Announces Milestone KarXT NDA Acceptance for Schizophrenia, Zai Lab
Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announces that China’s National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for KarXT as a treatment for schizophrenia in adults. This announcement marks an important step for Zai Lab in its mission to address the substantial unmet medical needs of the over 8 million people living with schizophrenia in China.
Schizophrenia is a chronic and often debilitating mental illness that significantly affects a person’s thoughts, emotions, and behavior. It manifests through positive symptoms, such as hallucinations and delusions, negative symptoms, including social withdrawal and lack of emotional expression, and cognitive impairments, such as memory and concentration deficits. These symptoms severely impact daily functioning and quality of life, creating a critical need for more effective treatment options.
Zai Lab announces that current treatment options, primarily antipsychotic medications, often come with limited efficacy and undesirable side effects. As a result, many patients discontinue their medication prematurely, leading to relapses and a prolonged path to recovery. This gap in treatment options emphasizes the pressing need for new therapies, particularly in regions like China, where fewer than half of schizophrenia patients receive adequate treatment.
Dr. Rafael Amado, President and Head of Global Research and Development at Zai Lab, announces the importance of KarXT in addressing these challenges. “There are more than 8 million patients living with schizophrenia in China who face significant unmet needs due to the limited efficacy and undesirable side effects of current treatment options,” Dr. Amado explains. He announces that KarXT, in clinical trials, has demonstrated statistically significant reductions in schizophrenia symptoms while maintaining a tolerable safety profile. “If approved, KarXT has the potential to redefine the treatment landscape,” Dr. Amado states.
KarXT is a novel treatment for schizophrenia, representing the first new class of treatment for the condition in several decades. Dr. Gang Wang, Dean of Beijing Anding Hospital and leading principal investigator for the Phase 3 China study, announces that there is excitement surrounding KarXT as a potentially transformative treatment option. “We are excited about this potentially transformative treatment option in clinical settings to benefit these patients as early as possible,” said Dr. Wang.
The NDA submission for KarXT is supported by data from several clinical trials, including a Phase 1 China Pharmacokinetics (PK) study and the Phase 3 China study (ZL-2701-001), along with data from the global EMERGENT clinical programs. Zai Lab announces that the Phase 3 study in China met its primary endpoint, demonstrating a statistically significant 9.2-point difference in the reduction of schizophrenia symptoms compared to the placebo.
At Week 5, patients treated with KarXT showed a 16.9-point improvement in the Positive and Negative Syndrome Scale (PANSS) total score, compared to a 7.7-point improvement in the placebo group. Zai Lab announces that all key secondary efficacy endpoints were met, showing significant improvements in symptoms related to positive and negative schizophrenia, as well as other clinical measures.
The safety profile of KarXT in the Phase 3 study was consistent with previous trials, with no new or unexpected safety concerns. Zai Lab announces that common treatment-emergent adverse events included vomiting, tachycardia, nausea, systemic hypertension, dizziness, and diarrhea. These findings align with results from global Phase 3 trials (EMERGENT-2 and EMERGENT-3), where KarXT also demonstrated statistically significant reductions in schizophrenia symptoms compared to placebo.
In September 2024, the U.S. Food and Drug Administration (FDA) approved COBENFY™ (xanomeline and trospium chloride) for the treatment of schizophrenia in adults. Zai Lab announces that COBENFY is notable for not carrying the typical warnings and precautions associated with atypical antipsychotics, including a boxed warning, making it an appealing alternative for patients.
KarXT, developed in collaboration with Karuna Therapeutics (now part of Bristol Myers Squibb), is a combination of two compounds: xanomeline, a muscarinic acetylcholine receptor agonist, and trospium chloride, a muscarinic acetylcholine receptor antagonist. Zai Lab announces that unlike traditional antipsychotic medications that primarily target dopamine or serotonin receptors, xanomeline works by stimulating muscarinic receptors in the central nervous system, offering a potentially different and more effective approach to treating psychiatric conditions like schizophrenia and Alzheimer’s-related psychosis.
Zai Lab announces that it holds an exclusive license to develop, manufacture, and commercialize KarXT in Greater China, including mainland China, Hong Kong, Macau, and Taiwan.
Schizophrenia affects nearly 24 million people worldwide, and while treatment with antipsychotics is the standard, many patients still struggle with limited efficacy and troublesome side effects. In China, fewer than half of those with schizophrenia receive treatment, and even fewer experience adequate symptom relief with current therapies. Zai Lab announces that the need for more effective treatments with improved safety profiles remains critical.
Zai Lab’s dedication to addressing these gaps in treatment announces its broader mission to develop innovative therapies in areas of high unmet need, including oncology, immunology, neuroscience, and infectious disease. Through its focus on groundbreaking research and development, Zai Lab announces its goal to make a significant positive impact on human health both in China and globally.
