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Clinical Trial News

AeroRx Therapeutics Reports Positive Phase 2a Results for First Nebulized LABA/LAMA Combination Therapy AERO-007 in COPD

  • AeroRx Therapeutics announced positive Phase 2a proof-of-concept results for AERO-007, the first nebulized LABA/LAMA combination therapy in development for COPD maintenance treatment.
  • The randomized, placebo-controlled trial in 16 adults with moderate-to-severe COPD demonstrated clinically meaningful and statistically significant improvements in FEV1 with both low and high doses.
  • AERO-007 combines FDA-approved bronchodilators indacaterol and glycopyrrolate via standard jet nebulizer, addressing an unmet need for patients who struggle with handheld inhalers.
  • The therapy showed rapid-onset and sustained 24-hour bronchodilation with good tolerability, potentially serving millions of COPD patients who remain symptomatic on current inhaler-based treatments.

Major Pharmaceutical Companies Lead Global Renal Failure Clinical Trials Landscape in 2025

  • A comprehensive 2025 clinical trials report reveals the global landscape of renal failure research, featuring data from over 80 clinical trial registries worldwide.
  • Ten major pharmaceutical companies including Bayer, AstraZeneca, Amgen, Sanofi, and Novartis are identified as prominent sponsors driving renal failure therapeutics development.
  • The report analyzes clinical trial distribution across G7 and E7 countries, providing insights into regional research patterns and enrollment trends over the past five years.
  • Clinical trials data encompasses various phases, trial statuses, and endpoint classifications, offering strategic intelligence for investment and research planning decisions.

Fresenius Medical Care Partners with Azenta to Advance Precision Kidney Disease Care Through Genomic Analysis

Precision MedicineAI
  • Frenova, Nephronomics, and GENEWIZ by Azenta Life Sciences have formed a strategic collaboration to advance genomic analysis of Frenova's My Reason® genomics research program.
  • The My Reason® program has registered over 35,000 participants with biospecimens, aiming to reach 50,000 participants over the next two years to build the world's most comprehensive kidney disease database.
  • The collaboration will leverage whole genome sequencing and AI/machine learning models to identify novel disease subtypes, protective genetic variants, and therapeutic targets for cardio-kidney-metabolic diseases.
  • GENEWIZ will provide DNA sequencing technologies and long-term sample storage through Azenta's global biorepository network to extract insights from the genomic and clinical data registry.

BeOne Medicines Receives EMA PRIME Designation for BTK Degrader BGB-16673 in Waldenström's Macroglobulinemia

Orphan Drug
  • BeOne Medicines received PRIME designation from the European Medicines Agency for BGB-16673, an investigational BTK degrader for treating Waldenström's macroglobulinemia patients previously treated with BTK inhibitors.
  • The EMA's Committee for Medicinal Products for Human Use issued a positive opinion on the EU Orphan Drug Designation application for BGB-16673 in WM, with a final decision expected in the coming weeks.
  • BGB-16673 is designed to overcome resistance by degrading both wild-type and mutant forms of BTK, representing the most advanced BTK protein degrader currently in clinical development.
  • The FDA has also granted Fast Track Designation to BGB-16673 for treating relapsed or refractory chronic lymphocytic leukemia, small lymphocytic lymphoma, and mantle cell lymphoma.

A Dose-Escalation and Expansion Study of BGB-16673 in Participants With B-Cell Malignancies

NCT05006716RecruitingPhase 1
BeiGene
Posted 9/13/2021

UNLV Researchers Achieve 99% Pancreas-Specific mRNA Delivery Using Novel ENDO Nanoparticle Platform

Targeted Therapy
  • UNLV researchers developed ENDO (Endogenous Targeting Lipid Nanoparticles) that achieve 99% selectivity for pancreatic delivery of mRNA therapeutics via intravenous injection.
  • The breakthrough technology exploits Vitamin D receptors on pancreatic cell surfaces to route nanoparticles specifically to the pancreas, marking the first material capable of such precision.
  • This targeted delivery system could revolutionize treatment for diabetes and pancreatic cancer by enabling direct mRNA therapy to pancreatic cells while minimizing systemic side effects.
  • The platform shows potential for expansion to other difficult-to-reach organs including the brain and heart, with commercialization efforts already underway through UNLV's Office of Economic Development.

Lung Cancer Experts Convene to Address Clinical Uncertainties in Rapidly Evolving Treatment Landscape

Targeted Therapy
  • The third annual Bridging the Gaps in Lung Cancer consensus meeting brings together approximately 30 global experts to identify and address unresolved clinical challenges in lung cancer management.
  • Key knowledge gaps persist in optimal duration of adjuvant targeted therapy, management of patients with complete pathologic response after neoadjuvant chemoimmunotherapy, and treatment guidelines for leptomeningeal disease.
  • The meeting's unique format focuses exclusively on clinical ambiguity rather than established standards, with consensus recommendations evolving annually as new trial data and biomarker validation studies emerge.
  • New features include livestreaming and social media integration to broaden reach and demonstrate variability in expert perspectives across different healthcare systems.

Comprehensive Safety Management Strategies Transform PI3K Inhibitor Treatment Delivery in Metastatic Breast Cancer

  • Proactive toxicity management for PIK3CA inhibitor combinations requires early patient contact after the first week of therapy with specific education about hyperglycemia warning signs and immediate treatment interruption protocols.
  • Multiple studies demonstrate that clinical benefits persist despite dose reductions, making sustained treatment delivery the primary goal rather than maintaining maximum doses for long-term therapy success.
  • Effective management extends beyond hyperglycemia to include comprehensive monitoring of skin rash, stomatitis, and diarrhea, with prophylactic interventions using antihistamines and dexamethasone mouth rinses.
  • Future treatment algorithms will incorporate emerging oral SERDs and individualized approaches based on disease characteristics, with sequential therapy strategies preserving options for patients throughout their treatment journey.

AbbVie in Advanced Talks to Acquire Mental Health Company Gilgamesh Pharmaceuticals for $1 Billion

  • AbbVie is reportedly in discussions to acquire clinical-stage mental health therapeutics company Gilgamesh Pharmaceuticals in a deal valued at approximately $1 billion.
  • The acquisition would strengthen AbbVie's psychiatric disorder pipeline, building on an existing partnership where Gilgamesh could receive up to $1.95 billion in option fees and milestone payments.
  • Gilgamesh is developing neuroplastogen compounds for depression, anxiety, and PTSD, with positive preliminary results from a mid-stage trial of GM-2505 for major depressive disorder.
  • The potential deal reflects AbbVie's broader acquisition strategy, having spent over $20 billion on acquisitions since 2023 as its flagship Humira treatment lost patent protection.

Osteosarcoma Market Poised for Significant Growth Through 2034 as Novel Therapies Enter Pipeline

Monoclonal AntibodyOrphan DrugRare Disease
  • The osteosarcoma market in the 7MM is expected to grow significantly by 2034, driven by extensive R&D activities and the entry of novel therapies including OST-HER2, Naxitamab 15-096, and HALMB-0168.
  • OS Therapies plans to submit a Biologics License Application for OST-HER2 to the US FDA by 2025, with the potential to receive a Priority Review Voucher upon approval.
  • The market faces challenges including chemotherapy resistance in approximately half of patients and limited treatment options for relapsed or refractory cases.
  • Currently, MEPACT (mifamurtide) remains the only approved therapy specifically for osteosarcoma, highlighting significant unmet medical needs in this rare cancer.

Inka Health Partners with OneMedNet to Develop AI-Powered External Control Arms for Cancer Drug Development

AIReal World EvidenceOncology
  • Inka Health has been selected by OneMedNet to lead a data analytics initiative using real-world data to develop external control arms as a faster, more cost-effective alternative to traditional clinical trials.
  • The project will deploy Inka Health's proprietary SynoGraph platform to build and validate an external control arm for the Keynote-189 trial of pembrolizumab in non-small cell lung cancer.
  • This collaboration aims to accelerate market entry for new cancer therapies by offering a more efficient path to generate evidence needed for regulatory and reimbursement approvals.
  • The initiative positions both companies in the rapidly expanding Real-World Evidence market, projected to exceed $4.6 billion globally by 2030.

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