Scholar Rock Announces Positive Final Results from Phase 1 Clinical Trial of SRK-015 in Healthy Volunteers

11 June 2019

Scholar Rock announced that SRK-015 was well-tolerated in healthy volunteers across all tested doses and shows robust and durable target engagement, supporting evaluation in the Phase 2 trial in SMA, which is currently ongoing.

SRK-015 is a muscle-directed therapy that has the potential to address motor function impairment in patients with SMA. The European Commission has granted Orphan Medicinal Product Designation to SRK-015 for the treatment of SMA.

Phase 1 of this randomised, double-blind, placebo-controlled clinical trial was designed to evaluate the safety and tolerability of intravenously administered SRK-015, assess its pharmacokinetic (the effect the body has on the drug) and pharmacodynamic (the effect the drug has on the body) profiles and inform dosing for the Phase 2 trial

Forty adult healthy volunteers were enrolled to receive a single dose of SRK-015 or placebo, and evaluated doses of 1, 3, 10, 20 and 30 mg/kg. 

In addition, 26 adult healthy volunteers were enrolled to receive SRK-015 or placebo, every two weeks for a total of three doses (Day 0, 14 and 28) of 10, 20 and 30 mg/kg.

Consistent with previously announced interim findings, the final results showed that SRK-015 successfully engages with its target, a protein which inhibits muscle growth, and no apparent safety signals were observed across all tested doses.  Detailed results from this Phase 1 trial will be presented at the Cure SMA Annual Conference, which will take place between 28th June and 1st July in Anaheim, California. 

Dosing of patients in the Phase II clinical trial (called TOPAZ) in SMA Types 2 and 3 began in May. Scholar Rock is hoping to enrol approximately 55 patients across the U.S., Canada, and Europe.

Further information

The press release

The Phase 2 announcement