BioCentury
ARTICLE | Company News

Scholar Rock developing lead candidate for SMA

June 28, 2017 9:41 PM UTC

Scholar Rock LLC (Cambridge, Mass.) said it will develop its lead candidate SRK-015 to treat spinal muscular atrophy. CEO Nagesh Mahanthappa told BioCentury the company plans to submit an IND and begin Phase I testing by mid-2018 of the candidate, an inhibitor of latent myostatin (MSTN; GDF8) activation.

The company had said it planned to develop SRK-015 to treat muscle diseases and primary myopathies, but Scholar Rock had not named any specific indications. The company's programs target growth factors in cell and tissue microenvironments to generate specific therapeutic effects at the site of disease (see BioCentury Innovations, Sept. 3, 2015 & BioCentury Extra, Sept. 23, 2015)...