Improving lives is the foundation of our values and our ultimate endpoint. From raising disease awareness to funding research, we strive to champion what matters most to patients and families, cultivating a support system that responds to their physical, mental and emotional needs for well-rounded health.
See our progress
Follow our product candidates on their journeys to fighting serious diseases.
Spinal Muscular Atrophy
Apitegromab
Inhibit the activation of latent myostatin to promote a potentially clinically beneficial increase in muscle function
Oncology
SRK-181
Potential to overcome primary resistance to checkpoint inhibitors to reach a larger patient population
Anemias
Target RGMc—a co-receptor of BMP6—to potentially treat diseases associated with dysregulation of iron metabolism
Fibrosis
Inhibit TGFβ signaling to potentially offer a powerful new approach to suppressing pro-fibrotic signaling in multiple organs
“We focus on the patients here; they’re the reason we’re at Scholar Rock.”
Peter McLaughlin
Senior Director, IT
Clinical Trials
We focus on the discovery and development of safe and effective therapies to find optimal solutions for those who need it.
How it Works
A clinical trial is a type of research involving human volunteers (also called participants) that follow strict protocols to evaluate whether a new drug is safe and effective in people. Clinical trials are usually conducted in multiple phases, each of which are designed to answer specific scientific questions. Data from these studies are then submitted to regulatory agencies, (e.g. US Food & Drug Administration), to gain approval of the new drug or treatment.
Find the safest dose
Test for beneficial effects and undesirable effects
Monitor adverse reactions
Test long term safety, in a diverse patient population
Phase 1
Focus on the study drug’s safety and tolerability. Phase 1 trials evaluate multiple doses in a small number of individuals that are, often, healthy volunteers.
Phase 2
Focus on the study drug’s safety and efficacy in patients with the disease or condition being studied. If a study drug is found to be reasonably safe in Phase 1 trials, it will then be investigated in Phase 2.
Phase 3
Focus on the study drug’s safety and efficacy, oftentimes compared to standard-of-care or control groups. Phase 3 trials typically evaluate much larger numbers of patients and follow the most rigorous protocols of the different phases. A study drug that passes Phase 3 can be submitted to regulatory agencies for approval to be marketed to the general public.
Phase 4
Pursue ongoing evaluation and monitor the study drug in patients, even after receiving regulatory approval.
Expanded Access Policy
As we advance our investigational therapies through clinical development, our goal is to provide access to these therapies at the appropriate time and in the correct manner for patients.
December 13, 2024
Scholar Rock Reports New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
November 15, 2024
Scholar Rock Reports New Employee Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)
November 12, 2024
Scholar Rock Reports Third Quarter 2024 Financial Results and Highlights Business Progress
Events
View More EventsNovember 12, 2024
Scholar Rock 3Q 2024 Financial Results and Business Update Conference Call