

58.1 (25/43)

*Excludes 7 patients who performed the MFM20 assessment (only patients who performed the full MFM32 assessment are included in the analysis) and one patient who had dropped out of the study prior to the Month 12 visit.