NEUROCRINE BIOSCIENCES, INC.

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.

Negotiated prices for 10 Medicare drugs fell from initial net prices, with reductions ranging from 8% to 42%, but all except insulin aspart remained more expensive in the U.S. than in peer countries. The study highlights the need for continued assessment of future negotiation rounds, especially with potential inclusion of Part B drugs, to maximize savings.

DelveInsight's report on the Gene and Cell Therapies Targeting CNS Disorders market forecasts growth to 2034, highlighting key companies and therapies, including Bayer AG's Phase Ib clinical trial success for Parkinson's disease and uniQure's positive trends in Huntington's disease trials. The market, valued at approximately USD 1 billion in 2021, is expected to surge due to increasing disease prevalence and pipeline product launches.

Bristol Myers Squibb's Cobenfy, approved by the FDA in September, marked a significant breakthrough in schizophrenia treatment, while AbbVie's muscarinic candidate emraclidine failed mid-stage trials, positioning BMS as the clear winner. Cobenfy's efficacy in three registrational trials contrasts with emraclidine's failure to show significant symptom improvement in Phase II trials, leading to a 12% drop in AbbVie's stock. BMS's acquisition of Karuna Therapeutics for $14 billion in December 2023 further solidified its lead in the schizophrenia market.

Day 2 of the 2024 Huntington Study Group Conference featured talks on palliative care, clinical trial challenges, and updates from various drug development companies, emphasizing the importance of early intervention and the use of biomarkers in HD treatment.

AbbVie's failed Phase 2 trial for schizophrenia drug emraclidine benefits Bristol Myers Squibb's Cobenfy, which was approved by the FDA in September.

AbbVie's $9 billion acquisition of Cerevel Therapeutics, centered on experimental schizophrenia drug emraclidine, failed in two clinical trials, causing a 12% drop in AbbVie's stock and a 12% rise in Bristol Myers Squibb's shares. Analysts suggest this failure may impact other companies researching muscarinic antipsychotics, with Neurocrine Biosciences and Neumora Therapeutics seeing stock fluctuations. Despite the setback, some analysts remain optimistic about the muscarinic class for schizophrenia treatment.

AbbVie's stock dropped 13% after emraclidine, a schizophrenia drug from its $8.7 billion Cerevel Therapeutics acquisition, failed in phase 2 trials. The failure benefits Bristol-Myers Squibb, whose Cobenfy received FDA approval in September, and Neurocrine Biosciences, which had a successful phase 2 trial for its M4 agonist.

Dr. Husseini Manji, former J&J neuroscience head, developed Spravato, a first-in-class depression medication approved by the FDA in 2019. Despite initial skepticism, Spravato is now poised for blockbuster status. Psychiatric drug development, once abandoned by Big Pharma, is experiencing a renaissance with over 160 mental health medications in clinical trials. Manji advocates for precise diagnoses and targeted treatments. Recent setbacks include Alto Neuroscience's ALTO-100 failing to beat a placebo in a mid-stage trial for major depressive disorder. Emerging precision approaches include targeting orexin receptors, AMPA modulation, kappa opioid receptors, and inflammation reduction.

Beam Therapeutics reports a patient death in BEAM-101 trial for sickle cell disease, unrelated to the therapy. Sana Biotechnology refocuses on autoimmune assets, reducing workforce. Ring Therapeutics expands in Singapore with new deals. Drug regulators criticized for ignoring forced labor in supply chains. OSE Therapeutics' IL-7 therapy shows positive results in ulcerative colitis trial. Japan's PMDA opens second overseas office in Washington.