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Clinical Trial News

Leucid Bio's Lateral NKG2D CAR-T Therapy LEU011 Receives MHRA Approval for Solid Tumor Clinical Trial

  • Leucid Bio has received MHRA clinical trial authorization for LEU011, a novel lateral CAR-T therapy targeting NKG2D ligands expressed in over 80% of human tumors.
  • The Phase 1/2 AERIAL trial will evaluate LEU011 in patients with relapsed or refractory solid tumors, with the first patient expected to be dosed in Q4 2023.
  • LEU011 previously received the Innovation Passport designation from MHRA, highlighting its potential as a breakthrough therapy for solid tumors with limited treatment options.

Travere Therapeutics' Filspari Nears, But Misses, Primary Endpoint in IgA Nephropathy Trial

  • Travere Therapeutics' Filspari narrowly missed statistical significance in a Phase 3 trial for IgA nephropathy, a rare kidney disorder, with a p-value of 0.058.
  • The trial involved 404 patients randomized to receive Filspari or irbesartan, an existing blood pressure medication used to manage the disease.
  • While early data showed a 50% reduction in urine protein levels, the 24-month data on kidney function decline narrowly missed the primary endpoint.
  • Filspari received accelerated approval in February based on earlier biomarker data, with full approval contingent on these confirmatory trial results.

FDA Grants Orphan Drug Designation to Immix Biopharma's NXC-201 for AL Amyloidosis

  • Immix Biopharma's NXC-201, a CAR-T cell therapy, receives Orphan Drug Designation from the FDA for treating Amyloid Light Chain (AL) Amyloidosis.
  • The designation provides Immix Biopharma with benefits including potential market exclusivity, tax credits, and a waiver for prescription drug user fees.
  • NXC-201 is currently in Phase 1b/2a clinical trials and has demonstrated a 100% hematologic response rate in AL amyloidosis patients in early trials.
  • AL Amyloidosis, a rare disorder affecting 30,000-40,000 individuals in the US and Europe, has a market projected to reach $6 billion by 2025.

Study of IMX-110 in Combination With Tislelizumab in Patients With Advanced Solid Tumors

NCT05840835RecruitingPhase 1
Immix Biopharma, Inc.
Posted 2/3/2023

NXC-201 (formerly HBI0101) Multiple Myeloma

NCT04720313Active, Not RecruitingPhase 1
Hadassah Medical Organization
Posted 1/1/2021

First RoActemra Biosimilar Tyenne Receives EU Approval, Marking Milestone for Fresenius Kabi

  • The European Commission has granted approval for Tyenne, the first biosimilar version of Roche's blockbuster drug RoActemra (tocilizumab), developed by Fresenius Kabi for multiple inflammatory conditions and severe COVID-19.
  • Tyenne is approved for all RoActemra indications, including rheumatoid arthritis, systemic juvenile idiopathic arthritis, and cytokine release syndrome, representing a significant advancement in accessible treatment options.
  • This approval marks Fresenius Kabi's third biosimilar success and aligns with their Vision 2026 strategy to generate €1 billion in additional revenue every three years.

Neuralink Begins Human Trials of Brain Implant for Paralysis and ALS

  • Neuralink has initiated recruitment for its first-in-human clinical trial, following FDA approval, targeting individuals with quadriplegia due to cervical spinal cord injury or amyotrophic lateral sclerosis (ALS).
  • The study aims to evaluate the safety of Neuralink's wireless brain chip and the surgical robot used for implantation, with the potential to enable users to control computers with their thoughts.
  • Neuralink envisions that successful implementation of their brain chip technology will provide enhanced abilities, such as improved reasoning and vision, and allow streaming music directly to the brain.
  • The company has faced criticism regarding its animal testing practices, which it denies, stating that experiments were conducted on animals near the end of their lives.

Extended-Course Nirmatrelvir/Ritonavir Shows Promise in Treating Long COVID Symptoms

A series of case studies highlights the potential benefits of extended-course nirmatrelvir/ritonavir treatment for individuals suffering from Long COVID, with some patients reporting significant improvements in symptoms. The treatment, typically used for acute COVID-19 infections, was administered outside of the acute phase, offering new insights into managing Long COVID.

RECOVER-VITAL: Platform Protocol to Measure the Effects of Antiviral Therapies on Long COVID Symptoms

NCT05595369CompletedPhase 2
Kanecia Obie Zimmerman
Posted 7/26/2023

EMA Panel Recommends Against Renewing Translarna Approval for Duchenne Muscular Dystrophy

  • The European Medicines Agency's CHMP has issued a negative opinion on renewing the conditional approval of Translarna for Duchenne muscular dystrophy (DMD).
  • PTC Therapeutics plans to request a re-examination of the decision, citing Translarna's established safety and efficacy profile in treating nonsense mutation DMD.
  • Translarna remains available in the EU while PTC seeks review, with a final decision expected after re-examination in January 2024.
  • The CHMP's decision was based on Phase 3 trial data that did not show significant improvement in the primary analysis population.

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

NCT03179631CompletedPhase 3
PTC Therapeutics
Posted 7/6/2017

Cloud-Based Data Analytics Emerges as Key Driver for Life Sciences Innovation and Drug Development

  • Life sciences organizations are facing unprecedented challenges in managing vast amounts of clinical, genomic, and image data, necessitating advanced analytics solutions for efficient drug development.
  • Cloud migration enables centralized data management and machine learning capabilities, significantly accelerating drug discovery processes and improving supply chain optimization.
  • Implementation of modern cloud-based platforms, supported by experienced partners, is revolutionizing critical business operations and enhancing decision-making across the pharmaceutical development lifecycle.

Alzheimer's Disease Treatment Advances: Lecanemab and Donanemab Offer New Hope

• Lecanemab (Leqembi) received full FDA approval in July 2023 and has shown success in slowing Alzheimer's progression, marking a significant milestone in treatment. • Eli Lilly's donanemab demonstrated a 35% reduction in cognitive decline by targeting amyloid protein, offering another promising treatment option for Alzheimer's. • These new drugs, while not cures, represent a crucial step forward, potentially leading to earlier diagnoses, increased awareness, and further research into Alzheimer's. • Challenges remain in ensuring broad access, managing costs, and establishing necessary infrastructure for diagnosis and treatment, particularly in low- and middle-income countries.

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