Biohaven Completes Enrollment in Pivotal Phase 3 Study of Taldefgrobep Alfa in Spinal Muscular Atrophy
- Taldefgrobep alfa is the only myostatin inhibitor in clinical development that targets both myostatin and activin A signaling, two key regulators of muscle mass and adipose tissue
- RESILIENT, the pivotal clinical trial in spinal muscular atrophy, was designed to test the efficacy and safety of taldefgrobep alfa as adjunctive therapy to increase muscle in SMA patients treated with standard of care nusinersen, risdiplam or onasemnogene abeparvovec-xioi
Taldefgrobep is an investigational, muscle-targeted recombinant protein with the potential to enhance muscle mass and strength in people living with SMA when used in combination with other approved treatments. Taldefgrobep targets myostatin, a natural protein that limits skeletal muscle growth. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.
RESILIENT is a Phase 3 placebo-controlled, double-blind trial designed to evaluate the efficacy and safety of taldefgrobep at 48 weeks as an adjunctive therapy for participants who are already taking a stable dose of nusinersen, risdiplam and/or have a history of treatment with onasemnogene abeparvovec-xioi, compared to placebo. The study is not restricted nor limited to patients based on ambulatory status or classification of SMA.
Taldefgrobep was granted EU Orphan Drug Designation, along with both Fast Track and Orphan Drug Designation by the
"While we have had good progress with current therapies, a high unmet need for safe and effective supportive treatments for SMA remains, as many patients still experience significant weakness and reduced levels of functioning. We appreciate the partnership of investigators, patients, and researchers to expedite the development of new efficacious therapies that will work in combination with current options to help restore muscle strength and function," said
About Taldefgrobep alfa
Taldefgrobep alfa (BHV-2000) is a fully human recombinant protein specifically designed to inhibit the signaling of myostatin and activin A; two key regulators of muscle and adipose tissue. Taldefgrobep binds myostatin and acts as an Activin 2b receptor antagonist. Taldefgrobep's novel mechanism of action offers the potential for meaningful reductions in fat mass, increased lean mass, and improvements in multiple metabolic parameters.
About Spinal Muscular Atrophy (SMA)
Spinal muscular atrophy (SMA) is a rare genetic neurodegenerative disorder characterized by the loss of motor neurons, atrophy of the voluntary muscles of the limbs and trunk and progressive muscle weakness that is often fatal and typically diagnosed in young children. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. Globally, SMA affects approximately 1 in 11,000 births, and about 1 in every 50 individuals is a genetic carrier.
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