Just days after an upbeat R&D event, Neurocrine Biosciences has found itself having to report a phase 3 failure.
The company’s Ingrezza (valbenazine), approved to treat certain uncontrolled movement conditions, failed to make a significant difference in a phase 3 trial for patients with dyskinesia due to cerebral palsy (CP), Neurocrine announced Monday.
Ingrezza didn’t outperform placebo on improving involuntary, jerky movements of the body after 14 weeks of treatment, causing the phase 3 trial to miss its primary endpoint. The study, dubbed Kinect-DCP, also did not meet key secondary endpoints, according to Neurocrine.
Dyskinetic CP is a subtype of CP, which is a nonprogressive neurological disorder that affects body movement and posture. Dyskinetic CP accounts for about 15% of all CP cases and is marked by various involuntary movements, including dystonia, chorea and athetosis.
Ingrezza is a vesicular monoamine transporter 2 inhibitor indicated for two other neurological disorders with uncontrollable movements: tardive dyskinesia and chorea associated with Huntington’s disease.
Currently, there are no approved therapies to treat uncontrolled movements in CP. While disappointing, the miss in dyskinetic CP has little impact on Neurocrine’s business within analysts’ expectations. In a Dec. 16 note, Jefferies analysts figured Ingrezza only had a 10% chance at success in dyskinetic CP. William Blair analysts labeled the fail’s effect on their valuation of Neurocrine as “minimal.”
The value of the company is currently driven by commercialization of Ingrezza and the launch progress of newly FDA-approved Crenessity in a genetic condition that affects the adrenal glands. In the third quarter, Ingrezza sales increased by 12% year over year to reach $687 million, while Crenessity generated $98 million following its FDA nod in December 2024.
On the pipeline side, the company’s focus is on two late-stage candidates, osavampator and direclidine. Neurocrine advanced osavampator, a potential first-in-class AMPA modulator, into phase 3 trials in major depressive disorder at the beginning of the year, with an initial readout expected in 2027. Direclidine, an M4 agonist, is also slated for a phase 3 readout in 2027 for schizophrenia.
The company is developing two next-generation VMAT2 inhibitors, led by NBI-1065890, which will enter phase 2 soon in tardive dyskinesia, according to Neurocrine’s R&D day update Dec. 16.
Neurocrine is also entering the obesity ring, unveiling at the R&D event a CRF2 agonist set to enter the clinic in the first half of 2026. The company plans to combine the drug, coded NBIP-’2118 with its own “triple G” agonist, as both a co-formulation and in a single-molecule conjugate form.