The first patient with spinal muscle atrophy (SMA) enrolled in Biohaven’s phase 3 trial of taldefgrobep alfa has been reported.

The placebo-controlled, double-blind, randomized RESILIENT trial is designed to evaluate the efficacy and safety of taldefgrobep as an adjunctive treatment for individuals who are already receiving a stable dose of nusinersen or risdiplam, or who have previously been treated with onasemnogene abeparvovec-xioi, compared with placebo.

Taldefgrobep is an investigational, muscle-targeted recombinant protein that has shown the potential to enhance strength and muscle mass when combined with other approved therapies. The agent targets myostatin—a natural protein that limits the growth of skeletal muscle via 2 distinct mechanisms: lowering myostatin directly and blocking key downstream signaling mechanisms.

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Among children and adults with a variety of neuromuscular disorders, active myostatin can limit the skeletal muscle growth required to attain functional and developmental milestones. Also known as BMS-986089, taldefgrobep alfa is a modified adnectin that is designed to specifically bind to myostatin.

“There have been great strides in advancing therapeutics in this challenging disease. Yet, there remains a significant unmet need to address the residual weakness and functional impairments, such as difficulty walking, that are caused by [SMA],” Irfan Qureshi, MD, senior vice president of neurology at Biohaven, said.

It is anticipated that Biohaven will enroll approximately 180 patients with SMA in the RESILIENT trial.

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SMA is a rare genetic neurodegenerative disorder that is characterized by atrophy of the voluntary muscles of the limbs and trunk, the loss of motor neurons, and progressive muscle weakness that is frequently fatal and is usually diagnosed among young children.

In patients with SMA, insufficient production of the survival of motor neuron (SMN) protein has been reported. The SMN protein, which is essential for the survival of motor neurons, is encoded by 2 genes—SMN1 and SMN2. In the United States, SMA affects approximately 1 in 11,000 births. Around 1 in every 50 individuals in the United States is a genetic carrier of SMA.


Biohaven enrolls first patients in phase 3 trial of taldefgrobep alfa in spinal muscle atrophy (SMA). News release. Biohaven Pharmaceuticals; July 7, 2022.