Positive Interim Results from Phase I Trial Announced by Scholar Rock

18 March 2019

Scholar Rock have announced positive interim results from an ongoing Phase I trial in healthy volunteers of their lead compound, SRK-015.

SRK-015 is a muscle-directed therapy that has the potential to reverse or restrict muscle atrophy and weakening, and is therefore a drug that may prove beneficial for SMA patients. Consequently, SRK-015 has received Orphan Drug Status in the US (click here for more information) and Orphan Medicinal Product Designation in Europe (click here for more information) for the treatment of SMA, which can expedite the drug development processes.

The Phase I trial was designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous injections (into the bloodstream) of SRK-015 in healthy adult volunteers (click here for more information).

The interim trial results indicate that SRK-015 is well tolerated with no apparent safety issues. Additionally, the drug was shown to successfully interact in a sustained manner with the protein it was designed to target, resulting in a desired effect.

Rather than attempting to replace the SMN protein that is lacking in SMA patients, SRK-015 can target an important protein pathway that usually restricts muscle growth and function. By altering this pathway, SRK-015 can potentially release the brakes on muscle cell growth leading to an increase in muscle size and function.

The latest positive results provide sufficient evidence to advance SRK-015 to a proof-of-concept Phase II trial with SMA patients, which is expected to commence in the coming months.

The trial is called TOPAZ, will involve 50-60 patients with SMA Types 2 and 3, and will test injections of SRK-015 every four weeks.

Interim safety and efficacy analyses from this Phase II trial are expected in the first half of 2020, allowing results to be generated from patients receiving the drug for at least six months.


Additional Information

Scholar Rock Press Release