SCHOLAR ROCK HLDG DL-,001

Scholar Rock confirmed that the FDA requires post‑marketing conditions for ISEMBYLD, including a study on safety in pregnancy and 15‑day adverse‑event reporting for bone fractures. Management had previously (and incorrectly) stated that no such conditions existed. The stock then fell 6.4% to $51.85 and declined 9.7% on the week. Scholar Rock said it will carry out the requested studies and reporting in line with FDA requirements; analysts subsequently adjusted ratings and price targets. » More on de.investing.com

The FDA approved ISEMBYLD (apitegromab-mstn) as the first muscle-specific treatment for spinal muscular atrophy in adults and children aged two and older who are already receiving an SMN2-directed therapy. The news pushed Scholar Rock’s stock up about 9% pre-market to $60.45 and converts the company into a commercial biopharma company. Several analysts sharply raised price targets and reiterated buy ratings; apitegromab is projected to generate billions in sales by 2035. Scholar Rock reports roughly $492 million in cash and has independent fill-and-finish and manufacturing capabilities for the product launch. » More on de.investing.com

The FDA has approved Isembyld (Apitegromab‑mstn) for the treatment of spinal muscular atrophy in patients aged two and older. The approval is based on the phase‑3 SAPPHIRE study, in which patients receiving Isembyld in addition to standard therapy showed a significant improvement on the Hammersmith scale after one year; 34.2% achieved an increase of at least three points versus 13.5% with placebo. The US commercial launch has begun, the stock climbed more than 14% to an all‑time high, and Scholar Rock is shifting from a clinical biotech company to a commercial drugmaker. » More on deraktionaer.de