Scholar Rock (SRRK.US) stock surges 335% as its SMA candidate drug succeeds in Phase 3 trial

Scholar Rock Holding Corp. (SRRK.US) saw its stock price soar 335% during Monday's trading, driven by the successful achievement of the primary endpoint in the third phase trial of its SMA candidate drug apitegromab. The positive result indicates that the apitegromab monoclonal antibody significantly improves patient motor function, particularly at week 52, with a statistically significant improvement compared to the placebo group.

In the SAPPHIRE study, patients receiving apitegromab showed an average improvement of more than 3 points on the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52, compared to only 12.5% of patients receiving placebo.

In addition, early improvements in motor function were observed in patients using apitegromab as early as week 8, and this improvement persisted throughout the 52-week study period.

Given these positive data, Scholar Rock plans to submit regulatory applications for apitegromab to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the first quarter of 2025.

This move is expected to bring new treatment options for SMA patients and may further propel Scholar Rock's growth in the biopharmaceutical sector.