Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare, severe neuromuscular disease resulting in irreversible loss of motor neurons and progressive muscle wasting that causes continuous motor function decline throughout life.
The motor unit has two key parts: the motor neuron and the muscle. SMA affects muscle which is required to fuel motor function and activities of daily living like breathing, eating, holding one’s head upright and walking. Other treatment approaches were designed to address the motor neuron and do not directly address the muscle. Despite advances in SMN-targeted treatments, challenges remain in preserving motor function over time, demonstrating how muscle remains at the center of unresolved needs in SMA.
Muscle strength and motor function gains are amongst the top 3 unmet needs in SMA – underscoring the importance of approaches that target both the motor neuron and muscle for people living with SMA. Myostatin inhibition harnesses the body’s natural pathways to directly target the muscle.
Our Pipeline
We have worked to turn our scientific discoveries into a pipeline of therapies aimed at helping people with diseases where growth factors play a central role.
Myostatin: A Closer Look
Myostatin is a protein expressed primarily in skeletal muscle to negatively regulate muscle growth. When myostatin is activated, it works alongside other growth factors and hormones to maintain appropriate muscle mass. Learn how we are using our proprietary platform to selectively target myostatin. By selectively turning myostatin “off” and allowing muscle growth to occur, there is potential to build muscle mass.
Learn how we are using our proprietary platform to selectively target myostatin
Clinical Trials in SMA
OPAL
Spinal Muscular Atrophy
Initiated, recruiting
OPAL is a Phase 2 randomized, double-blind study designed to evaluate apitegromab in patients under two years of age with SMA who have been or are continuing to be treated with any currently approved SMN treatment, including onasemnogene abeparvovec.
ONYX
Spinal Muscular Atrophy
Active, not recruiting
ONYX is an ongoing open-label, multi-center extension study designed to assess the long-term safety and efficacy of apitegromab in participants with Type 2 and Type 3 SMA who completed the TOPAZ or SAPPHIRE trials.
SAPPHIRE
Spinal Muscular Atrophy
Completed
SAPPHIRE was a randomized, double-blind, placebo-controlled Phase 3 clinical trial that evaluated the safety and efficacy of apitegromab in non-ambulatory patients with Types 2 and 3 SMA who were receiving either nusinersen or risdiplam.
TOPAZ
Spinal Muscular Atrophy
Completed
The TOPAZ trial was a proof-of-concept, open-label Phase 2 trial evaluating the safety and efficacy of apitegromab in patients with Type 2 and Type 3 SMA.
Expanded Access Policy
At Scholar Rock, our goal is to provide access to our therapies at the appropriate time and in the correct manner for patients.
