Scholar Rock Summarises Progress To Date With SRK-015

14 January 2020

SRK-015 is an experimental therapy directed at the muscle, that aims to reverse or restrict the atrophy and weakness experienced by SMA patients. SRK-015 works by specifically inhibiting an important protein called myostatin, which can restrict the growth and function of muscles. By targeting myostatin, SRK-015 can potentially release the breaks on muscle cell growth leading to an increase in muscle size and function.

Progress to date:

The preliminary pharmacokinetic and pharmacodynamic analysis of the TOPAZ trial included data from 29 patients across the three cohorts. These patients had received one dose of SRK-015 and were evaluated for four weeks as of the data cut-off.

The preliminary results demonstrated:

  • Presence of latent myostatin in patients with SMA, further supporting the relevance of this drug target in SMA
     
  • Robust and dose-dependent engagement between SRK-015 and latent myostatin
     
  • No clinically significant safety signals had been observed as of the data cut-off for this preliminary analysis.

Enrolment for the Phase 2 trial, which is evaluating the safety and efficacy of SRK-015, has now been completed.

Key Priorities for 2020:

In mid-2020, Scholar Rock plans to report on the interim safety and efficacy data from 6 months’ treatment exposure to SRK-015, from all three cohorts.

Top-line data for the full 12-month treatment period are expected in the fourth quarter of 2020 or in the first quarter of 2021.
 

Further information

The press release
 

More about the trials:

SCHOLAR ROCK REPORTS PRELIMINARY PHARMACOKINETIC AND PHARMACODYNAMIC DATA FROM TOPAZ PHASE 2 TRIAL OF SRK-015 FOR THE TREATMENT OF SMA

FIRST COHORT DOSING COMPLETED IN SRK-015 PHASE 1 CLINICAL TRIAL

SRK-015 (Scholar Rock) Updates

Clinical Trials