Neurocrine's Movement Disorder Drug Fails Final Trial

• The drug, already marketed as Ingrezza for other movement disorders, was being tested in the second most common form of cerebral palsy characterized by uncontrolled movements
• Chief Medical Officer Sanjay Keswani called the results 'disappointing,' highlighting the unmet medical need in dyskinetic cerebral palsy
• Valbenazine is currently approved in the U.S. for tardive dyskinesia and Huntington's disease-related chorea, generating significant revenue for Neurocrine

Neurocrine Biosciences announced Monday that its drug valbenazine failed to meet the primary endpoint in a Phase 3 trial for dyskinetic cerebral palsy, causing disappointment for patients lacking approved treatment options. The 14-week study showed no significant improvement in chorea (involuntary movements) compared to placebo in patients with this condition, which represents the second most common form of cerebral palsy.

The setback impacts Neurocrine's efforts to expand valbenazine's therapeutic applications beyond its current FDA-approved uses. The drug, marketed as Ingrezza, is already approved in the U.S. for treating tardive dyskinesia and chorea associated with Huntington's disease. Dyskinetic cerebral palsy affects the brain's muscle control, causing uncontrolled, involuntary movements that differ from other cerebral palsy types characterized by muscle stiffness or poor coordination.

Chief Medical Officer Sanjay Keswani acknowledged the disappointing results, emphasizing the unmet medical need in this patient population. The company plans to present complete study data at a future scientific meeting, though specific timing was not disclosed.

This trial failure represents a notable setback for Neurocrine's pipeline expansion strategy and leaves dyskinetic cerebral palsy patients without new treatment options. The market implications include potential reassessment of valbenazine's broader therapeutic potential and possible impact on Neurocrine's growth projections, though the drug's existing approvals remain unaffected. The announcement may prompt investors to reevaluate the company's development pipeline and future revenue opportunities in movement disorders beyond its established indications.