Core Viewpoint - Nxera Pharma's partner Neurocrine Biosciences has initiated a Phase 3 clinical trial for NBI-1117568, an investigational treatment for schizophrenia, following positive Phase 2 results [1][6]. Company Overview - Nxera Pharma is a biopharma company focused on developing specialty medicines for unmet medical needs, leveraging its NxWave™ discovery platform [11][12]. - The company has a pipeline of over 30 active programs targeting neurology, neuropsychiatry, metabolic diseases, and immunology [12]. Clinical Development - NBI-1117568 is the first oral muscarinic M4 selective orthosteric agonist in clinical development for schizophrenia, with a unique mechanism aimed at improving safety and efficacy [5][8]. - The Phase 3 study will enroll approximately 280 patients and will evaluate the drug's efficacy using the Positive and Negative Syndrome Scale (PANSS) and Clinical Global Impression of Severity (CGI-S) scale [2][6]. Financial Aspects - No milestone payment is due from Neurocrine to Nxera at the start of the Phase 3 trial, but a US$15 million milestone payment is expected upon dosing the first patient [3]. Market Context - Schizophrenia affects approximately 24 million people globally, with annual costs exceeding $150 billion in the U.S., highlighting the significant need for effective treatments [10].

Core Insights - Neurocrine Biosciences has initiated a Phase 3 registrational program for NBI-1117568, an investigational oral muscarinic M4 selective orthosteric agonist aimed at treating schizophrenia, following positive Phase 2 data reported in August 2024 [1][2][3] Company Overview - Neurocrine Biosciences is a neuroscience-focused biopharmaceutical company dedicated to developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a diverse portfolio including FDA-approved treatments and a robust pipeline [8] Clinical Development - The Phase 3 study is a global double-blind, placebo-controlled trial targeting approximately 280 patients with schizophrenia experiencing acute exacerbation or relapse of symptoms, with primary and secondary endpoints focused on PANSS and CGI-S scale improvements [2][3] - NBI-1117568 is the first oral muscarinic M4 selective orthosteric agonist in clinical development for schizophrenia, potentially offering a novel mechanism with an improved safety profile [3][4] Market Need - Schizophrenia affects approximately 24 million people globally, with annual costs exceeding $150 billion in the U.S., highlighting the significant need for innovative treatment options [6][7] Clinical Results - The Phase 2 study demonstrated a statistically significant reduction in PANSS total score with a placebo-adjusted mean reduction of 7.5 points and an 18.2-point reduction from baseline, indicating the efficacy of NBI-1117568 [6]

Group 1 - The article discusses Neurocrine Biosciences (NASDAQ: NBIX) and highlights a previous analysis regarding a buying opportunity following a stock price drop related to schizophrenia data [2] - The author operates the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and includes a library of over 600 biotech investing articles [2] - The service offers a model portfolio of more than 10 small and mid-cap stocks, along with live chat and various analysis and news reports to assist healthcare investors [2] Group 2 - The article does not contain any specific financial data or performance metrics related to Neurocrine Biosciences or the broader biotech industry [1][3][4]

Company Overview - Neurocrine Biosciences, Inc. (NASDAQ: NBIX) is a San Diego-based drug developer that completed its initial public offering (IPO) in 1996, raising $36.75 million [2]. Investment Insights - The investment group Haggerston BioHealth provides insights for both novice and experienced biotech investors, including catalysts to watch for, buy and sell ratings, product sales forecasts, and integrated financial statements [2]. Analyst Background - The article is authored by a biotech consultant with over 5 years of experience covering biotech, healthcare, and pharma, having prepared detailed reports on over 1,000 companies [2].

Core Insights - Neurocrine Biosciences presented new data indicating that all patients with tardive dyskinesia achieved a therapeutic dose with INGREZZA (valbenazine) from the start of treatment, contrasting with only about half of patients on deutetrabenazine reaching a therapeutic dose within six months [1][5][6] Group 1: Study Findings - The retrospective cohort study included 3,527 patients treated with INGREZZA, 2,166 with deutetrabenazine BID, and 326 with deutetrabenazine XR, assessing them over a six-month period [2][3] - Results showed that 100% of patients on INGREZZA reached a therapeutic dose immediately, while only 47.5% of those on deutetrabenazine BID and 54.3% on XR achieved this within six months [6] - The average time to reach a therapeutic dose for deutetrabenazine patients was three to four weeks, with nearly 10% unable to maintain the therapeutic dose after reaching it [6] Group 2: Treatment Implications - INGREZZA's ability to provide a therapeutic dose from day one without titration may reduce the burden on clinicians and patients associated with sub-therapeutic dosing [2][5] - Fewer patients on INGREZZA experienced dose changes compared to those on deutetrabenazine, with 33.7% of INGREZZA patients needing a dose change after the first month, compared to 48.1% for BID and 54.0% for XR [6] Group 3: About Tardive Dyskinesia - Tardive dyskinesia is a movement disorder characterized by uncontrollable and repetitive movements, often resulting from the use of certain antipsychotic medications [4][7] - It is estimated to affect at least 800,000 adults in the U.S., highlighting the significant need for effective treatments [7] Group 4: About INGREZZA - INGREZZA is a selective VMAT2 inhibitor approved for treating tardive dyskinesia and chorea associated with Huntington's disease, uniquely offering a therapeutic dose from day one [8][9] - The medication is available in 40 mg, 60 mg, and 80 mg capsules, and can be taken alongside most stable mental health regimens [10][19]

Core Insights - The Motley Fool aims to make the world smarter, happier, and richer through its financial services [1] Company Overview - Founded in 1993, The Motley Fool is a financial services company [1] - The company reaches millions of people monthly through various channels including premium investing solutions, free guidance, market analysis on Fool.com, top-rated podcasts, and its non-profit foundation [1]

Core Insights - Neurocrine Biosciences presented new data from the KINECT® 4 study showing that the majority of patients treated with INGREZZA® (valbenazine) capsules achieved remission of tardive dyskinesia (TD) [1][7] Study Findings - The KINECT 4 study involved 163 participants with moderate to severe TD and underlying psychiatric conditions, who received 48 weeks of treatment with INGREZZA [4] - A post-hoc analysis indicated that 59.2% of participants achieved remission, with 58.6% on the 40 mg dose and 59.5% on the 80 mg dose [8] - Significant improvements in the Abnormal Involuntary Movement Scale (AIMS) total scores were observed, with mean baseline scores decreasing from 12.4 (40 mg) and 15.1 (80 mg) to 2.1 and 2.5, respectively, at Week 48 [8] Treatment Efficacy - The findings suggest that INGREZZA is an effective long-term treatment option for TD, regardless of the underlying psychiatric condition [2][3] - Improvements in TD symptoms were consistent across different psychiatric diagnoses, with remission rates of 57.7% for schizophrenia or schizoaffective disorder and 62.5% for mood disorders [8] Safety and Tolerability - INGREZZA was generally well tolerated, with treatment emergent adverse events occurring in ≥ 5% of participants being urinary tract infection (8.5%) and headache (5.2%) [5] - Changes in psychiatric stability and vital signs were generally small and not clinically significant [5] About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating adults with tardive dyskinesia and chorea associated with Huntington's disease [10][11] - The drug is unique in that it offers a therapeutic dose from day one without the need for titration [10]

Core Insights - Neurocrine Biosciences presented data from the KINECT®-HD study demonstrating significant improvements in chorea symptoms in adults with Huntington's disease using once-daily INGREZZA® (valbenazine) capsules [1][6][8] Study Findings - The KINECT-HD study was a Phase 3, randomized, double-blind, placebo-controlled trial involving 128 adults aged 18 to 75 diagnosed with motor-manifest Huntington's disease [4][5] - The primary efficacy endpoint was the Unified Huntington's Disease Rating Scale (UHDRS®) Total Maximal Chorea (TMC) score, which showed a placebo-adjusted mean reduction of 3.2 units (P<0.0001) after 12 weeks of treatment [5][7] - Statistically significant improvements in chorea severity were observed in both upper and lower extremities, with P<0.05 for each region [7] Patient Impact - The analysis indicated that more participants experienced meaningful improvements in chorea, with TMC score shifts from ≥2 at baseline to ≤1 at maintenance with INGREZZA compared to placebo [7] - The most significant improvements were noted in the arms and legs, which were the most severely affected at baseline [2][3] Safety Profile - Treatment-emergent adverse events were consistent with the known safety profile of valbenazine, with common adverse reactions including somnolence, sedation, urticaria, rash, and insomnia [8][20] - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating chorea associated with Huntington's disease and tardive dyskinesia [9][10] Company Overview - Neurocrine Biosciences is focused on developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders, with a portfolio that includes FDA-approved therapies for various conditions [22][24]

Core Insights - Neurocrine Biosciences has initiated a Phase 1 clinical study for NBI-1140675, an investigational oral selective second-generation small molecule inhibitor of VMAT2, aimed at treating neurological and neuropsychiatric conditions [1][2] - The company aims to expand its portfolio of VMAT2 inhibitors, building on the success of valbenazine, which is approved for tardive dyskinesia and chorea associated with Huntington's disease [2] Company Overview - Neurocrine Biosciences is a leading biopharmaceutical company focused on neuroscience, dedicated to developing treatments for neurological, neuroendocrine, and neuropsychiatric disorders [3] - The company has a diverse portfolio that includes FDA-approved treatments for various conditions and a robust pipeline with multiple compounds in mid- to late-phase clinical development [3]

Core Insights - Neurocrine Biosciences announced positive top-line data from the Phase 4 KINECT-PRO study, demonstrating the effectiveness of INGREZZA (valbenazine) in improving the physical, social, and emotional impacts of tardive dyskinesia (TD) in patients, regardless of the severity of TD or underlying psychiatric conditions [1][2][4] Group 1: Study Overview - The KINECT-PRO study is the first to utilize multiple clinically validated scales to assess the patient-reported impact of INGREZZA on TD [1][3] - The study enrolled 59 patients who received once-daily doses of INGREZZA (40 mg, 60 mg, or 80 mg) for up to 24 weeks, with 52 patients completing the study [2][5] - The primary objective was to evaluate changes in patient-reported physical and socio-emotional impacts of TD, while the secondary objective focused on clinician- and patient-reported changes in TD severity [3][4] Group 2: Results and Findings - Significant and sustained improvements were observed in all three patient-reported outcome measures, with improvements noted as early as four weeks at the lowest dose of 40 mg [4] - AIMS scores indicated sustained reductions in involuntary movements across different TD severity levels and underlying psychiatric conditions [4] - The safety and tolerability profile of INGREZZA remained consistent with previous studies, with no new safety concerns identified [4] Group 3: About INGREZZA - INGREZZA is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor approved for treating adults with tardive dyskinesia and chorea associated with Huntington's disease [8][9] - The drug is designed to be taken once daily without the need for titration, offering a therapeutic dose from day one [8][10] - INGREZZA is available in 40 mg, 60 mg, and 80 mg capsules, and a sprinkle formulation is also available for patients who have difficulty swallowing [10][19]