Neurocrine Biosciences has only days later been forced to announce a phase 3 failure following a series of upbeat R&D.
Ingrezza (valbenazine), the approved medication of the company, to treat some uncontrolled movement conditions, did not give a significant difference, according to a phase 3 trial in patients with dyskinesia caused by cerebral palsy (CP), Neurocrine announced Monday.
The phase 3 trial was not able to achieve its primary endpoint because Ingrezza did not outperform placebo on the enhancement of involuntary, jerky body motions after 14 weeks of treatment. Neurocrine also said that the study, which was referred to as Kinect-DCP, failed to achieve material secondary endpoints.
Dyskinetic CP is a type of CP, which is a nonprogressive neurological disorder that interferes with body movement and posture. Dyskinetic CP represents approximately 15 percent of all CP, and it is characterized by several involuntary movements, such as dystonia, chorea, and athetosis.
Ingrezza is a vesicular monoamine transporter 2 blocker that is used to treat two other conditions that are characterized by uncontrolled movements: tardive dyskinesia and chorea in Huntington’s disease.
At present, no treatment is approved that can be used to treat uncontrolled movements in CP. The miss in dyskinetic CP is not a successful one, though it does not have much influence on the business of Neurocrine in the expectations of the analysts. In a Dec.16 note, Jefferies analysts estimated only a 10 percent success rate of Ingrezza in dyskinetic CP. According to William Blair analysts, the impact of the failure on their valuation of Neurocrine was minimal.
Commercialization of Ingrezza and initiation process of novel FDA-approved Crenessity in a genetic disorder that impacts on adrenal glands are the current sources of value to the company. In the third quarter, Ingrezza sales grew by 12 per cent year over year to $687 million, and Crenessity brought in 98 million after gaining FDA approval in December 2024.
The company has two late-stage candidates, which are osavampator and direclidine, on the pipeline side. In major depressive disorder, beginning the year, neurocrine initiated the phase 3 trials of AMPA modulator, potentially first-in-class, neurocrine advanced osavampator, and the preliminary readout is anticipated in 2027. M4 agonist direclidine is also planned to undergo a phase 3 readout in 2027 in schizophrenia.
Neurocrine executes two next-generation VMAT2 inhibitors, the first being NBI-1065890, as reported in the R&D day update on Dec. 16, which will be entering phase 2 shortly in tardive dyskinesia.
Neurocrine is also no exception as it is approaching the obesity market, presenting at the R&D event a CRF2 agonist that will be in the clinic by the first half of 2026. The company has intended to co-formulate the drug, NBIP-’2118, with its own agonist (single-molecule conjugation), which is a triple G.
Despite the setback in dyskinetic cerebral palsy, Ingrezza continues to demonstrate value in Neurocrine’s established indications. The drug’s consistent performance in tardive dyskinesia and Huntington’s disease chorea underscores its clinical reliability and reinforces confidence among healthcare providers and patients. Analysts highlight that this track record positions Ingrezza as a cornerstone therapy, allowing Neurocrine to continue investing in its pipeline of novel neurological and rare disease treatments.
Moving forward, Neurocrine is expected to leverage the data and insights from the Kinect-DCP trial to refine patient selection strategies and explore potential combination therapies. These efforts aim to optimize treatment outcomes and identify niche patient populations that may benefit from Ingrezza in the future.
The company’s broader pipeline remains robust, with multiple late-stage candidates targeting major depressive disorder, schizophrenia, and rare genetic disorders. By advancing therapies such as osavampator, direclidine, and next-generation VMAT2 inhibitors, Neurocrine is positioning itself for sustainable long-term growth beyond Ingrezza.
Additionally, Neurocrine’s strategic initiatives in obesity and endocrine disorders signal a commitment to diversifying its portfolio while leveraging existing expertise in neurology. These upcoming programs complement Ingrezza’s market presence and demonstrate the company’s ability to innovate across multiple therapeutic areas.
