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Clinical Trial News

Neurizon's NUZ-001 Demonstrates Neuroprotective Effects in Huntington's Disease Zebrafish Model

  • NUZ-001 and its active metabolite NUZ-001 Sulfone showed significant neuroprotective effects in a zebrafish model of Huntington's disease, preventing developmental abnormalities and neuronal cell death.
  • Treatment with both compounds rescued key biomarkers including BDNF expression and haemoglobin production following huntingtin protein knockdown in the disease model.
  • The findings strengthen NUZ-001's potential as a platform therapy for multiple neurodegenerative diseases beyond its current ALS development program.
  • Neurizon plans to advance additional preclinical studies in mammalian models of Huntington's disease to further validate the therapeutic potential.

uBriGene Launches GMP-Compliant iPSC Banks to Accelerate Cell Therapy Development

  • uBriGene Biosciences has launched fully characterized, ready-to-use induced pluripotent stem cell (iPSC) banks developed using proprietary RNA-LNP reprogramming technology to support clinical applications worldwide.
  • The iPSC banks are generated in GMP-compliant cleanrooms using healthy donor fibroblasts from the USA and undergo rigorous quality control testing to confirm genomic stability and pluripotency markers.
  • According to Dr. Xiulian Sun, CTO and Founder at uBriGene, these banks can save clients 1-2 years and reduce uncertainty associated with donor cell sourcing and reprogramming processes.
  • The banks are available in both research-use only and GMP-compliant formats, with additional gene editing services offered for disease modeling and therapeutic development customization.

Quiver Bioscience Names Co-Founder Graham Dempsey CEO to Advance Nav1.7-Targeting ASO for Chronic Pain

Leadership Change
  • Quiver Bioscience appointed co-founder Graham Dempsey, PhD as CEO to lead the company's transition from discovery platform to clinical-stage therapeutics development.
  • The company's lead antisense oligonucleotide targeting Nav1.7 for chronic neuropathic pain is approaching development candidate selection in 2025 with IND-enabling studies to follow.
  • Quiver is scaling its AI-driven CNS drug discovery platform while advancing a pipeline including a UBE3A-targeting ASO for chromosome 15q duplication syndrome.
  • The Nav1.7 ASO demonstrates efficacy in preclinical models with favorable CNS tolerability, potentially overcoming limitations of existing Nav-targeted small molecules.

Elite Pharmaceuticals Achieves Bioequivalence for Generic Anticoagulant in $27 Billion Market

FDA Approval
  • Elite Pharmaceuticals reported positive bioequivalence results for an undisclosed generic anticoagulant targeting a branded product with $27 billion in annual sales.
  • The crossover bioequivalence study in healthy subjects demonstrated that Elite's generic formulation is bioequivalent to the branded reference product.
  • The company is preparing to file an Abbreviated New Drug Application with the FDA, though commercialization depends on patent resolution and regulatory approval.
  • No generic competitor currently exists in this anticoagulant market, presenting a significant commercial opportunity for Elite upon successful market entry.

D&D Pharmatech's DD01 Shows Rapid Liver Fat Reduction in MASH Phase 2 Trial, Matching Competitor Results in Quarter of the Time

  • D&D Pharmatech's DD01 achieved 75.8% of patients with at least 30% liver fat reduction in just 12 weeks, matching Boehringer Ingelheim's servodutide results that required 48 weeks of treatment.
  • The dual GLP-1/glucagon receptor agonist demonstrated superior tolerability with only 9.09% treatment discontinuation due to gastrointestinal side effects compared to 20% for competing drugs.
  • DD01 showed significant improvements in non-invasive MASH markers including liver stiffness and proC3 levels, with 48.5% of patients achieving normal liver fat levels within 12 weeks.
  • The drug's unique pharmacokinetic profile allows for rapid therapeutic dosing without lengthy titration periods, potentially offering advantages in clinical management of MASH patients.

A Trial Assessing Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ZP7570

NCT06000891TerminatedPhase 1
Zealand Pharma
Posted 9/15/2023

A Study to Evaluate DD01 in Overweight/Obese Subjects With MASLD/MASH

NCT06410924Active, Not RecruitingPhase 2
Neuraly, Inc.
Posted 6/13/2024

Circle Pharma's CID-078 Receives FDA Orphan Drug Designation for Small Cell Lung Cancer Treatment

  • Circle Pharma announced that the FDA has granted Orphan Drug Designation to CID-078 for treating small cell lung cancer, a highly aggressive cancer affecting 13-15% of lung cancer cases.
  • The designation provides seven years of market exclusivity upon approval, tax credits for clinical trials, and eligibility for FDA research grants to support development.
  • CID-078 is an orally bioavailable macrocycle that targets cyclin A and B proteins, with a Phase 1 clinical trial currently enrolling patients with advanced solid tumors.
  • Small cell lung cancer has a poor prognosis with high recurrence rates despite existing treatments, highlighting the urgent need for new therapeutic options.

Safety/Efficacy Study of CID-078 in Patients With Advanced Solid Tumor Malignancies

NCT06577987RecruitingPhase 1
Circle Pharma
Posted 8/14/2024

NextCure and Simcere Zaiming Form Strategic Partnership for CDH6-Targeting ADC SIM0505

Oncology
  • NextCure gains global rights to SIM0505, a novel antibody-drug conjugate targeting CDH6 for solid tumors, while Simcere Zaiming retains Greater China rights.
  • The partnership includes potential milestone payments up to $745 million plus tiered royalties, with U.S. Phase 1 trials expected to begin in Q3 2025.
  • SIM0505 features a proprietary topoisomerase 1 inhibitor payload designed for broad anti-tumor activity with high systemic clearance to improve the therapeutic window.
  • Initial Phase 1 clinical data is anticipated in the first half of 2026, following ongoing dose escalation studies in China.

Incyte's INCA033989 Shows 86% Response Rate in CALR-Mutant Blood Cancer Trial

Monoclonal AntibodyPrecision Medicine
  • Incyte's experimental monoclonal antibody INCA033989 demonstrated an 86% complete or partial hematologic response rate in patients with CALR-mutant myeloproliferative neoplasms receiving doses of 400 mg or higher.
  • The company announced a precision medicine collaboration with Qiagen to develop next-generation sequencing diagnostics for detecting CALR mutations, the second most common driver of MPNs.
  • Clinical data from dose-escalation studies showed 89% of evaluable patients had reduced mutant CALR variant allele frequency, with 21% achieving partial molecular response within three treatment cycles.
  • Stifel upgraded Incyte to 'Buy' rating with a $107 price target following the encouraging clinical results presented at the European Hematology Association congress.

Pierre Fabre Acquires Global Rights to Next-Generation EGFR Inhibitors for NSCLC Treatment

Targeted TherapyPrecision MedicineOncology
  • Pierre Fabre Laboratories acquired worldwide rights to PFL-721 and PFL-241 from Antares Therapeutics, expanding their oncology pipeline with mutant-specific EGFR inhibitors targeting unmet needs in non-small cell lung cancer.
  • PFL-721 is a dual EGFR exon 20 and HER2 exon 20 inhibitor transitioning to dose optimization in first-in-human trials, while PFL-241 is a brain-penetrant fourth-generation EGFR inhibitor addressing C797S resistance mutations.
  • EGFR mutations drive approximately 14-38% of NSCLC tumors globally, representing a significant patient population with substantial therapeutic needs.
  • The acquisition consolidates Pierre Fabre's complete ownership of their R&D portfolio including exarafenib and PFL-002, positioning the company to advance precision medicine development for cancer patients.

Revolutionary Blood Test Enables Celiac Disease Diagnosis Without Gluten Challenge

  • A new blood test can accurately diagnose celiac disease even in patients already following a gluten-free diet, eliminating the need for painful gluten challenges that worsen symptoms.
  • The test measures interleukin-2 (IL-2) immune response to gluten in blood samples, achieving 90% sensitivity and 97% specificity in detecting celiac disease.
  • Researchers tested 181 volunteers and found the IL-2 signal strength correlates with symptom severity, potentially predicting how severely patients might react to gluten exposure.
  • The breakthrough could help diagnose millions of undiagnosed celiac patients worldwide who avoid current testing due to the debilitating diagnostic process.

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