Scholar Rock (SRRK) Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 SMA

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Go back to Scholar Rock (SRRK) Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 SMA

Scholar Rock Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 Spinal Muscular Atrophy

October 27, 2020 7:00 AM EDT

- Motor function improvements were observed for all three SRK-015 treatment cohorts in the primary efficacy endpoints (Hammersmith scale scores) at six-months; top-line data from the 12-month treatment period are anticipated in 2Q21

- SRK-015 dose response in the primary efficacy endpoint was observed in the randomized, double-blind cohort, with high dose attaining a 5.6 point mean improvement at six-months over baseline compared to low dose attaining a 2.4 point mean improvement over baseline

- First clinical data showing the potential therapeutic benefits of Scholar Rocks innovative scientific... More