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Scholar Rock Shines On TOPAZ Data, Stock Hits New High

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Shares of Scholar Rock (SRRK) soared to a new high in Monday's trading, on the back of positive six-month interim analysis results from the company's phase II trial of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy, dubbed TOPAZ.

Spinal muscular atrophy is an inherited neuromuscular disease caused by a mutation in the SMN1 gene, which is located on chromosome 5 and is characterized by muscle weakness. Scholar Rock's investigational SRK-015 is designed to improve muscle strength and motor function in patients with Spinal Muscular Atrophy.

In the TOPAZ trial, motor function improvements were observed for all three SRK-015 treatment cohorts in the primary efficacy endpoint of Hammersmith scale scores at six-months. The high dose of SRK-015 attained a 5.6 point mean improvement at six-months over baseline while the low dose attained a 2.4 point mean improvement over baseline.

The study involved 58 patients with Type 2 and Type 3 spinal muscular atrophy in the U.S. and Europe.

The 12-month results of the TOPAZ study are anticipated in the second quarter of 2021.

It is estimated that 10,000 to 25,000 children and adults in the United States have spinal muscular atrophy, according to the SMA Foundation. Novartis' gene therapy Zolgensma, Biogen's Spinraza, and Roche's Evrysdi are the FDA-approved treatments.

SRRK is up 76.90%, trading at $24.27.

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