Investing.com — Shares of Scholar Rock Holdings skyrocketed 320% on Monday following the announcement of optimistic outcomes from its Part 3 SAPPHIRE scientific trial for apitegromab, a remedy for spinal muscular atrophy (SMA).
The trial met its major endpoint, exhibiting statistically vital enhancements in motor operate as measured by the Hammersmith Practical Motor Scale Expanded (HFMSE).
Scholar Rock defined in its press launch that 30.4% of sufferers receiving apitegromab skilled a larger than three-point enchancment in comparison with solely 12.5% of these on a placebo.
Dr. Jay Backstrom, President and CEO of Scholar Rock, expressed enthusiasm for the outcomes, stating, “The results clearly demonstrate robust and clinically meaningful improvement in motor function in patients with SMA.”
The corporate plans to submit a U.S. Biologics License Utility and European Union advertising and marketing authorization software within the first quarter of 2025.
The corporate added that was no new security findings have been noticed within the SAPPHIRE scientific trial and the profile was in keeping with that noticed within the Part 2 TOPAZ scientific trial.
Following the information, Truist analysts stated in a be aware that the research confirmed a clinically significant enchancment of 1.8 factors in comparison with placebo, reinforcing the unmet want for efficient SMA remedies.
They reiterated a Purchase ranking for Scholar Rock and elevated the worth goal from $20 to $36 per share.
“We see this as a validation of the company’s TGFbeta platform and of their strategy to evaluate anti-myostatin (a drug that improves muscle) on top of a drug that targets the genetic cause of the disease,” stated Truist.
“We also see these data as a positive read-through to the company’s obesity program with readout in 2Q2025,” they added. As a reminder, whereas apitegromab is being evaluated in a Ph2 trial, firm plans to develop SRK-439, a subsequent gen anti-myostatin drug that has been engineered particularly for CVD in weight problems.”