MedPath

Tagged News

FDA's Approval of MRD as End Point Transforms Multiple Myeloma Research and Treatment

Drug ApprovalMonoclonal AntibodyOncology
  • The FDA's Oncology Drugs Advisory Committee unanimously approved minimal residual disease (MRD) as an end point for accelerated approval of multiple myeloma therapies in April 2024, potentially reducing trial timelines from 10-15 years to just 3 years.
  • Pharmaceutical companies have rapidly adapted by implementing MRD as a coprimary end point in new trials and amending existing protocols, with the CEPHEUS trial being the first major study to read out with MRD as a coprimary endpoint.
  • Researchers are now exploring MRD applications beyond drug approval, including using MRD status to guide treatment decisions, developing improved blood-based detection technologies, and expanding the approach to other hematologic malignancies.

A Study of Daratumumab, Carfilzomib, Lenalidomide, and Dexamethasone in Patients With Newly-Diagnosed Multiple Myeloma

NCT04268498Active, Not RecruitingPhase 2
University of Miami
Posted 2/11/2020

A Study Comparing Daratumumab, VELCADE (Bortezomib), Lenalidomide, and Dexamethasone (D-VRd) With VELCADE, Lenalidomide, and Dexamethasone (VRd) in Participants With Untreated Multiple Myeloma and for Whom Hematopoietic Stem Cell Transplant is Not Planned as Initial Therapy

NCT03652064Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 11/6/2018

Daratumumab, VELCADE (Bortezomib), Lenalidomide and Dexamethasone Compared to VELCADE, Lenalidomide and Dexamethasone in Subjects With Previously Untreated Multiple Myeloma

NCT03710603Active, Not RecruitingPhase 3
Stichting European Myeloma Network
Posted 12/14/2018

A Study of Subcutaneous Daratumumab Versus Active Monitoring in Participants With High-Risk Smoldering Multiple Myeloma

NCT03301220Active, Not RecruitingPhase 3
Janssen Research & Development, LLC
Posted 11/7/2017

CellCentric Secures $120 Million Series C Funding to Advance Oral Multiple Myeloma Treatment

Oncology
  • CellCentric has raised $120 million in Series C funding to advance inobrodib, a first-in-class oral p300/CBP inhibitor for multiple myeloma treatment.
  • The funding will support a Phase II/III study with potential for accelerated approval, as well as trials combining inobrodib with bi-specific antibodies and in maintenance settings.
  • Inobrodib has received FDA Fast Track and orphan drug designation, with promising efficacy and safety data presented at the American Society of Hematology Annual Meeting.

GT Biopharma Advances GTB-3650 Phase 1 Trial to Second Cohort Following Promising Immune Activation Signals

Oncology
  • GT Biopharma has successfully completed dosing in the first cohort of its Phase 1 trial for GTB-3650, a second-generation TriKE immunotherapy, with no safety or tolerability issues observed.
  • Both patients in Cohort 1 demonstrated early biological signs of immune activation, including increased natural killer cell activity and expansion, supporting the therapy's mechanism of action.
  • The trial, which will evaluate approximately 14 patients across seven dose cohorts, aims to treat relapsed or refractory CD33-expressing hematologic malignancies through enhanced NK cell-mediated cancer destruction.

HM2023-05: GTB-3650 Trike for High Risk MDS and R/R AML

NCT06594445RecruitingPhase 1
Masonic Cancer Center, University of Minnesota
Posted 11/19/2024

Eascra Biotech Secures $100,000 MassVentures Grant for Novel Nanoparticle Drug Delivery Platform

Gene EditingOncology
  • Eascra Biotech has received a $100,000 Stage I SBIR Targeted Technologies grant to advance their Janus Base Nanoparticles (JBNps) delivery platform for hard-to-reach tissues.
  • The company has leveraged microgravity research, completing five missions on the International Space Station, becoming the first commercial entity to produce medical nanoparticles in space.
  • Eascra's proprietary technology can deliver RNA therapeutics to challenging tissues like cartilage and solid tumors while maintaining mRNA stability at room temperature, eliminating cold chain requirements.

Fujifilm Validates Novel Peptide-Oligonucleotide Conjugates for Targeted Cancer Cell Delivery

OncologyDrug Discovery
  • Fujifilm has successfully validated peptide-oligonucleotide conjugates that selectively target cancer cells, utilizing cyclic peptides from their proprietary library containing trillions of variants.
  • The company developed a cyclic peptide that binds strongly to integrins overexpressed on cancer cell surfaces with a low dissociation constant of 1.6 nM, enabling higher cellular uptake compared to non-conjugated oligonucleotides.
  • This breakthrough addresses delivery challenges in oligonucleotide-based therapies and will be presented at TIDES USA 2025 in San Diego, highlighting Fujifilm's expansion of global drug discovery services for peptide therapeutics.

Gustave Roussy Launches UMBRELLA Trial: Using Blood-Based Tumor DNA to Revolutionize Cancer Follow-Up Care

OncologyPrecision MedicineAI
  • Gustave Roussy has initiated UMBRELLA, a groundbreaking phase III trial that personalizes post-treatment cancer monitoring based on minimal residual disease (MRD) detected through blood-based circulating tumor DNA analysis.
  • The trial will evaluate two strategies: pre-emptive immunotherapy (tislelizumab) for MRD-positive patients and reduced follow-up schedules for MRD-negative patients with non-metastatic lung, colorectal, pancreatic, and soft tissue sarcomas.
  • UMBRELLA represents the first French multi-cancer trial using MRD status for therapeutic stratification, aiming to enroll over 700 patients across 10-11 centers in a collaborative effort involving Veracyte, IntegraGen, and BeiGene.

tislelizUMaB in canceR Patients With molEcuLar residuaL Disease

NCT06332274RecruitingPhase 3
Gustave Roussy, Cancer Campus, Grand Paris
Posted 4/16/2025

Surgical Robotics Advance: EndoQuest and Virtuoso Begin First Human Trials for Minimally Invasive Procedures

Oncology
  • EndoQuest Robotics has initiated its pivotal Paradigm trial for an endoluminal robotic system designed to perform endoscopic submucosal dissection for colorectal lesions through natural body openings.
  • Virtuoso Surgical has successfully completed first-in-human procedures using its robotic endoscopy system with needle-sized manipulators to perform en bloc bladder tumor resections at The Chinese University of Hong Kong.
  • Both robotic systems aim to address technical challenges in minimally invasive procedures, potentially reducing the need for more invasive surgeries and improving patient outcomes.

CytomX Doses First Patient in Combination Trial of CX-801 with Keytruda for Metastatic Melanoma

OncologyTargeted Therapy
  • CytomX Therapeutics has dosed the first patient in a Phase 1 trial combining its masked interferon alpha-2b (CX-801) with Merck's Keytruda in patients with metastatic melanoma.
  • The novel combination aims to address the high unmet need in PD-1 refractory melanoma by localizing interferon's potent immune-stimulating effects to tumors while minimizing systemic toxicities.
  • Initial Phase 1a translational and biomarker data from this study are expected in the second half of 2025, potentially advancing a new approach in combination immuno-oncology therapy.

First In Human Study of CX-801 in Advanced Solid Tumors

NCT06462794RecruitingPhase 1
CytomX Therapeutics
Posted 8/30/2024

Johnson & Johnson and Karolinska Institutet Celebrate Decade of Collaboration Advancing Medical Innovations

OncologyNeurology
  • The ten-year partnership between Johnson & Johnson Innovative Medicine and Karolinska Institutet has yielded significant scientific advances across nine disease areas, with researchers working side by side since 2015.
  • Over 150 researchers have participated in the collaboration, producing more than 30 scientific papers and 50 presentations at international conferences, focusing on critical medical needs in oncology, neurology, immunology, and ophthalmology.
  • The partnership exemplifies successful innovation through academic-industry collaboration, with both organizations now exploring new opportunities to develop innovative therapies for complex conditions like Alzheimer's disease and cancer.

Astellas to Present New Long-Term Survival Data for Cancer Therapies at ASCO 2025

Oncology
  • Astellas will showcase 16 abstracts at the 2025 ASCO Annual Meeting, including two oral presentations highlighting long-term survival data for its oncology portfolio.
  • New post-hoc analyses will feature five-year overall survival data for XTANDI (enzalutamide) in metastatic hormone-sensitive prostate cancer and exploratory analyses for PADCEV (enfortumab vedotin) in urothelial cancer.
  • The presentations underscore Astellas' commitment to delivering meaningful clinical outcomes for patients with advanced prostate and bladder cancers through innovative treatment approaches.

A Study of Enzalutamide Plus Androgen Deprivation Therapy (ADT) Versus Placebo Plus ADT in Patients With Metastatic Hormone Sensitive Prostate Cancer (mHSPC)

NCT02677896CompletedPhase 3
Astellas Pharma Global Development, Inc.
Posted 3/9/2016

Darolutamide in Addition to ADT Versus ADT in Metastatic Hormone-sensitive Prostate Cancer

NCT04736199Active, Not RecruitingPhase 3
Bayer
Posted 2/23/2021

Enzalutamide in First Line Androgen Deprivation Therapy for Metastatic Prostate Cancer

NCT02446405Active, Not RecruitingPhase 3
University of Sydney
Posted 3/1/2014

Safety and Efficacy Study of Enzalutamide Plus Leuprolide in Patients With Nonmetastatic Prostate Cancer (EMBARK)

NCT02319837Active, Not RecruitingPhase 3
Pfizer
Posted 12/17/2014

MedPath

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

© 2025 MedPath, Inc. All rights reserved.