SMA is a rare genetic disease that typically affects young children. Currently, there is no cure for spinal muscular atrophy (SMA), however, various pharmaceutical firms across the world are working to discover a novel, innovative treatment for the disorder. Support and treatment is offered by various firms to help patients with the disorder to at least have the best quality of life.

Scholar Rock, a US based biopharmaceutical company headquartered in Cambridge, reportedly announced that preclinical and Phase 1 data for SRK-015 would be showcased at World Muscle Society Congress (WMSC) which is being conducted between October 1 and October 5, 2019 at Copenhagen, Denmark.

SRK is an extremely selective inhibitor used against myostatin activation. Myostatin is a negative muscle mass regulator and is presently being examined inside the TOPAZ Phase 2 clinical trial to treat patients with Type 3 and Type 2 SMA.

CEO and President of Scholar Rock, Nagesh Mahanthappa, Ph.D, stated that collectively, the Phase 1 as well as preclinical healthy volunteer data has offered the base for further advancing the SRK-015 to the company’s TOPAZ Phase 2 clinical trial.

Mahanthappa added that the company is thrilled about the potential role of SRK-015 as the first muscle-directed treatment that addresses the prolonged functional deficits that by some patients suffering with SMA experience.

Mahanthappa further stated that the company looks forward to the data from forthcoming clinical read-outs of the TOPAZ study, beginning with the primary PK/PD results by 2019 end and interim efficacy as well as interim safety and efficacy data during the first half of 2020.

The preclinical data which would be showcased at WMS shows enhanced muscle strength following the administration of muSRK-015P, the mouse analog of the SRK-015, in mouse models of late and early SMN restoration.

Treatment with muSRK-015P resulted in approximately 20%-51% growth in maximal torque, at over ≥ 40 Hz, of the subject’s plantar flexor muscle group along with a higher percentage of the muscle fibers in comparison with placebo treatment.

The phase 1 trial to be showcased at WMS shows sustained and strong target engagement, including durable saturation, following the administration of SRK-015 in both multiple-ascending and single-ascending dose portions of the study.

Source credit: http://investors.scholarrock.com/news-releases/news-release-details/scholar-rock-present-preclinical-and-phase-1-clinical-data-srk