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Clinical Trial News

Third-Line mCRC Treatment Landscape Evolves with Multiple Sequential Options Offering Modest Survival Benefits

  • The management of treatment-refractory metastatic colorectal cancer has evolved with three distinct therapeutic options, each offering modest but meaningful survival benefits of 2-6 months progression-free survival and 2-4 months overall survival improvements.
  • The SUNLIGHT trial demonstrated that combining TAS-102 with bevacizumab improves outcomes compared to TAS-102 alone, making this combination the preferred approach with meaningful progression-free survival of 5.6 months and overall survival of 10.8 months.
  • The FRESCO-2 trial validated fruquintinib as a VEGF tyrosine kinase inhibitor option for patients who had progressed on both regorafenib and TAS-102, providing multiple sequential treatment strategies.
  • Treatment decisions should balance survival benefits against toxicity profiles with careful patient selection, as quality of life considerations become paramount in this heavily pretreated population.

Phase III Study of Trifluridine/Tipiracil With and Without Bevacizumab in Refractory Metastatic Colorectal Cancer Patients

NCT04737187CompletedPhase 3
Taiho Oncology, Inc.
Posted 11/25/2020

A Study of Efficacy and Safety of Fruquintinib (HMPL-013) in Participants With Metastatic Colorectal Cancer

NCT04322539CompletedPhase 3
Hutchison Medipharma Limited
Posted 8/12/2020

Ambroxol Shows Promise in Slowing Parkinson's Disease Dementia in Phase II Clinical Trial

  • A 12-month clinical trial involving 55 participants with Parkinson's disease dementia found that Ambroxol, a European cough medicine, was safe and well-tolerated while reaching therapeutic levels in the brain.
  • Psychiatric symptoms worsened in the placebo group but remained stable in those taking Ambroxol, and participants with high-risk GBA1 gene variants showed improved cognitive performance.
  • The blood marker GFAP, linked to brain damage, increased in the placebo group but stayed stable with Ambroxol treatment, suggesting potential brain protection.
  • The findings offer hope for a new treatment avenue for Parkinson's dementia, which affects about half of Parkinson's patients within 10 years of diagnosis.

Neurocrine Biosciences Initiates Phase 1 Trial for Long-Acting CAH Treatment NBIP-01435

Rare Disease
  • Neurocrine Biosciences has launched a Phase 1 first-in-human clinical study evaluating NBIP-01435, a long-acting corticotropin-releasing factor type 1 receptor antagonist for congenital adrenal hyperplasia treatment.
  • NBIP-01435 represents the first investigational peptide from Neurocrine's biologics pipeline to advance to clinical trials, administered as a subcutaneous injection.
  • The compound builds on Neurocrine's CAH expertise following their December 2024 FDA approval of crinecerfont, the first new CAH treatment in 70 years.
  • CRF1 antagonism has demonstrated potential to improve androgen control while enabling lower, more physiological glucocorticoid dosing regimens for CAH patients.

Major Pharma Companies Face Massive Revenue Losses as Patent Cliff Hits Key Blockbusters

  • The pharmaceutical industry is experiencing one of the biggest patent cliffs since 2010, with blockbuster drugs including Merck's Keytruda, BMS's Eliquis, and Johnson & Johnson's Darzalex losing market exclusivity between 2025-2030.
  • Several top pharma companies face revenue decreases of up to 62% by 2030, with Johnson & Johnson's Stelara already losing $1.2 billion in sales due to biosimilar competition in the second quarter of 2025.
  • Companies are implementing defensive strategies including patent litigation, acquisitions, and pipeline strengthening, though up to 67% of new drug launches fail to meet sales targets according to EY analysis.

SOMAÍ Pharmaceuticals Acquires RPK Biopharma, Strengthening Global Cannabis Manufacturing Leadership

  • SOMAÍ Pharmaceuticals has completed a strategic acquisition of RPK Biopharma (Holigen), significantly enhancing its manufacturing capacity and global distribution capabilities in the medicinal cannabis sector.
  • The acquisition positions SOMAÍ as one of the few fully vertically integrated Multi-Country Operators with best-in-class cultivation, manufacturing, and distribution assets across multiple international markets.
  • This strategic move reinforces SOMAÍ's global leading position in the medicinal cannabis industry, expanding its operational footprint and strengthening its competitive advantage in the rapidly growing international cannabis market.

ICH Releases Draft E20 Guideline on Adaptive Clinical Trial Designs for Global Harmonization

  • The International Council for Harmonisation (ICH) has released draft guideline E20 "Adaptive Designs for Clinical Trials" for public consultation, marking the first harmonized global framework for adaptive trial designs in drug development.
  • The guideline addresses four key principles for confirmatory trials including adequacy within development programs, proper trial planning, controlling erroneous conclusions, and maintaining reliable treatment effect estimates.
  • Stakeholders have until November 30, 2025 to provide comments on the draft, which aims to provide clarity on terminology, benefits, and implementation challenges of adaptive designs while maintaining regulatory evidence standards.
  • The draft follows five and a half years after the FDA's 2019 guidance on adaptive designs, representing a significant step toward global regulatory harmonization in innovative clinical trial methodologies.

Radiotherapy-Immunotherapy Combinations Show Promise for Enhanced Cancer Treatment Through Abscopal Effects

  • Radiotherapy combined with immune checkpoint inhibitors demonstrates synergistic effects by generating tumor antigens and activating immune responses, with stereotactic body radiotherapy (SBRT) at 24 Gy/3F showing optimal efficacy in preclinical studies.
  • Low-dose radiotherapy (LDRT) emerges as a promising approach to remodel immunosuppressive tumor microenvironments without damaging lymphocytes, with growing clinical interest particularly in combination therapies.
  • Clinical trials in non-small cell lung cancer reveal increasing innovation in radiotherapy techniques and patient selection, with 65.3% of trials focusing on dose optimization and durvalumab being the most commonly studied immune checkpoint inhibitor.

Protagonist Therapeutics Advances PN-477 Triple Agonist for Obesity Treatment, Targeting Same Mechanism as Eli Lilly's Retatrutide

  • Protagonist Therapeutics has selected PN-477, a novel triple agonist peptide targeting GLP-1, GIP, and GCG receptors, as a development candidate for obesity treatment with both oral and injectable formulations.
  • The drug directly competes with Eli Lilly's retatrutide using the same triple-receptor mechanism, with Phase 1 clinical studies expected to begin in the second quarter of 2026.
  • PN-477 is designed to offer optimal weight loss with improved gastrointestinal tolerability and better fat-to-lean mass ratio, available as both a once-daily oral pill and once-weekly injection.
  • The company positions PN-477 as a potential best-in-class treatment that could address current market gaps in obesity therapeutics, representing a potential blockbuster opportunity in the competitive weight-loss drug market.

Artelo Biosciences Stock Surges Over 130% Following Positive Preclinical Depression Data and First-in-Human Pain Trial Results

  • Artelo Biosciences shares soared over 130% after reporting positive preclinical data for ART12.11 in stress-induced depression, showing efficacy comparable to Pfizer's Zoloft.
  • The company's ART26.12 non-opioid pain treatment candidate demonstrated favorable safety and pharmacokinetic profiles in its first-in-human study for persistent pain.
  • ART26.12 is being developed for chemotherapy-induced peripheral neuropathy, targeting a chronic pain market expected to exceed $159 billion by 2030.
  • All adverse events in the human trial were mild, transient, and self-resolving, with predictable dose-dependent linear absorption confirmed across the evaluated range.

FDA Mandates New Safety Warnings for ADHD Stimulants in Children Under Six

  • The FDA is revising labeling for all extended-release ADHD stimulants to include warnings about weight loss and adverse reactions in children under six years old.
  • Clinical trial data revealed that younger children have higher drug levels in their bodies and experience more side effects compared to older children.
  • Extended-release stimulants are not approved for children under six, but healthcare professionals can prescribe them off-label for ADHD treatment.
  • The agency determined that benefits may not outweigh risks in this younger patient population, prompting the new "limitation of use" labeling requirement.

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