Scholar Rock is a biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our lead product candidate, SRK-015, a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We have successfully completed a Phase 1 clinical trial in healthy volunteers and initiated a Phase 2 proof-of-concept trial (named TOPAZ) in patients with later-onset (Type 2 and Type 3) SMA in the second quarter of 2019. In addition, we are planning to initiate a Phase 1 trial for our second product candidate, SRK-181, a highly specific inhibitor of TGFbeta1, in patients with solid tumors in mid-2020. We believe SRK-181 has the potential to render resistant solid tumors vulnerable to immune checkpoint inhibitors such as anti-PD1/PDL1 therapies and drive tumor regression through combination therapy. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia. Source
No articles found.
Neovasc is a specialty medical device company that develops, manufactures and mark...
Neovasc is a specialty medical device company t...
Intec Pharma is a clinical-stage biopharmaceutical company focused on developing d...
Intec Pharma is a clinical-stage biopharmaceuti...
Helius Medical Technologies, Inc. (Helius) is a neurotech company in the medical d...
Helius Medical Technologies, Inc. (Helius) is a...
PLx Pharma Inc. (Nasdaq: PLXP), is a late-stage specialty pharmaceutical company i...
PLx Pharma Inc. (Nasdaq: PLXP), is a late-stage...
At Bellerophon Therapeutics, we aim to transform the lives of people suffering fro...
At Bellerophon Therapeutics, we aim to transfor...
MyoKardia is a clinical-stage biopharmaceutical company pioneering a precision med...
MyoKardia is a clinical-stage biopharmaceutical...
Epizyme, Inc. is a clinical stage biopharmaceutical company creating novel epigene...
Epizyme, Inc. is a clinical stage biopharmaceut...
Celyad is a biopharmaceutical company, specialized in CAR-T cell therapy, that is ...
Celyad is a biopharmaceutical company, speciali...
CytoDyn is a publicly traded late stage biotechnology company developing innovativ...
CytoDyn is a publicly traded late stage biotech...
Sarepta Therapeutics is a U.S. commercial-stage biopharmaceutical company focused ...
Sarepta Therapeutics is a U.S. commercial-stage...
Join the National Investor Network and get the latest information with your interests in mind.