Six questions raised by Scholar Rock’s standout SMA data

The unexpected news that a myostatin inhibitor from Scholar Rock led to functional improvements in non-ambulatory spinal muscular atrophy patients after standard treatments stopped working sent the company’s shares up 362% on Monday, but the data raise a suite of questions about how and when to use the mAb.

Scholar Rock Holding Corp. (NASDAQ:SRRK) reported that apitegromab, which targets the pre- and latent forms of myostatin in muscle cells, led to an average 1.8-point improvement on the Hammersmith Functional Motor Scale – Expanded (HFMSE), which assesses motor ability of children and adults with Type 2 and 3 SMA. ...