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Clinical Trial News

Anti-Obesity Drugs Show Promise in Young Children, but Long-Term Effects Remain Unknown

  • A recent study shows that liraglutide, a GLP-1 mimic, led to a BMI decrease in children aged 6-11, offering a potential treatment for early-onset obesity.
  • While trials indicate significant weight loss in adolescents using drugs like semaglutide and liraglutide, concerns persist regarding long-term impacts on growth and puberty.
  • Experts emphasize the need for diverse studies and prolonged monitoring to address ethical considerations and potential side effects in young children using GLP-1 mimics.
  • The use of BMI as a metric for measuring progress has disadvantages, and weight-related health problems should be considered.

Keytruda Demonstrates Significant Overall Survival Benefit in Early-Stage TNBC

  • Phase III KEYNOTE-522 trial shows Keytruda plus chemotherapy significantly improves overall survival (OS) in high-risk early-stage triple-negative breast cancer (TNBC).
  • The Keytruda regimen reduced the risk of death by 34% compared to chemotherapy alone, with a five-year OS rate of 86.6% versus 81.7%.
  • The study reinforces Keytruda's role as a perioperative therapy, showing consistent OS benefits across pre-specified subgroups, including tumor size and PD-L1 expression.
  • Immune-mediated adverse events were more frequent in the Keytruda arm, but the OS benefit supports its commercial adoption with careful patient management.

Study of Pembrolizumab (MK-3475) Plus Chemotherapy vs Placebo Plus Chemotherapy as Neoadjuvant Therapy and Pembrolizumab vs Placebo as Adjuvant Therapy in Participants With Triple Negative Breast Cancer (TNBC) (MK-3475-522/KEYNOTE-522)

NCT03036488Active, Not RecruitingPhase 3
Merck Sharp & Dohme LLC
Posted 3/7/2017

BridgeBio's BBO-8520: A Novel KRAS(G12C) Inhibitor Shows Promise in NSCLC

  • BridgeBio's BBO-8520 is a selective, covalent KRAS(G12C) inhibitor designed to target the 'ON' state of the protein, potentially enhancing clinical efficacy.
  • Presented at the 2024 AACR Annual Meeting, BBO-8520 is currently undergoing a Phase I trial in patients with advanced non-small-cell lung cancer (NSCLC).
  • Preclinical data suggests that BBO-8520 may overcome resistance to existing KRAS(G12C) inhibitors, offering a new therapeutic avenue for KRAS(G12C)-driven cancers.

Oncology Accelerated Approvals Often Based on Non-Comparative Trials

  • A recent study highlights that oncology accelerated approvals frequently rely on non-comparative trials assessing response rates, raising questions about long-term efficacy.
  • The reliance on single-arm trials for accelerated approvals may lead to uncertainties regarding the true clinical benefit compared to existing treatments.
  • Post-marketing requirements and commitments are crucial to confirm the efficacy and safety of oncology drugs initially approved based on limited data.
  • Regulatory agencies face the challenge of balancing the need for rapid access to promising therapies with the necessity for robust evidence of clinical benefit.

Neuralink Receives FDA Breakthrough Device Designation for Vision Implant

  • Neuralink has been granted Breakthrough Device designation by the FDA for its innovative vision implant, aiming to restore sight to individuals with blindness.
  • The implant works by directly stimulating the brain, bypassing damaged eyes, and holds potential for treating various vision impairments.
  • This designation expedites the development and review process, facilitating quicker access to the technology for patients in need.
  • Neuralink's technology represents a significant advancement in neurotechnology, offering hope for restoring visual function and improving the quality of life for the visually impaired.

Bubs Australia Pursues Permanent US Market Access with Clinical Trial

  • Bubs Australia is seeking permanent FDA approval to sell its infant formula in the US market beyond the current temporary authorization expiring in October 2025.
  • A clinical trial enrolling 400 infants is underway to demonstrate that babies fed Bubs formula grow at a similar rate to those fed cow milk formula.
  • Bubs has significantly grown its US revenue, now exceeding China, and aims to achieve breakeven EBITDA in FY25 with continued sales growth.
  • The company's future hinges on securing permanent access to the US market, making the clinical trial and regulatory approval critical milestones.

Senseonics' Eversense 365, the First One-Year CGM, Receives FDA Approval

  • Senseonics and Ascensia Diabetes Care received FDA approval for Eversense 365, the first continuous glucose monitoring (CGM) system offering a full year of use.
  • Eversense 365 is a fully implantable device for adults with type 1 and type 2 diabetes, minimizing life disruption compared to short-term CGMs.
  • The system is designated as an integrated CGM (iCGM), allowing integration with insulin pumps for automated insulin delivery, enhancing diabetes management.
  • Expected to launch in the US in Q4 2024, Eversense 365 aims to improve time in range and lower A1c levels, offering freedom and peace of mind to patients.

Madrigal's Resmetirom Nears Full Approval for NASH Cirrhosis Treatment Following Phase 3 Enrollment Completion

  • Madrigal Pharmaceuticals completed patient enrollment for its MAESTRO-NASH OUTCOMES trial, evaluating resmetirom for compensated NASH cirrhosis.
  • The Phase 3 trial involves 845 patients and aims to measure the progression to liver decompensation events over two to three years.
  • Positive outcomes from the trial could support full approval of Rezdiffra for noncirrhotic NASH and expand its indication to cirrhotic patients.
  • Resmetirom, a liver-directed THR-β agonist, has already received accelerated approval for NASH with moderate to advanced liver fibrosis.

A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis

NCT06215716RecruitingPhase 3
Akero Therapeutics, Inc
Posted 12/1/2023

A Study Evaluating Efruxifermin in Subjects With Non-invasively Diagnosed Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Nonalcoholic Fatty Liver Disease (NAFLD)/Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)

NCT06161571Active, Not RecruitingPhase 3
Akero Therapeutics, Inc
Posted 11/10/2023

A Study of Efruxifermin in Subjects With Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis (NASH) (Symmetry)

NCT05039450Active, Not RecruitingPhase 2
Akero Therapeutics, Inc
Posted 7/30/2021

A Study Evaluating Efruxifermin in Subjects With Compensated Cirrhosis Due to NASH/MASH

NCT06528314RecruitingPhase 3
Akero Therapeutics, Inc
Posted 9/4/2024

A Phase 3 Study Evaluating the Safety and Efficacy of Denifanstat in Patients With MASH and F2/F3 Fibrosis

NCT06594523WithdrawnPhase 3
Sagimet Biosciences Inc.
Posted 3/1/2025

ADVENT-HF Trial Shows Promise for Peak Flow-Triggered ASV in Heart Failure Patients with Sleep Apnea

  • The ADVENT-HF trial demonstrated that peak flow-triggered adaptive servo-ventilation (ASV) devices improved sleep quality and quality of life in heart failure patients.
  • Unlike the SERVE-HF trial, ADVENT-HF did not find an increased risk of mortality with ASV use; mortality trended lower, though not statistically significant.
  • The study highlights that ASV devices are not generic, with efficacy and safety varying based on manufacturer-specific algorithms and triggering mechanisms.
  • ADVENT-HF used Philips Respironics BiPAP autoSV Advanced, which differs from the minute ventilation-triggered ASV used in SERVE-HF, potentially explaining the divergent outcomes.

Neuralink's Blindsight Implant Receives FDA Breakthrough Device Designation

  • Neuralink's Blindsight implant, designed to restore vision, has received Breakthrough Device Designation from the FDA, expediting its development and review.
  • The implant aims to enable sight in individuals who have lost both eyes and their optic nerve, potentially benefiting those blind from birth.
  • Early vision with Blindsight may resemble low-resolution Atari graphics, but Neuralink anticipates future enhancements, including infrared and ultraviolet vision.
  • Neuralink is also developing brain implants for paralyzed individuals to control digital devices, with ongoing clinical trials evaluating their effectiveness.

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