SRK-015

Summary

Scholar Rock is developing SRK-015, a drug that specifically blocks the latent form of myostatin in order to increase muscle mass and potentially treat spinal muscular atrophy (SMA). Myostatin is a member of the TGF-beta superfamily of growth factors, and the specificity of SRK-105 is intended to prevent the drug from blocking the myostatin receptor, activated myostatin, or any other member of this superfamily, thus greatly reducing the risk of side effects.

SRK-015 is currently in phase 1 clinical trials, and while this drug is being developed for a specific disorder, its application for other, age-related disorders is obvious. It could potentially be used to combat sarcopenia, the age-related loss of muscle mass and strength that many older people experience and that leads to frailty and increased risk of falls and injury. Scholar rock is planning a Phase 2 trial in patients with later-onset SMA in the first quarter of 2019.

References: Pirruccello-Straub, M., Jackson, J., Wawersik, S., Webster, M. T., Salta, L., Long, K., … & Mahanthappa, N. K. (2018). Blocking extracellular activation of myostatin as a strategy for treating muscle wasting. Scientific
reports, 8(1), 2292.

Website: Scholar Rock

Article: FDA Grants Orphan Drug Status to SRK-015 for SMA