By Christy Santhosh
(Reuters) -Scholar Rock said on Monday its experimental drug apitegromab met the main goal of a late-stage trial in patients with a type of rare genetic disease, sending the company’s shares surging in premarket trading.
Apitegromab was statistically significant in improving measures of motor function such as standing and crawling, compared to a placebo, in patients with spinal muscular atrophy.
The company said it would file a marketing application for the drug with the U.S. Food and Drug Administration in the first quarter of 2025.
The drug is also being evaluated in a mid-stage trial to preserve lean muscle mass in obese individuals on GLP-1 weight-loss drugs.
SMA, the leading genetic cause of infant deaths, affects about 1 in 10,000 people, according to government data. It prevents the body from producing a protein necessary for neuromuscular development and leaves children too weak to walk, talk and swallow.
If approved, analysts at Wedbush project sales of $50 million to $55 million for apitegromab in 2026.
Shares of Scholar Rock, which had a market capitalization of nearly $600 million as of Friday’s close, tripled to $22.50 in premarket trading.
(Reporting by Christy Santhosh; Editing by Pooja Desai)
