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Immix Biopharma and its subsidiary Nexcella have advanced their CAR-T therapy, NXC-201, for relapsed/refractory AL amyloidosis to an expansion cohort in the U.S. trial NEXICART-2. After successfully dosing patients in the initial cohort, the new cohort will explore higher doses of the therapy, aiming to expand enrollment. Previous trials showed promising results, with high overall response rates in patients. This marks a key milestone for CAR-T in treating AL amyloidosis, which currently lacks FDA-approved treatments https://lnkd.in/eRyghAtA

Enterprise Therapeutics has dosed the first patient in its pivotal Phase IIa clinical trial, which is investigating the efficacy and safety of ETD001 — an epithelial sodium channel (#ENaC) inhibitor designed to enhance hydration and mucus clearance in patients with cystic fibrosis. The study is also focusing on the suitability of such a treatment for the 10% of cystic fibrosis patients who are ineligible for CFTR modulators. #cysticfibrosis #clinicaltrials #CFTRmodulators #ENaCinhibitors https://lnkd.in/env3Jv_N

🚀 Exciting News from Kyverna Therapeutics! 🌟 #FDA #FastTrack #InnovationInHealthcare #MultipleSclerosisTherapy #KyvernaTherapeutics 🔬 The FDA has just granted fast track designation to Kyverna Therapeutics' groundbreaking CAR T-cell therapy, KYV-101, specifically aimed at treating multiple sclerosis (MS). This is a major leap forward in autoimmune disease treatment, showcasing Kyverna's commitment to innovative healthcare solutions. 👩🔬KYV-101, an autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell therapy, is designed to target the CD19 protein on the B cell surface - a key player in various autoimmune diseases. This therapy represents a novel approach, modifying patients' T cells to effectively identify and eliminate B cells. 🧬 Originating from a collaborative effort with the US National Institutes of Health, the CAR in KYV-101 has been engineered for enhanced tolerability and has already shown promise in a 20-subject Phase I oncology trial. 🔎 Following the FDA's fast track designation for KYV-101 in myasthenia gravis last month, this new status for MS treatment accelerates the development and review process, addressing a critical unmet medical need. 🌍 As a clinical-stage biopharmaceutical leader, Kyverna is not stopping here. Plans are underway for further trials of KYV-101 in systemic sclerosis, myasthenia gravis, and multiple sclerosis, reinforcing their dedication to transforming the treatment landscape for autoimmune diseases. 💡 Kyverna CEO Peter Maag remarks, “This marks another important milestone in our endeavor to change the treatment paradigm with KYV-101.” 🔗 Join us in celebrating this significant advancement in healthcare and stay tuned for more updates on Kyverna’s journey in revolutionizing autoimmune disease treatment! #HealthcareInnovation #CARTherapy #AutoimmuneDisease #ClinicalTrials #Biopharmaceuticals #KyvernaUpdate #HealthTech #MedicalAdvancement

Advances in CAR T-Cell Therapy and Bispecifics for Relapse/Refractory Multiple Myeloma: Insights From ESMO Congress 2024: The efficacy and safety profiles of CAR T-cell and bispecific antibody therapies have led to their investigation in earlier lines of therapy, with several new treatments in development. #finance #pharmacy #lifesciences

Phase I/II data for Regeneron Pharmaceuticals’ costimulatory bispecific antibody were disappointing, with only one complete response when used as a combination treatment with Libtayo for solid tumors. One additional patient, who had liver metastasis, reached partial response after the cut-off. Nine patients had stable disease. Almost all patients developed TEAEs, though only 35% of participants saw grade 3 or 4 TEAEs. Toxicities deemed related to the study drug arose in 90% of patients, but only 7% of these side effects were grade 3 or 4. In addition, 5% of patients dropped out of the study due to side effects. There have been no treatment-related deaths so far. #biotech #biopharma #ASCO24 #solidtumor

NEJM: Results From Targeted Therapy for Ulcerative Colitis Study "Phase II Study Shows That Monoclonal Antibody Treatment Developed by Cedars-Sinai Researchers Is Effective for Moderate to Severe Ulcerative Colitis "An international placebo-controlled study led by Cedars-Sinai suggests that a targeted drug therapy that was developed by researchers at Cedars-Sinai is safe and effective at helping people with moderate to severe ulcerative colitis reach clinical remission. "Results from the multicenter Phase II study, ARTEMIS-UC, were published in The New England Journal of Medicine." https://lnkd.in/gSB6GqFP Dermot McGovern MD, PhD, FRCP(Lon), FACG, AGAF Cedars-Sinai #IBD #crohns #ulcerativecolitis #colitis

Longitude portfolio company Vera Therapeutics, Inc. shared exciting news at the recent European Renal Association (ERA) Congress: data presentations from its Phase 2b ORIGIN trial of atacicept in immunoglobulin A nephropathy (IgAN) showing that atacicept stabilized kidney function through 72 weeks and led to rapid improvements in hematuria. “For the first time in this field, we presented 72-week data from our Phase 2b ORIGIN trial showing stable kidney function over the duration of treatment. In addition, we presented data showing that atacicept leads to hematuria resolution in significantly more patients compared with placebo. The impact on hematuria was seen as early as 4 weeks after treatment initiation, which could have important implications for patients with acute kidney inflammation. The evolving atacicept data package supports our belief that atacicept may offer comprehensive disease modification to patients with IgAN,” said Vera Founder and CEO Marshall Fordyce, M.D. Explore more of the data: https://lnkd.in/eRcDMqd5 #biotech #ERA24

Regulatory agencies such as the FDA and EMA may require repeated testing of HLA-genotypes for selecting T cell receptor (TCR)-based therapeutics which is why companies and not-for-profit institutions call for a change in regulation: https://lnkd.in/dGAWUqHh. TCR-based therapeutics refer to treatments that utilize TCRs as targets for developing biotherapeutics and are dependent on the HLA type, which is fixed. TCR-based therapeutics can target peptides derived from Tumor Associated Antigens (TAAs). Confirming the presence of these specific TAAs directly (by immunopeptidomics) or indirectly (using sequencing data), still requires expert platforms. HLA typing by itself, is more straightforward and therefore should not be considered as a companion diagnostic which would result in additional regulatory hurdles, the authors argue. Next to developing TCR-based therapeutics, identification of MHC-I and MHC-II presented peptides can give very useful, patient-specific information on possible immunogenicity issues of used biologicals. ImmuneSpec is an immunopeptidomics service provider that enables both identification of TAAs from tumor samples as well as immunogenicity profiling of TCR-biotherapeutics. Want to know more about ImmuneSpec’s advanced immunopeptidomics platform and its use cases in immunogenicity, (neo)antigen discovery and target validation? Get in touch at info@immunespec.com #CancerResearch #Immunopeptidomics #HLAtyping

1. PharmAust has rebranded as Neurizon Therapeutics, seeing a share price increase of approximately 90% year-to-date. 2. The company is advancing its lead drug, monepantel, now called NUZ-001, targeting neurodegenerative diseases, starting with ALS. 3. A Phase 2/3 clinical trial for NUZ-001 is set to begin in Q1 2025 as part of the HEALEY ALS Platform Trial. 4. NUZ-001 has received orphan drug designation from the FDA, providing benefits such as tax credits and market exclusivity. 5. The drug inhibits the mTOR pathway to help clear misfolded proteins in ALS, potentially slowing disease progression. 6. Early results from trials show NUZ-001 may reduce ALS symptom decline by 39% and improve patient survival rates significantly. 7. The market for ALS therapies exceeds $2 billion annually, with a high demand for effective treatments due to the lack of current cures. 8. Neurizon's strategy focuses on improving patient access, partnering with neurologists, and exploring the drug's potential for other neurodegenerative diseases.

Sirius begins patient dosing in phase 1 trial of next─generation, long─acting factor XI siRNA anticoagulant to treat thromboembolic disorders