NEUROCRINE BIOSCIENCES, INC.
🇸🇪Sweden
- Country
- 🇸🇪Sweden
- Ownership
- Public
- Established
- 1992-01-01
- Employees
- 1.4K
- Market Cap
- $15.4B
- Website
- http://www.neurocrine.com
Biopharma Q1 Updates: New Drug Launches and FDA Extension for Cytokinetics' Aficamten
• Neurocrine Biosciences, Madrigal Pharmaceuticals, and Vanda Therapeutics provided investors with key updates on their recent drug launches during Q1 earnings calls.
• Cytokinetics announced an FDA review extension for aficamten, their investigational cardiac myosin inhibitor, potentially impacting their timeline for market entry.
• The Q1 earnings season highlights significant catalysts across the biopharma sector as companies navigate regulatory pathways and initial commercialization challenges.
Crinecerfont Shows Promise in Pediatric Congenital Adrenal Hyperplasia Across All Patient Subgroups
• Phase 3 CAHtalyst pediatric study demonstrates that crinecerfont (CRENESSITY) effectively reduces glucocorticoid dosing while maintaining or improving androstenedione levels in children with classic congenital adrenal hyperplasia.
• The CRF1 receptor antagonist showed consistent benefits across all patient subgroups regardless of demographic characteristics, baseline androstenedione levels, or baseline glucocorticoid dose.
• By enabling reduced reliance on high-dose glucocorticoids, crinecerfont addresses both the hormonal imbalances of CAH and the complications associated with traditional steroid treatments.
Shionogi to Acquire Japan Tobacco's Pharmaceutical Subsidiaries for $1.1 Billion
• Shionogi & Co. plans to acquire Japan Tobacco's pharmaceutical subsidiaries Torii Pharmaceutical and Akros Pharma for ¥160 billion ($1.1 billion) to strengthen its global R&D capabilities.
• The acquisition, negotiated since early 2024, will proceed through a tender offer beginning June 18 with a share price of ¥6,350 to acquire at least 11.89% of the minority stake.
• This strategic move represents significant consolidation in the Japanese pharmaceutical sector and expands Shionogi's market presence amid increasing global competition.
CRENESSITY Shows Consistent Efficacy Across Pediatric CAH Subgroups While Reducing Glucocorticoid Doses
• New analyses from Neurocrine Biosciences' Phase 3 CAHtalyst Pediatric study demonstrate CRENESSITY (crinecerfont) consistently reduced glucocorticoid doses while maintaining or improving androstenedione levels across all patient subgroups.
• The study showed significant reductions in androstenedione levels at Week 4 (-9.3 nmol/L vs placebo, p=0.0002) and glucocorticoid doses at Week 28 (-23.5% vs placebo, p<0.0001), representing a major advancement in congenital adrenal hyperplasia management.
• CRENESSITY, a CRF1 receptor antagonist approved by the FDA in December 2024, offers a non-glucocorticoid mechanism to address both hormonal imbalances and reduce reliance on high-dose steroids that cause significant long-term complications.
Neurocrine Biosciences Advances Novel Muscarinic M4 Agonist to Phase 3 Trials for Schizophrenia
• Neurocrine Biosciences has initiated a Phase 3 registrational program for NBI-1117568, an oral muscarinic M4 selective orthosteric agonist, following positive Phase 2 results in schizophrenia patients.
• The global double-blind, placebo-controlled trial will enroll approximately 280 adults with schizophrenia experiencing acute symptom exacerbation, with PANSS score reduction as the primary endpoint.
• NBI-1117568 represents a novel mechanism of action that achieved clinically meaningful results in Phase 2, showing an 18.2-point reduction from baseline in PANSS scores with minimal gastrointestinal and cardiovascular side effects.
Sodium Channel Blockers Pipeline Surges with 20+ Pharmaceutical Companies Developing Novel Therapies
• The sodium channel blocker market is expanding rapidly due to increasing prevalence of neuropathic pain and rising demand for selective treatments targeting Nav1.7 and Nav1.8 inhibitors.
• Over 20 pharmaceutical companies including SK Biopharmaceuticals, Vertex Pharmaceuticals, and Praxis Precision Medicines are advancing 25+ pipeline candidates through various clinical stages.
• Recent developments include FDA fast-track designation for Latigo's LTG-001 for acute pain and Hisamitsu Pharmaceutical's milestone achievement with RaQualia's novel transdermal sodium channel blocker.
Neurocrine Biosciences Appoints Dr. Sanjay Keswani as New Chief Medical Officer
• Neurocrine Biosciences has appointed Dr. Sanjay Keswani as Chief Medical Officer effective June 2, 2025, succeeding Dr. Eiry W. Roberts who served in the role for seven years.
• Dr. Keswani brings over 20 years of pharmaceutical industry experience as a physician-scientist with expertise across multiple therapeutic areas and will lead clinical development and medical affairs activities.
• Prior to joining Neurocrine, Dr. Keswani served as President and CEO of ImmunoBrain and held senior leadership positions at Hoffman La Roche and Bristol-Myers Squibb.
First Patients Receive Groundbreaking Autologous Stem Cell Treatment for Parkinson's Disease in Phase 1 Trial
• Mass General Brigham launches pioneering Phase 1 trial using patients' own reprogrammed stem cells to replace damaged dopamine neurons in Parkinson's disease, with three of six participants treated successfully.
• The innovative treatment converts patients' blood cells into induced pluripotent stem cells (iPSCs), which are then transformed into dopamine neurons, eliminating the need for immunosuppression.
• The FDA-approved trial, based on three decades of research at McLean Hospital, will monitor patients for 12 months to assess safety and potential improvements in Parkinson's symptoms.
Phase 4 KINECT-PRO Trial Shows Ingrezza Significantly Improves Quality of Life in Tardive Dyskinesia Patients
• Neurocrine Biosciences' phase 4 KINECT-PRO study demonstrates sustained improvements in physical, social, and emotional impacts of tardive dyskinesia with Ingrezza treatment across all severity levels.
• The 24-week trial showed significant patient-reported benefits starting as early as 4 weeks at the lowest dose (40mg), with 52 out of 59 enrolled patients completing the study.
• Results revealed consistent efficacy regardless of underlying psychiatric conditions, with improvements measured across multiple validated clinical scales including AIMS and TD Impact Scale.
FDA Grants Fast Track Status to Neuren's Novel Pitt Hopkins Syndrome Treatment
• Neuren Pharmaceuticals receives FDA Fast Track designation for their investigational treatment targeting Pitt Hopkins syndrome, a rare neurodevelopmental disorder affecting 1 in 34,000-41,000 people.
• The Fast Track status aims to expedite the development and review process for this potential first-ever approved treatment for Pitt Hopkins syndrome.
• Company shares responded positively to the announcement, rising 5.4% to $13.77, marking a significant development in addressing this serious unmet medical need.
Major Pipeline Expansion in Schizophrenia Treatment: 55+ Companies Developing 60+ Novel Therapies
• DelveInsight's latest pipeline report reveals over 55 pharmaceutical companies actively developing 60+ innovative drug candidates for schizophrenia treatment, indicating significant industry investment in addressing this serious mental illness.
• Several promising candidates are advancing in clinical trials, including Lyndra's weekly oral risperidone (LYN-005), AbbVie's selective M4 receptor PAM emraclidine, and Kynexis's cognitive function-targeting KYN-5356.
• Multiple clinical trials are scheduled for early 2025, including LB-102 for acute schizophrenia, Click Therapeutics' digital therapeutics, and Cerevel's emraclidine safety study, demonstrating diverse therapeutic approaches.
Revive Therapeutics Focuses R&D on Bucillamine for Infectious Diseases and Nerve Agent Exposure
• Revive Therapeutics is prioritizing the development of Bucillamine for medical countermeasures against nerve agent exposure, leveraging its antioxidant properties.
• A research partnership with Defence R&D Canada is evaluating Bucillamine's potential to mitigate nerve agent-induced brain injury by enhancing GABA receptor function.
• Revive is also exploring Bucillamine as a treatment for Long COVID, with plans to submit a Phase 2 study protocol to the FDA by the end of Q1 2025.
• The FDA has provided feedback on study design for Long COVID, recommending a new IND application referencing data from a previous Phase 3 COVID-19 trial.
Vigil Neuroscience's VG-3927 Shows Promise in Phase 1 Alzheimer's Trial, Advancing to Phase 2
• Vigil Neuroscience reports positive Phase 1 data for VG-3927, an oral TREM2 agonist, showing a favorable safety profile in healthy adults and Alzheimer's patients.
• The trial demonstrated robust, dose-dependent reductions in sTREM2 levels in cerebrospinal fluid, indicating strong target engagement and potential neuroprotective effects.
• Vigil plans to advance VG-3927 into a Phase 2 clinical trial in Q3 2025, using a 25mg once-daily oral dose based on Phase 1 findings.
• VG-3927's unique mechanism aims to enhance microglial function without increasing inflammation, offering a differentiated approach to Alzheimer's treatment.
Boehringer Ingelheim Expands Schizophrenia Pipeline with Sosei Heptares and Autifony Collaborations
• Boehringer Ingelheim licenses GPR52 agonists from Sosei Heptares for €755m, targeting a range of schizophrenia symptoms including positive, negative, and cognitive dysfunction.
• The collaboration with Autifony Therapeutics focuses on Kv3.1/3.2 positive modulators, with Boehringer funding R&D and holding an option to purchase AUT00206, currently in Phase 1b.
• These deals complement Boehringer's existing schizophrenia pipeline, including iclepertin (GlyT1 inhibitor) in Phase 3 and a digital therapeutic (DTx) in late-stage testing.
AI-Discovered Drug SOM3355 Shows Promise in Treating Huntington's Disease
SOM Biotech's AI-discovered drug, SOM3355, has shown promising results in a phase 2 trial for Huntington's disease, significantly improving symptoms of chorea. The drug, originally developed for high blood pressure, could offer a new treatment option with fewer side effects compared to existing medications.
FDA Approves Neurocrine's Crenessity (crinecerfont) for Congenital Adrenal Hyperplasia
• The FDA has approved Crenessity (crinecerfont) as an adjunctive treatment for congenital adrenal hyperplasia (CAH) in adults and children, marking a significant advancement.
• Crenessity is the first and only classic CAH treatment that directly reduces excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production.
• Clinical trials demonstrated that Crenessity enables lower glucocorticoid doses while maintaining or improving androgen control in CAH patients.
• Neurocrine Biosciences expects Crenessity to be commercially available soon, offering a new treatment option for individuals with classic CAH.
Nxera Pharma Initiates Phase 3 Trial of Daridorexant for Insomnia in South Korea
• Nxera Pharma has enrolled the first patient in a Phase 3 clinical trial in South Korea to evaluate daridorexant for treating insomnia.
• The trial aims to secure marketing authorization from South Korea's Ministry of Food and Drug Safety, with results expected in 1H 2026.
• Daridorexant, approved in Japan as QUVIVIQ™, is already available in the US, Europe and Japan, targeting overactive wake signaling in insomnia.
• Insomnia affects 15-25% of South Korean adults, highlighting the need for effective treatments like daridorexant to improve sleep quality.
Xenon Pharmaceuticals Advances Azetukalner Program for Epilepsy and MDD
• Xenon Pharmaceuticals is progressing its Phase 3 epilepsy program with azetukalner, anticipating topline data from the X-TOLE2 study in the second half of 2025.
• The company plans to initiate the X-NOVA2 Phase 3 trial for azetukalner in major depressive disorder (MDD) by the end of the year, expanding its therapeutic focus.
• Xenon is expanding its ion channel portfolio, with multiple candidates advancing towards IND filings in 2025, targeting Kv7, Nav1.7, and Nav1.1.
• Long-term data from the X-TOLE open-label extension study of azetukalner in focal onset seizures will be presented at the American Epilepsy Society (AES) annual meeting.
FDA Approves Cobenfy, a Novel Schizophrenia Treatment Targeting Cholinergic Receptors
• The FDA has approved Cobenfy (xanomeline and trospium chloride) as the first new class of drug for schizophrenia in over 30 years, offering a novel approach to treatment.
• Cobenfy targets muscarinic receptors, unlike traditional antipsychotics that focus on dopamine, potentially reducing side effects like weight gain and movement disorders.
• Clinical trials demonstrated Cobenfy significantly reduced schizophrenia symptoms compared to placebo, marking a transformative moment in managing this challenging condition.
• Expected to launch in late October, Cobenfy offers a new option for adults with schizophrenia, with ongoing studies exploring its potential in Alzheimer's psychosis and other conditions.
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