Scholar Rock

Scholar Rock is a late-stage biopharmaceutical company focused on developing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders like obesity, and other serious diseases where protein growth factors play a fundamental role. Its lead product candidate, apitegromab, is an investigational fully human monoclonal antibody inhibitor of myostatin activation that selectively binds to the pro- and latent forms of myostatin in skeletal muscle. Apitegromab is being evaluated in a pivotal Phase 3 SAPPHIRE trial for the treatment of non-ambulatory Types two and three SMA patients receiving SMN therapy. The company completed enrollment for the SAPPHIRE trial in September 2023 and expects to announce top line data in the fourth quarter of 2024. If successful and approved, Scholar Rock anticipates launching apitegromab commercially in 2025.

The company also has a preclinical investigational myostatin inhibitor, SRK-439, with high affinity for pro- and latent myostatin, being developed initially for obesity treatment. Scholar Rock plans to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 receptor agonist in obesity patients in mid-2024, subject to IND clearance, to inform the clinical development of SRK-439. Data from this trial is expected in mid-2025, and the company aims to file an IND for SRK-439 in obesity in 2025.

Additionally, Scholar Rock's pipeline includes SRK-181, a selective inhibitor of latent TGF-β1 activation being evaluated in a Phase 1 DRAGON proof-of-concept trial to overcome resistance to checkpoint inhibitor therapy in advanced cancers. As of August 2023, SRK-181 demonstrated favorable tolerability and promising anti-tumor activity in heavily pre-treated clear cell renal cell carcinoma patients resistant to anti-PD-1 therapy. The company believes the DRAGON trial supports proof-of-concept for SRK-181 and is completing enrollment in December 2023 while continuing treatment for patients remaining on study.