First Cohort Dosing Completed in SRK-015 Phase 1 Clinical Trial

08 June 2018

Pharmaceutical company Scholar Rock has announced that dosing of the first cohort has been completed in a Phase I trial of the experimental drug, SRK-015.

The Phase I trial has been designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous injections of SRK-015 in healthy adult volunteers.

The trial will evaluate single- and multiple-ascending doses and is placebo-controlled and double-blind, meaning that volunteers will be allocated to either a treatment or placebo group, and that this will be concealed from the volunteers and people administering the treatment.

Recently granted Orphan Drug Designation for the treatment of SMA by the US Food and Drug Administration (click here for more information), SRK-015 is an SMN-independent, muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by those with SMA.

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. If this is successful, this could benefit people with SMA.

If SRK-015 proves safe, Scholar Rock intend on starting a Phase II trial at the start of 2019, which will further assess the safety of the drug and begin to determine efficacy in patients with later-onset SMA. In this future trial, it is expected that SRK-015 will be tested in isolation or in combination with an approved SMN-dependent therapy.

Further Information

Scholar Rock Press Release