SRK-015 for Spinal Muscular Atrophy (SMA)

SRK-015, our most advanced product candidate, is a selective and local inhibitor of the activation of myostatin. Based on existing research on the mechanism of myostatin in muscle growth and strength, Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically beneficial increase in muscle strength. Scholar Rock is developing and investigating SRK-015 as a potential treatment to improve muscle strength and motor function in patients with Spinal Muscular Atrophy (SMA).

Clinical Development of SRK-015 for SMA

Scholar Rock completed a Phase 1 clinical trial of SRK-015 in healthy volunteers, and announced positive interim results in February 2019.  In the Phase 1 trial, SRK-015 was well-tolerated with no apparent safety signals. Pharmacodynamic (PD) data showed SRK‑015 was able to successfully engage latent myostatin in a robust and durable fashion, providing proof-of-mechanism for this unique therapeutic approach of targeting the latent form of myostatin. SRK-015 also displayed a well-behaved pharmacokinetic (PK) profile with a serum half-life of 23-33 days across the evaluated doses. These favorable interim results supported the advancement of SRK-015 to a Phase 2 proof-of-concept clinical trial.

TOPAZ Phase 2 Clinical Trial in SMA

Scholar Rock believes that SRK-015 has the potential to be the first muscle-directed therapy to address muscle atrophy in SMA patients and could be used both as a monotherapy or together with any approved SMN upregulator therapy.

In the first quarter of 2019, we initiated a Phase 2 proof-of-concept clinical trial, TOPAZ, to evaluate the safety and efficacy of SRK-015 in patients with Type 2 and Type 3 SMA.  The trial is anticipated to enroll approximately 55 patients between the ages of 2 and 21 in the US, Canada and Europe across three distinct cohorts.  All patients will receive SRK-015 once every four weeks either as a monotherapy or in conjunction with an approved SMN upregulator treatment over a 12-month treatment period.  The primary efficacy endpoints will measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA.

For more information on the TOPAZ trial, please refer to

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