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 motor function. 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 has completed a Phase 1 clinical trial of SRK-015 in adult healthy volunteers. SRK-015 was observed to be well-tolerated with no dose-limiting toxicities identified up to the highest evaluated dose of 30 mg/kg. 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 patients with SMA and could be used both as a monotherapy or in conjunction with any approved SMN upregulator therapy.

The safety and efficacy of SRK-015 is being investigated in the TOPAZ Phase 2 proof-of-concept clinical trial in patients with Type 2 and Type 3 SMA.  The trial enrolled 58 patients between the ages of 2 and 21 in the U.S., and Europe across three distinct cohorts.  All patients 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.

In the fourth quarter of 2020, we plan to report interim efficacy, safety, pharmacokinetic (PK), and pharmacodynamic (PD) results for patients across the three cohorts who have progressed through at least six months of the treatment period. Top-line data for the full 12-month treatment period are anticipated in the first half of 2021.

For more information on the TOPAZ trial, please refer to

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