Spinal Muscular Atrophy
We are committed to addressing the SMA community’s top remaining unmet needs by advancing fundamentally different treatment approaches for people living with SMA around the world.
Other treatment approaches were designed to address the motor neuron component of the disease 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 is required to fuel motor function, as motor function relies not only on motor neuron signaling but also muscle responsiveness – underscoring the importance of treatment approaches that directly address both the motor neuron and muscle.
Clinical Trials
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.
