Apitegromab (SRK-015)
Spinal Muscular Atrophy (SMA)
Key Facts
About Scholar Rock Holding
Scholar Rock is a public biotech company leveraging its unique understanding of protein growth factor activation to develop novel therapeutics. Its core technology platform targets the latent, inactive forms of proteins, aiming for greater specificity and reduced side effects compared to targeting the mature, active forms. The company's most advanced program, apitegromab, has shown promising Phase 2 results in SMA and is advancing into pivotal trials, positioning Scholar Rock as a key player in neuromuscular therapeutics. Beyond SMA, the company is advancing programs in oncology and immuno-oncology.
View full company profileAbout Scholar Rock Holding
Scholar Rock is a public biotech company leveraging its unique understanding of protein growth factor activation to develop novel therapeutics. Its core technology platform targets the latent, inactive forms of proteins, aiming for greater specificity and reduced side effects compared to targeting the mature, active forms. The company's most advanced program, apitegromab, has shown promising Phase 2 results in SMA and is advancing into pivotal trials, positioning Scholar Rock as a key player in neuromuscular therapeutics. Beyond SMA, the company is advancing programs in oncology and immuno-oncology.
View full company profileTherapeutic Areas
Other Spinal Muscular Atrophy (SMA) Drugs
| Drug | Company | Phase |
|---|---|---|
| Evrysdi (risdiplam) | PTC Therapeutics | Approved |
| Taldefgrobep alfa | Biohaven | Phase 3 |