MedPath

Clinical Trial News

Patritumab Deruxtecan BLA Receives CRL from FDA Due to Manufacturing Inspection

  • The FDA issued a Complete Response Letter (CRL) for patritumab deruxtecan, intended for EGFR-mutated non-small cell lung cancer, due to findings at a third-party manufacturing facility.
  • The CRL did not raise concerns regarding the efficacy or safety data submitted in the Biologics License Application (BLA) for the drug.
  • Patritumab deruxtecan is a HER3-directed antibody-drug conjugate being developed by Daiichi Sankyo and Merck for previously treated NSCLC.
  • Daiichi Sankyo and Merck plan to address the FDA's feedback to make patritumab deruxtecan available to patients with limited treatment options.

Verona Pharma's Ohtuvayre (ensifentrine) Receives FDA Approval for COPD Maintenance Treatment

• The FDA has approved Verona Pharma's Ohtuvayre (ensifentrine) for maintenance treatment of chronic obstructive pulmonary disease (COPD) in adults, marking a novel advancement. • Ohtuvayre is the first inhaled COPD treatment in over two decades with a new mechanism, acting as a dual inhibitor of PDE3 and PDE4 enzymes for bronchodilation and anti-inflammatory effects. • Clinical trials (ENHANCE-1 and ENHANCE-2) demonstrated significant improvements in lung function and reduced exacerbations, supporting the drug's efficacy and safety profile. • Verona Pharma plans to launch Ohtuvayre in Q3 2024, backed by substantial funding, aiming to address the unmet needs of millions suffering from daily COPD symptoms.

Star Therapeutics' VGA039 Shows Promise as Subcutaneous VWD Therapy

  • Star Therapeutics presented Phase 1 data for VGA039, a potential subcutaneous treatment for von Willebrand disease (VWD), at the ISTH Congress.
  • The VIVID 1 study in healthy volunteers demonstrated favorable safety, tolerability, and nearly 100% subcutaneous bioavailability of VGA039.
  • VGA039 exhibited dose-dependent increases in thrombin generation, supporting its potential as a universal hemostatic agent for bleeding disorders.
  • A multinational Phase 1 study (VIVID 2) is underway in VWD patients to further evaluate VGA039's efficacy and safety.

Study of VGA039 in Healthy Volunteers and Patients With Von Willebrand Disease (VIVID)

NCT05776069RecruitingPhase 1
Vega Therapeutics, Inc
Posted 3/16/2023

Industry Survey Reveals Healthcare Mistrust as Major Barrier to Clinical Trial Diversity

  • A third of pharmaceutical executives identify systemic healthcare mistrust as a significant obstacle to achieving diverse clinical trial enrollment, according to a new Trinity Life Sciences survey.
  • Despite minorities comprising over 40% of the US population, only 5-10% of clinical trial participants represent minority groups, highlighting a critical representation gap in medical research.
  • GSK emerges as an industry leader with 100% of its phase 3 trials incorporating diversity plans by end of 2022, while many companies await clearer FDA guidance on diversity requirements.

AbbVie's ABBV-951 (Foscarbidopa/Foslevodopa) Receives Complete Response Letter from FDA

  • AbbVie received a Complete Response Letter (CRL) from the FDA for its New Drug Application for ABBV-951 (foscarbidopa/foslevodopa).
  • The FDA's response cited observations from a third-party manufacturer inspection, unrelated to ABBV-951 or any AbbVie medicine.
  • The CRL does not raise concerns regarding the safety, efficacy, or labeling of ABBV-951, including the delivery device.
  • AbbVie is committed to addressing the FDA's concerns and bringing this therapy to advanced Parkinson's disease patients.

AskBio Initiates Phase 2 Trial of AB-1005 Gene Therapy for Parkinson's Disease

  • AskBio's REGENERATE-PD, a Phase 2 clinical trial, has begun recruitment to evaluate AB-1005 for moderate-stage Parkinson's disease.
  • AB-1005 is an investigational adeno-associated virus 2 (AAV2) glial cell line-derived neurotrophic factor (GDNF) gene therapy.
  • The Phase 2 trial follows encouraging Phase 1b results, which demonstrated that AB-1005 was well-tolerated with no related serious adverse events.
  • The study will enroll approximately 87 subjects across sites in the United States, UK, and Europe to assess the efficacy and safety of AB-1005.

A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

NCT06285643RecruitingPhase 2
AskBio Inc
Posted 6/11/2024

GDNF Gene Therapy for Multiple System Atrophy

NCT04680065Active, Not RecruitingPhase 1
Brain Neurotherapy Bio, Inc.
Posted 10/3/2023

Generative AI Poised to Transform Clinical Trial Efficiency with 40% Faster Regulatory Submissions

  • Generative AI technology shows promise in accelerating clinical trials by streamlining patient cohort selection, improving recruitment, and enhancing trial design processes with up to 30% increased efficiency in initial drug target assessment.
  • The technology can reduce regulatory submission timelines by 40% through automated documentation generation, while potentially decreasing patient drop-off rates by 5-10% through personalized engagement strategies.
  • Despite concerns about data accuracy and privacy compliance, healthcare organizations are implementing GenAI solutions with proper safeguards, though ROI considerations require careful evaluation of operational costs and development timelines.

FDA Grants Priority Review to Ionis' Olezarsen for Familial Chylomicronemia Syndrome

  • The FDA has accepted Ionis Pharmaceuticals' NDA for olezarsen with Priority Review for treating adults with familial chylomicronemia syndrome (FCS).
  • The FDA has set a target action date of December 19, 2024, for olezarsen and does not plan to hold an advisory committee meeting.
  • Olezarsen is an investigational RNA-targeted medicine designed to lower triglyceride levels by reducing the production of apoC-III.
  • Phase 3 trials are fully enrolled to evaluate olezarsen for severe hypertriglyceridemia (sHTG), with data expected in the second half of 2025.

Riliprubart Shows Promise as First-in-Class Treatment for Chronic Inflammatory Demyelinating Polyneuropathy

Rare DiseaseNeurologyMonoclonal Antibody
  • Sanofi's riliprubart demonstrated significant disease-controlling benefits across all patient cohorts in a Phase 2 study for chronic inflammatory demyelinating polyneuropathy (CIDP), including those who failed standard treatments.
  • The complement C1s inhibitor showed sustained efficacy for up to 48 weeks, with 87-92% of participants experiencing improvement or disease stabilization after 24 weeks of treatment.
  • Riliprubart reduced neurofilament light chain levels by 35% across all cohorts, suggesting potential reduction in nerve damage, while also improving patient-reported fatigue and quality of life outcomes.

A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

NCT06290141RecruitingPhase 3
Sanofi
Posted 8/21/2024

A Study to Test the Effects and Safety of Riliprubart in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) for Which the Usual Treatments do Not Work

NCT06290128RecruitingPhase 3
Sanofi
Posted 7/12/2024

N-Power Medicine Secures Series B Funding to Enhance Oncology Clinical Trial Access

  • N-Power Medicine raised Series B funding led by Merck Global Health Innovation Fund to expand its oncology clinic network and biopharmaceutical collaborations.
  • The company's integrated data platform streamlines clinical trials and routine patient management, aiming to accelerate drug development timelines.
  • N-Power's platform includes the Kaleido Registry, which has enrolled over 7,000 patients, and AI-enabled workflow automation to support oncologists.
  • The funding will further N-Power's mission to integrate clinical research into everyday patient care, making trials more accessible to oncologists and patients.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.