Analysts said the outcome is disappointing because there are no approved treatments for dyskinetic cerebral palsy, but the setback had little impact on Neurocrine’s valuation.
A Phase III trial of Neurocrine Biosciences’ valbenazine in dyskinetic cerebral palsy has missed its primary and key secondary endpoints.
The study randomized children and adults with the nonprogressive neurodevelopmental disorder to take either valbenazine, which Neurocrine sells as Ingrezza in other indications, or placebo orally once a day for 14 weeks. Neurocrine’s primary endpoint looked at changes in the severity of chorea, the involuntary, jerky movements associated with the condition, from baseline to the final two weeks of the trial.
San Diego-based Neurocrine said the trial missed its primary endpoint but has yet to share data. Secondary endpoints tracked the effect of valbenazine on other motor symptom measures and different aspects of dyskinetic cerebral palsy (DCP).
William Blair analysts said in a note to investors that the failure is disappointing because there are no approved treatments for DCP. While Neurocrine’s press release lacked a comment on the future of valbenazine in DCP, the analysts removed the indication from their assessment of the value of the vesicular monoamine transporter 2 inhibitor.
DCP was a small part of the Neurocrine valuation calculated by the analysts, who said the removal of the opportunity had a minimal impact on their target share price or thesis. Neurocrine shares fell 1.55% to $145.25 in premarket trading Tuesday. The analysts said investors will be focused on the company’s execution and commercial performance in 2026.
Neurocrine markets Ingrezza in tardive dyskinesia and chorea associated with Huntington’s disease. Product sales rose 12% to $687 million in the third quarter. Investors saw the Inflation Reduction Act negotiations for Teva Pharmaceuticals’ rival drug Austedo as a potential headwind for Ingrezza going into the reveal of the new price last month.
With Teva agreeing to a 38% price cut, BMO Capital Markets analysts said in a note to investors that the “relatively low discount” reads as a positive for Neurocrine’s Ingrezza. Eric Benevich, Neurocrine’s chief commercial officer, said at a Piper Sandler event this month that the situation is “very manageable,” adding that the company expects to maintain coverage in 2027 and continue to grow.
The launch of Crenessity, which won FDA approval in congenital adrenal hyperplasia one year ago, is the company’s other commercial focus. Neurocrine reported Crenessity sales of $98 million in the third quarter. Matt Abernethy, Neurocrine’s chief financial officer, said at the Piper Sandler event that the company is “not satisfied with where we’re at” with the drug, adding that “we’re going to accelerate that path to peak.”
Beyond its commercial drugs, Neurocrine has late-stage programs in neuropsychiatry and an early-stage pipeline in obesity that it discussed at a recent R&D day. BMO analysts said the event featured “positive neuropsych updates followed by puzzling disclosures around new efforts in obesity.” The analysts expressed concern about Neurocrine’s ability to generate a return on investment in the fast-moving obesity field.
