Successful SAPPHIRE Phase III Study
The pivotal Phase III SAPPHIRE study for apitegromab in spinal muscular atrophy met the primary endpoint with a 1.8 point improvement on the Hammersmith Functional Motor Scale, with a statistically significant p-value of 0.0192. 30% of patients on apitegromab achieved a 3-point improvement, compared to 12.5% for placebo.
Strong Financial Position
Scholar Rock completed an upsized follow-on offering of $345 million, leading to a pro forma September 30 cash balance of approximately $463 million, enabling them to scale up operations and focus on key priorities.
Regulatory and Commercial Preparation
Plans are in place to submit the BLA and MAA for apitegromab in Q1 2025, with a commercial launch anticipated in Q4 2025, assuming regulatory approval.
Advancements in Obesity Treatment
The nonclinical data for SRK-439, a novel anti-myostatin program in obesity, shows promising results in preserving lean muscle mass during weight loss, with plans to submit the IND by mid-2025.